Portfolio updates –ADC data readouts, AVROBIO, Chiasma

Crucial ADC readouts at WCLC 2019

The coming months will be very important for the ADC industry which has been struggling to bring forward effective treatments, especially for solid tumors. While there have been some positive news (mostly utilizing Seattle Genetics’ technology like the recent  update on GSK’s BCMA program in multiple myeloma), existing platforms are still limited by side effects. In order to address this need, the industry has to find new technologies that would minimize off -target toxicities.

To me, the most intriguing ADC platform today is that of Daiichi Sankyo, which has been quietly developing its next generation ADC platform at a time when most of its peers de-prioritized their ADC programs.

Daiichi’s bet started to pay off with DS-8201, now partnered with AstraZeneca (AZN), which demonstrated unprecedented efficacy in breast cancer. The company has 6 additional ADC programs, including two in clinical testing:  DS-1062 (anti-TROP2) and U3-1402 (anti-HER3). Both programs will report data at WCLC 2019 next month that, based on previous results, could demonstrate impressive efficacy.

The TROP2 program looks particularly promising based on preliminary findings presented at ASCO 2019. The study was able to reach high doses not typically achieved with ADCs (8 mg/kg) without severe toxicity issues (although side effects were clearly observed) and demonstrate very encouraging dose-dependent efficacy. If the trend continues and no safety show stoppers emerge, the 6-8 mg/kg cohorts may demonstrate a >50% response rate, which is unprecedented for an ADC in NSCLC. Response rate could actually be higher in TROP2 expressors as the trial did not include TROP2 expression as an entry criterion.


Data for DS-1062 and U3-1402 will be crucial for the entire ADC field as they could potentially validate Daiichi’s ADC platform and provide the first differentiated next-gen ADC platform in over a decade. Most importantly, they might mark the start of the next innovation cycle in the ADC space.

Positive results for DS-1062 will have a material impact on Immunomedics (IMMU), which has a TROP2 ADC. While Immunomedics’ lead indication is TNBC (triple negative breast cancer), their ADC could become irrelevant even before an expected approval next year as both ADCs use Topo-1 inhibitors as payloads. If DS-1062’s data are as good as expected, it will be very hard to justify Immunomedics’ valuation of ~$2.5B. Therefore, I would stay out of the stock going into WCLC 2019 given the asymmetric risk/reward profile.

AVROBIO – Back in the game

I was previously very skeptic about AVROBIO’s (AVRO) Fabry program (AVR-RD-01) given the decline in enzyme (AGA) levels and VCN seen 3-6 months post treatment. Based on the trajectory at the time I thought transduced cells will eventually vanish after ~2 years which could make AVR-RD-01 irrelevant.

I am now much more optimistic following  last month’s update that demonstrated stabilization of the slope of AGA levels and VCN in two patients 18 and 28 months post treatment, respectively (three additional patients did not have long enough follow up).

While more data are needed (# of patients and longer follow up) the new data suggests AVR-RD-01 may have a long lasting “tail” of enzyme activity. Enzyme levels generated by AVR-RD-01 appear clinically meaningful based on substrate reduction to levels below what had been achieved with ERT in these patients. Importantly, patient #1 is ERT free for 10 months with no significant changes in substrate levels, which unequivocally demonstrates the treatment’s activity with ERT’s “masking” effect.


Substrate levels are considered a good surrogate for long term tissue damage (mainly kidney and heart) but the clinical impact will have to be evaluated in the long run. A read-across from AVROBIO’s P2 in ERT-naïve patients further supports the notion that levels achieved in P1 will lead to clinical improvement.

The company presented biopsy data from an ERT-naive patient that had a dramatic reduction in substrate in the kidney, which is the approvable endpoint for Fabry. Enzyme levels in this patient were similar to long term expression levels seen in patients in P1. In theory, long term clinical outcomes should be better with AVR-RD-01 vs. ERT as the prior produces constant, stable enzyme levels vs. the typical peak/trough profile with ERT.

Things look much better for AVROBIO today but long term durability will remain an overhang for a long time as the company aspires to provide life-long treatments. Risk is still high but with the new data demonstrating stabilization or near-stabilization of enzyme levels in P1 coupled with kidney biopsy data in P2 and experience with other bone marrow transplant technologies, the company is attractive at the current valuation.

AVR-RD-01  is competing with AAV-based treatments for Fabry from  Amicus (FOLD), Abeona (ABEO) and Freeline. These treatments from are more straightforward to administer as a “drug in a vial” but their distribution profile and durability are still unclear.

Chiasma – Positive P3 but market unimpressed

Chiasma (CHMA) reported positive P3 data for its oral octreotide program, Mycapssa, in acromegaly. Despite the clear efficacy across every endpoint, the stock price is now lower than the price prior to the announcement.

Overall, results look very good to me with a clear superiority over placebo, which they demonstrate for the first time (previous P3 was a single arm study). The study, which involved switching stabilized patients from an injectable to Mycapssa, clearly demonstrated that in a significant portion of patients the disease can be controlled with Mycapssa: 58% of patients in the Mycapssa maintained an IGF-1 response (≤ 1.0 x ULN) vs. 19% in the placebo arm.

The drug’s eventual market penetration is a matter of debate but given the pain and inconvenience associated with injectables, Mycapssa should be able to capture a significant market share.  It is easy to understand why patients and physicians will prefer to start with oral and only if patients are not controlled, switch or add an injectable drug. Initial uptake is hard to predict but the eventual conversion to an oral alternative is highly likely.

Looking at rescue treatment and drop off rates during the study provides more color Mycapssa’s use in real life. 25% of patients in the active arm received rescue treatments vs. 68% in the control arm. Importantly 90% of patients who completed 36 weeks on Mycapssa opted to stay on the drug as part of an open label extension study.

The lackluster stock reaction can be attributed to future competition from Crinetics (CRNX), which has an oral agent (CRN00808) with encouraging P1 data including good PD modulation. As a highly selective and potent small molecule dosed once daily, CRN00808 represents a major threat to Chiasma but it is still in P2 and has no data in patients. One important advantage Chiasma has is the fact it utilizes a known and approved product with years of clinical experience. As a novel small molecule, Crinetics faces off target risks that will have to be assessed in long term studies.

In summary, Chiasma is now a de-risked story with a decent chance of capturing 25-50% of the acromegaly market. The indication represents a ~$900M  global opportunity so even a 20% chunk that translates to $180M in annual sales makes Chiasma a good risk/reward bet with a market cap of $216M.

Portfolio updates

I am starting a new position in AVROBIO based on the recent update in Fabry. I am also adding follow-on positions in Madrigal (MDGL) and Viking Therapeutics (VKTX), and selling Nightstar (NITE) following the Biogen (BIIB) acquisition.

Portfolio holdings – Aug 25, 2019

Biotech portfoio - 25-8-2019 - after changesBiotech ETFs - 25-8-2019

239 thoughts on “Portfolio updates –ADC data readouts, AVROBIO, Chiasma

  1. Ohad…Speaking of antibody drug conjugates…what are your thoughts on MRSN or STRO programs/molecules?

  2. ONCE – the merger with Roche postponed to Sept. 3
    the stock now is ~ 100$
    what do you think on adding ONCE and gaining an easy 10%?

  3. Robert goulet (MRSN/STRO) –

    MRSN – So far their technology has solved hte main problem of therapeutic window. Just like previous technologies, they can’t dose escalate easily and efficacy isn’t stellar.
    STRO – Their pipeline is too early, I am interested in their FRa program but need to wait for clinical data. They should have updated results at ASH for the CD74 program.

    Alex –
    MDGL/VKTX – I view both as long term de-risked holdings and potential M&A candidates at these valuations.
    AGTC , prefer to wait for more data.
    NERV – Prefer to wait for P3 readout this year, high risk event.

    Alex (ONCE) – I also think the deal with go through eventually but don’t know how to factor in risk/reward.


  4. immu-132, the trop-2 adc had similar response rates at this stage for their development. Their dose is 10 mg/kg and they are much ahead in development with their BLA to be resubmitted early 4th qtr.

  5. Ohad,

    thanks for your write-up, always interesting.

    Regarding ADCs, do you think ZYME’s ADC-Tech from their Kairos takeover has good chances of success? They started with licensing their tech (one small deal so far, and it sounded as they are expecting additional deals).
    I am very much looking forward to ZW49 first data later this year… Do you think it can compete with DS-8201..?

    Then another one, Belbuca (buccal film – oral formulation of buprenorphine) from BDSI – long-acting opioid schedule III substance – has a really nice growth trajectory, see below. Most opiods are schedule II. They have done a good job of getting coverage during the last few years.

    Q1 2018 to Q2 2019 TRx:
    27k -> 35k -> 44k -> 56k -> 65k -> 80k

    For this year they are guiding now 90 – 93 mio. USD net turnover for Belbuca (plus Symproic which they now distribute in the USD which gives a total guidance of 101 – 105 mio USD). Long-term they are now guiding 425 – 500 mio. USD annual peak sales for Belbuca and Symproic.

    They are valued at about 380 mio. USD. Sounds relatively cheap?

  6. Ohad
    Nice blog!
    Especially you added to stocks I like:)

    Is there any catalysis for MDGL & VKTX this year? It looks there will be some readouts next year. What is the reason to add now, not wait, let’s say until December?

    No position in IMMU yet, on my watch list though, but curious why positive readout with the same target TROP2 should be negative? They both validates each other, as in NASH, no?.
    You do not declare VKTX ‘irrelevant’ bc it will be 2 years behind MDGL. Not picking on you, just trying to learn.

  7. hi again Ohad,

    one more regarding CHMA

    If their TPE tech is good/valid, didn’t they conclude any license deals for their tech (maybe they have but I am not aware of it).

    Also I am wondering a little why they don’t have any advanced plans for Mycapssa in other indications. IP protection for this drug isn’t that long if I remember correctly.

    thanks as always for your insights!

  8. Alex, re ONCE
    I also think deal goes through (caveat emptor, worthless opinion, just an uninformed hunch) but doubt by Sept 3. You probably know this but for others Sep 3 is just the expiry date of the current tender offer, which they have already pushed back several times while waiting for FTC (and UK’s CMA?) to OK the deal. As far as I’m aware there is no new news on that front other than Roche saying recently words to the effect of apologies for delay but that they still expect deal to go through by end of year and are fully committed to it. If anyone knows anything else love to hear it.

  9. provocateur (IMMU) – I don’t think IMMU-132 demonstrated a >50% response rate at any stage but first let’s see if actual data for DS-1062 are that strong. Yes, they are ahead in development for TNBC but given the similar MOA, DS-1062 could eventually displace it.

    Christian (ZYME) – Yes, I am still optimistic about their ADC platform, preclinical package looks very good but we’ll have to wait for P1 data.

    Sorry, don’t know BDSI…

    andre –

    MDGL/VKTX – No major catalysts this year I am aware of but their valuations are attractive at these levels imo and programs are de-risked at least to some degree.

    IMMU – The main difference is that I assume (needs to be proven eventually) DS-1062 will be much more effective than ‘132. I cannot say that about either MGL-3196 or VK2809 at this stage.

    Christian (CHMA) – That’s a good question, I am not aware of any other programs or partnerships. I presume it’s a consequence of business decisions and probably technical challenges in finding an appropriate cargo and indications where the technology can reliably work.

    Alain (STML) – Overall launch is going very well but market opportunity is limited.


  10. I noticed the following patent application for CHMA:
    United States Patent Application 20190240284
    August 8, 2019


    Methods are disclosed of treating or preventing hypotension (e.g., neurogenic orthostatic hypotension or postprandial hypotension) or portal hypertension (e.g., bleeding esophageal varices associated with portal hypertension) or ascites (e.g., ascites associated with liver cirrhosis), by oral administration to affected subjects of compositions comprising a therapeutically effective amount of terlipressin or a pharmaceutically acceptable salt thereof.
    Chiasma, Inc. Waltham

    TERLIPRESSIN is a peptide hormone effective in the pituitary gland, Maybethe next peptide that should be made available orally.
    Terlipressin is an analogue of vasopressin used as a vasoactive drug in the management of low blood pressure. It has been found to be effective when norepinephrine does not help. Its currently not available in the US.

  11. Ohad , thanks for all you do. Do you happen to have an opinion on CCXI which has upcoming phase 3 data in Anca vasculitis 4th Q . A modest mkt cap of $396m a little over half of it in cash.

  12. Hello Ohad!
    Nice to read the update!
    Saw some skepticism about mdgl and the design of the Ph3 as they will be testing two doses- it seems in case the preferred dose generates a safety signal. Any concerns with their trial design and the rationale for also using a lower dose than the go ahead dose?

    Regarding CHMA – will they remain independent or do you expect a large rare diseases company to acquire them? And what would be valuation? 5x top annual revenues? So between $1 and $1.5B – or is this at the moment a mirage?

    What do you need to see from APTO MYC program to become a believer?

    Finally what are the implications for ESPR of the MDCO RNAI ldl lowering drug results?



  13. Hi Ohad,

    Thanks for your thorough analysis as always.
    This might be a naive question, but is it normal for companies like VKTX and MGDL and the likes to be run by less than 20 people? I know that they are outsourcing most of the clinical trial works, but with a portfolio of a few molecules 14 or 17 people seem far too few to support them. Is this the norm in the industry?

  14. Ruedi Schmid (CHMA) – Thanks, inteersting to see they are still issuing patents. I haven’t heard anything from the company about additional programs.

    Paul (CCXI) – I don’t have a strong opinion there, indication and biology are complex so high technical risk imo.

    Dan (MDGL) – I think it’s perfectly normal to think about safety in this setting of chronic small molecule treatments for non-life threatening diseases, don’t see anything wrong with it. Safety is a major risk here, no doubt as the bar is set very high…

    CHMA – I don’t expect anybody to buy them before approval (given previous attempts), probably potential acquirers would want to wait to see market uptake of several Qs. I plan to add next year if price stays at this level. I think a 5x multiple is reasonable.

    APTO – I would like to see safety and efficacy data that demonstrate clinical activity (not just biomarkers)

    ESPR – I don’t think these drugs compete in the same market, different pt subsets imo.

    Erin (VKTX/MDGL) – Agree, it does sound quite low. Not sure if this figure is up to date.


  15. AUPH – Phase 3 results for voclosporin nearing, what do you think are their chances?

  16. Good, excellent results for ESPR today- clean and effective drug – the combo almost as effective as PCSK9 drugs with the addies benefit is also lowers inflammation by 25%. Maybe time to add to the position?

  17. Hi Ohad thanks a lot for the very nice note as always. Great readings!
    Daiichi TROP2 ADC programs will have oral presentation or posters at WCLC 2019?
    Any link?
    Thanks Bruno

  18. Hi Ohad,

    As a follow up to your comment regarding company size, I think the figures are current. If you look at the most recent VKTX form 10-Q (http://ir.vikingtherapeutics.com/sec-filings) , under the stated risks it is shown that they only have 14 full time employees. Given that they have 5 candidates in the pipeline, I have a hard time understanding how the trials are thoroughly managed. Might be typical for small biotechs, but I personally was shocked at the small scale.

  19. Hi Ohad,

    As a follow up to the company size comment, the figures for VKTX are current. In their recently released form 10-Q they stated having only 14 employees as a risk. Not sure if this is the industry standard, but managing 5 drug candidates with 14 people seems rather interesting… Do you think this can be a significant factor impacting the success of VKTX candidates?

  20. Alex (AUPH) – I am not optimistic given the mixed p2b results but hope I am wrong…

    Dan (ESPR) – Agree, data look great and the combo should be an attractive option for statin failures. I don’t think this can compete with PCSK9i but these are two distinct patient populations.

    Bruno – Thanks. Both will be oral presentations, see link:


    Erin (VKTX) – It is quite low but looks like they are handling things fine to date.


  21. Hi Ohad,
    Any reason why you’re waiting to increase CHMA position, as you mention that you’re planning to do it if price remains around these levels? I mean, is there a compelling event you’re waiting for before increasing?
    Are you planning to close ABEO position or do you see it as attractive at this level? Market cap is <$80M
    Thanks for all the great analyses and info!

  22. Hey Ohad
    Thanks for the replies!
    Any opinions on TGTX CD20 drug for MS? Roche’s drug has had a great launch and it seems that ph2 results for TGTX are on par with that drug but with shorter infusion times- could be large market opportunity given projections for Roche’s drug are $7B in top annual sales

  23. Alex (ABEO) – Not aware of anything fundamental, probably weakness related to financial position.

    Aviv (CHMA) – The way I see it the company can only have bad news in the next several months (like regulatory or CMC issues) so I prefer to wait and see they have a PDUFA date. A takeout is always an option but a low likelihood one IMO.
    ABEO- Plan to hold my position and wait for the next funding round.

    Dan (TGTX) – I was never a big fan of their approach to target established markets with products that are clearly active but not well differentiated.


  24. Ohad,

    ABEO provided some strategic view this morning but no timeline or funding raise were determined. The uptick volume is huge. Do you think it’s good time to average down or still waiting for the funding raise or partnership announcement? Thanks!

  25. AGLE

    great data, ultra orphan and clear path to approval/high commercial value . EV is 120m. How do you see it?


    Also derisked Stock Trading very low. Any opinion?


  26. Ohad, any opinion on AFMD?

    I agree with previous poster who requested that you put purchase price for your stocks in the table.

  27. Richard, I think it is very easy (using excel) to get the purchase price on the table. I think Ohad is using generic finance portfolio and they have limited capabilities. Using excel just subtract the “Total Chg” from the “Market Value” and divide that with the number of stocks held and you will get the purchase price. Then just copy the equation for the entire table.

  28. Hi Ohad,

    Any thoughts on Kura’s MLL and ERK inhibitor programs? Looks like the market is only valuing Tipifarnib. I believe the ERK class has disappointed in the past, do you think Kura’s intravenous dosing and increased receptor occupancy time could lead to success?

    Thank you

  29. Ohad
    Are you still following IMGN?
    On ESMO Sep 27 – data for Mirvetuximab+CARBOPLATIN+ BEVACIZUMAB.
    On ASH Dec 7 – data for IMGN632 in AML and BPDCN
    Any upside from these data?
    Is it now the right time to jump in or IMGN is lost cause 4ever :(

  30. Hi Ohad
    Do you have any opinion on Zealand Pharma (ZEAL)? Just recently announced that Boehringer Ingelheim will proceed with their GLP-1/glucagon dual agonist into phase II. Also, announced a private placement today, the large shareholder in the company Van Herk Investments B.V invest >80 mio $. Phase III readout for potential best-in-class GLP-2 agonist for short bowel syndrome and another peptide, Dasiglucagon, close to NDA for severe hypoglycemia and in development for several other indications. Do you see some potential here?


  31. CHMA 09/06/19 S-3 Simplified registration form
    What do you think about the potential capital increase?

  32. IMMU-132 side effect profile clearly better than the Ds-1062a. Recommended dose in Trop2ADC (8 mg/kg) saw major occurence of ILD toxicity in NSCLC

  33. Ohad

    Daiichi reported some safety issues in their news release today. Is there a crossover to ZYME which is also selling off.

  34. Hello Ohad,
    Have you read the Reuter brief news on AVEO vs Sorafenib? It says HR of 0.99 with 20 patients progression free vs 2 for Sorafenib in rénal cell carcinoma…looks good to me what do you think taking into account how well tolerated Tivo is!

  35. Ohad
    Any comments on the ADVM data?
    The market reaction is negative. But not all look bad – they had substantial reduction in retina thickness -52 from 370 baseline. Does it mean that it might actually work and longer follow up is needed for vision improvement

  36. Hello Ohad,
    I know you like companies with an “unique selling point”. Can you take a look at Valneva SE? They have the only Lyme vaccine in clinical development.

  37. What is your take on PBYI? The annual sales of Neratinib is expected to 220-240M in 2019 while the market cap is only 450M (2x sales). Plus, what is the chance of approval for Neratinib in the 3rd line Her2+ breast cancer based on the recent Phase III data? How does Neratinib compare to the new ADCs in the field?


  38. Hi Ohad,

    ADVM dropped big-time and is still dropping. Had sold a large part of my holdings (unfortunately not at the $16 peak) but still holding and bought a few more (too early). Will appreciate your comments on the trial results, potential for further data to show better efficacy and valuation. Are you planning to hold / add?Thanks as always for your insight.

    Nice little gain for FCSC bag-holders (like me) who averaged down when it dropped. Any sense in holding on until the deal is closed?

  39. Any opinion on EIGR? They have four BTDs for 4 different regimens. The valuation is very very low <300 M. How do their HDV programs compare others?

  40. hi Ohad, abstract for PRS-060 (Pieris) was released this weekend. All doses including the smallest 2mg dose showing >= 25% FeNO reduction! Presentation of results will be on Oct 1 (late breaking abstract). Basically we are talking inhalable Dupixent…

    GOSS has a much higher market cap with less FeNO reduction with their oral drug…

    PRS-060 is partnered with AZ, and Pieris has co-development/co-commercialisation rights for the US on which they can decide after phase 2a. Anticalins could really play out in various lung diseases. AZ already drew 4 of their 5 options for anticalins, and Pieris also has some own programs. market cap right now 280 million with about 100 mio. cash (no LT-debt) as of mid-year 2019.

    What do you think about the company, still too early for you?

  41. OHAD
    In light of DS-1062’s somewhat disappointing safety data, do you think IMMU-132 has regained the upper hand in the TROP-2 ADC scene? You had mentioned in one of your previous blogs that a milder safety profile will lead to combo opportunities. Surely IMMU-132 has an advantage in this regard. Do you think IMMU is a buy at this time?
    Thanks for all the continued help and insight that your blog provides.

  42. Hello Ohad,

    $CLLS now at 52 wk low. Market cap $ 485 M. Cash was in June $ 425 M.

    There seems to be no confidence in CLLS?


  43. Hello Ohad,

    Any thoughts on adaptimmune’s TCR engineering approach with MAGE A4 with CD8 receptor to target solid tumors? Some decent pre-clinical data, but will the technology overcome TME? Or any safety concerns?

    I would like to start a position here as it seems no one cares about it anymore and seems completely beaten down.


  44. Gene Tx

    Ohad,All of your stocks get sold and are very low right now.

    Chance of the Century ? Or are you more skeptical about Gene therapy in General than you were before? Hmmm…

    Doesnt look too Good right now?

  45. Les (SRPT) – Not for me. I am getting quite nervous about this program given the limited functional data to date and risk of transgene dilution, hope this works out eventually….

    Canela (ABEO) – Still waiting for the smoke to clear, the partial hold isn’t helping…

    Karlos AGLE/UROV – Sorry, don’t know them well.

    Richard Baker (AFMD) – I don’t see a lot of value in targeting NK cells, so far clinical profile with their CD16/CD30 isn’t very attractive. NK based CARs are more interesting IMO.

    DJ Chudasama – Thanks.

    John (KURA) – Thanks. I can definitely understand why the market ascribes limited value to these programs. I personally really like the MLL program, it has a strong biological rationale but it’s a high bar with this novel modality. More skeptic about their IV ERK program and ERK inhibitors in general. Cantor recently put out a deep dive on this target, so far nothing stands out.

    andre (IMGN) – Yes, still following them as I am interested in next gen ADC technologies and beleive mirv works. The triple data look underwhelming, don’t have high hopes for ‘632 because it utilizes a super potent payload that is too toxic IMO.

    Lasse Vedel Jørgensen (ZEAL) – Sorry, not following them, T2D is out of scope for me.

    Jonas (CTMX) – Agree they are becoming cheap but so far I can’t find any differentiation wit their masking technology. Perhaps with their bispecific programs where the approach makes a lot of sense but that’s still far.

    Robert (APVO) – Traded around cash levels but I can’t find a lot to hold on to in their pipeline. CD123 T cell engager doesn’t seem differentiated and the targeted IL10 program is interesting but early.

    Richard Baker (MGNX) – They have a lot of irons in the fire but I can’t find anything truly valuable in the pipeline. Margetuximab’s P3 data don’t look great so far and HER2 will become very competitive and the IO pipeline is not delivering so far.

    Rüdi (CHMA) – Not sure what this means, they already completed a fundraising.

    kelven (ARDX) – Sorry, not following them.

    Provocateur (IMMU) – Yes, IMMU-132 is clearly safer but I still view 1062 as a major threat as ILD may be addressed with dosing regimen optimization and moving to less advanced patients.

    Dave (ZYME) – Not aware of any reason for the selloff as the technologies aren’t related.


  46. Biotech

    How do you see the sector in the Future? Looks like investing in biotech isnt fun anymore?


  47. hi ohad,

    are you going to protect your portfolio with more short in the future? sector doesn’t look good with XBI and IBB breaking important resistances.

    where do you see gene therapy stocks in the future? you still like the technology and market opportunities? how do you see the durability problem?

    Thank you for the work.


  48. Swiss private bio ADC Therapeutics filing for a 150m$ IPO on Nasdaq. do you like their technology and ADCs in program? looks good.

  49. Ohad
    Any thoughts about recent OVID data in rare epilepsy. It look that the drug OV935 is working – constant improvement of seizure frequency from 48% @ w12 to 90% at week 48, and seizure free for 250 days and 150 days (n2).

  50. Hello Ohad! I would appreciate your comments on todays {Sat. Sept. 28th } press release from ZYME . Thanks !

  51. Ohad
    Very impressive SGEN data. Can this brings some live into stagnated ADC field – as an PD-1 combo?
    Any other promicing ADC combos to follow?

  52. Hi Ohad,

    You forgot to comment on ADMV for last run of questions. Could you please comment?


  53. Georges Robitaille (AVEO) – Stopped following the story, seems to me the field has moved on and not sure tivo has room in today’s RCC market.

    andre (ADVM) – Still a head scratcher to me… the signal is there but some of the data are tricky to reconcile. The time without rescue in some of these patients is definitely longer than what you would expect, but there were some troubling data on a flexible definition of progressors and whether patients were deprived of rescue shots…
    not concerned about limited retinal thickness changes as these were “dry” patients but baseline values appear quite high so not sure what to expect.

    Toby (Valneva) – Sorry, don’t know the space well.

    Liang (PBYI) – I would stay away despite the attractive PS given disappointing sales and limited IP protection. New ADCs are probably more relevant in the advanced/metastatic setting and neratinib’s sales are predominantly in adjuvant setting so no direct competition imo.

    Les (ADVM?) – See above, I need to dig deeper but plan to hold for now and wait for the next update on Oct 12.

    Congrats on FCSC ! Not familiar with the details here.

    Liang (EIGR) – Sorry, not following them closely.

    Christian (PIRS) – Clear FeNO reductions but not sure durability and extent of effect are sufficient, plus they will compete with an established Sub Q treatment. Might have a place in the market, still not sure yet…
    Agree about valuation , very low EV. Too bad they focus on IO which has been challenging so far.

    Declan (IMMU) – Despite 1062’s safety issues I still believe it will become the dominant TROP2 ADC eventually with some dose and regimen optimization. Efficacy is so good there is room dose reductions while mainitaning good efficacy.

    Toby (CLLS) – Yes, sentiment is bad, driven perhaps to some extent by next gen allo CAR formats (NK, GD etc.). They also said they will wait a while before releasing more data for CD19 and CD123 programs.

    Adam (ADAP) – I am cautious on TCRs for solid tumors without meaningful technical improvements. So far, results have been disappointing unfortunately.

    Jonas – Yes, not a good period for GTx to say the least… My view hasn’t changed and I still believe GTx will become an important class of drugs, a lot of issues to overcome of course…


  54. Hi Ohad, AVRO has dropped significantly (as has everything else) since your entry. Any plans to add?

  55. Hi RAM – Value your opinion on the market dynamics- where are you on Investment vs cash position in both biotech and tech? I’m unfortunately underwater in biotech – stayed invested thinking situation would reverse or acquisitions would increase and prices would rebound but they are just hitting the floor. Even BIS is not great protection.

  56. Hi Ohad,

    thanks! Regarding PIRS:

    “Too bad they focus on IO which has been challenging so far.”

    Well actually it seems that their own focus is increasigly on lung diseases, and I think that their anticalins have the greatest potential there. Should be totally 7 respiratory programs now they are working on, including the ones for AstraZencea (where they have co-development option for 2 of them)

    meanwhile enterprise value down to some $ 70 million or so….

    some valuations out there already seem pretty absurd

  57. Equalizer – Yes, sentiment doesn’t look so good but hard to predict as a lot depends on the general market (where a correction is also due imo).

    Muhab (IBB/XBI) – I don’t plan to add more short ETFs, comfortable with what I have + cash.
    Yes, I am still a big believer in GTx and I think we reached a point where challenges have manifested themselves (incl durability) but hope they will be overcome with next gen products.

    Muhab (ADC) – I am quite negative on them as they utilize ultra potent PBD payloads that have proven to be too toxic (SGN33A etc.). Valuation they expect (1.5B+) is unrealistic IMO.

    Gerhard (MRNS) – Sorry, stopped following them.

    andre (OVID) – Good preliminary data but hard to interpret these small studies in epilepsy. (see old SAGE and MRNS studies)

    bouschka (ZYME) – They clearly havea signal in tumors not amenable to Herceptin which is encouraging, durability is still an issue IMO but this drug should find a place in biliary cancer at least.

    andre (SGEN) – Yes, very good response rate that needs to be validated in a randomized study . So far ADC combination regimens have been disappointing and hard to implement given overlapping tox. IMGN’s mirv had a disappointing readout at ESMO.

    Les (AVRO) – I want to see more follow up from the P1 (ERT pretreated) to make sure persistence is there.

    Christian (PIRS) – Yes, it does look anticalins have an added value in lung delivery vs. systemic delivery where they have a short half life. Agree valuation is cheap but prefer to wait for now.


  58. Hi Ohad, what are you thoughts on NERV’s seltorexant readouts now that all P2s are complete? Do you believe there is a path in MDD+insomnia? Market reaction has been pretty severe given that at least the Insomnia indication represents a tangible path forward.

  59. Ohad, do you have an opinion on Ymabs?

    they have 2 programs in pivotal trials. valuation now somewhat better due to the correction…. market cap right now about $ 850 mio.

    thanks for your feedback!


  60. Hi Ohad, what’s your opinion on valuation of RETA? They have multiple phase 3 trials on going and will have readouts in 2H2019. If success/fail, up 20% vs down 80%? Some gave them up 80% (Citi) if positive on P3 but does that sound realistic?

    Also, how about PHAS? They have good data on Phase 2 but the stock got crushed because of some articles published in NEJM. Thanks!

  61. Ohad
    SRPT data – did they answer your concern about functional data?
    STML launch – how is going? Initial numbers were good but the stock is dropping since then.
    HRTX offering – is it attractive at that level?

  62. Hi Ohad, have you looked at RIGL?
    They have Tavalisse (SYK inh.) already approved for ITP. Sales are early but growing (10 m in last Q). They also have partnered it with good conditions in other parts of the world.
    I would assume that there is still a need for people failing thrombopoietin receptor agonists like Promacta and Nplate and a new MOA.
    Also DOVA good aquried recently for their 3d. to market TRA. Any thoughts?

  63. Hey Had,

    Results for CAPR Hope-2 trail in DMD have just been presented (6 months results) and they look highly encouraging.

    The market cap is $10 million and an end of phase2 typeB meeting with FDA is scheduled for later this year; some people are speculating there is a chance for early approval just on this ph2 result given how underserved this population is (teens and young adults, as opposed to infants and small children with most GTx) and also given positive results in various physical assessments and measurable (confirmed by independent party).

    Any opinions? The stock tanked this morning but is recovering. Shorts are betting the company will have to raise money and, given the low marks cap, that dilution is going to very painful. However, the upcoming FDA meeting is could be a transformational catalyst.

    Any opinions on the results they just highlighted in their PR?


  64. Ric, the lack of any institutional ownership in CAPR tells you all you need to know about that one.

  65. Paul,

    I disagree. It´’s definitely a very low float and market cap stock, but that doe snot mean that their technology or science has no merits…

  66. Paul, also RMAT designation for CAPR’s infusions, so obviously the FDA thinks there is some merit to it

  67. Ohad
    interesting market reaction – I thought RNGX 314 had very good data. Improved visual acuity (+4 letters) and decreased retinal thickness (–68µm), but the stock is down a lot.
    ADVM couldn’t demonstrate any improvement but the stock is up.
    What is your take on the data?

  68. Xbi nbi

    How far do you think the indizes will go down further? You still confortable with your longs these days? Protect Portfolio more ?

  69. Hey Ohad

    Any opinions on the RLMD ph2 results, with stat significant decreases in MADRS compared to placebo

    This is the oral version of Sparvato.


  70. Christian,
    Just saw this note on EXEL. Looks like generics are coming along. Long way off but EXEL needs to come up with something else.
    Guggenheim analyst Michael Schmidt lowered his price target on Exelixis shares to $25 from $33 after the company disclosed its receipt of a Paragraph IV certification notice letter from MSN Pharmaceuticals regarding an ANDA for a generic version of Cabometyx. Based on his talks with a patent attorney, Schmidt thinks a mid-2026 launch of a generic version of Cabometyx represents “an unlikely bear case,” with loss of exclusivity in the window of 2027-2030 seen by him as a more realistic base case. Schmidt keeps a Buy rating on Exelixis shares.

    Read more at:

  71. Jonathan, the only silver lining (if one can call it that : ( we continue our research,make investing choices and hopefully Ohad’s opinions align with our decisions. best to you – C.B.

  72. Ohad/Andre – hope you are both doing well. Did you see the update on ARVN today in targeted protein degradation with their PROTAC tech? Appears to be safe with no grade 2/3/4 AEs, and PK shows good dose response. No efficacy data until 2020 though. The stock is up 15-20% at the time I’m writing this. Have you been considering getting into this space early, like you did with gene therapy?

  73. Hammertime should charge us some money for access here if that’s what it takes to get more of his time..as always..thanks for all you do Ohad.

  74. Manish (NERV) – Recent news were negative for MIN202 for depression but I actually became more optimistic about this drug as an insomnia treatment. 101 is still a high risk program approaching P3 readout. I plan to wait to P3 readout before adding more based on 202 for insomnia.

    Christian (Ymabs) – Don’t know them well but looked at their pipeline and didn’t find anything compelling so far. GD2 has been around for years as a target for naked antibodies with more advanced programs in development and limited market potential, the radio-conjugates are IgG based which is not the right format IMO, lastly, CD3 engagers for solid tumors have a narrow therapeutic window.

    Canela (RETA) – Sorry, don’t know them well, the MOA and data have always looked a little iffy to me but looks like the market really likes them and FA results were a nice surprise.

    PHAS – Not familiar with them either.


    SRPT data – if you are referring to the LGMD2 results, I think they were directionally positive but limited in terms of sample size and lack of a control arm.
    STML – Agree, launch looks fine but overall potential is still limited.
    HRTX – I am watching them closely as I think they have a differentiated product vs. PCRX. Waiting for a better entry point next year.

    Christian (NERV) – See above. I plan to hold into P3 readout for 101 despite the inherent high risk and long term feel more comfortable about 202 for insomnia where results are quite strong imo.

    kmastra (RIGL) – Haven’t been following them for a while. They definitely deserve credit for salvaging the drug after the RA failures by finding a niche indication. I was under the impression their data weren’t strong but with a 300M market cap, worth another look.

    Ric (CAPR) – I am not a fan of their approach and don’t find data convincing.

    Bouschka (ADVM/RGNX) – So I have been following both programs closely and am still trying to wrap my head around the data. Both data sets have strengths and weaknesses in terms of rescue shots, retinal thickness and protein levels. I don’t think there is a clear winner and data sets are still quite small.

    andre (RGNX/ADVM) – Yes, hard to explain market reaction as there is definitely a signal of efficacy. I am still trying to understand why clinical efficacy is stronger given the protein levels they achieve. I guess a lot depends on patient population and baseline treatment experience.

    Michael Goodman (MTEM) – High risk pipeline with risk of immunogenicity and a lot of competition for all their programs.

    Sako (XBI/NBI) – Yes I still think a significant correction is warranted but am comfortable with selectively owning small and mid cap biotechs in conjuction with a large cash position and BIS as ~10% of the portfolio.

    RLMD – Still didn’t have a chance to see the data. Will check

    Christian (EXEL) – Still following them but not sure they have a significant upside at the current valuation. Next major catalysts are PD1 combination data in renal and bladder but market is becoming competitive.

    Jonathan – Thanks. Indeed, including a busy holiday season here in Israel :)
    Started working on a new post, will try to post within 2 weeks.

    Nick (ARVN) – Update is quite positive IMO demonstrating PROTACS are safe at exposures approaching therapeutic levels. No efficacy readout but this is a major landmark for the field. I prefer to still wait on the sidelines.

    Paul – Thanks, no plans to charge for access, I have enough people I need to report to in return for a salary… 😉


  75. Ohad
    While checking RLMD, can you compare them to AXSM, both with NMDA receptor antagonist for MDD. Also SAGE 217 targets MDD, different MOA though.
    Would be very helpful if you comment on the MDD field.

  76. Ohad,

    To be clear, it is so much more than your opinion I (we) value. Your analysis and investment decisions are unmatched anywhere. True dat. Thank you.

  77. Alex (XENE) – Next year they should have P2 efficacy data for both epilepsy programs. I think both have a a high likelihood of success, it is encouraging they haven’t announced any amendments/termination due to safety so far.

    andre (RLMD/AXSM/SAGE) – Personally, I find NMDA inhibitors for MDD challenging to assess as the mechanistic link is still unclear and it is still hard to elucidate the controbution of NMDA to esketamine’s actvity (other NMDA drugs should have worked but they actually don’t which tells us there is something we don’t understand).

    Bouschka (MRUS) – Interesting twist with NRG1 fusions, efficacy isn’t stellar so far but signal is there. Still on the sidelines.

    Alex (STML) – This is more a commercial story.

    conrad – Thanks!

    Richard Baker (KURA) – Looks like their allele frequency hypothesis holds up, which is very good. The data set includes 10 patients, they have 3 or 4 responders + anti tumor activity is several more so this looks like an approvable drug. Population is smaller than originally anticipated, not sure how big it is right now.


  78. Ohad, do you have an opinion on DRNA? Received $200 million upfront today from Roche for an HCB drug in Phase I. Now has over $500 million in cash and a slew of partnerships.

  79. hi Ohad!

    you were very critical towards XEN496 for Xenon, targeting pediatric epilepsy KCNQ2-EE

    are you still negative on this program – or isn’t it a smart move with limited risk..?

    you see the main value in 1101 and afterwards 901, correct?

    thanks for all your work

  80. Hi Ohad,

    Any comments on Innate Pharma? $IPHA or $IPH in France market?

    Seem to have a lot of cash, big collaboration with AstraZeneca, and a future Anti-Siglec 9 program which now becomes interesting.


  81. Ohad
    How you view NK companies FATE, AFMD. What you would be looking for before buying anyone of them, if at all.

    What about BLCM – will be presenting some early data on CAR-NK. The stock is up 50% since the announcement of the presentation of preclinical data?!?
    If CAR-NK is going to be a game changer, is it a good idea to buy into the stock now or wait for Ph1 data.

  82. Hi Ohad / All (realizing Ohad is likely unable to comment),

    Anyone have any commentary on this ARQL SNSS sell off post abstract release supposedly tied to the loxo data? the public markets are bizarre sometimes.

    Saw this on twitter and wanted to stress test with the comments crew…ARQL IC50 to BTK is 0.85nm and SNSS is 3nm. So Vecabrutinib at 300mg or 400mg should match ARQL at 65mg, correct? Veca at 100mg and 50mg on C481S, comparable to ARQL-531 at 15mg and 20mg. By this math it seems hard to compare SNSS to ARQL until we see the 300-400mg data at ASH.

    Seems like a buying opportunity for SNSS and ARQL. ARQL has a decent head start and nothing has really changed for SNSS with the 300mg data still not out. Sure lilly is a sizable player but their data is early.

    Assuming ARQL gets to market first, with SNSS and Loxo as fast followers, Lilly definitely has the commercial edge (vs. SNSS unless they get bought or partner) but the data seems too early to count out SNSS….anyone have a market size estimate for non-covalant btks?

    sorry for rambling but any feedback would be helpful.


  83. Arql has sicker patients, more mature data than loxo data. Appears to be a good buying opportunity especially if it dips below $7.00 with ASH coming with more detail on Arql 531. Plan on putting a buy limit order Friday.

  84. ARQL has good data so far and a lot will depend on their own dataset imho. If they show good durability (no loss of PRs) and even better response rates on CLL C481S mutated patients and also RT (not sure if LIly has any) i think that market will see a clear path forward for this compound. Perhaps at ASH there could be an update on FDA discussion and i would expect that there could be a fast path to market for C481S CLL and also RT (if data at ASH is good).
    But one has to consider the Loxo compound as a real competitor. But data is early and also here a lot will depend on their updated dataset (will PR-L translate to PR?) at ASH. So far it is hard to compare both datasets but i find it a bit irritating that Lily used PR-L and especially that they used 87% tumor responses and not simply PR.
    Not so sure about implications on SNSS but to me they still have to show any efficacy and with ARQL being further ahead and Lily having much better financial possibilities it could be hard for them to find a path forward.

  85. kmastra…those were the exact concerns that arql dropped though not really worried about snss and Loxo data is way too early and confusing PR-L that may or may not reach PR. But by the time there is more clarity, arql may be much higher though. Unless I am missing something, it seems like a good r/r.

  86. Ohad
    CNST – what do you think of their epigenetic inhibitors. Their BET + JAK has very nice data for ASH. They plan to start a big registration Ph 3 in 2020, First line / second-line transfusion-dependent myelofibrosis could be a big market opportunity, considering INCY revenue.

  87. Hello Ohad,
    I just re-entered a small position in OVID, and I went back through this Open Forum to January the of 2018- where you mention that you were awaiting a better entry point. At that date- OVID WAS VALUED AT ABOUT $234M. Today, it is valued at about $122M.
    Ovid seems to be preparing for a very busy 2020 with multiple read outs. Do you still consider them to be investment worthy? Thanks again for your efforts with this blog. This has got be be Martian sent!

  88. Dear Ohad,

    Any opinions on SNSS now that they cleared 300mg dove without adverse events ? Maybe the AE at the first does was just a glitch?

  89. snss has horrible management, toxic financing, and their drug appears to be inferior to Arqule. Arqule’s shares dropped in part because of arql abstract release and potential competition from loxo’s drug….though we will find out if this drop was justified or not. However, after followings ohad blog for years, I would imagine that he would still consider Arqule overvalued and high risk (but he obviously can’t fit Arqule). I am a buyer near $7 now.

  90. I don’t think ARQL is to expensive for Ohad – in fact their company is owning quite a lot if that has not changed yet, and purchases also around 10 dollars if I remember correctly

  91. Hi Ohad
    Any opinion on SWTX GSI technology.
    Ph1 and. 2 results look nice – very high PR+SD rate. The targeted population is small but the drug looks approveable – BTD, fast track, already started registration Ph3. Discontinuation is low – just 1 / 17 due to AE.
    The combo w/ BCMA ADC targets much larger population but they have only pre- clinical data for that.

  92. Christian….I have been waiting for arql to get close to $7 to buy and then today happens where its up more than 10%. The stocks that I was interested in buying have unfortunately all gone up this week…eidx, atnx, and arql. Kinda frustrating.

  93. Sam I know how this feels. Just on Monday I wanted to place an order for KRTX when it opened some 70% or so higher. I hoped it would come back a bit, so I waited… It went up about 450% on this day!

    grrrrr headbang grrrrr

  94. Ohad, ZYME data ZW25 at ESMO Asia – what is your take on it?

    looking forward to first ZW49 data!


  95. Hi any thoughts on Agios’s recent IDH1 inhibitor data in Cholangiocarcinoma and Glioma? Noticed the updated response rate for glioma was 31% and 90% disease control

  96. Ohad, do you think any companies are interesting in the allogenic CAR-T space? Allo, fate, Atra, Cyad, clls?

  97. Hi Ohad,

    Do you have an opinion on Neoleukin (NLTX) a de novo IL2/IL15 agonist?

    Also, Milestone Pharma (MIST) working on a nasal calcium channel blocker for SVT/AFIB


  98. Hi Ohad,

    Could you talk about ICPT and NGM in regards to Nash, and how they compare to VKTX and MDGL.


  99. Good day for RNAi field -ALNY, ARWR, SLN, DRNA…
    I have them on my “watch list” since I guess forever.
    Except for a small position in ALNY, I never found a good entry.

    Hope GT will follow sooner or later. A solid acquisition will change the sentiment.
    I bet on QURE and FOLD, which might mean that – they may never be acquired.

  100. Helllo Ohad,

    Heidelberg Pharma AG has an ADC technology with an ultra-potent toxin. The toxin (Amanitin) has a unique biological mode of action.

    Can you take a look?

  101. Dear Ohad,

    Hope you are having a good end of the year.

    Any opinions of AXSM and their 3-4 readouts in the coming weeks?

    Interesting to note that KRTX, which rose 400% on positive Ph2 in Schizophrenia is combining two drugs: a muscarinic receptor agonist called xanomeline and trospium, an approved muscarinic receptor antagonist–this strategy seems to closely follow what AXSM is doing: combining two drugs to achieve (hopefully for AXSM in coming readouts) powerful effects in CNS.
    Any opinion on KRTX and AXSM?
    Last time you chimed in you were not too keen on technology — combining existing drugs.

    And what about CCXI results?



  102. Hi Ohad
    do you think INCY could be a candidate for a purchase in view of their recent updates? or they too expensive?

  103. Hammertime, I’d love to get your take on $CYCN (recent spinoff from ironwood) following this ‘nutty’ selloff…lol!. Recent insider buying in open mkt($227k at $1.82) and the $IRWD founder chose to join the smaller co and trading well below cash. They own SGC stimulator drug that the president developed and I noticed Merck paid $1 billion for similar drug.

    It’s a pipeline in a drug and they plan to out-license the diabetic nephropathy portion of pipeline (failed due to irregularities at one study site where patients had either no drug or too low drug in their plasma) and proceed with the sickle cell indication as well as vascular dementia.

  104. Many biotech companies, even very speculative ones, have substantially gone up that I wouldnt be surprised if Ohad’s said that the company in his next post became over valued and to wait for another buying opportunity. However, it would still be an interesting read and he posts soon but tends to post them on Sundays.

  105. Ohad
    Nice deal for XENE. It established them as a name in epilepsy
    But was it smart strategically to give their best asset – Na channel?

  106. Hey Andre,

    I agree on XENE question, especially $50M upfront is not much… market has not reacted too well either. This, in my opinion, raises question about what management is thinking… Especially considering they are keeping program they inlicensed, one of which is off patent, but giving away the internally developed 901.

    Makes you wonder….

  107. For me it was always 1101 that seemed the most important program…. they fully own it. 496 is more a side-show….. I remember Ohad didn’t like that deal.

  108. but 901 after 1101 though riskier. so, I think deal is “ok”. gives them longer cash runway and they can focus on 1101 (and 496..)

  109. Astellas Pharma to buy Audentes Therapeutics (BOLD) for $60 per share
    congratulations to all and thank you Ohad

  110. Hi Ohad, congratulations again for BOLD and thanks. RGNX jumped in response and QURE too. Maybe time to buy more RGNX at the current low price.

    CLVS has had a great run… congrats to all those (including me 😊) who cost averaged and loaded up between $3-6. Looking for it to run back up to $30 from
    It’s current $15 price.

    MDCO always looked great to me and it’s acquisition was great news. Hope ESPR follows suit. Congrats on KRYS as well and hoping ABEO sees better days ahead.

    Would love to hear your opinion. Thanks again!

    Curious, what they have in common …
    Probably nothing, just coincidence

    Ric & Christian
    I think the XENE mgmt massage is clear – get 1101 approved and sell the company. All r&d activity will be limited to support and will be paid by NBIX for 3 years.

  112. Thank you for all Ohad!! Was just re-reading your comments on ALNY and siRNA last January.

  113. 1. Awhile ago you mentioned CARA. Data released today. Thoughts about it and your general thesis?

    2. VKTX/MDGL – once the hottest, now dead. Was the NASH hype just hype? There are investors that dumped hundreds of millions during the high of hype and are down -75% in 1 year on 9 figures

    3. RLMD/AXSM – similar to my question #2 above, is MDD just the hype of 2019? Will 2020 for MDD become the NASH of 2019?

    4. APLS thoughts?

  114. Hey Ohad, Would love to know your thoughts about TBIO’s mrna drug for CF?

    Also, do you have an opinion on Hookipa and their arenavirus platform?

  115. Ohad – How much interest do you think big Pharma would have in ADVM/RGNX Wet AMD assets? I would imagine a successful gene therapy for that disease would be a massive drug.

    Also, do you like PTCT/FOLD/RARE for gene therapy exposure?


  116. Hi Ohad,

    I’d like to get your opinion on CRSP, you mentioned previously their programs are too early. Now some preliminary data on SCD etc rare blood diseases have showed very promising results. Is it time to have some exposure in gene editing technology? Thanks so much!

  117. Dear Ohad,

    good raise this morning by RLMD @ $30 per share, 3.3M shares
    Any opinions on their ph2 results?

  118. Ohad
    AUPH delivered outstanding results.
    Most importantly – mortality was one in the voclosporin arm vs five in the control.
    So the ph 2 imbalance was a fluke !?!
    Considering 10B market opportunity, what about jumping back into AUPH.

  119. Andre,

    where do you have the 10 billion market opportunity from?

    After all the company did a good job of explaining the death imbalances. Their explanation sounded good to me, and together with the fact that there were no further deaths for the drug arms in the second part of P2 study and that deaths were anyway lower in the higher dose group convinced me to sit this out with (unfortuantely) a bit reduced exposure.

    Safety profile looks really clean in the P3 study. Percentage with CR is a bit lower than the low-dose (= p3 dose) in P2, but still very ok imo:

    – CR placebo 24%, low dose (= P3 dose) 49%, high dose 40%
    – CR placebo 22,5%, low dose 40,8%

    Ohad and others, what could this company be worth?? More than yesterday for sure, but would be nice to hear your inputs

  120. Christian,
    Looking into my old notes from 2017.
    200K pts x $50k -> potential $10B/ year peak sales
    Not sure how accurate it is since I didn’t follow auph in the last 2 years.
    About what the co is worth – I would say ~2B today, considering 70% coverage, 70% probability for approval and 10% per year discount.

  121. $CYCN (Ironwood spinoff) CEO mentioned at conference on monday they have developed the only SGC stimulator to cross the blood brain barrier(Bayer has adempas for PAH). Phase 1 proof of concept study out 1st week of Jan 2020 at medical conference).

  122. Hey Ohad!

    Yeah SAGE failure not good and makes me nervous about AXSM Imminent read out- difference is that AXSM has not outsourced trial, which might translate in better control. But yeah, SAGE failure a reminder of how hard an indication depression is, and that ph2 results are no guarantee of successful ph3.

    What kind of molecule is sage-217 anyway- seen people say that there is actually very little innovative about it, just a modified benzo-mimetic with high risks of addiction. Any views on this?


  123. hi Dan,

    I think that the MDD readout should not be thaaat difficult since it compares to Plaecebo (GEMINI-trial).

    STRIDE-trial next year seems to be a much higher hurdle, but that’s sometimes next year so I don’t care 😉


  124. Christian,

    I actually think that placebo response will be high (similar to BUP response)- I also like odds of Stride TRD ph3 trial better than Gemini 3 for MDD as it is two stage phase3 trial where in stage 2 they randomise inadequate responders – closely following the sequential parallax comparison design which generally eliminates placebo (and in this case BUP) responders.

  125. Dan, thanks for your valuable insights. do you really think placebo effect can be as strong as BUP would do, and still then AXS-05 despite small n showed very good improvement compared to BUP arm in P2? Do you know which delta in MADRS between placebo and AXS-05 would allow to call it a success?

  126. Ohad, I just noticed that you became a partner at Pontifax meanwhile. My greatest appreciation! I am very happy for you! Thanks a lot for all your valuable insights, I am well aware of how time consuming it is for you, which makes your commitment even more special!

  127. Hey Christian,

    There is a likelihood of high placebo response in Gemini 3 because patient enrolled in the trial are aware of the success of the ASCEND phase 2, and this will boost their expectations and placebo effect. This is the nature of the challenge in CNS drug development: high placebo response. I expect (or fear) that the placebo effect for Gemini 3 might be in line with Sage Mountain trial, namely a 10 point decrease on MADRS–which is what the BUP arm achieved in ASCEND. Management has made clear that they have recruited patients with a minimum MADRS score of 25 and have been diligent about double-checking who is admitted, including identifying nd excluding professional trial patients (which supposedly is quite a problem in the USA). In ASCEND, AXSM-05 achieved a 17 point reduction -7 delta compared to BUP… Crossing my fingers we can still get close to that delta for Gemini 3.

  128. I suppose the development strategy for AXSM-05 in MDD seems to have been executed as best as possible:
    1. phase 2 comparing 05 to active drug
    2. phase 3 comparing 05 to placebo (and reminding everyone: you will get placebo or the active drug, unlike in Ascend! yes, you may be given placebo which has no effect! –which may help manage expectations and placebo effect to some degree?)


  129. hi Dan,

    thanks a lot for your comments!

    yes it is also my impression that they are designing really smart trials. I found the crossover study for AXS-12 also very interesting for example. Gives you effectively an n of 40 with 20 (ok 21) people and increases the reliability of the results because of the crossover.

    The high placebo response in depression is certainly always a high risk for sure. I am still hoping it will come in at least as positive as the P2 with active comparator, and it is good to know that either a positive Gemini or a positive Stride study will be enough for the NDA in MDD.

    Let’s keep fingers crossed for this week… It will go down a lot in case of Gemini failure, though it is good that there is more promising stuff in the pipe with AXS-07 and AXS-12 and other indications like AD for AXS-05 which should put a floor somewhere…. (where? haha)

  130. hi again,

    found this quite interesting:
    “Sinyor et al (14) reported that, if there was no placebo control in an antidepressant trial comparing two antidepressants, the magnitude of symptom reduction was 65.7%. If the trial included two antidepressant treatments and one placebo arm (33% placebo exposure risk), the magnitude of symptom reduction with the antidepressants was 57.7%, while that with placebo was 44.6%. If the antidepressant trial included one antidepressant arm and one placebo arm (50% placebo exposure risk), the magnitude of symptom reduction with antidepressant was 51.7% and that with placebo was 34.3%.
    In short, the apparent therapeutic effect of antidepressants is related to the risk of exposure to placebo, when this is known to clinicians and depressed patients from the consent form. If you lower the risk of exposure to placebo, then the apparent therapeutic effect with the antidepressants and placebo is greater.”
    The comments made by Brown (15) regarding the experience of patients assigned to placebo are pertinent. He states: “The capsule they receive is pharmacologically inert, but hardly inert with respect to its symbolic value and its power as a conditioned stimulus. In addition, placebo-treated patients receive all the components of the treatment situation common to any treatment, i.e., a thorough evaluation; an explanation for distress; an expert healer: a plausible treatment; a healer’s commitment, enthusiasm, and positive regard; an opportunity to verbalize their distress”.
    Indeed, Frank has argued that these elements of the treatment situation are the active ingredients of all the psychotherapies (16). Since antidepressant clinical trials involve extensive evaluations, long visits, many experts and “new and exotic treatments”, it is not surprising that, under such conditions, the differences between active treatments and inactive treatments including sham acupuncture and placebo are, at best, small”
    This finding translates into the design of a prospective antidepressant trial as follows. This trial should compare a known effective dose of the test antidepressant to placebo (50% placebo risk), a two treatment option. Each treatment arm should consist of a minimum of 120 depressed patients and should be implemented at a maximum of twelve investigative sites.
    The effect size of current antidepressant trials that include patients with major depressive episode is approximately 0.30 (modest), and this fact needs to be heeded for future antidepressant trials.
    (source is PMC4592645 from ncbi.nlm.nih.gov)

    Regarding Gemini, hurdle for placebo as comparator in the P3 is at least lower than it were with BUP (instead of placebo in the same study), and their study is designed in accordance with the above recommendations. So, fingers crossed!

  131. MRK buys ARQL $20 a share
    APTO and SNSS only two small cos with reversible BTKi
    Interesting to see what j&J and ABBvie will do now…

  132. Missed it…but that’s ok. I started investing in biotech because of very sick family members and tried to do research on what medicine approved or yet to be approved would help them. Obviously the perfect investment would be one you make money, contribute to its research, and a cure for an illness….but that would be almost s miracle in my case…lol. Anyways congratulations to anyone who invested in it to made money and that this money brings themselves and others happiness.

  133. Ohad, BIG CONGRATUATIONS to you and your colleagues! This will for sure be a big payday for you.

    And Ohad, thank you very much! Without your knowledge on this name I would not be in it today.

    Thank you!


  134. Yes Ohad!
    well done on ARQL–and a huge thanks for your invaluable insight into biotech!

  135. ARQL data looks excellent—wondering if MRK is getting too good a deal? Also considering 531 is not the only drug…

  136. Hi Dan,

    What’s your take on APTO? SNSS had another failure… any thoughts on their future? It went up as well on ARQL buyout as did APTO

    Hi Ohad, would love your thoughts on APTO as well.

  137. I have been an investor in APTO for over a year now. They are finally getting clinical data – albeit very slowly – they seem over cautious. The next two-three months will be crucial to see if they an maintain a clean safer profile and comical response.

  138. Dan,

    APTO is a Canadian biotech (enough said) with limited financial resources (dilution coming soon). Plus, they are VERY early-stage What is so great about them in your estimation?

  139. Comical response :) I meant clinical

    Hey Richard
    They have two unique molecules and have been very diligent in their development. The two molecules, if successful, could address large markets. 806 is a highly diversified reversible BTK and FLT3 inhibitor that supposedly also inhibits many cancer rescue pathways. So far (very early stages) it has a clean safety profile and has shown signs of clinical activity – inflection point is coming as more patients take the drug and efficacy and durability will be in focus.
    Risky because it is early stages – but market cap under $200M and considering ARQL story has a lot of upside if the molecule turns out effective and reasonably safe.

  140. Ohad, any chance for a competitive bid in ARQL? data looked great.. and there is also Miransertib

  141. Yeah, RE ARQL price rose above MRK offering today…
    I am going to hold on to the shares. The strange thing is they announced buyout before releasing data and allowing market to decide fair value, which I think was poor strategic decision by management…

  142. Interesting to see $ARQL bouncing today over the $20 MRK offer.
    Is there any chance of a second bidder coming over for a bid war?
    Hard to see it happen especially with the all cash offer, but maybe BMY want to stay in the run!? Thoughts?

  143. Options are not pricing any other bidder for Arqule. In fact, huge call and put selling. I may buy itm options if they come cheap enough. However, I would rather hold other companies with much more potential with ground breaking science and exceptional management…getting much harder nowadays. Still like reading his articles because they are also like little stories of possible miracles in the making (at least that is how I feel).

  144. Ohad,
    Happy Holidays.
    2 Questions:
    What’s your current sense about MTEM? Last time I asked you were interested but waiting to see more. They’ve traded well since but that doesn’t necessarily reflect on the Science.
    From a strictly trading perspective, what current positions to you feel are still undervalued and should be added to. Of course, if you have new positions that you’re considering adding, that is always appreciated and would be a great gift for the holidays…..Cheers

  145. Hey Christian!

    Yeah, I think it looks good!

    Maybe only small issue is the 6% discontinuation, but otherwise looks like an amazing result also considering so few drugs have gotten approved in the indication in decades, and this is a new MOA. Likelihood of doing quite well commercial, also considering the SAGE setback.

    Result als bodes well for next readings (Migraine and TRD), management is executing at a high level.

  146. Folks,
    Thanks to one of you who first highlighted AXSM on this blog. What a run it’s had and 2 or 3 more results yet to come. Certainly made up for SAGE and WVE losses. Thanks guys.

    Ohad- would love to hear your impressions on AXSM results. Are you a believer now?

  147. I believe it wasn’t right for Arqule management to sell just days before positive results were released….Arqule would of had a much brighter future as an independent company. It seems like it is going to be a difficult to invest in this biotech tape.

  148. Hey Richard,

    what do you say now about limited financial resources for APTO? they just raised $60M+ and stock is up nonetheless.


  149. Re: APTO

    That’s a good sign, Dan, no doubt. I may have to dip my toes in the water on this one, although I just hate Canadian biotechs. LOL

  150. Hi, Ohad.
    Do you think MGTA (magenta) is too early to consider, preclinical but very intriguing pipelines?

    Again I asked you if you would consider to start biotech fund a couple years ago. I’d like to be the first to vest.

  151. Hi Richard
    Not all Canadian bios are bad. ZYME and AUPH are two examples of something good coming from Canada.
    APTO may join them, if higher doses confirm the preclinical data.

  152. Hi everybody – Really sorry for disappearing for the last couple of months. Things have been quite hectic here (not just ARQL) so I barely had time to spend on the public side. I think I’ll have to change the format in order to keep this site active so going forward I’ll try to post shorter, more concise posts. I’ll start replying to questions in portions.

    Alex (STML) – I plan on holding it, it’s a niche profuct that might fit a hem player, so far launch is going well, IMGN’s CD123 program is a comp threat to watch.

    Richard Baker (PRVB) – Don’t know them well.

    Jens (ZYME) – I though they looked pretty good, obviously hard to interpret single arm combination studies. In contrast to other HER2 programs, ZW25 has a very benign safety profile which makes it more amenable for combination regimens. Should be better than Herceptin IMO.
    Regarding the link, ZYME’s ADC platform is also my favorite one among publicly traded companies. I hope they go to site specific conjugation in the future.

    Richard Baker (DRNA) – Very impressive! Missed the siRNA train unfortunately as valuations are already high but the siRNA basket (ALNY, ARWR, DRNA) have made a huge comeback.

    Christian (XENE) – Yes, I always prefer innovative products with a differentiated clinical profile. I like both 901 and 1101 as de-risked high impact compounds.

    John (IPHA) – Haven’t been following them for a while but so far all attempts to activate innate cells failed. Agree about Siglecs becoming interesting as a target class but so far the ones with clinical validation are Siglec 8 (ALLK) and 15 (NXTC).

    Alex (ESPR) – No idea. Looks like the market remains uninterested, need to wait for launch and CVOT data.

    andre (FATE/AFMD) – NK field is still a “show me” story to me. So far, clinical validation is limited. There are some interesting NK-CAR platforms for allo products but the most interesting stuff I know is actually private with some cool next gen technologies.

    Karlo (STML) – So far launch is going well, I see they had data at ASH in MF with some efficacy signals but I am not sure it was good enough, especially for a chronic disease like MF.

    Richardb (ANAB) – Very disappointing outcome after the initial P2 readout in AD, chart is painful to watch. Not sure there is any value left here.

    biocqr (NXTC) – This is a real head scratcher for me… Of all IO agents, this is the only one that generated promising monotherapy data but would like to see more responses given the problematic nature of the first PR.

    andre (CNST) – I was really intrigued by their ASH abstract headline, really caught me by surprise as I ascribed limited value to BET inhibitors. Didn’t delve into the story but the data have enough red flags typical to these single arm combo studies in hem diseases like MF and MDS. See below some very important critical analyses that made me skeptical:



  153. Lawrence (OVID) – Yes, I liked the OVID story but since they put out data that were inconclusive in Angelman. Another example of challenges with CNS programs and GABA biology in particular (see SAGE recently). Still onthe sidelines here.

    andre (SWTX) – I think valuation is too high, GSIs are an interesting twist on targeting Notch biology in cancer but I prefer to wait . Combination with an ADC in myeloma does not look viable to me, there are better options.

    chrissy – Will try to post more regularly with shorter posts next year.

    Les (MDCO) – Same here. MDCO deal is super impressive, not sure I would pay that but definitely could disrupt the sleepy PCSK9 market. So far people seeem to count ESPR out but I still think it’s a potential blockbuster and the only oral drug with an effect on LDL and CRP. We’ll see…

    Kay – Nothing comes to mind. There have been so many IPOs …

    Christian (ZYME) – See above, I like it but the real asset in the pipeline is ZW49 imo.

    Dan G (AGIO) – Overall I would say results are positive but not stellar. They can’t file for cholangiocarcinoma though…

    Chriss (MDCO/ESPR) – It means nobody is intersted in buying ESPR before seeing launchor CVOT data IMO.

    Jake (BPMC/DCPH) – Both agents look really good, not sure who the winner is but the combined valuations are quite high…

    Tim – I think the real intersting allo companies are still private, but prefer to not discuss….


  154. Joe M (NLTX) – Looks like another engineered CD25-sparing IL2 (with apparently some IL15 activity as well). Similar story to THOR, looks like they are still preclinical so it’s too early for me.
    Not following MIST closely.

    Robert (ICPT/NGM/VKTX/MDGL) – So ICPT has a drug that works in NASH but magnitude of effect is modest plus there are safety liabilities (pruritis, LDL) that may prevent wide adoption IMO. NGM has an active molecule based on their P2 studies but effect isn’t as stellar as originally reported and their frequent dosing regimen makes it a non-viable product IMO. VKTX and MDGL work on the same target and are effective, safe and oral but clinical profile needs to be proven in larger studies.

    Ronald Abrams (CALA) – So far clinical data are underwhelming. It appears that cancer metabolism or cancer immunometabolism are challenging fields…

    Toby – They should start P1 next year, I think. Their payload is interesting but require clinical de-risking given past issues with amanitin and related compounds.

    Ric – So I clearly bet on the wrong psychiatric program… AXSM and KRTX both performed very well but I didn’t delve into the data so no concrete opinion there. I still always prefer novel drugs that can be protected from generic competition, especially in non-orphan indications where regulatory exclusicity is limited. Don’t know how strong their patents are.

    CCXI – Haven’t been following the story closely.

    Alex (INCY) – I think they became too big without a clear growth driver after epacadostat failed. Jakafi is very lucrative but as asmall molecule will come off patent at some point so not a lot of long term growth.

    Alex (CYCN) – Sorry, don’t know them well.

    andre (XENE) – I didn’t like the deal and preferred them to keep all rights to 901 and raise more money via equity. Upfront wasn’t huge (50M), royalties of 10-15% are not bad but also not great, milestones look big but very back end loaded. Hope they keep the KV7 programs…

    paul/jossi/RAM (BOLD) – Thanks! A good deal for BOLD at a nice premium.

    Les (RGNX) – Thanks. RGNX seems to be dependent on its AMD program, so far data look good but somewhat ambiguous. I plan on holding it as an AAV ETF.

    Kirk Hartley (ALNY) – Yes, nice to see how the siRNA story evolved from being toxic 5-6 years ago to being super hot today.

    Kay (CARA) – Data were disappointing, it makes it less attractive as the potential of the IV drug is significant but still limited.
    VKTX/MDGL – I plan on holding the stocks, I think the drugs work.
    RLMD/AXSM – Not sure…
    APLS – I didn’t find data to be very convincing.

    Max (TBIO) – Historically CF has been a very tough nut to crack with oligos due to delivery into lung epithelium and so far data are hard interpret so not sure they solved that problem.

    Hookipa – sorry don’t know them well.

    Josh (ADVM/RGNX) – Agree about opportunity but I don’t see massive uptake for a subretinal injection unless duration and success rate is out of this world. ADVM use intravitreal but data re not clear cut so far.

    PTCT/FOLD/RARE – Yes I like the GTx exposure but not the valuations 😉

    Jinyu (CRSP) – Yes, there is clear poc for ex vivo gene editing programs (also DTIL) but I prefer to wait to see long term effects

    Dan (RLMD) – Sorry, not following closely.

    druz (EXEL) – I think they need something beyond cabo to move higher. Liver is becoming more crowded after atezo+avastin P3 data.

    andre (AUPH) – Yes well done. My concerns have been proven wrong as I thought the drug had safety liabilities. Not sure it’s a 10B opportunity but clearly a significant one, still competing with generics here.

    John (CYCN) – Not following them.


  155. Hi Ohad, Thanks very much for your posts. Great to hear from you. NERV failure… what do you think are their future prospects?

  156. Ohad…thanks again for your insights. To double up on the question from Jonas, I looked at the JCI insight study and it looks quite promising for the phase III RDEB study. Would you still recommend waiting for the funding raise? I bought about 1/2 the stock I plan to buy and hoping it will come down a little more, but I think the valuation and upcoming studies seem promising…..
    Once again…thanks for your generosity and insight.

  157. Hi Ohad, I noticed that Agios received Breakthrough designation for Tibsovo in MDS. What did you think about their data in Beta Thal with their PK activator at ASH? Seemed early but very promising. Thanks

  158. Given the m&a activity in Gene therapy the question is not if but only When the Next Company will get acquired.

    Where do you see the highest likelihood of a m&a? ABEO RGNX?

  159. Dear Ohad,
    Nice to read you once again! And I guess you have been good busy!

    Any opinions on MDNAF (another Canadian biotech;) and their ph2b result she which showed a decisive improved survival over avastin and standard of care? Market cap at $60M seems very interesting – I started small position 3 weeks ago and they just released additional data and ceo is taking about partnerships.

  160. Regarding ABEO: until recently this was my worst investment with huge losses, but I added large number of shares when the stock was in the $2s and now I am finally back at breakeven.
    Valuation is still low $250M, so I think with continued development of programs this could soar back to teens. Funding overhang still a risk- but maybe they will find a partner for one of the programs or raise money in a few weeks when market cap is decent again – keeping dilution in check?

  161. Hi Dan,
    Same here on ABEO 😊. Great day today and looks like it’s recovering. Good to be positive again after being very negative. Same story with CLVS. Did not make sense when they dipped so low.

  162. Looks like ABEO issuing common stock tomorrow, so personally I will lock in my final position after the dust settles. Richard, I dont follow NERV, but what convinced me (other than seeing it on Ohad’s buy list :)) is Figure 2 in the JCI insight article. Recall that the three month window is the primary endpoint for the RDEB phase III trial. What I dont know is how much the valuation is dependent on their other studies, which are encouraging but less clear cut at this time with regard to arresting developmental delays.
    I will check out NERV–I do want to thank everyone in this group for the discussions as they have helped me immensely and, of course, especially Ohad as a guide and host.

  163. Dang, just when we thought ABEO was back it’s tanking again. Back to being negative for a while. Bad… these days you just have to sell 20% for every 20% rise.

  164. I dont disagree and glad I bought originally at that price. I am probably going in still for the other half at the same price given the science. But, I will admit that it is a big gamble….

  165. Wow! I could not have timed my post about ABEO any better :( disgusting dilution just when the stock had reached $5. What was management thinking of doing? terrible.

  166. Hi folks, anyone have an opinion on KRTX vs AXSM? There was/is an opportunity to switch some AXSM to KRTX. Both names look very promising but seems like a good idea.

  167. Les/paul (SAGE) – Indeed very disappointing. Still need to re-assess the story based on 547. No plans to add more for now.

    Dan (SAGE/AXSM) – Yep, corroborating P2 is very challenging in CNS indications. So far so good for AXSM, though…
    I liked 217 as it looked diffferentiated from BZD because it modulates different subtypes of GABA but these things are always iffy in CNS.
    Dan on December 6, 2019 at 8:08 am said:Edit

    Höl (MGTX) – I don’t think they are expensive but they gave away a lot of the upside in their Oph pipeline, parkinson’s data are interesting but don’t know how clinically meaningful (not disease modifying) and whether effect will be corroborated in future studies.

    Marc – Thanks! Indeed, still trying to find the right balance…

    Christian (ARQL) – Thanks! Indeed, our biggest exit ever in absolute $ :)
    Thanks everybody for the kind words.

    Saldo (STML) – Not sure efficacy in MF is interesting enough.

    Frank (MTEM) – Initial efficacy with their CD20 program was better than expected but I still don’t think it’s a viable drug given competition and immunogenicity. The VRTX collaboration is interesting, though. A lot of activity in new conditioning regimens.


  168. It seems like its probably going to be difficult to find attractive risk/reward names. Even something like Arqule is trading above bo price of $20 and speculative names trading near highs and even some companies with disappointing results were not published “enough”. Any attractive opportunities?

  169. hi Ohad,

    PR from MDGL from 11.12.:
    “Madrigal Pharmaceuticals Announces Publication in The Lancet of Positive Phase 2 Results for Resmetirom (MGL-3196) for the Treatment of Non-alcoholic Steatohepatitis (NASH)”

    on this day, the stock made a high-volume pretty harsh down day, and since then fallen further 10% (so from 120 to 90 USD in a few days)

    EV some 900 mio. USD which seems not very expensive for a promising P3 asset in NASH

    Do you know why the stock dropped so harshly, what did investors not like?


  170. hi again Ohad,

    by the way, this additional P3 trial that Madrigal just started, isn’t it a bit strange that they test 80mg and 100mg and placebo? This is taking statistical power, no? and shouldn’t they know by now which dose is appropriate?

    how is it with IP-situation for this drug?

  171. Hi Ohad
    Can you look into BTAI – novel MoA, selective alpha-2a receptor agonist
    It the light the of the recent explosive development in CNS like KRTX and AXSM, BTAI looks fairly modest with 150M valuation for Ph 3 company with data readout (mid-2020); NDA submission (2H 2020), and product launch mid 2021.

    Ph2 data with BXCL501 met all primary and secondary endpoints with p< 0.0001
    Looks like a low risk CNS investment for 2020, or … I am missing something?

  172. Hello Ohad !!! GOOD to have you back.Could you provide an updated comment on Arcus { RCUS }? Thanks in advance !

  173. Ohad….. Arcus caught my attention not only for their Original Venture Backing, but for their stellar leadership,and recent Genentech collaboration.Any comments appreciated !

  174. Aviv (MDGL) – I still like their drug and think it works. Plan to add more next year.

    Gene H (MGTA) – Early but I like the story, they were one of the first to recognize the need for new conditioning regimens instead of the primitive ones we currently have.

    andre/Dan – Agree, ZYME and XENE are two of my favorite stocks.

    Richard Baker (KURA) – Yes, I still like them although the HRAS H&N opportunity is smaller than initially thought.
    SYBX – I am following them but still skeptic around most microbiome programs, their PKU program makes sense but so far nothing has been validated in humans.

    Les (NERV) – So the past months made me more cautious on CNS/psychiatry drugs but I still think they have an insomnia drug with an attractive profile so if P3 for ‘101 next year fails I plan on adding.

    Brian G (AGIO) – Agree , looks quite interesting, nice to see several agents going after hemoglobin beyond EPO. It is an oral agent so could be advantageous vs. XLRN.

    Franco – Very hard to predict….

    Ric (MDNAF) – Don’t know them but data seem preliminary and hard to interpret (single arm, subset analysis etc.)

    Richard Baker (NERV) – I don’t think last week’s failure has readacross on 101. It’s still a high risk binary readout but 202 represents a fallback program so I feel comfortable owning a small portion into P3 readout.

    Les – AGTC should present data next month, don’t know what to expect but NITE’s XLRP results generated an efficacy signal that still hasn’t been corroborated with new patients.
    MRNS – Haven’t been following them but not optimistic, especially after SAGE’s failure.


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