ArQule –An ultra-rare opportunity emerges

With most of its market cap covered by cash, ArQule (ARQL) and its pipeline are receiving very limited appreciation. The negative sentiment stems from skepticism regarding tivantinib,  currently in phase III for liver cancer (partnered globally and in Japan with Daiichi Sankyo and Kyowa Hakko Kirin, respectively). The skepticism is based on the drug’s 2012 failure in lung cancer as well as uncertainty about its mechanism of action.

The market also assigns no value to ArQule’s two early stage programs, which are close to completing phase I. The more advanced program, ARQ092, is an Akt inhibitor with what appears to be a better clinical profile compared to the first generation of Akt inhibitors (e.g. MK-2206) but still no clear route to market. In contrast to the case with tivantinib, which many claim is not a true Met inhibitor, ARQ092 appears like a bona fide Akt inhibitor with similar pharmcodynamic behavior to other Akt inhibitors in development.

ARQ092 as a fast follower

Roche and AstraZeneca (AZN) have the most advanced Akt inhibitors in development (both in randomized phase II trials). Roche’s GDC-0068 (licensed from Array Biopharma [ARRY]) is being evaluated in three large trials in breast, gastric and prostate cancer. Astra’s AZD5363 is in several randomized trials that are funded predominantly by research institutes. Results from the above studies should start to generate data in 2015 and could inform phase III strategy for Akt inhibitors as they assess multiple regimens and predictive biomarkers.

As I discussed last year, ARQ092 can be regarded as a fast follower behind Roche and AstraZeneca, whose Akt inhibitors are in multiple large phase II studies. The big advantage in having a fast follower drug is that its development is informed by experience gained with more advanced compounds. This includes indication, biomarkers for patient selection, combination regimens and mitigation of toxicity.

While ARQ092’s development will continue to focus on oncology and rely on experience generated by Roche and AstraZeneca, ArQule might pursue a parallel effort in a group of ultra-rare diseases. This strategy may enable ArQule to differentiate its Akt inhibitor and potentially beat competing drugs to the market.

On the last quarterly call, Arqule’s management indicated it will present preclinical data with ARQ092 in an undisclosed ultra-rare disease at the 2104 meeting of American Society of Human Genetics (ASHG). According to CEO Paolo Pucci “there will be some pretty extensive preclinical work done for 092, our Akt inhibitor, in a very rare and terribly debilitating disease…“.

Akt-related rare diseases

Based on the abstract released on the ASHG website, the ultra-rare indication is Proteus syndrome (PS), a condition characterized by asymmetric and disproportionate growth of certain tissues in the body. The most famous Proteus syndrome patient is Joseph Merrick, known as the elephant man.

Akt was implicated in Proteus syndrome following a landmark publication from a group at the NIH which showed that 90% of tested patients carry an activating mutation in Akt, which is also observed in various cancer types. The presence of the same mutation in cancer and Proteus syndrome is logical, as both cases involve uncontrolled growth and proliferation.

The abstract describes experiments (conducted by the same NIH group who identified the Akt mutation in 2011) using cells from PS patients. Treatment with ARQ092 led to a rapid shutdown of Akt signaling and resulted in a reduction in cell viability, which appeared meaningful but not dramatic (25-40%). These findings, albeit preliminary, represent the first proof of concept for Akt inhibitors in rare diseases (to my knowledge) and may pave the way to clinical testing of ARQ092 in PS.

Interestingly, other rare diseases are also characterized by hyperactivation of Akt that leads to uncontrolled growth of certain tissues. These diseases are characterized by mutations in other components of the PI3K-Akt-mTOR pathway and share some clinical features with PS. These include diseases with activating mutations in PI3K (PIK3CA-related overgrowth spectrum) or inactivating mutations in PTEN (PTEN Hamartoma Tumor Syndrome).

Commercial opportunity – modest but meaningful

Although there are still a lot of uncertainty associated with ARQ092’s potential development in PS and other PI3K-related diseases, this development is a clear positive. Rare diseases are regarded as attractive due to the high unmet need, low regulatory requirements, short timelines and high pricing. Another important aspect that distinguishes rare genetic diseases from cancer is their monogenic nature (caused by a single genetic defect). This is in contrast to cancer, which is typically driven by multiple mutations. Having a disease with a clear etiology significantly increases likelihood of finding effective treatments.

It is also important to acknowledge the uncertainties associated with developing ARQ092 in PS other PI3K-related diseases. On top the obvious challenge of translating an effect on cells in a tube to patients, a primary challenge would be safety. Akt inhibitors lead to hyperglycemia (increased blood glucose) and skin toxicities. To date, the glucose side effect appears manageable but the skin toxicities are dose limiting and have hampered development of Akt inhibitors. Based on phase I results for ARQ092 and AZD5363, this issue was overcome with the new Akt agents and creative dosing regimens. It remains to be seen whether ARQ092’s safety profile will be mild enough to be given to young patients (including infants) on a chronic basis.

Competition is another risk to bear in mind, although it is unclear whether Roche and AstraZeneca will pursue such an ultra-rare opportunity. In addition, there are many drugs in development or approved which inhibit the PI3K pathway including PI3K inhibitors and mTOR inhibitors. Akt inhibitors may have a better clinical profile but this is still an open question. Lastly, as no company has tried to pursue these indications, the clinical trial design needed for approval is unknown.

The commercial opportunity is also unclear as most of the relevant diseases are very rare and the exact prevalence is unknown. PS is believed to have a known prevalence of 100-200 patients worldwide. Most other indications range from 20 to 200 patients each, with Cowden syndrome (1 in 200,000 people) as the only exception. Therefore, the immediate market opportunity is probably ~500 patients which may grow to ~1000 with time as diseases without approved drugs are often under-diagnosed.

A 500-1000 prevalence is in the low range of other rare disease programs, which typically address a market of thousands or more. For example, BioMarin’s (BMRN) Vimzim is approved for Morquio A which has a global prevalence of ~3000 patients. There are cases, however of companies which decided to pursue ultra rare diseases with a very low prevalence. Ultragenyx’s (RARE) UX003 program, which targets a disease with a prevalence of 200 in developed countries, is a recent example. Analysts forecast peak sales of $75M for UX003, which could serve as a good conservative benchmark for ARQ092’s opportunity in PS and other PI3K-related diseases. This is on top of the potential for Akt inhibitors in cancer, which is significantly larger.

Q4 is going to be an important quarter for ARQ092, with preclinical results in PS at ASHG (October 18-22) and clinical data in cancer at EORTC (November 18-21). These readouts may increase awareness of ARQ092 as well as its worth in the eyes of investors. The commercial opportunity in PS is modest and timelines for approval in cancer are long, but for a company with an enterprise value of $2.5M, ARQ092 may become an important valuation driver.

72 thoughts on “ArQule –An ultra-rare opportunity emerges

  1. Thanks for this research Ohad!
    I had listened to Pucci’s conference call and I liked it. It seemd teh company was doing a lot or through and innovative reserach, also in characterization of ittues for met positive negative, if I remeber correctly. I thought ARQL seemed focused and driven, and even excited, also regarding the two ph1 programs you have mentioned. That was the biggest take away for me.

    Do you think it would be worth adding some ARQL? It is still at an all-time low.

    Regarding NPSP – did you see what a zoo thsi week has been? now the FDA has issued positive notes, and teh stock regained everything it lost afte the reckless feuerstein tweet. A lot of people got burned for nothing.

    Dan

  2. Hi Ohad,

    Thanks for a very interesting post. What do you think of Genmab- SGEN collaboration?

    Chris

  3. Dan – Yes I think ARQL is extremely undervalued but that was the case during the past 2 years and the stock continued to slide. At the moment the negative sentiment around tivantinib overshadows anything positive that could come out of the Akt and FGFR programs. I honestly believe that had tivantinib been terminated, Arqule’s market cap would have been higher today.
    Bottom line, I might wait for data readouts for 092 in Oct/Nov to get a better sense on where things are headed.

    Ohad

  4. Ohad
    interesting article in Forbes about Nant system backed by billionaire Soon-Shiong. They claim 260 times more genes to be tested compared to 343 genes by FMI, faster and at lower costs?! They already signed up for the Nant system Providence Health & Services with 34 hospitals in Oregon, California, Alaska, Washington State and Montana.
    Do you see this development as a serious completion to FMI? This guy has deep pockets plus a number of strong investors – Verizon, Celgene, BlackBerry.

  5. andre – This sounds very futuristic and immature, hard for me to call it competition because I don’t know what the landscape would look like and what FMI’s performance will be by then.

    Ohad

  6. Ohad, GSK has an AKT inhibitor afuresertib that showed activities in MM and other hematologic maglinancies. It is the only one early stage compound NVS excercised from GSK oncology assets buyout.

  7. JQ – Thanks, they have good preliminary data in terms of activity in myeloma. Interestingly, in their trials they give the drug daily and looks like skin toxicity is not a major issue (neither is hyperglycemia) yet they still see activity.

    Ohad

  8. Ohad – any further thoughts on Array? It continues to slide, but for no real reason. I see tremendous upside if NVS returns the MEK inhibitor, which seems to be the most likely outcome. I suppose they may need to generate cash if that happens – which could lead to a good buying opportunity also.
    Mark

  9. Mark – I completely agree, when ARRY gets MEK162 back it will have full rights for a p3 program with multiple p2 trials in process. The main problems in this scenario are : a) many of the combination trials are with Novartis’ compounds b) ARRY will have to raise a lot of cash to support the program.

    I see a lot of upside from the partnered pipeline as well, plenty of cards to turn next year.

    Ohad

  10. hi Ohad,

    again a CMO change at ARRY. the last one was appointed only a little more than a year before.

    what do you think of it, does it maybe have to do with ARRY520?

    what’s your guess how much money ARRY will burn approx. until they know whether 520 is a competitive program or not?

  11. Christian – The guy they brought as the new CMO is very good from what I hear.
    Don’t know what the background for the change is but overall it’s a positive change.
    Re: ARR520, ARRY should have response rate data for AAG-low patients at ASH or 1H:15. I am not very optimistic but the attention will move to binimetinib by then.

    Dan – Agree.

    Ohad

  12. re ARRY – I am not so sure that NOV will return the program.

    if data look good they might prefer to sell Mekinist (I wonder why they have not excluded that program from GSK deal)

    in any case, the uncertainty is not good as we can see.

  13. if program is really returned, my favourable scenario would be a buyout.

    don’t think ARRY is able to market themselves

  14. Christian – If NVS decides to keep binimetinib that would be a huge surprise. Actually, Array is in a position that every decision NVS takes should be regarded as a positive, as long as it will end the uncertainty. If ARRY gets the program back, it can do a lucrative Ex-US deal, no need for a buyout.

    Ohad

  15. it will be interesting to see how it plays out – and I hope it won’t take very long until we know (I guess we should know soon after the NOV GSK deal is approved by authorities).

    Ohad, have you ever considered a position in Ablynx? market cap looks rather low (444 mio. EUR, about 230 or so in cash). i am not familiar with acquired TTP, but the program looks interesting and is fully owned. and there are quite a few partner deals. with totally > 3 billion theoretical milestone payments plus royalties.

  16. Ohad
    do you have an opinion about MGNX? They have Ph 3 data for margetuximab (HER2) in gastric cancer in December.
    What do you think about their Dual-Affinity Re-Targeting (DART) platform. It looks to me that the GILD collaboration gives some credibility to this program, correct?
    thanks–andre–

  17. Christian – Agree about the TTP data set as Ablynx’s most interesting asset but I still worried about their pipeline which is mostly “me too”.

    andre (MGNX) – From what I understand they will have p2 data for margetuximab. Don’t have high expectations for this program plus there may be better HER2 targeting alternatives. The DART platform is very impressive, probably the most popular technology for bispecific antibodies in the industry.

    Alex (AVEO) – Doesn’t look like anything has changed there. The cachexia program will take time to generate value and the other 2 antibodies are not that interesting imo.

    Ohad

  18. Since we are talking antibody companies, what about KBIO? Has anything changed there? The valuation is so low.
    And ArGen-x – the Llama antibodies company that recently went public? They have several programs, including immuno-oncology, and have partnerships with Roche and Shire (AbbVie now). The CD70 program (argx-110), I suppose is rivaling CLDX CD27 antibody, but nobody seems to talk about that.
    Thanks

  19. Hallo Ohad,

    after Dan I wan’t to mention another new IPO: Affimed Therapeutics. The plan was an IPO price in the range of $11-13. They only get $7. Do you see this as an opportunity for investors? However to date they have only Phase I data for one compound and will start the Phase II trial this year. Another compound is in Phase I. “Affimed has three proprietary platform technologies based on its proprietary tetravalent antibody architecture characterized by 4 binding domains.”
    Thanks Toby

  20. Hey Toby
    Affimed looks good. I like their two lead programs. Small trials, but seems like they have somethng unique.

  21. Hey Ohad,
    looking fowrward to ESMO: Obviously COBRIM results will be important for EXEL.
    But also NVS will present PII data of MEK162 as singleagent in patients with melanoma with BRAF and KRAS. With background of ARRY getting MEK162 back: I would say that these results are important, right? What would be a good result?
    http://clinicaltrials.gov/show/NCT01320085
    Thans Ike

  22. hi ohad, are you still planning to add to your position in EXEL prior to the ESMO data release? i’m hoping for some strong data to get the stock moving on an uptrend! thanks.

  23. Dan – KBIO is indeed very cheap with no value ascribed to its pipeline. They should have randomized p2 data for the pseudomonas antibody next year.
    Re: ArgenX, cool technology but targets are not attractive imo, don’t think that the cd70 program competes with cdx1127, which is an agonist of cd27.

    Toby – Affimed bispecific technology is intriguing and have advantages over other formats (tetra vs bivalent). So far the cd30 results are disappointing, show that nk and macrophages may not be potent enough…

    Richard (RXDX) – I m closely watching them as the most advanced trk inhibitor, should have more data at ESMO. LOXO is a serious threat. I like their RET programs which are highly selective.

    Martin – OMED is still overvalued imo, would waituntil clinical poc.

    Dan S – I added some EXEL personally, will reassess the entire opportunity after ESMO.

    Dan – FMI continues to slide but I am still very enthusiastic about them. They are the leader of an exploding field, won’t remain independent for long imo.

    Ohad

  24. Hello Ohad!
    When I look at the shares of Clovis I see them falling deeper and deeper. Can you guess why? I can’t find anything on their page. Do you see this as a good entry point?

    Thanks for your advice!

    Tom

  25. Hey Ohad
    how low can ARQL go, we thought it was cheap at $1.30, but now it even touched $1.10 are you adding some more here?
    also, have you ever looked at KPTI? the valuations seems generous to me.
    Thankds
    Dan

  26. Tom -My guess is that CLVS will continue tobe volatile going into EORTC. AZD9291’s data next week may also have a strong impact.
    Yes, I consider current price as attractive following CEO’s comments about metformin being sufficient to deal with hypetglycemia.

    Dan (ARQL) – that’s a good question for which i have no answer. Turns out that the negative sentiment around tivantinib is overshadowing the potential of the Akt and FGFR programs.

    Ohad

  27. Ohad
    ESPR had a nice move in the last 2-3 day. Is the run due to the presentation today? Did they say something what fired the market?
    In May you wrote about several stocks. 3 are doing exceptionally well – BLUE, AGIO, XNPT. What drives AGIO – they are very early in the development.

    ONO (OPHLY) – do you like them on that level.
    thanks –andre–

  28. andre – Don’t know what’s behind the run-up in ESPR, volume is high so perhaps a new institutional is accumulating. ESPR typically has very thin trading so price is sensitive.

    Yeah, BLUE and AGIO have been doing very well since but unfortunately I missed the ride.

    Re Ono, my impression is that both nivo and Kyprolis are fully priced in.

    Ohad

  29. Great call on AMBI. Looks like CLVS news looks good and waiting on EXEL update. As always, thanks for your insight.

  30. Sorry, posted too quickly about AMBI. Can you provide any insight into the CVR in the agreement and whether you plan on holding your shares? Thanks again.

  31. Dan S – Thanks. The deal is great for Daiichi which gets a p3 asset plus multiple combination avenues for a cheap price. Valuation is somewhat disappointing but I guess that was the highest bid and the BOD probably chose the quickest way out. Still not sure about the CVR.

    Re CLVS – AZN’s data at ESMO still look good, PFS is lower than expected (<10 months)but overall it's a great drug. Let's wait for CLVS' update later in the year.

    EXEL - data are due tomorrow, must have a differentiated profile to become a major drug.

    Ohad

  32. Hey Ohad
    very good call on Ambit – but I agree with you, the valuation is low. The share price has gone so low, for anybody who bought in the last two month it;s been a great return, but for shareholders who have bought earlier the return is very low. Do you think the board will approve it?

    so then the logical question is: which is teh next company in p3 that might get acquired? ay ideas?

    Have you looked at the EXEL press release. What is your take and what kind of market penetration will the drug have. Any ideas on the valuation for Exel? Thanks, and keep up the good work!
    Dan

  33. Dan – I think it’s a done deal. With respect to other companies with p3 programs, there are not a lot of cheap options out there but I expect several companies with pivotal p2 to garner a lot of interest. These include LOXO, RXDX, MRTX and STML.
    My top acquisition target is still FMI.

    EXEL – Results look robust, still didn’t have a chance to delve deeper but at first glance the new GSK data also look strong. Now it will all come down to overall survival.

    Ohad

  34. Hey Ohan
    have you looked at AVEO’s press release this morning, they have identified a target population /biomarker for ficlatuzumab. The results look good to me.
    Dan

  35. Dan – imo LOXO, MRTX, RXDX all have intriguing programs but valuation is rich given the early stage of their programs. The only one with clinical poc is RXDX in several subsets of NSCLC but activity in ALK tumors may be limited and initial activity in a CRC patient with Trk fusion needs to be corroborated.

    AVEO – It’s hard to take a retrospective analysis of 35 patients out of 188 seriously. The cachexia program is interesting but early.

    Ohad

  36. Exel appears that it did not get the two month PFS advantage over GSK to create a differentiation between the two combos…was hoping for this

  37. Ohad explain the independent review median PFS of 11.3? Is this two month advantage you were looking for? How is this different from the 9.9%?

  38. Robert – Agree, its PFS benefit is slightly superior from a HR perspective but not more than that.
    PFS can be assessed by the treating physician or by indpendent reviewers (IRC). IRC assessment is viewed as more reliable and conservative as it is not impacted by physician bias (especially in cases where the physician knows if the patient is on the active arm or the control arm). In this case, the better PFS per IRC is surprising but not unprecedented, may emanate from physicians’ ability to determine progression based on clinical signs (clinical progression) and not only based on a CT scan.

    Ohad

  39. Hi Ohad,

    What are your thoughts on the 12 month median OS for ARRY’s MEK162 in NRAS melanoma? Do the PFS and OS results bode well for the P3 outcome? Thanks.

    Rick

  40. Kimi – Thanks.

    Rick (ARRY) – It is hard to call Binimetinib’s data impressive but given the lack of other tratment options even limited efficay is better than nothing. It’s hard to put the PFS and OS in perspective so this doesn’t change my view of this program.

    Ohad

  41. wow on ambi…didnt see that…does everyone with a jak2 become acquired? jeez

    ohad is arry ahead of all meks when it comes to nras? …but kras is where the $$ is, correct?

  42. Hey Ohad
    re EXEL – it shot up and now the sp is settling down – is this a buying opportunity?
    thanks
    Dan

  43. Robert – Yes ARRY has th most advanced melanoma NRAS program. There are many other subsets where MEK inhibitors may come in handy, KRAS+ NSCLC is viewed as the largest opportunity but BRAF+ colon and RAS+ ovarian cancer are also attractive indications imo.

    Dan – Not sure, need to see GSK’s and Roche’s data set first.

    Ohad

  44. Loxo spiked today. Any thoughts on which is the better buy and undervalued of the 3? Loxo, rxdx and mrtx? Thanks Ohad

  45. hi everybody,

    if I see it correctly, AMBI shares can be bought for approx. 15 USD now. that is the price that will be refunded in cash to stock holders once the transaction is closed.

    BUT the CVR comes in addition, doesn’t it?? so in effect the CVRs would come for free?!?

    thanks for your opinions

    Christian

  46. I was thinking just the same, Christian
    it seems like a low risk entreprise. Pay pay 20-30 cent now for the potential $4,50 payout.

  47. Hello Ohad
    I used to be an investor in NSPH, sold my shares 6 months ago and just looked back at share price yesterday – it imploded to $0.58 – market cap is $45M. The company has struggled to sell more systems to hospitals and other clients and are still far from break-even. It seems they will need to find some cash just to stay afloat, ehnace the decline (market cap used to be 200M not long ago. I wonder what you view is on systems like NSPH, for rapid detection/assays at hospital or in labs. I ask this espcially knowing that youa re a big fan of technology for screeninga nd customized medicine (FMI) and I know the two technologies are remarkably different, however, isn’t it expected thats ystems such as NSPH will find a broader use? Thanks for your opinion, as always
    Dan

  48. Hello Ohad,
    Seattle Genetics is getting hammered today as a result of a downgrade by Bank of America. BAC says Aethera data not as positive as expected and gave a $34 negative price target. Your thoughts?

  49. Christian
    I have some limited experience with CVRs.
    After the transaction is closed the stockholders will receive $15 cash plus one CVR note per AMBI share. This note will trade on the stock exchange as a regular share. You can trade it at any time or wait to collect 4.50 royalties after meeting some or all milestones. After receiving any one of the milestone payments the value of the CVR will drop correspondingly. After the last payment the value of the CVR will become zero.

    After Sanofi bought Genzyme they provided in addition to the $74 purchase price a CVR which still trades as a normal share. The stock symbol is GCVRZ.
    You may look into this sec.gov document for good explanation of the CV rights:
    EX-99.A.43 2 b85162exv99waw43.htm
    Still the conditions of the CVR are not know, but they may have an expiration date, i.e. if the FDA approval is not obtained by a certain date, the CVRs may expire, so the right look like royalty options
    –andre–

  50. Ohad, you said that RXDX had a high valuation, but I don’t see that, unless you consider $157 million high. What did you think of their presentation as ESMO?

  51. Re AMBI (Dan, Chrstian, Ike, andre) – Indeed looks like the CVR will not be traded on the stock exchange so the onlty way to get exposure to it is owning the stock prior to finalization of the deal. There are 2 tranches, each relating to 50% of the CVR potential, looks like the second scenario will occur before the first one:

    “A holder of a CVR will be entitled to a cash payment of $2.25 upon the first commercial sale in the United States following marketing authorization of a product for remission induction in patients with acute myeloid leukemia (“AML”), either alone or in combination with any other therapy, if the product’s marketing authorization does not require that a patient have received at least one prior systemic therapy for AML, plus an additional $2.25 upon the first commercial sale in the US following marketing authorization of a product for the treatment of relapsed or refractory FLT3-positive AML in patients who have received at least one prior systemic therapy for AML. ”

    Ohad

  52. Hey Ohad
    regarding FMI – who do you think are potential suitors? I do not think thse woudl be drug companies (would create conflict of interest) – possibly diagnostics and assay reagent makers as BD, Quiagen, Coulter, Illumina?
    Thanks
    Dan

  53. Dan – Agree, potential acquirers are diagnostics companies, especially those selling companion Dx markers (Abbott, Roche, Qiagen etc.)

    Ohad

  54. Ohad, what did you think of the RXDX presentation at ESMO? Price has come down a bit in the last couple days. Would you be a buyer?

  55. Richard – I am somewhat ambivalent about RXDX’s data. On the one hand they have clear activity across the different mutations (ALK, ROS1, TrkA), including the firs response in a patient with a Trk-fusion. On the other, I fear their compound will be inferior to competing drugs which appear more selective and potent. For Trk, LOXO might have a better molecule that can inhibit teh target more potently (without penetrating the brain). For ALK/ROS1 there are multiple drugs that appear more efficacious based on cross trial comparisons.

    Ohad

  56. EORTC abstracts are out:

    P 100: First-in-human study with ARQ 092

    Efficacy is only mentioned for the intermittent schedule. Of the 27 pts, one with lymphoma had a partial response, 21 had stable disease including 3 pts with 18–20% tumor reduction. Safety profile is reported to be in line with other drugs in the class.

    P 169: First-in-human study of ARQ 087

    Best response in 32 of 40 evaluable pts was stable disease (80%). Eleven of 32 pts had stable disease for ≥16 wks [adrenocortical (2), renal cell, endometrial, ovarian carcinomas, choroidal melanoma, pleural mesothelioma, liposarcoma, osteosarcoma and chondrosarcoma]. ARQ 087 was well tolerated with manageable, mostly grade 1/2 AEs.

    Ohad, do you view these results as encouraging for a P1 trial? Thanks as always!

    Deaglan

  57. Deglan – I would say they are ok, so far no major surprises.

    ARQ092 – we already knew about the PR in the lymphoma patient, the other cases of tumor shrinkage are encouraging. The most important finding is the safety profile which proves that the drug safe enough to be pursued and implies it truly inhibits Akt.
    ARQ087 – The lack of efficacy is not surprising, this is similar to other FGFR inhibitors (note this molecule is not very selective so its future is unclear imo). The lack of hyperpohsphatemia is an advantage (can be explained by the lower inhibition of FGFR4) but also calls into question whether good FGFR inhibition was achieved.

    I was surprised by the amount of preclinical data ArQule has this year. I counted at least 5 abstracts on one or both drugs in combination looking at biomarker, distribution or synergism. Couldn’t find anything dramatic but good to see they have been busy setting the stage for combination and biomarker-defined trials.

    Ohad

Leave a Reply

Your email address will not be published. Required fields are marked *