Biomarin – testing the boundaries of evidence-based medicine

As an outside observer to the rare disease community, I find the recent acquisition of Prosensa (RNA) by Biomarin (BMRN) puzzling. To me, Prosensa’s drisapersen was just another case where promising phase II results were not corroborated in phase III. This happens frequently with oncology drugs (metmab, iniparib, tivantinib, palifosfamide etc.) and typically leads to termination of the program. Judging by Biomarin’s optimism, drisapersen’s fate may be different, which raises questions regarding approval of drugs despite negative P3 outcome. Continue reading

Array gets binimetinib back (phenomenal deal terms)

After months of uncertainty regarding the fate of binimetinib, Array (ARRY) announced it regained full rights for the drug from Novartis (NVS). Binimetinib (MEK162) was originally partnered with Novartis in 2010 (discussed here) and has been aggressively pursued since. Novartis had to return binimetinib back following the acquisition of GSK’s (GSK) oncology portfolio which included Mekinist, a MEK inhibitor approved for melanoma. Although binimetnib has a high likelihood of returning to Array, the decision is still subject to final approvals for the GSK-Novartis deal. Continue reading

Biotech portfolio update – Three readouts and an acquisition

There was a lot of activity in my coverage universe in the last two weeks, including positive data readouts for three companies and an acquisition announcement. However, of the four cases, only two resulted in share appreciation.

Ambit – To keep or not to keep (the CVR)

Two weeks ago, Ambit (AMBI) agreed to be acquired by Daiichi Sankyo in a deal that included a $15 upfront payment and $4.5 in Contingent Value Right (CVR) per share. The CVR represents a milestone-like mechanism in which Ambit’s shareholders may eventually get additional payments equal to 30% of the initial purchase price. Continue reading

Exelixis following the prostate cancer failure

Yesterday, Exelixis (EXEL) announced Cometriq (cabozantinib) failed to improve overall survival in a pivotal study in prostate cancer. Cometriq is already approved for MTC (medullary thyroid cancer, a rare tumor type) and prostate cancer was viewed as the drug’s most significant opportunity given the large commercial opportunity ($1B+).

Following the failure in prostate cancer, investor focus moves to Exelixis’ MEK inhibitor (cobimetinib, partnered with Roche) and Cometriq in other indications. Although the news are obviously disappointing, Exelixis still has several irons in the fire, which will provide important catalysts over the next 12 months. I previously discussed these catalysts earlier this year. Continue reading

Immuno-oncology – Key themes for 2014

A lot has been written about the immuno-oncology (cancer immunotherapy) field and how it is expected to revolutionize cancer treatment. In 2013, excitement around immuno-oncology and PD-1 antibodies in particular reached record high levels. In 2014, the trend is expected to continue on several fronts. These include potential approvals, new combination regimens, new indications and new targets.

Below is a review of key catalysts and drivers for immuno- oncology in 2014. Continue reading

Biotech portfolio update – 2013 summary and 2014 preview

Seattle Genetics (+65% in 2013)

In 2013, Seattle Genetics’ (SGEN) Adcetris reached market saturation in its approved labeling (relapsed/refractory HL), shifting market attention to label expansion. These include DLBCL, where Adcetris showed impressive efficacy in highly refractory patients (42% response rate, PFS of 5 months) and CTCL (73% response rate). Adcetris is in phase III for earlier stages of HL as well as CTCL, which are viewed as the next opportunity to grow sales. The company will outline its registration strategy for DLBCL in early 2014. Continue reading

Biotech portfolio update – Array, Morphosys, Onyx and Synta

Array Biopharma – Following Puma’s footsteps

Last month Array Biopharma (ARRY) announced a licensing deal with Oncothyreon (ONTY) for ARRY-380, a selective HER2 kinase inhibitor for the treatment of HER2+ breast cancer. ARRY-380 is regarded as an insignificant program, evidenced by the modest deal size ($10M upfront) and the lack of market reaction, but this could change once investors make the connection between ARRY-380 and  Puma Biotechnology’s (PBYI) neratinib (EGFR/HER2 inhibitor). Although neratinib is more advanced, backed by more clinical data and probably has broader potential, ARRY-380’s selectivity profile could differentiate it in certain clinical settings. As the market is clearly excited with neratinib (Puma has a market cap of ~$1.6B), some of the excitement could eventually be tunneled toward Array as well. Continue reading

Major trends in cancer drug development – Notes from TAT 2013

Earlier this month, I attended the TAT (Targeted anticancer therapies) congress in Paris. This conference focuses exclusively on targeted therapies for cancer, one of the most active areas in drug development. As a small conference (~500 participants), it does not generate a lot of high profile clinical data, still, it is a great opportunity to “feel the pulse” of oncology drug development. Speakers include clinical oncologists, basic scientists and industry researchers, which provide a fairly broad spectrum with respect to existing and upcoming trends.

Here, I focus on three major themes from the meeting: PD-1 inhibitors, antibody drug conjugates (ADCs) and cancer metabolism. Continue reading

AVEO Pharmaceuticals– Attractive following recent de-risking events

AVEO (AVEO) is down 45% in less than 3 months due to uncertainties around its lead program, tivozanib. Tivozanib was recently submitted for FDA approval in renal cancer based on positive phase III data showing superiority over the approved drug, Nexavar. Although tivozanib led to superior progression-free survival (PFS) and was substantially safer than Nexavar, investors are concerned about Regulatory and market positioning risks. Continue reading

Exelixis – First partnered program enters phase III

Roche recently disclosed a decision to start phase III with GDC-0973 (formerly XL518), a MEK inhibitor licensed from Exelixis (EXEL) to Genentech 6 years ago. The trial is expected to start next month and will evaluate GDC-0973 in combination with Roche’s Zelboraf in patients with BRAF-mutated melanoma. This is an extremely positive catalyst for Exelixis, who now has a second drug in pivotal studies with a high likelihood of success and a substantial market opportunity.  Continue reading