3 Things In Biotech, May 4: Merck's New Combo, Alnylam Jump Starts, Ocata Gone Forever?
Summary
- Merck finally breaks out the combos in the tougher lung cancer.
- Alnylam gets a potential headstart from the FDA on a new RNA therapy.
- Astellas' Ocata show is a no-show.
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Welcome to another edition of "3 Things In Biotech," a daily digest dedicated to helping you keep pace with the fast-moving world of pharmaceutical and biotechnology research.
Merck finally breaks out the combos in the tougher lung cancer
Company: Merck (MRK)
Therapy: Pembrolizumab
Disease: Squamous cell non-small cell lung cancer (NSCLC)
News: MRK has announced findings from its KEYNOTE-407 study, which is assessing the addition of pembrolizumab to standard chemotherapy for patients with squamous cell NSCLC. This differs from KEYNOTE-189, which looked at patients with non-squamous disease. KEYNOTE-407 has met its endpoint of overall response rate. Furthermore, based on the data it's gathered so far, MRK has submitted a supplemental drug application for approval of this combination in first-line treatment.
Looking forward: Stretching ever further, now into squamous cell disease. Just a few years ago, this was an extremely poor prognosis tumor type, and the emergence of immunotherapy has arguably impacted this subset of NSCLC most of all. I am eager to see a full data readout from this study, but I expect it will yield good results, and that the FDA will accept and approve the combo sometime in 2018.
Very good news for building the lung empire for pembrolizumab ever wider.
Alnylam gets a potential headstart from the FDA on a new RNA therapy
Company: Alnylam (ALNY)
Therapy: Lumasiran
Disease: Primary hyperoxaluria type 1 (PH1)
News: ALNY announced that it has reached an understanding with the FDA on a pivotal trial design for its RNA therapy lumasiran for the treatment of PH1. The primary endpoint for this study will be reduction in urinary oxalate at 6 months, and it will enroll around 25 patients. The company hopes to start this trial in mid-2018, which would set it up for data readouts in time for a 2020 NDA.
Looking forward: Lumasiran has already demonstrated some promising early-stage clinical data for PH1, and it's a testament to these results that the FDA is allowing such a small trial to be registrational. It also speaks to the rarity of this disease, but it is now becoming firmly in ALNY's modus operandi to target rare disorders...and successfully.
Great news for ALNY, if you ask me, but the near-term sales of its amyloidosis program have higher weight at this time.
Astellas' Ocata show is a no-show
Company: Astellas (OTCPK:ALPMY)
Therapy: MA09
Disease: Stargardt's disease
News: It appears as though ALPMY is letting old Ocata's MA09 candidate die quietly, as it has been removed from its pipeline for development. MA09 was an embryonic stem cell-derived retinal pigmented epithelium that was intended to replace deficient tissue to treat macular disorders like AMD and Stargardt's disease.
Looking forward: This would seem to be bad news, and it might confirm the rumors swirling at around the time of the Ocata buyout that there were legal or clinical issues with the technology. The Astellas Regenerative Institute continues RPE research, as there are studies ongoing for a product called ASP7317. I know many shareholders have been very interested to follow this research further. Needless to say, it's a good thing that there is a big pharma supporting the show now!
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