Alnylam shares dip on a decision to scrap accelerated pitch on givosiran; Loxo gets a ‘breakthrough’ at the FDA

Alnylam $ALNY has decided to put a hold on its plans to shoot for an accelerated approval of givosiran and instead start a rolling submission in search of a full approval. Investors didn’t care for what looked like a slowdown, driving shares down by 1.8%. But at least one analyst was holding fire this morning. Stifel’s Paul Matteis noted: “This is not a surprise to us: this plan is, in our opinion, a byproduct of faster than expected enrollment of the phase III ENVISION trial, and we’d be buying the stock on any weakness that ensues.” This drug has been lined up as Alnylam’s second RNAi drug intended for the market. The drug is designed to treat acute hepatic porphyria.

Sarepta Therapeutics $SRPT has lined up another gene therapy for its pipeline. The Cambridge, MA-based biotech in-licensed US rights to Lysogene’s program for Sanfilippo syndrome type A, with the French company set to begin a pivotal trial before the end of this year. Sarepta is handling over $15 million for the regional rights to LYS-SAF302, with another $2.5 million for an equity stake in the company. Next year they’ll follow up with an additional $19 million, with milestones running up to $125 million in the deal.

Loxo Oncology $LOXO is going to get some help in its quest to hurry along the development of LOXO-292. The FDA has come through with a breakthrough drug designation for its RET inhibitor. Loxo’s drug is up against a rival from Blueprint Medicines $BPMC. And now the punters will give Loxo an edge in that showdown. The BTD program calls on regulators to keep an open-door policy to a therapy to help accelerate its passage to the market. And it’s helped do that for a number of cancer drugs now being sold.

HiFiBiO Therapeutics has acquired H-Immune Therapeutics, an early-stage biotechnology company and a spin-out of the French Atomic Energy Commission engaged in immuno-oncology R&D work.


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Sr. Manager, Regulatory Affairs, CMC
CytomX Therapeutics San Francisco, CA
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Flatiron Health New York City or San Francisco

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