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Oct 05, 2015
 |  BioCentury  |  Finance

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Investors' top names, milestones and meetings in 4Q15

For investors who are not inclined to sit it out until the volatility abates, the fourth quarter is bursting with late-stage catalysts. There are no fewer than 17 PDUFA dates on the calendar before year end, and at least 11 Phase III data events.

The most eagerly anticipated event of 4Q15 - an FDA panel of Duchenne muscular dystrophy therapies - also is unconfirmed, as the agency still hasn't set a meeting date.

In addition to the bevy of regulatory and late-stage clinical milestones, investors agree the American Society of Hematology (ASH) meeting in December should provide much awaited visibility on durability of gene therapies and chimeric antigen receptor (CAR) T cell therapeutics.

Dialing up DMD

Investors who spoke to BioCentury are watching the reviews of BioMarin Pharmaceutical Inc. and Sarepta Therapeutics Inc. as an indicator of FDA's thinking on what constitutes sufficient evidence for approval in Orphan diseases beyond just DMD.

CEO Jean-Jacques Bienaimé has said BioMarin expects FDA to hold an advisory committee meeting this year for drisapersen. The antisense oligoribonucleotide that induces exon 51 skipping on the dystrophin gene has a Dec. 27 PDUFA date.

Sarepta has a Feb. 26 PDUFA date for its eteplirsen, a phosphorodiamidate morpholino oligomer (PMO) targeting exon 51. Numerous analysts and buysiders have speculated that an FDA panel likely would review both molecules together.

Deerfield's Jim Flynn said it will be interesting to see how FDA weighs drisapersen, which has a large database but a failed Phase III trial, and eteplirsen, which has fewer patients but a lot of patient advocate support.

Drisapersen missed the primary endpoint of six-minute walk distance (6MWD) and multiple secondary endpoints in the Phase III DEMAND III trial in 186 ambulant boys. BioMarin has attributed the results to different enrollment criteria and testing methods than were used in Phase II studies, in which pooled data showed a significant improvement on 6MWD.

Sarepta's 12-patient Phase IIb study met its primary endpoint of increasing the percentage of dystrophin-positive muscle fibers from baseline at week 48. The company kicked off 4Q15 with updated data showing effects were sustained at three years. The news sent Sarepta up $3.32 to $39.59 last week.

"If Sarepta gets approval on a dozen patients it will have implications across Orphan diseases," Flynn said.

He said both reviews are "really close calls," but he thinks BioMarin has enough data for approval.

"It's really going to come down to the message from the top of FDA," he said.

Loncar Investments' Brad Loncar said he is "biased towards" Sarepta. "As small as their study is, I think it's compelling because they have dystrophin levels that you can look at," he said.

Loncar said one could argue both companies are trying to get approval on "unprecedented" types of data. "It'll say a lot about the FDA's mindset," he said.

EcoR1's Oleg Nodelman is going far upstream in the DMD space. "I'm excited about FibroGen, which has DMD assets that nobody really knows about," he said.

This year, FibroGen Inc. expects to start a Phase I trial of FG-3019, a...

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