Doug Ingram, Sarepta CEO

Sarep­ta won't need FDA ad­comm, boost­ing Duchenne gene ther­a­py's odds

On its quest to get the first gene ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy ap­proved by the FDA, Sarep­ta won’t have to face an FDA ad­vi­so­ry com­mit­tee af­ter all.

The biotech dis­closed in its quar­ter­ly up­date Tues­day af­ter­noon that af­ter a mid-cy­cle re­view of SRP-9001 with the FDA, in which it an­swered ques­tions about chem­istry, man­u­fac­tur­ing and con­trols, the agency said it does not plan to hold an ad­comm. A de­ci­sion on the ther­a­py is due by May 29.

The pos­si­bil­i­ty of an ad­comm has been top of mind for both the com­pa­ny and in­vestors since the FDA grant­ed pri­or­i­ty re­view and set the PDU­FA date for SRP-9001, which would be the fourth Duchenne drug from Sarep­ta to win ac­cel­er­at­ed ap­proval based on a bio­mark­er. When the pri­or­i­ty re­view was grant­ed in No­vem­ber, Sarep­ta said it ex­pect­ed an ad­comm to be held.

The news that one won’t be nec­es­sary sent the stock up 20% in pre-mar­ket trad­ing Wednes­day.

The ther­a­py works on the same con­cept as the three ap­proved drugs Sarep­ta al­ready has on the mar­ket — Ex­ondys 51, Vyondys 53 and Amondys 45 — ex­cept that the trio of treat­ments spurs func­tion­al dy­s­trophin pro­duc­tion by telling the body to skip over prob­lem­at­ic ex­ons, or parts of the DNA.

But more than six years af­ter its first ap­proval, the com­pa­ny has yet to of­fer de­fin­i­tive ev­i­dence that the dy­s­trophin pro­duc­tion ac­tu­al­ly trans­lates to clin­i­cal ben­e­fit. While con­fir­ma­to­ry tri­als for the ear­ly drugs are still un­der­way, ini­tial da­ta on SRP-9001 were mixed. EM­BARK, the pro­posed con­fir­ma­to­ry tri­al for the gene ther­a­py, is ful­ly en­rolled and, ac­cord­ing to CSO Louise Rodi­no-Kla­pac, is on track for a read­out in the fourth quar­ter of 2023.

On a call with an­a­lysts Tues­day, CEO Doug In­gram cit­ed the “keen in­ter­est” in the ap­pli­ca­tion as the rea­son for shar­ing the up­date, but of­fered few oth­er de­tails and said the com­pa­ny is still work­ing with reg­u­la­tors on re­main­ing ques­tions.

“The di­vi­sion for­mal­ly in­formed us as well at the mid-cy­cle meet­ing that they see no sig­nif­i­cant safe­ty is­sues that have been iden­ti­fied,” he said on the call.

An in­tra­mus­cu­lar in­jec­tion, SRP-9001 is de­signed to de­liv­er a gene that will pro­duce a short­ened ver­sion of dy­s­trophin, the key pro­tein miss­ing in Duchenne pa­tients that leads to loss of mus­cle func­tions over time, which can of­ten be dead­ly. Duchenne af­fects rough­ly 1 in 3,500 male births world­wide.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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FDA ap­proves Nar­can opi­oid over­dose re­ver­sal spray for over-the-counter sale

The FDA today approved Emergent BioSolutions’ Narcan brand naloxone nasal spray for over-the-counter sales. The nod was expected and comes on the heels of a unanimous 19-0 advisory committee vote in favor of approval last month.

The move to OTC means the opioid overdose reversal agent will now be available on grocery, convenience and gas stations shelves, as well as potentially for purchase online.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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See­los Ther­a­peu­tics 'tem­porar­i­ly' stops study in rare neu­ro dis­or­der for busi­ness rea­sons

Microcap biotech Seelos Therapeutics is halting enrollment of its study in spinocerebellar ataxia type 3 (also known as Machado-Joseph disease) because of “financial considerations,” and in order to focus on other studies, the company said today, adding that the pause would be temporary.

The study will continue with the patients who have already enrolled, and the data from them will be used to decide whether to continue enrolling others in the future.

Alec­tor cuts 11% of work­force as it dou­bles down on late-stage neu­ro pro­grams part­nered with GSK, Ab­b­Vie

A month after revealing plans to concentrate on its late-stage immuno-neurology pipeline, Alector is trimming its headcount by 11%.

The layoffs will impact around 30 employees across the organization, the company disclosed in an SEC filing, adding that the plan will “better align the company’s resources” with the new strategy. With $712.9 million in cash, cash equivalents and investments as of the end of 2022, Alector believes the reserves will now get it through 2025.

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Hugo Peris, Spiral Therapeutics CEO

Hear­ing-fo­cused biotech grabs trio of pro­grams from Oton­o­my's fire sale

Otonomy may be shutting down, but the lessons learned there will live on at another biotech working on new treatments for hearing loss.

San Francisco-based Spiral Therapeutics has bought certain assets related to three of Otonomy’s programs, ranging from data, patent rights, and know-how to inventory. That includes data around Otonomy’s twice-failed lead program, OTO-104 (Otividex), a sustained-exposure formulation of dexamethasone.

CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.