Doug Ingram, Sarepta CEO

In re­ver­sal, Sarep­ta to face FDA’s pan­el of out­side ex­perts on Duchenne gene ther­a­py

Sarep­ta now will have a pub­lic hear­ing with the FDA’s pan­el of out­side ex­perts on its Duchenne mus­cu­lar dy­s­tro­phy gene ther­a­py, it said Thurs­day af­ter­noon. The an­nounce­ment comes just two weeks af­ter Sarep­ta said the FDA told the com­pa­ny it had no plans for an ad­vi­so­ry com­mit­tee meet­ing.

A date has not been set for the ad­comm meet­ing, but it will be held some­time be­fore the FDA’s dead­line of May 29 to de­cide whether or not to grant the ther­a­py ac­cel­er­at­ed ap­proval. In an in­vestor call, Sarep­ta CEO Doug In­gram em­pha­sized that the de­ci­sion was not re­lat­ed to any new da­ta or analy­sis.

The meet­ing, he said, will like­ly fo­cus on the clin­i­cal tri­al’s sur­ro­gate end­point — ex­pres­sion of dy­s­trophin, an en­zyme that pro­tects mus­cles when they ex­pand and con­tract that peo­ple with Duchenne do not make func­tion­al ver­sions of, lead­ing their mus­cles to at­ro­phy over time. The ther­a­py, known as SRP-9001, de­liv­ers a gene that en­codes for a short­ened ver­sion of dy­s­trophin and is meant to be a long-term treat­ment for the dis­ease.

“I think the fact that this is one of the first times that this ac­cel­er­at­ed ap­proval path­way and sur­ro­gate end­points are be­ing used for an in vi­vo gene ther­a­py plays some role in this de­ci­sion, and then some part of it just re­lates to the fact that the di­vi­sion does have the right to change its mind on things. And I think that, you know, that hap­pened here,” In­gram said dur­ing the call.

When the FDA ac­cept­ed Sarep­ta’s ap­pli­ca­tion for ac­cel­er­at­ed ap­proval in No­vem­ber, the com­pa­ny had said that an ad­comm was ex­pect­ed.

Sarep­ta has three oth­er drugs for Duchenne mus­cu­lar dy­s­tro­phy that were all grant­ed ac­cel­er­at­ed ap­proval based on sur­ro­gate end­points. None have been con­vert­ed to a full ap­proval as their con­fir­ma­to­ry tri­als are still run­ning. The three pre­vi­ous drugs are in­jectable drugs that tar­get cer­tain mu­ta­tions of Duchenne.

In an emailed state­ment, FDA CBER spokesper­son Paul Richards said a de­ter­mi­na­tion was made late in the re­view process that in­put from the agency’s gene ther­a­py ad­vi­so­ry com­mit­tee would be im­por­tant to ob­tain.

“We rec­og­nize the tremen­dous in­ter­est in Sarep­ta Ther­a­peu­tics’ gene ther­a­py to treat in­di­vid­u­als with Duchenne Mus­cu­lar Dy­s­tro­phy,” said the state­ment. “We have there­fore worked ex­pe­di­tious­ly to sched­ule an ad­vi­so­ry com­mit­tee meet­ing, which will be an­nounced in the Fed­er­al Reg­is­ter in the near fu­ture, in or­der to fa­cil­i­tate the eval­u­a­tion of the safe­ty and ef­fec­tive­ness of the prod­uct in a time­ly man­ner.”

The flip-flop on whether to hold an ad­comm comes as the FDA faces a num­ber of changes. The FDA’s Of­fice of Tis­sues and Ad­vanced Ther­a­pies, or OTAT, has turned in­to the Of­fice of Ther­a­peu­tic Prod­ucts, or OTP, un­der PDU­FA VII. The new of­fice will have more funds to work with and will be ex­pand­ing its work­force, and out­go­ing OTAT di­rec­tor Wil­son Bryan said that a ma­jor re­or­ga­ni­za­tion was un­der­way.

Ac­cord­ing to In­gram, OTAT said there were no plans for an ad­comm, but OTP re­versed course in the re­cent meet­ing.

In ad­di­tion, FDA’s neu­ro­science chief Bil­ly Dunn, who had a hand in the ap­proval of the pre­vi­ous Duchenne drugs, left the reg­u­la­to­ry agency at the end of Feb­ru­ary. The first Duchenne treat­ment, Sarep­ta’s Ex­ondys 51, was ap­proved by the FDA af­ter an ad­vi­so­ry com­mit­tee vot­ed against it.

Sarep­ta’s stock $SRPT fell 20% in af­ter hours trad­ing.

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.

Lu­pus drug de­vel­op­ment mar­ket heat­ing up, while FDA links with ad­vo­ca­cy group to fur­ther ac­cel­er­ate re­search

The long-underserved systemic lupus erythematosus (SLE) market is suddenly buzzing with treatment possibilities. Less than two years after AstraZeneca’s approval for Saphnelo — the first new SLE drug in a decade and joining just one other approved in GSK’s Benlysta – the pipeline of potential drugs numbers in the dozens.

Although most are very early stage — Spherix Global Insights estimates five in Phase II/III — the pharma R&D enthusiasm is catching on among doctors, patients and advocacy groups. On Wednesday, the Lupus Research Alliance and the FDA formed a novel private-public partnership called Lupus Accelerating Breakthroughs Consortium (Lupus ABC) to help advance lupus clinical trial success.

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Mass­a­chu­setts judge dis­miss­es law­suit against Bio­gen over failed launch of Alzheimer's drug Aduhelm

A Massachusetts federal judge on Wednesday dismissed a class action lawsuit filed by investors against Biogen and several of its current and former executives over the company’s failed Alzheimer’s drug, Aduhelm (aducanumab).

The investors argued that Biogen’s contact with the FDA was unlawful and that the company made 25 false and misleading statements, including statements about the rollout and price of the drug.

Af­ter safe­ty re­view, EMA mir­rors FDA with up­dat­ed rec­om­men­da­tions for JAK in­hibitors

The EMA released updated recommendations today for the use of JAK inhibitors (JAKi) after reviewing data from several clinical trials that showed increased incidents of issues in certain patients who have rheumatoid arthritis and other risk factors.

The EMA noted malignancy, major adverse cardiovascular events (MACE), serious infections, venous thromboembolism (VTE) and mortality in some patients.

Stéphane Bancel, Moderna CEO (AP Photo/Markus Schreiber)

Mod­er­na so­lid­i­fies deal with Kenya to build mR­NA man­u­fac­tur­ing fa­cil­i­ty

The mRNA player Moderna is further cementing its presence on the African continent.

Moderna announced on Thursday that it has finalized an agreement with Kenya’s government to partner up and bring an mRNA manufacturing facility to the east African nation. The new facility aims to manufacture up to 500 million doses of vaccines annually. Moderna also said the new facility will have the ability to spike its production capabilities to respond to public health emergencies on the continent or globally.

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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Sulagna Bhattacharya, Nanoscope Therapeutics CEO

Nanoscope’s eye dis­ease gene ther­a­py shows mixed re­sults in PhII

Dallas-based biotech Nanoscope Therapeutics unveiled Phase II results on its gene therapy for a rare eye disease Thursday morning.

In the RESTORE trial, 18 patients with retinitis pigmentosa got a gene therapy called MCO-010 while nine got placebo. On a vision test called the MLYMT, the treatment group had a one-point greater change over one year in their score compared to the placebo group, the primary endpoint of the study. However, the 95% confidence interval was 0.0 to 3.0, meaning the result was not statistically significant. The p-value was not provided.

Ivana Magovčević-Liebisch, Vigil Neuroscience CEO

FDA lifts par­tial clin­i­cal hold on Vig­il Neu­ro­science's TREM2 an­ti­body, re­mov­ing dos­ing cap

When Vigil Neuroscience filed its IPO papers in late 2021, the biotech revealed that the FDA had just cleared its Phase I trial — but with a partial clinical hold that limited dosing to under a certain level.

More than a year later, the FDA has lifted the hold.

Vigil is now free to dose VGL101, an antibody targeting TREM2, at levels higher than 20 mg/kg in its ongoing and future clinical trials in patients with adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP), an inherited condition that affects the brain and spinal cord.

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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