Joshua Brumm, Dyne Therapeutics CEO

An­oth­er Duchenne clin­i­cal hold gets lift­ed, this time at Dyne Ther­a­peu­tics

Clin­i­cal holds have ham­pered a host of tri­als test­ing treat­ments for Duchenne mus­cu­lar dy­s­tro­phy, but one biotech is get­ting the go-ahead to start its study five and a half months af­ter the ini­tial road­block.

The FDA cleared Dyne Ther­a­peu­tics’ first clin­i­cal tri­al in boys with a cer­tain type of Duchenne mus­cu­lar dy­s­tro­phy, the Mass­a­chu­setts biotech said Tues­day morn­ing, send­ing shares $DYN up near­ly 10% be­fore the open­ing bell. The reg­u­la­tor had re­quest­ed more clin­i­cal and non-clin­i­cal in­for­ma­tion back in Jan­u­ary, but the biotech has been mum on de­tails of what ex­act­ly the agency want­ed to look at be­fore sign­ing off on the Phase I/II tri­al.

In the months since the hold, Dyne has re­peat­ed­ly said it ex­pects to start the study in mid-2022, and the com­pa­ny re­it­er­at­ed that time­line Tues­day with the hold’s lift. The sec­ond half of the year be­gan mere days ago.

Dyne’s can­di­date, dubbed DYNE-251, will be test­ed in 30 to 50 boys aged 4 to 16 with Duchenne who have mu­ta­tions amenable to ex­on 51 skip­ping ther­a­py. Sarep­ta Ther­a­peu­tics mar­kets a drug, Ex­ondys 51, for the spe­cif­ic ex­on, among oth­er treat­ments avail­able for the pro­gres­sive mus­cle weak­ness dis­or­der.

The lift marks a key mo­ment for Dyne as it nears the clin­ic with DYNE-251 and my­oton­ic dy­s­tro­phy type 1 as­set DYNE-101, both slat­ed for mid-2022 tri­al starts. Once DYNE-251 en­ters hu­man stud­ies, Dyne hopes to progress ad­di­tion­al work in oth­er DMD ex­ons, in­clud­ing 53, 45 and 44, as part of its broad­er am­bi­tions to com­pete against oth­er DMD treat­ment mak­ers.

“To­day marks a sig­nif­i­cant step in our jour­ney to build a DMD fran­chise to serve peo­ple across the globe with Duchenne mu­ta­tions amenable to ex­on skip­ping. The clear­ance of our first IND is an im­por­tant achieve­ment for Dyne, and we ap­pre­ci­ate the part­ner­ship with the FDA through­out this process,” Dyne pres­i­dent and CEO Joshua Brumm said in a state­ment.

While there are a few treat­ments on the mar­ket for DMD from the likes of Sarep­ta, PTC Ther­a­peu­tics and an at­tempt from now-de­funct Marathon Phar­ma­ceu­ti­cals at turn­ing a cheap over­seas steroid in­to a steep stick­er price drug, the DMD com­mu­ni­ty has run in­to many hur­dles over the years in get­ting ad­di­tion­al ap­proved ther­a­pies.

Pfiz­er, in its at­tempt to catch up to Sarep­ta, was able to re­sume its Phase III gene ther­a­py tri­al in late April and, at the time, was on track to sub­mit for FDA ap­proval in late 2023. But oth­er bio­phar­mas have run in­to holds and elon­gat­ed paths to ap­proval in the months since.

Just last week, San­thera Phar­ma­ceu­ti­cals said its at­tempt at get­ting a green light for its DMD treat­ment has been pushed back four to six months. Two weeks ago, Sarep­ta’s next-gen Duchenne as­set got hit by a clin­i­cal hold in Part B of a Phase II study. Al­so last month, PTC’s years-long shot at get­ting the FDA to ap­prove its Eu­ro­pean-nod­ded DMD drug Translar­na hit an­oth­er set­back with mixed da­ta from a con­fir­ma­to­ry study.

One biotech, though, has pro­vid­ed some pos­i­tive news to the field in re­cent weeks, with Capri­cor Ther­a­peu­tics say­ing its DMD drug notched the pri­ma­ry end­point in an open-la­bel ex­ten­sion to its Phase II study.

Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

I hope your weekend is off to a nice start, wherever you are reading this email. As for me, I’m trying to catch the tail of the Lunar New Year festivities.

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Pfiz­er lays off em­ploy­ees at Cal­i­for­nia and Con­necti­cut sites

Pfizer has laid off employees at its La Jolla, CA, and Groton, CT sites, according to multiple LinkedIn posts from former employees.

The Big Pharma confirmed to Endpoints News it has let go of some employees, but a spokesperson declined to specify how many workers were impacted and the exact locations affected. Earlier this month, the drug developer had confirmed to Endpoints it was sharpening its focus and doing away with some early research on areas such as rare disease, oncology and gene therapies.

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Jake Van Naarden, Loxo@Lilly CEO

Lil­ly en­ters ripe BTK field with quick FDA nod in man­tle cell lym­phoma

Eli Lilly has succeeded in its attempt to get the first non-covalent version of Bruton’s tyrosine kinase, or BTK, inhibitors to market, pushing it past rival Merck.

The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

Filip Dubovsky, Novavax CMO

No­vavax gets ready to take an­oth­er shot at Covid vac­cine mar­ket with next sea­son plans

While mRNA took center stage at yesterday’s FDA vaccine advisory committee meeting, Novavax announced its plans to deliver an updated protein-based vaccine based on new guidance.

Vaccines and Related Biological Products Advisory Committee (VRBPAC) members voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all future vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

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CBER Director Peter Marks (Susan Walsh/AP Images)

FDA ad­vi­so­ry com­mit­tee votes unan­i­mous­ly in fa­vor of bi­va­lent Covid shots re­plac­ing pri­ma­ry se­ries

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all current vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

The vote marks an effort to clear up confusion around varying formulations and dosing schedules for current primary series and booster vaccines, as well as “get closer to the strains that are circulating,” according to committee member Paul Offit, professor of pediatrics at the Children’s Hospital of Philadelphia.

FDA ap­proves an­oth­er in­di­ca­tion for Keytru­da, this time in the ad­ju­vant NSCLC set­ting

Merck’s blockbuster cancer treatment Keytruda has been handed another indication by the FDA.

The US regulator announced on Thursday that it has approved Keytruda to serve as an adjuvant treatment for non-small cell lung cancer (NSCLC), which is its fifth indication in NSCLC and 34th indication overall.

According to a Merck release, the approval is based on data from a Phase III trial, dubbed Keynote-091, which measured disease-free survival in patients who received chemotherapy following surgery. The data from Merck displayed that Keytruda cut down on the risk of disease recurrence or death by 27% versus placebo.

No­var­tis' ap­proved sick­le cell dis­ease drug fails to beat place­bo in PhI­II

Novartis’ sickle cell drug, approved in 2019 and branded as Adakveo, has failed an ongoing Phase III, according to preliminary results.

The Swiss pharma giant unveiled early data from the ongoing STAND Phase III study on Friday, saying that crizanlizumab showed no statistically significant difference between the drug at two different dose levels compared to placebo in annualized rates of vaso-occlusive crises that lead to a healthcare visit over the first year since being randomized into the trial.

Post-hoc analy­sis: EMA's CHMP re­jects Ipsen's po­ten­tial drug for rare ge­net­ic dis­ease

The European Medicines Agency’s Committee for Medicinal Products for Human Use on Friday rejected Ipsen Pharma’s potential treatment for a rare genetic disease known as fibrodysplasia ossificans progressiva (FOP), which causes extra bone to form outside the skeleton.

The EMA said on its website that it could not draw any firm conclusions on the benefits of the French biopharma’s Sohonos (palovarotene), which selectively targets the retinoic-acid receptor gamma (RARγ), “as the applicant’s conclusion was based on a post-hoc analysis which was neither scientifically nor clinically justified and pre-specified study objectives were not met.”

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Steve Harr, Sana Biotechnology CEO

Four years in, Sana gets first FDA go-ahead to bring can­cer treat­ment in­to the clin­ic

Sana Biotechnology is finally headed to the clinic.

Thursday afternoon, the biotech announced the FDA had cleared its application to start a clinical trial for its allogeneic, or “off-the-shelf,” CAR-T cell therapy targeting the antigen CD19 for patients with B-cell lymphomas and leukemias. Sana said its therapy, dubbed SC291, was designed to evade the immune system, which could help cell therapy produce a more durable response in patients, a concern that has followed such off-the-shelf therapies that use donor cells as opposed to a patient’s own cells.

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