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.

IDC: Life Sci­ences Firms Must Em­brace Dig­i­tal Trans­for­ma­tion Now

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

Paul Hudson, Sanofi CEO (Eric Piermont/AFP via Getty Images)

Up­dat­ed: Hit by an­oth­er PhI­II flop, Sanofi culls breast can­cer drug — sound­ing alarm for the class

Sanofi is officially giving up on its oral SERD.

The French drugmaker put out word Wednesday morning that it will discontinue the global development program of amcenestrant, the selective estrogen receptor degrader once billed as a top late-stage prospect. Having already failed a Phase II monotherapy test earlier this year, a combo with the drug also missed the bar in a second trial for breast cancer, triggering the decision to drop the whole program.

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Tom Barnes, Orna Therapeutics CEO

UP­DAT­ED: 'We have failed to fail': Mer­ck gam­bles $250M cash on a next-gen ap­proach to mR­NA — af­ter punt­ing its big al­liance with Mod­er­na

Merck went in deep on its collaboration with Moderna on new mRNA programs, and dropped them all over time, including their RSV partnership. But after writing off what turned out as one of the most successful infectious disease players in the business, Merck is coming in this morning with a new preclinical alliance — this time embracing a biotech that hopes to eventually outdo the famously successful mRNA in a new run at vaccines and therapeutics.

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Kate Haviland, Blueprint Medicines CEO

Blue­print met all its end­points in bid for ex­pand­ed Ay­vak­it la­bel — but stock trends low­er any­way

Blueprint Medicines announced this morning that the second part of its study on Ayvakit in non-advanced systemic mastocytosis (SM) — a rare disease in which a type of white blood cells known as mast cells builds up — met all endpoints, but the biopharma left key questions unanswered.

In 212 patients, with 141 in the treatment arm and 71 in the control arm, patients who got Ayvakit saw an average 15.6-point decrease in their symptom scores compared to a 9.2-point decrease in the placebo arm at 24 weeks. In an extension study, those on Ayvakit saw their symptom scores drop by 20.2 points by week 48.

Bayer's first DTC ad campaign for chronic kidney disease drug Kerendia spells out its benefits

Bay­er aims to sim­pli­fy the com­plex­i­ties of CKD with an ABC-themed ad cam­paign

Do you know the ABCs of CKD in T2D? Bayer’s first ad campaign for Kerendia tackles the complexity of chronic kidney disease with a play on the acronym (CKD) and its connection to type 2 diabetes (T2D).

Kerendia was approved last year as the first and only non-steroidal mineralocorticoid receptor antagonist to treat CKD in people with type 2 diabetes.

In the TV commercial launched this week, A is for awareness, B is for belief and C is for cardiovascular, explained in the ad as awareness of the connection between type 2 and kidney disease, belief that something can be done about it, and cardiovascular events that may be reduced with treatment.

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James Mock, incoming CFO at Moderna

Mod­er­na taps new CFO from PerkinElmer af­ter for­mer one-day CFO oust­ed

When Moderna hired a new CFO last year,  it didn’t expect to see him gone after only one day. Today the biotech named his — likely much more vetted — replacement.

The mRNA company put out word early Wednesday that after the untimely departure of then brand-new CFO Jorge Gomez, it has now found a replacement in James Mock, the soon-to-be former CFO at diagnostics and analytics company PerkinElmer.

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Etleva Kadilli, director of UNICEF’s supply division

GSK lands first-ever UNICEF con­tract for malar­ia vac­cine worth $170M

GSK has landed a new first from UNICEF the first-ever contract for malaria vaccines, worth up to $170 million for 18 million vaccine doses distributed over the next three years.

The vaccine, known as Mosquirix or RTS,S, won WHO’s backing last October after a controversial start, but UNICEF said these doses will potentially save thousands of lives every year.

“We hope this is just the beginning,” Etleva Kadilli, director of UNICEF’s supply division, said. “Continued innovation is needed to develop new and next-generation vaccines to increase available supply, and enable a healthier vaccine market. This is a giant step forward in our collective efforts to save children’s lives and reduce the burden of malaria as part of wider malaria prevention and control programmes.”

Joe Jonas (Photo by Anthony Behar/Sipa USA)(Sipa via AP Images)

So­lo Jonas broth­er car­ries Merz's new tune in Botox ri­val cam­paign

As the lyrics of his band’s 2019 pop-rock single suggest, Joe Jonas is only human — and that means even he gets frown lines. The 33-year-old singer-songwriter is Merz’s newest celebrity brand partner for its Botox rival Xeomin, as medical aesthetics brands target a younger audience.

Merz kicked off its “Beauty on Your Terms” campaign on Tuesday, featuring the Jonas brother in a video ad for its double-filtered anti-wrinkle injection Xeomin.

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Marisol Peron, Genmab SVP of communications and corporate affairs

Gen­mab launch­es cor­po­rate cam­paign am­pli­fy­ing its ‘knock your socks off’ an­ti­bod­ies

Genmab often talks about its “knock-your-socks-off” antibodies — and now the term is getting its own logo and corporate campaign.

The teal and purple logo for the acronym KYSO — Genmab pronounces it “ky-so” — debuts on Wednesday and comes on the heels of Genmab’s newly announced 2030 vision. That aspiration aims to expand Genmab’s drug development beyond oncology to include other serious diseases, while also doubling down on its own drug development.

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