Ilan Ganot, Solid Bio CEO (Solid Bio)

FDA keeps Sol­id Bio's trou­bled Duchenne MD gene ther­a­py on clin­i­cal lock­down in lat­est set­back

Much-ma­ligned Sol­id Bio­sciences re­ceived yet an­oth­er spate of bad news late Fri­day re­gard­ing its Duchenne mus­cu­lar dy­s­tro­phy gene ther­a­py pro­gram.

It’s been al­most nine months since the FDA an­nounced a sec­ond clin­i­cal hold on Sol­id Bio’s Phase I/II tri­al for an AAV-based Duchenne MD treat­ment. And af­ter ex­tend­ing that hold in May, US reg­u­la­tors again de­cid­ed they need­ed more in­fo with an­oth­er ex­ten­sion on Fri­day.

In­vestors re­act­ed some­what poor­ly to the news as the Cam­bridge, MA-based biotech’s stock price, which peaked at over $50 per share in Sep­tem­ber 2018, fell about 2.5 per­cent to $2.70 af­ter Fri­day’s clos­ing bell.

Sol­id Bio’s no­to­ri­ety dates all the way back to its IPO in Jan­u­ary 2018 when CEO Ilan Gan­ot did not dis­close its lead pro­gram was placed on a par­tial clin­i­cal hold un­til the com­pa­ny was wrap­ping up pric­ing. Around that time, the com­pa­ny al­so an­nounced that gene ther­a­py pi­o­neer James Wil­son re­signed from its sci­en­tif­ic ad­vi­so­ry board af­ter con­cerns arose in an­i­mal test­ing about se­vere tox­ic re­ac­tions, in­clud­ing liv­er dam­age, in high AAV dos­es.

A few months lat­er, the FDA placed its first clin­i­cal hold on the study af­ter the first pa­tient treat­ed ex­pe­ri­enced a “de­crease in platelet count fol­lowed by a re­duc­tion in red blood cell count, tran­sient re­nal im­pair­ment and ev­i­dence of com­ple­ment ac­ti­va­tion,” ac­cord­ing to a state­ment from the time.

But that hold was lift­ed in June 2018, as Sol­id Bio changed the study to add IV glu­co­cor­ti­coids af­ter treat­ment, amp up mon­i­tor­ing ac­tiv­i­ty and pro­vide eculizum­ab on an as-need­ed ba­sis to fight any com­ple­ment ac­ti­va­tion that may oc­cur in fu­ture.

Things seemed to be look­ing up, rel­a­tive­ly speak­ing, un­til Feb­ru­ary 2019 when the Phase I/II dose-as­cend­ing tri­al flopped in the clin­ic as biop­sies of pa­tients “showed low lev­els of mi­crody­s­trophin pro­tein ex­pres­sion.” Lat­er that year, the FDA placed its cur­rent hold on the tri­al af­ter an­oth­er pa­tient ex­pe­ri­enced se­ri­ous ad­verse ef­fects that close­ly re­sem­bled the ear­li­er set­back.

In or­der to weath­er that storm, Sol­id Bio laid off a third of its staff in­clud­ing its CMO and COO. Re­spond­ing to the hold’s ex­ten­sion in May, the biotech pro­posed clin­i­cal changes to the tri­al “de­signed to en­hance pa­tient safe­ty,” and com­mit­ted to im­prove­ments in the man­u­fac­tur­ing process.

SVB Leerink saw a sil­ver lin­ing at the time, not­ing that the FDA did not com­ment on any of the com­pa­ny’s clin­i­cal strat­e­gy pro­pos­als. The changes that were ul­ti­mate­ly made, how­ev­er, did not sat­is­fy reg­u­la­tors as the hold re­mains in place.

Gan­ot, a for­mer JP Mor­gan in­vest­ment banker, is a Duchenne MD dad and has said that has dri­ven him to find a cure for the rare, lethal dis­ease. By in­tro­duc­ing a syn­thet­ic dy­s­trophin trans­gene con­struct called mi­crody­s­trophin, via a vi­ral vec­tor, Gan­ot and Sol­id Bio hope to prove it can do what com­peti­tors like Sarep­ta and oth­ers have been try­ing for with one de­ci­sive in­ter­ven­tion.

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Warren Huff, Reata CEO

Rea­ta sug­gests Friedre­ich's atax­ia pro­gram could be de­layed, send­ing stock plung­ing

Reata Pharmaceuticals $RETA made waves last October when its drug omaveloxolone produced positive trial results in treating a rare neurological disorder, but the candidate’s path forward became much murkier Monday.

In a report of quarterly earnings, the biotech divulged that the FDA is considering delaying omaveloxolone’s NDA pending completion of a second trial. That could push back approval by at least a year given that the target population, individuals with Friedreich’s ataxia, is limited and progression of the hard-to-treat illness is notoriously slow. The Covid-19 pandemic would also hinder Reata’s ability to complete an additional trial.

DFC CEO Adam Boehler and Kodak CEO Jim Continenza (Kodak)

Covid-19 roundup: Cure­Vac beefs up its uni­corn IPO dreams as bil­lion­aire own­er takes this Covid-19 mR­NA play­er on a forced march to Nas­daq; Ko­dak's $765M deal is put on hold

When CureVac initially jotted down $100 million for its IPO raise a couple of weeks ago, it seemed small. The German mRNA player, after all, had jumped into a Covid-19 race that swelled the sails of Moderna and BioNTech by tens of billions. And after raising $640 million in a slate of deals, $100 million in a hot market like this seemed like a pittance in the bigger scheme of things.

Today, we got a look at a figure that probably comes closer to the game-changing number the top execs probably have in mind. Selling 15.3 million shares at the high end of their $14 to $16 range would net a $243 million bounty. Majority owner Dietmar Hopp is putting in another €100 million, bringing the total to around $350 million. And what are the chances they want to do even better than that?

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Eric Shaff (Seres)

UP­DAT­ED: Af­ter a 4-year so­journ, strug­gling mi­cro­bio­me pi­o­neer Seres claims a break­out PhI­II come­back. And shares re­spond in fren­zied spike

Almost exactly 4 years ago, Seres Therapeutics $MCRB experienced one of those soul-crunching failures that can raise big questions about a biotech’s future. Out front in their pursuit of a gut punch to C. difficile infection (CDI), the Phase II test was a flat failure, and investors wiped out a billion dollars of equity value that never returned in the years that followed.

Seres, though, pressed ahead, changing out CEOs a year ago — bidding Merck vet Roger Pomerantz farewell from the C suite — and pushing through a Phase III, hoping that amping up the dosage would make the key difference. And this morning, they unveiled a claim that they had aced the Phase III and positioned themselves for a run at a landmark FDA OK.

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Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

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Vi­da Ven­tures co-leads Dyne's $115M megaround for next-gen oli­go ther­a­pies aimed square­ly at mus­cles

Dyne Therapeutics started out last April with a modest $50 million to mine targeted muscle disease therapies from its in-house conjugate technology. The biotech has now convinced more investors that it’s got gems on its hands, closing $115 million in fresh financing to push its next-gen oligonucleotide drugs into the clinic.

Vida Ventures and Surveyor Capital led the round, joined by a group of other new backers including Wellington Management Company, Logos Capital and Franklin Templeton.

Eli Lil­ly teams with Pieris on HER2+ tu­mors; Op­di­vo + Yer­voy best chemo in mesothe­lioma

Despite the FDA putting a partial clinical hold on its lead program only a few weeks ago, Boston-based Pieris Pharmaceuticals is plowing forward with a new collaboration.

Pieris will work with Eli Lilly to further advance studies on PRS-343, a 4-1BB/HER2 bispecific for HER2-positive tumors, in combination with the latter’s ramucirumab and paclitaxel for the second-line treatment of patients with HER2-positive gastric cancer in a single-arm, Phase II study.

In­novent and Eli Lil­ly chal­lenge Mer­ck­'s mega-block­buster Keytru­da in non-small cell lung can­cer field

China-based Innovent Biologics and its multinational ally Eli Lilly shared Phase III evidence that their PD-1 inhibitor combo can delay the progression of nonsquamous non-small cell lung cancer.

But the drugmakers will face stiff competition in China from Merck’s Keytruda, the ruling PD-1 which is already approved to treat both squamous and nonsquamous NSCLC and boasts positive overall survival rates.

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