House com­mit­tee ad­vances bills aimed at or­phan ex­clu­siv­i­ty, gener­ic la­bel­ing and con­tin­u­ous man­u­fac­tur­ing

The House En­er­gy and Com­merce Com­mit­tee on Wednes­day ad­vanced 17 health-re­lat­ed bills for con­sid­er­a­tion by the House, sev­er­al of which have im­pli­ca­tions for the FDA’s over­sight of or­phan drugs, im­port­ed med­ical de­vices and la­bel­ing.

Frank Pal­lone

“These bills will ex­pand cov­er­age and ac­cess to care, strength­en men­tal health par­i­ty, pro­vide men­tal health sup­port to first re­spon­ders, en­hance trans­paren­cy and op­er­abil­i­ty of the Strate­gic Na­tion­al Stock­pile and im­prove the safe­ty of Amer­i­ca’s food, drugs and med­ical de­vices,” said Com­mit­tee Chair­man Frank Pal­lone (D-NJ).

One of the bills, H.R. 4712, the Fair­ness in Or­phan Drug Ex­clu­siv­i­ty Act, would re­quire drug­mak­ers seek­ing or­phan ex­clu­siv­i­ty for a prod­uct be­cause it would not be prof­itable to demon­strate that there is no rea­son­able ex­pec­ta­tion that they will re­coup the cost of de­vel­op­ing the drug with­in 12 years of mar­ket­ing in or­der to ob­tain or re­tain or­phan drug ex­clu­siv­i­ty.

An­oth­er bill, H.R. 5663, the Safe­guard­ing Ther­a­peu­tics Act, would give FDA the au­thor­i­ty to de­stroy im­port­ed adul­ter­at­ed, mis­brand­ed or un­ap­proved med­ical de­vices that it deems to be a threat to pub­lic health. The new au­thor­i­ty would build on the agency’s au­thor­i­ty to de­stroy adul­ter­at­ed, mis­brand­ed or coun­ter­feit drugs un­der the Food and Drug Ad­min­is­tra­tion Safe­ty and In­no­va­tion Act.

FDA would gain an­oth­er new au­thor­i­ty un­der H.R. 5668, the Mak­ing Ob­jec­tive Drug Ev­i­dence Re­vi­sions for New La­bel­ing Act of 2020, to re­quire drug­mak­ers to up­date the la­bel­ing of old­er gener­ic drugs when new sci­en­tif­ic ev­i­dence is avail­able re­gard­ing the drugs’ use, to re­flect ac­cept­ed us­es of the drug in clin­i­cal prac­tice that are not cur­rent­ly re­flect­ed in the ap­proved la­bel­ing and when the la­bel­ing does not meet cur­rent le­gal and reg­u­la­to­ry re­quire­ments.

Ad­di­tion­al­ly, H.R. 4866, the Na­tion­al Cen­ters of Ex­cel­lence in Con­tin­u­ous Phar­ma­ceu­ti­cal Man­u­fac­tur­ing Act of 2019, would di­rect FDA to des­ig­nate na­tion­al cen­ters of ex­cel­lence in con­tin­u­ous phar­ma­ceu­ti­cal man­u­fac­tur­ing that would work with the agency and in­dus­try “to craft a na­tion­al frame­work for con­tin­u­ous man­u­fac­tur­ing im­ple­men­ta­tion.” The bill would pro­vide $80 mil­lion in ap­pro­pri­a­tions for des­ig­nat­ed cen­ters of ex­cel­lence each year from FY2021-2025.

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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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