Aa­van­tiBio lands man­u­fac­tur­ing part­ner in Friedre­ich's Atax­ia pro­gram; NC Re­search Tri­an­gle lands an­oth­er CD­MO

Aa­van­tiBio and Re­silience have an­nounced a col­lab­o­ra­tion to man­u­fac­ture a pipeline of ther­a­pies, in­clud­ing Aa­van­tiBio’s Friedre­ich’s Atax­ia pro­gram, the com­pa­ny an­nounced Thurs­day.

The ade­no-as­so­ci­at­ed vi­ral vec­tor-based can­di­date is cur­rent­ly in Phase I/II tri­als in the US and Eu­rope, and Re­silience will pro­vide cell lines and vi­ral banks for the drug’s first in-hu­man tri­als. The drug will be man­u­fac­tured at Re­silience’s 183,000-square-foot fa­cil­i­ty in Alachua, FL.

Friedre­ich’s Atax­ia is an in­her­it­ed neu­ro­mus­cu­lar dis­ease that can cause ner­vous sys­tem dam­age and cause pa­tients to have trou­ble mov­ing. The dis­ease usu­al­ly starts in child­hood, and leads to the de­gen­er­a­tion in the spinal cord, pe­riph­er­al nerves and cere­bel­lum.

In a state­ment, Re­silience CEO Rahul Singhvi said:

We are ex­cit­ed to work with an in­no­v­a­tive de­vel­op­ment-stage com­pa­ny like Aa­van­tiBio who is uti­liz­ing a unique AAV ap­proach to ad­dress the un­der­ly­ing caus­es of rare ge­net­ic dis­eases. Our high­ly-ex­pe­ri­enced team will work col­lab­o­ra­tive­ly with Aa­van­tiBio on process de­vel­op­ment and an­a­lyt­i­cal test­ing to en­able a seam­less tran­si­tion from de­vel­op­ment to man­u­fac­tur­ing.

NC Re­search Tri­an­gle lands an­oth­er CD­MO

An­oth­er week, an­oth­er biotech has moved its man­u­fac­tur­ing op­er­a­tions to North Car­oli­na.

Med­Pharm, a CD­MO that spe­cial­izes in top­i­cal and trans­der­mal phar­ma­ceu­ti­cal prod­ucts, will open a new lo­ca­tion in the Raleigh-Durham area, the com­pa­ny an­nounced Tues­day.

The new fa­cil­i­ty will dou­ble its ex­ist­ing foot­print, and sup­port de­vel­op­ment, clin­i­cal and small-scale com­mer­cial man­u­fac­tur­ing. The site is just a few miles from its cur­rent of­fice, and will meet all FDA, EMA and GMP re­quire­ments, the com­pa­ny said.

In a state­ment, CEO Eu­gene Ciolfi said:

Med­Pharm’s in­vest­ment in this fa­cil­i­ty is a clear demon­stra­tion of our con­tin­ued com­mit­ment to a glob­al vi­sion by ex­pand­ing client ser­vices down­stream in­to com­mer­cial man­u­fac­tur­ing. Med­Pharm’s newest fa­cil­i­ty is an ex­ten­sion of our de­vel­op­ment ser­vices and will al­so sup­port our client base with their clin­i­cal and com­mer­cial pipelines needs for top­i­cal and trans­der­mal prod­ucts glob­al­ly.

Un­pack­ing the Aduhelm de­ci­sion, Ver­tex's half full glass, a $525M J&J breakup, 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.

By now you have surely read about the FDA’s controversial approval of Biogen’s Alzheimer’s drug and all its reverberations. But I’d still recommend checking out the meaty recap below to make sure you didn’t miss all the angles that the Endpoints team has covered. If you’d rather look ahead, look no further than our three-day virtual panels next week at BIO, where we will discuss what the new normal means for every part of the industry.

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What does a clear ma­jor­i­ty of the bio­phar­ma in­dus­try think of the FDA ap­proval of ad­u­canum­ab? 'Hor­ri­fy­ing' 'Dan­ger­ous' 'Con­fus­ing' 'Dis­as­ter'

Over the years, we’ve become used to seeing a consensus emerge early in our industry polls at Endpoints News. And when we took the pulse of drug hunters on the heels of a controversial FDA approval for aducanumab this week, it became immediately apparent that the vast majority of our readers — heavily concentrated among biopharma staffers and execs — were incensed by what they had just witnessed.

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Aaron Kesselheim (Scott Eisen/AP Images for AIDS Healthcare Foundation)

Har­vard’s Aaron Kessel­heim re­signs from ex­pert pan­el in wake of ad­u­canum­ab OK, blast­ing FDA for ‘worst drug ap­proval de­ci­sion in re­cent U.S. his­to­ry'

A third member of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has resigned in the wake of Biogen’s controversial Aduhelm approval, slamming the agency as he left and further deepening the controversy surrounding the decision.

Harvard University professor Aaron Kesselheim quit in protest Thursday afternoon, calling the Aduhelm OK “probably the worst drug approval decision in recent U.S. history.” Kesselheim follows both Joel Perlmutter, a neurologist from Washington University in St. Louis, and David Knopman, a neurologist from the Mayo Clinic, out the door.

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David Knopman (Mayo Clinic via YouTube)

A sec­ond ad­comm mem­ber aban­dons his post in af­ter­math of con­tro­ver­sial ad­u­canum­ab de­ci­sion

As the fallout from the FDA’s approval of Alzheimer’s med aducanumab grows, a second member of the adcomm overseeing that drug’s review has walked away. But even with two experts now having resigned from that committee in protest, is there enough broad-level outrage to prevent another aducanumab from getting approved?

The FDA on Wednesday lost another member of its Peripheral and Central Nervous System Drugs Advisory Committee as Mayo Clinic neurologist David Knopman hit the exit over the agency’s decision to approve Biogen’s Alzheimer’s drug Aduhelm despite the committee’s near-unanimous vote against it.

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Reshma Kewalramani, Vertex CEO (BIO via YouTube)

UP­DAT­ED: Ver­tex strikes out on its lat­est big shot at a rare ge­net­ic dis­ease. But they're go­ing to keep on swing­ing

It’s been several months since Vertex culled one of its small molecules for alpha-1 antitrypsin deficiency (AATD), taking a big hit after evidence of liver damage surfaced in a key Phase II trial. Now we learned that the company has whiffed on its second shot, and there’s nothing left in the clinic to treat the rare genetic disease — but that won’t stop it from trying.

Despite avoiding the safety issues that plagued the last candidate, Vertex $VRTX is taking the axe to VX-864 after Phase II results revealed the magnitude of the drug’s response is “unlikely to translate into substantial clinical benefit.” As a result of the news, the company’s stock fell 12.5% after hours.

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As it ex­pands its foot­print, Mod­er­na reach­es deal to man­u­fac­ture Covid-19 vac­cine dos­es in Mid­dle East

While the UAE leads the world with the highest percentage of residents vaccinated, neighboring Saudi Arabia — home to nearly 35 million people — has lagged behind significantly. On Friday, Moderna announced that it has partnered with the Saudi pharmaceutical company Tabuk to manufacture its jab and future variant-specific boosters in the country.

Tabuk will hold marketing authorization for the vaccine in Saudi Arabia, and the agreement gives them the possibility of distributing future Moderna mRNA products.

FDA au­tho­rizes about 10M J&J vac­cine dos­es, trash­es 60M more from trou­bled Emer­gent plant

The FDA on Friday released about 10 million doses of J&J’s vaccine for use, and disposed of another 60 million doses that were manufactured at the now-shuttered Emergent BioSolutions facility in Baltimore where cross-contamination occurred.

The agency said it’s not yet ready to allow the Emergent plant to be included in the J&J EUA, but that may occur soon. FDA came to the decision to authorize some of the doses after reviewing facility records and quality testing results.

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

Months af­ter FDA re­jec­tion, Sanofi touts piv­otal win for rare dis­ease drug su­tim­limab as it preps to re­file

One of the pillar drugs of Sanofi’s $11.6 billion pickup of Bioverativ hit a big setback late last year when the FDA sent its application for approval back. Now, as Sanofi gears up to resubmit the drug for review, the drugmaker is touting pivotal data it hopes will help take it over the finish line.

Sanofi’s sutimlimab nailed all three of its primary endpoints in its Phase III CADENZA study for patients with cold agglutinin disease, a rare disorder that can cause severe anemia, without a recent history of blood transfusion, the French drugmaker said Friday. The topline results will be presented at this weekend’s virtual EHA meeting.

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Sadasivan Vidyasagar (Credit: University of Florida)

An API firm with a his­to­ry of J&J fund­ing scores a ma­jor loan to ad­vance its port­fo­lio

A Massachusetts-based company that manufactures APIs closed its Series A and B fundraising, with J&J among the list of investors. Now, the company is announcing another $49 million loan to help boost operations.

Entrinsic Bioscience — or EBS — expects to use the loan to fund programs for GI, airway and skin diseases. CEO Stephen Gatto called these programs a “blueprint for serial innovation” in a press release.