Amy­lyx to move for­ward with ALS pro­gram in Eu­rope, but FDA wants an­oth­er look; Hu­ma­cyte adds $50M in debt fi­nanc­ing

Amy­lyx is one of sev­er­al com­pa­nies look­ing to break through in the tough ALS field, and Wednes­day they an­nounced they’re mov­ing for­ward with reg­u­la­to­ry plans.

The Cam­bridge, MA-based biotech said they’re sub­mit­ting a mar­ket­ing ap­pli­ca­tion to the EMA for their AMX0035 pro­gram by the end of 2021. Wednes­day’s news comes a few weeks af­ter they re­vealed sim­i­lar plans to move for­ward with Cana­di­an health reg­u­la­tors by June 30.

In the US, how­ev­er, the FDA has re­quest­ed the com­pa­ny con­duct an ad­di­tion­al tri­al be­fore it sub­mits an NDA. As such, Amy­lyx is ex­pect­ing to launch a Phase III study for the can­di­date and be­gin en­rolling in the third quar­ter.

Amy­lyx’s EMA sub­mis­sion comes on the heels of a 137-per­son tri­al that mea­sured the rate at which pa­tients de­clined us­ing a clin­i­cal­ly val­i­dat­ed test. The av­er­age pa­tient start­ed at a base­line of 36 on the 48-point scale, and those in the ac­tive arm saw an av­er­age de­cline of 1.24 points per month. The place­bo group, mean­while, did so at an av­er­age of 1.66 points per month.

In all, the pa­tients in the tri­al arm scored on av­er­age 2.32 points high­er than place­bo af­ter 24 weeks, good for a p-val­ue of p=0.03.

The news of the FDA de­lay has al­ready prompt­ed crit­i­cism from the promi­nent ad­vo­ca­cy group, ALS As­so­ci­a­tion. In a state­ment, the group said the FDA is be­ing too cau­tious giv­en the ex­treme need for treat­ments, not­ing the agency has the abil­i­ty to con­di­tion­al­ly ap­prove treat­ments based on one clin­i­cal tri­al but is choos­ing not to do so here. That stands in con­trast, they say, to the ap­proach­es be­ing tak­en by Cana­da and the EU. — Max Gel­man

Hu­ma­cyte picks up $50M in debt fi­nanc­ing

Two months af­ter lin­ing up a $175 mil­lion SPAC deal to jump to Nas­daq, Hu­ma­cyte has added $50 mil­lion in debt fi­nanc­ing to fu­el its work on im­plantable bio­engi­neered tis­sue.

The Durham, NC-based biotech took the first $20 mil­lion of that debt, which was pro­vid­ed by Sil­i­con Val­ley Bank, and can draw down the rest as need­ed.

“This fi­nanc­ing fur­ther strength­ens Hu­ma­cyte’s fi­nan­cial and op­er­a­tional flex­i­bil­i­ty as we ad­vance the broad de­vel­op­ment of our pipeline, pre­pare for near-term Phase III da­ta read­outs of our po­ten­tial first-in-class Hu­man Acel­lu­lar Ves­sels in ar­te­ri­ove­nous vas­cu­lar ac­cess and vas­cu­lar trau­ma, and pre­pare to be­come a pub­licly trad­ed com­pa­ny,” said Hu­ma­cyte CEO Lau­ra Nikla­son.

The Al­pha Health­care Ac­qui­si­tion Corp. set up the SPAC. — John Car­roll

Stride­Bio part­ners with Duke for AAV gene ther­a­pies

Sarep­ta and Take­da-part­nered Stride­Bio has found an­oth­er part­ner in its quest to de­vel­op gene ther­a­pies.

The North Car­oli­na biotech is team­ing up with Duke Uni­ver­si­ty to uti­lize mul­ti­ple tech­nolo­gies to ad­vance their pro­grams, with an ini­tial pro­gram tar­get­ing the pe­di­atric neu­ro­log­i­cal dis­or­der al­ter­nat­ing hemi­ple­gia of child­hood. In­clud­ed in the deal are new­ly en­gi­neered AAV vec­tors from Duke re­searcher and Stride­Bio co-founder Ar­avind Asokan, the com­pa­ny said.

Stride­Bio has al­so se­cured ex­clu­sive rights for a new use of the IgG-de­grad­ing en­zyme IdeZ, in which it can po­ten­tial­ly clear neu­tral­iz­ing an­ti­bod­ies in con­junc­tion with AAV gene ther­a­py ad­min­is­tra­tion. And on top of that, it’s al­so li­censed a new AHC gene ther­a­py ap­proach from a sep­a­rate Duke re­searcher.

Un­der the agree­ment, Stride­Bio will fund the work against AHC and oth­er undis­closed tar­gets. There’s al­so a frame­work for oth­er new AAV gene ther­a­py pro­grams to be brought in­to the col­lab­o­ra­tion, with one tar­get­ing the “CNS vas­cu­la­ture” hav­ing al­ready been launched. Fi­nan­cial terms of the deal were not dis­closed. — Max Gel­man

Boehringer In­gel­heim col­lab­o­ra­tion for lung can­cer gets 5 more years

A col­lab­o­ra­tion be­tween Boehringer In­gel­heim and the Uni­ver­si­ty of Texas to ex­plore new mol­e­cules for the treat­ment of lung can­cer has been ex­tend­ed and ex­pand­ed, the com­pa­ny an­nounced.

The joint re­search will con­tin­ue for 5 more years. Boehringer teamed up with MD An­der­son Can­cer Cen­ter in 2019 to ex­plore mol­e­cules from KRAS — Kirsten rat sar­co­ma —  and TRAILR2 — TNF-re­lat­ed apop­to­sis-in­duc­ing lig­and re­cep­tor 2).

The agree­ment’s flex­i­bil­i­ty al­lows the team to ex­pand lung can­cer in­di­ca­tion pro­grams tar­get­ing the two genes. The col­lab­o­ra­tion has al­ready re­sult­ed in a pre­sen­ta­tion at the 2021 AACR An­nu­al Meet­ing and clin­i­cal tri­al ac­tiv­i­ties, the com­pa­ny said.

The part­ner­ship will help bring med­i­cines to both lung and gas­troin­testi­nal can­cer pa­tients, Boehringer’s head of can­cer re­search Nor­bert Kraut said. — Josh Sul­li­van

For­mer Ap­ple ex­ec tapped to head in­for­ma­tion for Eli Lil­ly

Eli Lil­ly will have a new chief in­for­ma­tion of­fi­cer for the first time in six years.

Dio­go Rau will join the com­pa­ny on May 17, to take the role head by 27-year com­pa­ny vet­er­an Aar­ti Shah. Shah’s re­tire­ment was an­nounced in 2020.

Rau comes from 10 years at Ap­ple, where he most re­cent­ly served as an IT ex­ec­u­tive for re­tail and on­line stores. Be­fore that, he was a part­ner with McK­in­sey & Com­pa­ny.

At Ap­ple, Rau led the de­vel­op­ment of tech­nol­o­gy sup­port­ing on­line and re­tail stores. At Eli Lil­ly, he’ll help grow the com­pa­ny’s use of da­ta and ma­chine learn­ing, CEO David Ricks said in a state­ment.

“Ma­chine learn­ing can open so many op­por­tu­ni­ties, and tech­nol­o­gy can strength­en our re­la­tion­ship with pa­tients and physi­cians,” Rau said in a state­ment. “I great­ly ad­mire Lil­ly’s mis­sion and val­ues, and I look for­ward to con­tribut­ing to the com­pa­ny’s long­stand­ing record of in­no­va­tion.” — Josh Sul­li­van

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His­toric drug pric­ing re­forms pass; Pfiz­er ac­quires GBT; The long search for non-opi­oid pain drugs; 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.

The Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

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Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

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Seagen interim CEO Roger Dansey and Daiichi Sankyo CEO Sunao Manabe

Paving the way for Mer­ck­'s buy­out, Seagen los­es ar­bi­tra­tion dis­pute with Dai­ichi over ADC tech

As Seagen awaits a final buyout offer from Merck that could be in the territory of $40 billion, Seagen revealed Friday afternoon that it lost an arbitration dispute with Daiichi Sankyo relating to the companies’ 2008 collaboration around the use of antibody-drug conjugate (ADC) technology.

But that loss likely won’t matter much when it comes to Merck’s deal.

After breaking off its pact with Daiichi in mid-2015, the two companies battled over “linker” tech — a chemical bridge between an ADC’s antibody component and the cytotoxic payload — that Seagen claims Daiichi would improve upon and implement in its current generation of ADCs.

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House pass­es his­toric drug pric­ing re­forms, lin­ing up decades-in-the-mak­ing win for Biden and De­moc­rats

The US House of Representatives today voted along party lines (all Dems voted for it), 220-207 to pass new, wide-ranging legislation that will allow Medicare drug price negotiations for the first time ever, and cap seniors’ drug expenses to $2,000 per year and seniors’ insulin costs at $35 per month.

Setting up a major victory for President Joe Biden, representatives returned from their summer recess to pass the Inflation Reduction Act, even as many noted the bill would only modestly reduce inflation.

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Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.

FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

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J&J to re­move talc prod­ucts from shelves world­wide, re­plac­ing with corn­starch-based port­fo­lio

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.

CSL is gathering its four business units under a unified brand identity strategy (Credit: CSL company site)

CSL brings Se­qirus, Vi­for un­der par­ent um­brel­la brand in iden­ti­ty re­vamp

CSL is gathering its brands under the family name umbrella, renaming its vaccine and newly acquired nephrology specialty businesses with the parent initials.

CSL Seqirus and CSL Vifor join CSL Plasma and CSL Behring as the four now uniformly branded business units of the global biopharma. The Seqirus vaccine division was formed in 2015 with the combination of bioCSL and its purchase of Novartis’ flu vaccine business. CSL picked up Vifor Pharma late last year in an $11.7 billion deal for the nephrology, iron deficiency and cardio-renal drug developer.

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