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

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

What lured Hal Bar­ron away?; Top FDA minds on ac­cel­er­at­ed ap­proval re­forms; ‘Dead wrong’ Aduhelm ad blitz; 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.

Nothing can really compete with Hal Barron’s departure from GlaxoSmithKline as the news of the week, but we do have plenty of original reporting and analysis from the Endpoints team in this edition. Enjoy and have a nice weekend.

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Mer­ck wins le­gal bat­tle over in­sur­ance cov­er­age af­ter ran­somware at­tack

Merck has emerged victorious from a years-long legal battle with insurers over the coverage of more than a billion dollars in losses from the malware NotPetya, with a New Jersey Superior Court judge concluding that the responsibility is on insurers to clarify their policies around cyber attacks.

The pharma giant was one of several victims of a global cyber attack back in 2017 that also hit Danish shipping company Maersk, American food company Mondelēz, French construction giant Saint-Gobain and even the systems monitoring the Chernobyl nuclear power stations, Bloomberg reported back in 2019.

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Crit­ics push back on Alzheimer’s As­so­ci­a­tion ad blitz to get Medicare to change its Aduhelm rul­ing: 'Dead wrong'

The latest Alzheimer’s Association advertising campaign encourages people to fight.

Not against the disease or for more research or treatments, but against the Centers for Medicare and Medicaid Services. More specifically, CMS’ recent reimbursement decision to only pay for Biogen and Eisai’s controversial Alzheimer’s drug Aduhelm for patients in clinical trials.

With CMS’ preliminary decision now in a 30-day comment period, patient advocates’ goal is to convince CMS to reverse its decision with a marketing blitz and public pressure.

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Dan O'Day, Gilead CEO (Jim Watson/AFP via Getty Images)

Fail­ing to con­firm clin­i­cal ben­e­fit, Gilead pulls 2 ac­cel­er­at­ed ap­proval in­di­ca­tions for can­cer drug

Gilead recently decided to pull two indications for its cancer drug Zydelig — in relapsed follicular B-cell non-Hodgkin lymphoma (FL) and relapsed small lymphocytic leukemia (SLL) — after failing to complete the confirmatory trials required as part of the accelerated approvals from 2014.

“As the treatment landscape for FL and SLL has evolved, enrollment into the confirmatory study has been an ongoing challenge,” Gilead said in a statement, noting it formally notified the FDA of its decision to voluntarily withdraw these indications.

Yingli Phar­ma brings small mol­e­cule re­search to the US in new pact with MD An­der­son; UCB's pso­ri­at­ic arthri­tis drug shows pos­i­tive PhI­II re­sults

Shanghai biotech Yingli Pharma wants to bring its small molecule drug research global — and a new pact with MD Anderson will help it get there.

Yingli and MD Anderson have inked a 5-year collaboration deal that will put its cancer candidates — some of which have already generated data in China — into trials in the US. The lead program is linperlisib, a PI3Kδ inhibitor that’s in a Phase III trial in follicular lymphoma, according to Yingli’s website. In the US, MD Anderson will work with Yingli to put the candidate in a Phase II trial for peripheral T cell lymphoma (PTCL), an uncommon and aggressive type of non-Hodgkin’s lymphoma.

Richard Pazdur (via AACR)

Time lim­its on ac­cel­er­at­ed ap­provals? FDA's on­col­o­gy chief Rick Paz­dur eyes po­ten­tial re­forms via in­ter­na­tion­al ap­proach­es

The spotlight on the accelerated approval pathway continues to shine bright, with the FDA’s top oncology official writing in an opinion that the pathway may be strengthened with bits and pieces of what other regulators in Europe and elsewhere have done with their expedited approval pathways, such as adding expiration dates for these faster approvals to ensure they confirm clinical benefit in a timely manner.

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Hal Barron, Endpoints UKBIO20 (Jeff Rumans)

'Al­tos was re­al­ly a once-in-a-life­time op­por­tu­ni­ty': Hal Bar­ron re­flects on his big move

By all accounts, Hal Barron had one of the best jobs in Big Pharma R&D. He made more than $11 million in 2020, once again reaping more than his boss, Emma Walmsley, who always championed him at every opportunity. And he oversaw a global R&D effort that struck a variety of big-dollar deals for oncology, neurodegeneration and more.

Sure, the critics never let up about what they saw as a rather uninspiring late-stage pipeline, where the rubber hits the road in the Big Pharma world’s hunt for the next big near-term blockbuster, but the in-house reviews were stellar. And Barron was firmly focused on bringing up the success rate in clinical trials, holding out for the big rewards of moving the dial from an average 10% success rate to 20%.

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Executive Director of the EMA Emer Cooke (AP Photo/Geert Vanden Wijngaert)

Eu­ro­pean Par­lia­ment signs off on strength­en­ing drug reg­u­la­tor's abil­i­ty to tack­le short­ages

The European Parliament on Thursday endorsed a plan to increase the powers of the European Medicines Agency, which will be better equipped to monitor and mitigate shortages of drugs and medical devices.

By a vote of 655 to 31, parliament signed off on a provisional agreement reached with the European Council from last October, in which the EMA will create two shortage steering groups (one for drugs, the other for devices), a new European Shortages Monitoring Platform to facilitate data collection and increase transparency, and on funding for the work of the steering groups, task force, working parties and expert panels that are to be established.

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