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

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Albert Bourla, Pfizer CEO (Efren Landaos/Sipa USA/Sipa via AP Images)

Pfiz­er makes an­oth­er bil­lion-dol­lar in­vest­ment in Eu­rope and ex­pands again in Michi­gan

Pfizer is continuing its run of manufacturing site expansions with two new large investments in the US and Europe.

The New York-based pharma giant’s site in Kalamazoo, MI, has seen a lot of attention over the past year. As a major piece of the manufacturing network for Covid-19 vaccines and antivirals, Pfizer is gearing up to place more money into the site. Pfizer announced it will place $750 million into the facility, mainly to establish “modular aseptic processing” (MAP) production and create around 300 jobs at the site.

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Vas Narasimhan, Novartis CEO (Thibault Camus/AP Images, Pool)

No­var­tis bol­sters Plu­vic­to's case in prostate can­cer with PhI­II re­sults

The prognosis is poor for metastatic castration-resistant prostate cancer (mCRPC) patients. Novartis wants to change that by making its recently approved Pluvicto available to patients earlier in their course of treatment.

The Swiss pharma giant unveiled Phase III results Monday suggesting that Pluvicto was able to halt disease progression in certain prostate cancer patients when administered after androgen-receptor pathway inhibitor (ARPI) therapy, but without prior taxane-based chemotherapy. The drug is currently approved for patients after they’ve received both ARPI and chemo.

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Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

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Af­ter M&A fell through, Ther­a­peu­tic­sMD sells hor­mone ther­a­py, con­tra­cep­tive ring for $140M cash plus roy­al­ties

TherapeuticsMD, a women’s health company whose one-time billion-dollar valuation seems a distant memory as its blockbuster aspirations petered out, is finally cashing out.

Australia’s Mayne Pharma is paying $140 million upfront to license essentially TherapeuticsMD’s whole portfolio, including two prescription drugs that treat conditions relating to menopause, a contraceptive vaginal ring as well as its prescription prenatal vitamin brands.

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Big week for Alzheimer’s da­ta; As­traZeneca buys cell ther­a­py start­up; Dig­i­tal ther­a­peu­tics hits a pay­er wall; 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.

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Yuling Li, Innoforce CEO

In­no­force opens new man­u­fac­tur­ing site in Chi­na

Innoforce is off to the races at its new site in the city of Hangzhou, China.

The Chinese CDMO announced last week that it has started manufacturing at the new facility, which was built to offer process development and manufacturing operations for RNA, plasmid DNA, viral vectors and other cell therapeutics. It will also serve as Innoforce’s corporate HQ.

The company said it’s investing more than $200 million in the 550,000-square-foot manufacturing base for advanced therapies. The GMP manufacturing facility features space for producing plasmids with three 30-liter bioreactors. For viral vector manufacturing, Innoforce also has 200- and 500-liter bioreactors at its disposal, along with eight suites to make cell therapies. The site also includes several labs and warehouse spaces.

FDA grants or­phan drug des­ig­na­tion to Al­ger­non's ifen­prodil, while ex­clu­siv­i­ty re­mains un­clear

As the FDA remains silent on orphan drug exclusivity in the wake of a controversial court case, the agency continues to hand out new designations. The latest: Algernon Pharmaceuticals’ experimental lung disease drug ifenprodil.

The Vancouver-based company announced on Monday that ifenprodil received orphan designation in idiopathic pulmonary fibrosis (IPF), a chronic lung condition that results in scarring of the lungs.  Most IPF patients suffer with a dry cough, and breathing can become difficult.