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

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

'Chang­ing the whole game of drug dis­cov­ery': Leg­endary R&D vet Roger Perl­mut­ter leaps back in­to work as a biotech CEO

Roger Perlmutter needs no introduction to anyone remotely involved in biopharma. As the R&D chief first at Amgen and then Merck, he’s built a stellar reputation and a prolific career steering new drugs toward the market for everything from cancer to infectious diseases.

But for years, he’s also held a less known title: science partner at The Column Group, where he’s regularly consulted about the various ideas the VCs had for new startups.

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UP­DAT­ED: Pfiz­er hits the brakes on their piv­otal tri­al for a BC­MA/CD3 bis­pe­cif­ic on safe­ty con­cerns while FDA road­block is hold­ing up Duchenne MD PhI­II

Pfizer’s ambitious plan to take a Phase II study of its BCMA CD3-targeted bispecific antibody elranatamab (PF-06863135) and run it through to an accelerated approval has derailed.

The pharma giant said in a release this morning that they have halted enrollment for their MagnetisMM-3 study after researchers tracked three cases of peripheral neuropathy in the ongoing Phase I. They are now sharing info with the FDA as they explore the red safety flag.

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FDA ex­tends re­search agree­ment with MIT-li­censed or­gan-on-chip sys­tems

The FDA on Wednesday extended its four-year agreement with CN Bio, a developer of single- and multi-organ-on-chip systems used for drug discovery, for another three years.

CN Bio said the scope of the research performed by the FDA’s Center for Drug Evaluation and Research has expanded to include the exploration of the company’s lung-on-a-chip system to help with the agency’s evaluation of inhaled drugs, in addition to the agency’s work on its liver model.

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In quest to meet user fee goals, FDA’s per­for­mance con­tin­ues down­ward trend

A recent update to the FDA’s running tally of how it’s meeting its user fee-related performance goals during the pandemic shows an agency that is not out of the woods yet.

The latest numbers reveal that for a second straight quarter in 2021, the FDA has met its user fee goal dates for 93% of original new drug applications, which compares with 94% and 98% for the previous two quarters in 2020, respectively.

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Cynthia Butitta (L) and Joe Jimenez

Is that an­oth­er IPO in the mak­ing? Ex-No­var­tis CEO Joe Jimenez and a lead Kite play­er take up new posts at an off-the-shelf ri­val to 2 pi­o­neer­ing drugs

Right on the heels of taking on a $160 million crossover round in a likely leap to Nasdaq, Century Therapeutics CEO Lalo Flores is now pushing ahead with the high-profile ex-Novartis chief Joe Jimenez as chairman.

Jimenez’s greatest fame at Novartis was earned for one of its weakest products, as their pioneering personalized CAR-T Kymriah won the honors for the first such drug to make it to the market. Now a host of players, including Century, are barreling in behind the frontrunners with allogeneic rivals that can be created for off-the-shelf use.

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Clo­vis spikes ovar­i­an can­cer com­bo af­ter 'un­der­whelm­ing' ef­fi­ca­cy; Servi­er wins pri­or­i­ty re­view for Tib­so­vo ex­pan­sion

Clovis Oncology updated investors with its first quarter results on Wednesday morning, and among its revelations proved some troubling news for a Bristol Myers Squibb-partnered program.

Pairing its experimental lucitanib drug with Bristol Myers’ Opdivo in an ongoing Phase Ib/II study, Clovis reported that “underwhelming efficacy” has led them to cease development for the combo in non-clear-cell ovarian cancer, execs said in Wednesday morning’s earnings call. Enrollment is continuing in other expansion cohorts, which include non-clear cell endometrial cancer, cervical cancer and clear-cell ovarian and endometrial cancers.

Joe Wiley, Amryt CEO

A biotech with a yen for pricey rare dis­ease drugs — and bar­gain base­ment shop­ping — adopts an­oth­er or­phan in lat­est M&A pact

After making it through a long, painful haul to get past a CRL and on to an FDA approval last summer, little Chiasma has found a buyer.

Amryt $AMYT, a company known for its appetite for acquiring expensive drugs for rare diseases at bargain prices, snagged Chiasma and its acromegaly drug Mycapssa (octreotide) capsules in an all-stock deal — with an exchange of 0.396 shares of Amryt for every share of Chiasma.

Ngozi Okonjo-Iweala, Director general of WTO (AP Photo/Keystone/Alessandro Della Bella)

Opin­ion: Waiv­ing Covid-19 vac­cine IP could save lives, but where is the man­u­fac­tur­ing ca­pac­i­ty?

Droves of House Democrats in Washington and members of the European Parliament have now glommed onto a major push by India and South Africa at the WTO to abolish all IP around Covid-19 vaccines.

At first blush, waiving this IP sounds like an easy win: More Covid-19 vaccines made locally for more people means more lives saved. Simple enough, especially as low-income countries have received just a tiny fraction of the world’s vaccine allotment so far.