News brief­ing: Ax­o­vant faces months of de­lay on lead Parkin­son's gene ther­a­py; Chi­nese CAR-T biotech nabs $100M

One of Ax­o­vant’s top gene ther­a­py prospects for its sec­ond act is hit­ting a road­block that could push its clin­i­cal time­lines back by al­most a year.

In an up­date, the biotech said it was in­formed about de­lays in CMC da­ta and third-part fill-fin­ish is­sues around mid-Oc­to­ber by its man­u­fac­tur­ing part­ner, Ox­ford Bio­med­ica. Ax­o­vant has been de­vel­op­ing a sus­pen­sion-based process for the Parkin­son’s drug; with that tak­ing longer than ex­pect­ed, it now be­lieves “it is un­like­ly that its planned ran­dom­ized, sham-con­trolled tri­al of AXO-Lenti-PD will en­roll pa­tients by the end of cal­en­dar year 2021.”

It had pre­vi­ous­ly ex­pect­ed the man­u­fac­tur­ing to be com­plete by the end of the year and to en­roll the first pa­tient by ear­ly 2021.

Ax­o­vant had li­censed the ther­a­py, AXO-Lenti-PD, from Ox­ford Bio­med­ica for $30 mil­lion up­front. Com­pris­ing a lentivi­ral vec­tor, It’s de­signed to de­liv­er three genes — for ty­ro­sine hy­drox­y­lase, cy­clo­hy­dro­lase 1, and aro­mat­ic L-amino acid de­car­boxy­lase — to re­store dopamine lev­els.

In Part A of the SUN­RISE-PD tri­al, Ax­o­vant had re­port­ed that two pa­tients who were giv­en the low­est dose saw an im­prove­ment in mo­tor func­tion af­ter be­ing washed out of oral lev­odopa ther­a­py.

The com­pa­ny said new fol­low-up da­ta on the sec­ond co­hort con­tin­ue to sup­port its hope for a one-shot Parkin­son’s treat­ment.

Faster, cheap­er CAR-T promis­es spur $100M fi­nanc­ing for Chi­nese play­er

Chi­nese CAR-T play­er Gra­cell Biotech­nolo­gies has se­cured $100 mil­lion — and the back­ing of mar­quee in­vestors — in a Se­ries C.

Welling­ton Man­age­ment, Or­biMed and Morn­ing­side (fresh­ly re­brand­ed 5Y Cap­i­tal) co-led the round. Vi­vo Cap­i­tal pitched in for the first time, joined by old-timers Temasek, Lil­ly Asia Ven­tures and King Star Cap­i­tal.

Based out of Shang­hai with a man­u­fac­tur­ing site in Suzhou, Gra­cell set out to dis­man­tle some of the con­straints lim­it­ing the first gen­er­a­tion of CAR-T ther­a­pies: high pro­duc­tion costs, lengthy man­u­fac­tur­ing process, lack of off-the-shelf prod­ucts and a short du­ra­tion of ef­fects.

Its tech plat­forms can gen­er­ate both au­tol­o­gous and al­lo­gene­ic cell ther­a­py can­di­dates, promis­ing a man­u­fac­tur­ing process un­der 24 hours, a way to tar­get two anti­gens at once and ge­net­ic tweaks to donor T cells.

6 Di­men­sions helped founder and CEO William Cao get it off the ground with $10 mil­lion Se­ries A three years ago, and a year lat­er Gra­cell scored $85 mil­lion more to push its pro­grams to­ward the clin­ic.

Cao had pre­vi­ous­ly found­ed and led Cel­lu­lar Bio­med­i­cine Group, which has a part­ner­ship with No­var­tis to de­vel­op and com­mer­cial­ize Kym­ri­ah in Chi­na as well as oth­er re­search pacts.

Gra­cell said the new cash will go to­ward both its Phase I prod­uct for re­lapsed or re­frac­to­ry T cell acute lym­phoblas­tic leukemia and oth­er pre­clin­i­cal can­di­dates.

J&J fun­nels two more IL-23 oral pep­tide drugs from Pro­tag­o­nist in­to pipeline 

Three years af­ter first buy­ing in­to Pro­tag­o­nist’s lead oral pep­tide, J&J is grab­bing two more IL-23 re­cep­tor an­tag­o­nists for its port­fo­lio.

“The IL-23 path­way is a val­i­dat­ed ther­a­peu­tic mech­a­nism, but oral­ly de­liv­ered ther­a­peu­tics for this class have not been avail­able,” Di­nesh Pa­tel, Pro­tag­o­nist pres­i­dent and CEO, said in a state­ment. “The ad­vance­ment of three dif­fer­ent oral co-de­vel­op­ment can­di­dates pro­vides us with sev­er­al strate­gic op­tions for de­vel­op­ment in mul­ti­ple in­di­ca­tions.”

PTG-200 (JNJ-67864238), the ini­tial drug J&J li­censed, is in a Phase II proof-of-con­cept study in mod­er­ate to se­vere Crohn’s dis­ease — one of its top in­flam­ma­to­ry bow­el dis­ease tar­gets.

PN-235 (JNJ-77242113) and PN-232 (JNJ-75105186) are both pre­clin­i­cal at the mo­ment, al­though PN-235 is ex­pect­ed to en­ter the clin­ic lat­er this year.

Ab­Cellera hip­sters Ko­di­ak Sci­ences re-up opthoma­l­o­gy dis­cov­ery pact

Ko­di­ak Sci­ences got in on the once be­low-the-radar Cana­di­an biotech Ab­Cellera be­fore it was cool, and ev­i­dent­ly, they were pleased with the re­sults.

Four years af­ter their first part­ner­ship, Ko­di­ak and Ab­Cellera have signed a new deal un­der which Ab­Cellera will use its mi­croflu­idics tech­nol­o­gy to de­vel­op an­ti­bod­ies against tar­gets that Ko­di­ak des­ig­nates. As with the pre­vi­ous part­ner­ship, those tar­gets are undis­closed, but Ko­di­ak is square­ly fo­cused on opthal­mol­o­gy, name­ly pre­vent­ing and treat­ing lead­ing caus­es of blind­ness.

Ab­Cellera spent the last decade qui­et­ly with some of the in­dus­try’s top drug de­vel­op­ers, in­clud­ing Lyell, Gilead, De­nali, Au­to­los, and No­var­tis. Fame came ear­li­er this year when Eli Lil­ly tapped them to de­vel­op a neu­tral­iz­ing an­ti­body against Covid-19, which is now in late-stage tri­als and which, de­spite mixed re­sults, the US gov­ern­ment just signed a block­buster con­tract to ac­quire in bulk.

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

$DNA is once again on NYSE; FDA clears Soliris chal­lenger for the mar­ket; Flag­ship’s think­ing big again with eR­NA; and more

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Gene ther­a­py from Bio­gen's $800M buy­out flops in mid-stage study, deal­ing blow to new am­bi­tions

The #2 candidate from Biogen’s $800 million ocular gene therapy buyout has failed in a mid-stage trial, dealing an early blow to the big biotech’s plans to revitalize its pipeline with new technologies.

Biogen announced that the candidate, an experimental treatment for a rare and progressive form of blindness called X-linked retinitis pigmentosa (XLRP), failed to sufficiently improve vision in patients’ treated eye — patients only received an injection in one eye — after a year, on a standard scale, compared to their untreated eye. The company said they saw “positive trends” on several secondary endpoints, including visual acuity, but declined to say whether the trial actually hit any of those endpoints.

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Vas Narasimhan (Photographer: Simon Dawson/Bloomberg via Getty Images)

No­var­tis whiffs on En­tresto study af­ter heart at­tacks — but that does­n't mean it's go­ing down qui­et­ly

If Novartis learned one thing from its interaction with the FDA over its latest heart failure approval for Entresto, it was that missing a primary endpoint may not be the nail in the coffin. Now, Entresto has missed again on a late-stage study in high-risk heart patients, and it’s already sowing the seeds for a path forward regardless.

Novartis’ Entresto couldn’t best standard-of-care ramipril in staving off a composite of deaths and heart failure events in patients with left ventricular systolic dysfunction and/or pulmonary congestion who have had a prior heart attack, according to topline data from the Phase III PARADISE-MI study revealed Saturday at the virtual American College of Cardiology meeting.

Michael Dell (Richard Drew, AP Images)

'Dude, you're get­ting a Del­l' — as a new deep-pock­et biotech in­vestor

What happens when you marry longtime insiders in the global biotech VC game with the family fund of tech billionaire Michael Dell, a synthetic biology legend out of MIT and Harvard and the former director of the NCI?

Today, the answer is a newly financed, $200 million biotech SPAC now cruising the industry for a top player interested in finding a short cut to Nasdaq.

Orion Biotech Opportunities priced their blank check company today, raising $200 million with Dell’s multibillion-dollar MSD group’s commitment on investing another $20 million in a forward-purchase agreement.

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BAR­DA slows its $9B en­gine for new Covid-19 ther­a­peu­tics

The Biomedical Advanced Research and Development Authority is cooling its jets in looking for new, potential Covid-19 treatments, at least in the near term.

An HHS spokesperson told Endpoints News via email, “to date, BARDA has obligated more than $9 billion for the development and/or purchase of 13 therapeutics, beginning in February 2020 with support to develop Regeneron’s monoclonal antibody therapeutic. Therapeutics are an important element of the COVID-19 response, and we are focused on the programs currently underway and/or in negotiation using the funds available to us.”

Scynex­is takes out $60M loan ahead of ex­pect­ed ap­proval; Com­pass Ther­a­peu­tics ac­quires new bis­pe­cif­ic an­ti­body in buy­out

As the PDUFA date for their vaginal yeast infections quickly approaches, Scynexis is taking out a loan to ensure it can hit the ground running for a potential commercial launch.

Scynexis closed terms on a $60 million loan with Hercules Capital and Silicon Valley Bank, the New Jersey biotech announced Friday, with its ibrexafungerp candidate set for a June 1 PDUFA. Approval of the program is anticipated, the company said, after which the oral antifungal will be branded as Brexafemme.

Bris­tol My­ers backs up its case for heart drug mava­camten as FDA weighs app in car­diomy­opa­thy

When Bristol Myers Squibb signed off on its $13 billion acquisition of MyoKardia back in October, it was making a big bet that lead drug mavacamten could prove a game changer in cardiac myopathy. Now, with the drug up for FDA review, Bristol Myers is backing up its case with new quality of life data.

Patients dosed with myosin inhibitor mavacamten posted a clinically significant increase in scores on the Kansas City Cardiomyopathy Questionnaire, a catch-all summary of symptoms and quality of life markers, over placebo at 30 weeks, according to data from the Phase III EXPLORER-HCM study presented Saturday at the virtual American College of Cardiology meeting.

UP­DAT­ED: Apel­lis bags FDA nod for Soliris chal­lenger with a dif­fer­ent path­way to PNH — but can it slay the gi­ant?

With a blockbuster rare disease giant in its sights in Alexion’s Soliris, small biotech Apellis has reason to think its competitor is worthy of the spotlight. Now, with the FDA on its side, Apellis will get its chance to be the David to Alexion’s Goliath.

The FDA on Friday approved Empaveli (pegcetacoplan), a C3 complement inhibitor the biotech thinks can prove a worthy challenger to Alexion’s C5 inhibitors Soliris and follow-up drug Ultomiris in rare disease paroxysmal nocturnal hemoglobinuria (PNH).