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.

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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Ted White, Verrica Pharmaceuticals CEO

'Hands may be tied': FDA slaps Ver­ri­ca with 3rd CRL due to prob­lems with con­tract man­u­fac­tur­er

The FDA has rejected Verrica Pharmaceuticals’ skin disease treatment for a third time — and once again the contract manufacturer is to blame.

The biotech emphasized that the only deficiency in the complete response letter is related to a general reinspection of the CMO, Sterling Pharmaceuticals, and has nothing to do specifically with its drug-device; the rest of the NDA is good to go.

CEO Ted White said the company is “extremely disappointed,” but will keep working toward approval.

Mihael Polymeropoulos, Vanda Pharmaceuticals CEO

Phar­ma com­pa­ny con­tin­ues its FDA law­suit spree, this time af­ter agency de­nies fast-track des­ig­na­tion

Vanda Pharmaceuticals is making a name for itself, at least in terms of suing the FDA.

The DC-headquartered firm on Monday filed its latest suit against the agency, with the company raising concerns over the FDA’s failure to grant a fast track designation for Vanda’s potential chronic digestive disorder drug tradipitant, which is a neurokinin 1 receptor antagonist.

Specifically, Vanda said FDA’s “essential point” in its one-page denial letter on the designation pointed to “the lack of necessary safety data,” which was “inconsistent with the criteria for … Fast Track designation.”

Mod­er­na seeks to dis­miss Al­ny­lam suit over Covid-19 vac­cine com­po­nent, claim­ing wrong venue

RNAi therapeutics juggernaut Alnylam Pharmaceuticals made a splash in March when it sued and sought money from both Pfizer and Moderna regarding their use of Alnylam’s biodegradable lipids, which Alnylam claims have been integral to the way both companies’ mRNA-based Covid-19 vaccines work.

But now, Moderna lawyers are firing back, telling the same Delaware district court that Alnylam’s claims can only proceed against the US government in the Court of Federal Claims because of the way the company’s contract is set up with the US government. The US has spent almost $10 billion on Moderna’s Covid-19 vaccine so far.

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Cracks in the fa­cade: Is phar­ma's pan­dem­ic ‘feel good fac­tor’ wan­ing?

The discordant effects of the Covid-19 pandemic on pharma reputation continues. While the overall industry still retains a respectable halo from its Covid-19 quick response and leadership, a new patient group study reveals a different story emerging in the details.

On one hand, US patient advocacy groups rated the industry higher-than-ever overall. More than two-thirds (67%) of groups gave the industry a thumbs up for 2021, a whopping 10 percentage point increase over the year before, according to the PatientView annual study, now in its 9th year.

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FDA ac­cepts pri­or­i­ty re­view of CSL-uniQure he­mo­phil­ia B gene ther­a­py; Fo­s­un Phar­ma nets an­oth­er deal

The first potential gene therapy for hemophilia B has gotten one step closer to reaching patients as the FDA has accepted CSL Behring’s BLA for its uniQure-partnered one-time treatment. The therapy, dubbed etranacogene dezaparvovec, was accepted under “accelerated assessment” by the European Medicines Agency in March.

A priority review will be conducted for the gene therapy intended for patients with the hereditary bleeding disorder, CSL said Tuesday morning. The Pennsylvania biotech did not indicate the regulator’s decision date, but a priority review generally means the treatment’s fate will be handed down within six months rather than 10.

Michael Corbo, Pfizer CDO of inflammation & immunology

UP­DAT­ED: Plan­ning ahead for crowd­ed ul­cer­a­tive col­i­tis mar­ket, Pfiz­er spells out PhI­II da­ta on $6.7B Are­na drug

Pfizer has laid out the detailed results behind its boast that etrasimod — the S1P receptor modulator at the center of its $6.7 billion buyout of Arena Pharma — is the winner of the class, potentially leapfrogging an earlier entrant from Bristol Myers Squibb.

Pivotal data from the ELEVATE program in ulcerative colitis — which consists of two Phase III trials, one lasting 52 weeks and the other just 12 weeks — illustrate an “encouraging balance of efficacy and safety,” according to Michael Corbo, chief development officer of inflammation & immunology at Pfizer. The company is presenting the results as a late breaker at Digestive Disease Week.

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Robert Califf (Michael Brochstein/Sipa USA via AP Images)

House Re­pub­li­cans at­tack Chi­na-on­ly da­ta in FDA sub­mis­sions, seek new in­ves­ti­ga­tion in­to re­search in­spec­tions

Three Republican representatives are calling on the FDA to take a closer look at the applications including only clinical data from China.

The letter to FDA commissioner Rob Califf late last week comes as the agency recently rejected Eli Lilly’s anti-PD-1 antibody, which attempted to bring China-only data but ran into a bruising adcomm that may crush the hopes of any other companies looking to bring cheaper follow-ons based only on Chinese data.

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Co­pay coupons gone wrong, again: Pfiz­er pays al­most $300K to set­tle com­plaints in four states

Pfizer has agreed to pay $290,000 to settle allegations of questionable copay coupon practices in Arizona, Colorado, Kansas, and Vermont from 2014 to 2018.

While the company has not admitted any wrongdoing as part of the settlement, Pfizer has agreed to issue restitution checks to about 5,000 consumers.

A Pfizer spokesperson said the company has “enhanced its co-pay coupons to alleviate the concerns raised by states and agreed to a $30,000 payment to each.”