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.

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

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UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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The flu virus (CDC)

Roche tacks on an­oth­er Xofluza in­di­ca­tion as flu sea­son meets pan­dem­ic

Xofluza was heralded as the first new flu drug in 20 years when it got the FDA OK back in 2018. But even so, Roche saw tough competition from cheaper Tamiflu generics that appeared to be nearly as — if not just as — effective.

Now, the pharma says the drug also can be used to prevent influenza after exposure, snagging a new approval and adding to Xofluza’s appeal as flu season meets the pandemic.

A poll sug­gests vac­cine da­ta boost­ed Pfiz­er's pub­lic im­age, but oth­er da­ta point to long road ahead

For much of the pharmaceutical industry, the pandemic presented an opportunity: to prove their value to the world and turn public opinion around on a business much of the country had come to disdain.

That theory — that helping pull the country from a pandemic could neutralize years of anger over high drug prices — was put to its biggest test this month, as three different drugmakers announced data from their Covid-19 vaccines, offering the first major evidence that industry-built inoculations could turn the tide of the outbreak in the US.

Covid-19 roundup: Rus­sia prices vac­cine 't­wo or more times cheap­er' than mR­NA shots; Sino­vac PhI­II da­ta ex­pect­ed in ear­ly De­cem­ber

The world can now purchase its first registered vaccine — at less than $10 per dose.

RDIF, the Russian sovereign wealth fund and an avid backer of Sputnik V, said the vaccine will be available internationally starting from March 2021. A two-dose regimen of the adenovirus-based vaccine, which it has said is more than 90% effective, will cost less than $20.

And they are not shy about inserting themselves right into a rivalry with Western frontrunners, namely Pfizer/BioNTech and Moderna.

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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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Bob Nelsen (Photo by Michael Kovac/Getty Images)

Bob Nelsen rais­es $800M and re­cruits a star-stud­ded board to build the 'Fox­con­n' of biotech

Bob Nelsen spent his pandemic spring in his Seattle home, talking on the phone with Luciana Borio, the scientist who used to run pandemic preparedness on the National Security Council, and fuming with her about the dire state of American manufacturing.

Companies were rushing to develop vaccines and antibodies for the new virus, but even if they succeeded, there was no immediate supply chain or infrastructure to mass-produce them in a way that could make a dent in the outbreak.

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