Cas­sa­va be­ing in­ves­ti­gat­ed by un­named 'gov­ern­ment agen­cies'; Rock­et Phar­ma gives up­dat­ed da­ta on Danon dis­ease

Cas­sa­va Sci­ences likes to fight fire with fire, with CEO Re­mi Bar­bi­er call­ing a cit­i­zen pe­ti­tion to halt de­vel­op­ment on its lead drug a “short at­tack” in Sep­tem­ber. But the noise sur­round­ing the saga has ap­par­ent­ly drawn the at­ten­tion of the US gov­ern­ment.

The biotech re­port­ed in an SEC fil­ing Mon­day that “cer­tain gov­ern­ment agen­cies” have asked Cas­sa­va to pro­vide them with “cor­po­rate in­for­ma­tion and doc­u­ments.” It’s not clear which agen­cies are in­volved in prob­ing Cas­sa­va, or if mul­ti­ple agen­cies are do­ing so, but the com­pa­ny says it’s co­op­er­at­ing.

Though Cas­sa­va re­port­ed that the gov­ern­ment said it had not in­formed the biotech that any wrong­do­ing had oc­curred, the com­pa­ny left the door open to po­ten­tial penal­ties.

“We can­not pre­dict the out­come or im­pact of any [of] these on­go­ing mat­ters, in­clud­ing whether a gov­ern­ment agency may pur­sue an en­force­ment ac­tion against us or oth­ers,” Cas­sa­va wrote in the fil­ing.

Cas­sa­va was orig­i­nal­ly ac­cused by the law firm La­ba­ton Sucharow in Au­gust of da­ta ma­nip­u­la­tion, with the share­hold­er rights ac­tivists pe­ti­tion­ing the FDA to stop de­vel­op­ment of the Alzheimer’s drug simu­fil­am. The biotech fought back with Bar­bi­er’s com­ments as well as an un­usu­al Q-and-A style press re­lease put out around that time. — Max Gel­man

Rock­et Phar­ma re­ports up­dat­ed PhI da­ta for Danon dis­ease

New Jer­sey biotech Rock­et Phar­ma­ceu­ti­cals has been work­ing on an in­ves­ti­ga­tion­al gene ther­a­py for gene dis­or­der Danon dis­ease — and the phar­ma an­nounced up­dat­ed da­ta from the Phase I tri­al to­day.

The tri­al was look­ing at a sin­gle IV in­fu­sion of gene ther­a­py RP-A501 at two dif­fer­ent dosages in five pa­tients — “low-dose” with three pa­tients (6.7e13 vg/kg) and “high-dose” with the oth­er two (1.1e14 vg/kg). While the gene ther­a­py was well-tol­er­at­ed at the low dose and con­ferred sus­tained clin­i­cal ben­e­fit, the same can’t be said for the high dose.

As pre­vi­ous­ly made pub­lic, dose-de­pen­dent tox­i­c­i­ty was seen in one of the two pa­tients on the high dose reg­i­men — and de­vel­oped throm­bot­ic mi­croan­giopa­thy that ful­ly re­solved with treat­ment, which in­clud­ed tran­sient he­modial­y­sis.

As such, the biotech will be cut­ting off the high­er dosage and stick­ing with the low­er amount mov­ing for­ward.

“We are ex­cit­ed to an­nounce pos­i­tive da­ta from our RP-A501 tri­al for Danon Dis­ease show­ing clin­i­cal, func­tion­al and bio­mark­er im­prove­ments at one year or be­yond and po­ten­tial ear­ly sep­a­ra­tion from the nat­ur­al course of dis­ease in adult and ado­les­cent pa­tients,” said Rock­et CEO Gau­rav Shah. “We have ini­ti­at­ed treat­ment in the pe­di­atric co­hort at the low-dose and an­tic­i­pate mov­ing as rapid­ly as pos­si­ble to­ward Phase II.” — Paul Schloess­er

Motric Bio launch­es from ex-No­var­tis CEO’s in­vest­ment shop

Af­ter No­var­tis’ for­mer CEO Joe Jimenez and NI­BR’s for­mer pres­i­dent Mark Fish­man co-found­ed Adi­tum Bio in 2019, they are launch­ing an­oth­er off­shoot — for the sev­enth time — with the launch of Motric Bio in a part­ner­ship with Mo­tor­phar­ma.

Mark Fish­man

In a joint state­ment, the com­pa­nies an­nounced the for­ma­tion of Motric Bio, a port­fo­lio com­pa­ny de­vel­op­ing a new ther­a­py for spas­tic­i­ty as­so­ci­at­ed with neu­ro­log­i­cal in­juries and dis­ease. Motric Bio suc­cess­ful­ly in-li­censed a myosin-2 in­hibitor from Mo­tor­phar­ma — and that in­hibitor has shown se­lec­tive in­hi­bi­tion of fast skele­tal mus­cle.

“This mol­e­cule has shown se­lec­tiv­i­ty to fast skele­tal myosin-2, with prac­ti­cal­ly no in­hi­bi­tion of car­diac and smooth mus­cle myosins,” Fish­man said in a com­pa­ny state­ment. “We be­lieve this could lead to greater ef­fi­ca­cy and few­er side ef­fects than the cur­rent stan­dard of care. We look for­ward to ad­vanc­ing this im­por­tant ther­a­py in­to the clin­ic to help these pa­tients.”

“This mol­e­cule is the re­sult of over 20 years of re­search to de­vel­op se­lec­tive myosin-2 in­hibitors,” said Mo­tor­phar­ma CEO An­na Rausch­er. “We are pleased to part­ner with Adi­tum Bio to form Motric Bio, en­abling this drug to en­ter the clin­ic to help pa­tients in need.” — Paul Schloess­er

In­FlaRx’s mAb for au­toim­mune dis­ease shows pos­i­tive re­sults

Ger­man bio­phar­ma In­FlaRx an­nounced pos­i­tive re­sults from its Phase II study of vilo­be­limab, a an­ti-C5a an­ti­body in pa­tients with AN­CA-as­so­ci­at­ed vas­culi­tis. Pa­tients went in­to re­mis­sion at sim­i­lar rates to stan­dard of care, while us­ing much less glu­co­cor­ti­coids — trans­lat­ing to an ef­fi­ca­cy and safe­ty pro­file in­ves­ti­ga­tors said they were en­cour­aged by.

The pri­ma­ry ef­fi­ca­cy eval­u­a­tion was de­fined by a 50% re­duc­tion on the Birm­ing­ham Vas­culi­tis Ac­tiv­i­ty Score (BVAS) and no wors­en­ing in any body sys­tem from base­line. Clin­i­cal re­mis­sion was the sec­ondary end­point.

“We are pleased to re­port that vilo­be­limab monother­a­py has shown sim­i­lar ef­fi­ca­cy com­pared to a stan­dard dose of glu­co­cor­ti­coids in pa­tients with AN­CA-as­so­ci­at­ed vas­culi­tis in this tri­al,” chief de­vel­op­ment of­fi­cer Ko­rin­na Pilz said in the press re­lease. “Vilo­be­limab treat­ment led to a con­sid­er­ably re­duced use of glu­co­cor­ti­coids, re­sult­ing in re­mark­ably less treat­ment emer­gent ad­verse events and a re­duc­tion in the glu­co­cor­ti­coid tox­i­c­i­ty in­dex.”

AN­CA-as­so­ci­at­ed vas­culi­tis is an au­toim­mune dis­ease that af­fects small blood ves­sels in the body. Vilo­be­limab is be­lieved to be the first mon­o­clon­al an­ti-C5a an­ti­body in­tro­duced in­to clin­i­cal de­vel­op­ment. Over 300 peo­ple have been treat­ed with vilo­be­limab in com­plet­ed clin­i­cal tri­als, the com­pa­ny says. — Josh Sul­li­van

Shoot­ing for clin­ic in 2022, Athos rais­es $15M in Se­ries A

Dim­itrios Il­iopou­los

Athos Ther­a­peu­tics closed its Se­ries A on Mon­day.

The biotech raised $15 mil­lion in the over­sub­scribed round, aim­ing to launch a Phase I tri­al of its lead com­pound in 2022. That can­di­date, ATH-63, is be­ing de­vel­oped for “in­flam­ma­to­ry bow­el dis­ease, lu­pus, oth­er au­toim­mune dis­or­ders, and se­lect can­cer in­di­ca­tions,” Athos said.

“I am de­light­ed at the progress Athos has made since our Seed round of $4.25MM in March of 2020,” CEO Dim­itrios Il­iopou­los said in a state­ment. “Those seed funds en­abled us to hire a stel­lar team, de­vel­op a pow­er­ful drug dis­cov­ery en­gine, per­form mul­ti-omics analy­sis of an­no­tat­ed pa­tient sam­ples from key IBD cen­ters around the world, file 8 patents, and cre­ate a med­i­c­i­nal chem­istry plat­form that pro­duced 100’s of high­ly se­lec­tive and safe com­pounds with ex­cel­lent phar­ma­co­log­ic pro­files.” — Max Gel­man

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

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Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

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In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

With on­ly burns to show in gene ther­a­py, Astel­las inks deal with AAV spe­cial­ist Dyno in push for a bet­ter cap­sid

On the hunt for a better AAV capsid for gene therapy, Eric Kelsic’s Dyno Therapeutics has set itself apart with its focus on machine learning to help speed discovery. Now, Japanese drugmaker Astellas — fresh off a slate of gene therapy burns — is taking a bet on Dyno as it looks to the future.

Astellas and Dyno will work together as part of an R&D pact to develop next-gen AAV vectors for gene therapy using Dyno’s CapsidMap platform directed at skeletal and cardiac muscle, the companies said Wednesday. Under the terms of the deal, Dyno will design AAV capsids for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the capsids.

As first Omi­cron case in US crops up, re­searchers won­der: which an­ti­bod­ies, vac­cines will hold up?

As Covid-19 drug and vaccine developers race to figure out which of their products might be hampered by the new variant, the CDC on Wednesday afternoon announced the first confirmed case of the Omicron variant (B.1.1.529) in the US, found in San Francisco.

The unidentified individual was a traveler who returned from South Africa on Nov. 22, 2021, was fully vaccinated, and had mild symptoms that the CDC described as improving. All close contacts have been contacted and have tested negative, the centers said.

Can South Dako­ta's trans­genic cows help treat the flu?; A speedy re­view for Mer­ck­'s Pre­vnar ri­val in kids

Wednesday brought another win for South Dakota’s biggest transgenic cow so far.

SAB Biotherapeutics, which develops treatments by collecting and distilling antibodies from cows with humanized immune systems, announced that its antibody treatment for flu passed an early-stage challenge study.

Volunteers were intentionally exposed to the flu virus and then given infusions of the SAB antibody treatment or placebo. Those who received the antibody treatment saw a significantly greater reduction in viral load and symptoms than those who received placebo. The company didn’t release numbers but said the p-value was 0.026.

Mod­er­na los­es lat­est bat­tle in key vac­cine de­liv­ery patent fight as fed­er­al ap­peal falls flat

The US Court of Appeals for the Federal Circuit on Wednesday rejected Moderna’s attempt to overturn key patents related to the delivery vehicle for its Covid-19 vaccine after the biotech sought to preempt a potentially risky infringement lawsuit.

For years, Moderna has been battling a tiny Pennsylvania biotech known as Arbutus over patents for a technology required to deliver its mRNA drugs and vaccines, known as lipid nanoparticles or LNP. Moderna is concerned there’s a substantial risk that Arbutus will assert the ’069 patent in an infringement suit targeting Moderna’s Covid-19 vaccine, particularly as Arbutus has boasted of its patent protection and refused to grant a covenant not to sue Moderna.

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Lan Huang, BeyondSpring CEO

Months af­ter shock­ing in­vestors with lung can­cer win, Be­yond­Spring's lead drug hits road­block at the FDA

BeyondSpring shocked investors in early August after its once-marginal lead drug suddenly showed a lot of promise in a common form of lung cancer. With hopes high, the FDA has now slammed the door on that drug in another indication — does that spell bad news for BeyondSpring’s Cinderella story?

The FDA issued BeyondSpring a complete response letter for its plinabulin in combination with granulocyte colony-stimulating factor (G-CSF) for the prevention of chemotherapy-induced neutropenia, effectively shutting down the drug’s immediate chances at a marketing approval, the biotech said Wednesday.

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