Marcio Souza, Praxis president and CEO

Prax­is rais­es $110 mil­lion to tar­get CNS dis­ease with Phase II drugs

Just two months af­ter burst­ing on­to the neu­ro­science scene, Prax­is Pre­ci­sion Med­i­cines has added $110 mil­lion in fund­ing to pur­sue ther­a­pies for cen­tral ner­vous sys­tem dis­or­ders.

Prax­is came out of stealth mode in May af­ter bank­ing $100 mil­lion in fund­ing and putting two drugs in Phase II de­vel­op­ment. While Big Phar­ma has strayed away from neu­ro­science, Prax­is set out to iden­ti­fy mu­ta­tions that cause epilep­sy in pa­tients who did not in­her­it the dis­ease. The com­pa­ny is now ap­ply­ing its dis­cov­er­ies to po­ten­tial treat­ments for oth­er CNS dis­eases, such as de­pres­sion, epilep­sy, move­ment dis­or­ders and pain syn­dromes. It has three drugs in the clin­i­cal pipeline.

“As was achieved in on­col­o­gy decades ago, re­cent ge­net­ic in­sights have pre­sent­ed mean­ing­ful op­por­tu­ni­ties to treat brain dis­or­ders in en­tire­ly dif­fer­ent and tar­get­ed ways based on the spe­cif­ic ge­net­i­cal­ly val­i­dat­ed path­ways dri­ving a pa­tient’s dis­ease,” Prax­is co-founder and board mem­ber Ki­ran Red­dy said in a pre­pared state­ment. “We are re­duc­ing these in­sights to prac­tice, to cre­ate nov­el med­i­cines that could fun­da­men­tal­ly al­ter the treat­ment path and out­comes for pa­tients with brain dis­or­ders.”

PRAX-114 — Prax­is’ lead pro­gram and one of its two Phase II drugs — tar­gets ma­jor de­pres­sive dis­or­der and per­i­menopausal de­pres­sion. Plans are in place to en­ter the drug, a GA­BA pos­i­tive al­losteric mod­u­la­tor, in a piv­otal tri­al this year. The oth­er Phase II drug, PRAX-944, is a T-type cal­ci­um block­er that ad­dress­es es­sen­tial tremor. The com­pa­ny told End­points News in May that it plans to have proof-of-con­cept re­sults for the lat­ter drug by the end of the year.

PRAX-562 is cur­rent­ly in Phase I de­vel­op­ment for the treat­ment of pe­di­atric epilep­sy and adult cepha­lal­gia.

“We are pleased with and ex­cit­ed by this fi­nanc­ing, which sup­ports our con­tin­ued mis­sion to de­vel­op best-in-class ther­a­pies that de­liv­er long-term val­ue to hu­man health,” Prax­is pres­i­dent and CEO Mar­cio Souza said in a news re­lease. “With three pro­grams cur­rent­ly in clin­i­cal de­vel­op­ment for five in­di­ca­tions, and ad­di­tion­al pro­grams in ear­li­er stages of de­vel­op­ment, we are mak­ing great strides to bring new treat­ments to pa­tients in the CNS space.”

A swath of in­vestors — old and new — sup­port­ed the Se­ries C1 fi­nanc­ing, led by Even­tide As­set Man­age­ment. The list in­cludes Vi­da Ven­tures, No­vo Hold­ings, Black­stone Life Sci­ences, OCV Part­ners, Avoro Cap­i­tal Ad­vi­sors, Sur­vey­or Cap­i­tal (a Citadel com­pa­ny), Point72, Cor­morant As­set Man­age­ment, Qatar In­vest­ment Au­thor­i­ty (QIA), Irv­ing In­vestors, Adage Cap­i­tal Man­age­ment, Veri­tion Fund Man­age­ment and Am­ple Plus Fund.

“At Even­tide, we be­lieve that by com­bin­ing a fo­cus on ther­a­peu­tic tar­gets de­fined through hu­man ge­net­ics with nov­el trans­la­tion­al tools and ef­fi­cient clin­i­cal de­vel­op­ment paths, Prax­is has the po­ten­tial to sig­nif­i­cant­ly im­pact pa­tients with CNS dis­or­ders,” Even­tide Chief In­vest­ment Of­fi­cer Finny Ku­ruvil­la, said in the re­lease. “The com­pa­ny has made sig­nif­i­cant progress in ad­vanc­ing a broad pipeline ad­dress­ing im­por­tant ar­eas for ther­a­peu­tic in­ter­ven­tion, and we are de­light­ed to sup­port the team in achiev­ing their mis­sion.”

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Vi­da Ven­tures co-leads Dyne's $115M megaround for next-gen oli­go ther­a­pies aimed square­ly at mus­cles

Dyne Therapeutics started out last April with a modest $50 million to mine targeted muscle disease therapies from its in-house conjugate technology. The biotech has now convinced more investors that it’s got gems on its hands, closing $115 million in fresh financing to push its next-gen oligonucleotide drugs into the clinic.

Vida Ventures and Surveyor Capital led the round, joined by a group of other new backers including Wellington Management Company, Logos Capital and Franklin Templeton.

Warren Huff, Reata CEO

Rea­ta sug­gests Friedre­ich's atax­ia pro­gram could be de­layed, send­ing stock plung­ing

Reata Pharmaceuticals $RETA made waves last October when its drug omaveloxolone produced positive trial results in treating a rare neurological disorder, but the candidate’s path forward became much murkier Monday.

In a report of quarterly earnings, the biotech divulged that the FDA is considering delaying omaveloxolone’s NDA pending completion of a second trial. That could push back approval by at least a year given that the target population, individuals with Friedreich’s ataxia, is limited and progression of the hard-to-treat illness is notoriously slow. The Covid-19 pandemic would also hinder Reata’s ability to complete an additional trial.

DFC CEO Adam Boehler and Kodak CEO Jim Continenza (Kodak)

Covid-19 roundup: Cure­Vac beefs up its uni­corn IPO dreams as bil­lion­aire own­er takes this Covid-19 mR­NA play­er on a forced march to Nas­daq; Ko­dak's $765M deal is put on hold

When CureVac initially jotted down $100 million for its IPO raise a couple of weeks ago, it seemed small. The German mRNA player, after all, had jumped into a Covid-19 race that swelled the sails of Moderna and BioNTech by tens of billions. And after raising $640 million in a slate of deals, $100 million in a hot market like this seemed like a pittance in the bigger scheme of things.

Today, we got a look at a figure that probably comes closer to the game-changing number the top execs probably have in mind. Selling 15.3 million shares at the high end of their $14 to $16 range would net a $243 million bounty. Majority owner Dietmar Hopp is putting in another €100 million, bringing the total to around $350 million. And what are the chances they want to do even better than that?

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Eric Shaff (Seres)

UP­DAT­ED: Af­ter a 4-year so­journ, strug­gling mi­cro­bio­me pi­o­neer Seres claims a break­out PhI­II come­back. And shares re­spond in fren­zied spike

Almost exactly 4 years ago, Seres Therapeutics $MCRB experienced one of those soul-crunching failures that can raise big questions about a biotech’s future. Out front in their pursuit of a gut punch to C. difficile infection (CDI), the Phase II test was a flat failure, and investors wiped out a billion dollars of equity value that never returned in the years that followed.

Seres, though, pressed ahead, changing out CEOs a year ago — bidding Merck vet Roger Pomerantz farewell from the C suite — and pushing through a Phase III, hoping that amping up the dosage would make the key difference. And this morning, they unveiled a claim that they had aced the Phase III and positioned themselves for a run at a landmark FDA OK.

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Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

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Eli Lil­ly teams with Pieris on HER2+ tu­mors; Op­di­vo + Yer­voy best chemo in mesothe­lioma

Despite the FDA putting a partial clinical hold on its lead program only a few weeks ago, Boston-based Pieris Pharmaceuticals is plowing forward with a new collaboration.

Pieris will work with Eli Lilly to further advance studies on PRS-343, a 4-1BB/HER2 bispecific for HER2-positive tumors, in combination with the latter’s ramucirumab and paclitaxel for the second-line treatment of patients with HER2-positive gastric cancer in a single-arm, Phase II study.

In­novent and Eli Lil­ly chal­lenge Mer­ck­'s mega-block­buster Keytru­da in non-small cell lung can­cer field

China-based Innovent Biologics and its multinational ally Eli Lilly shared Phase III evidence that their PD-1 inhibitor combo can delay the progression of nonsquamous non-small cell lung cancer.

But the drugmakers will face stiff competition in China from Merck’s Keytruda, the ruling PD-1 which is already approved to treat both squamous and nonsquamous NSCLC and boasts positive overall survival rates.

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