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.”

Eli Lilly CEO David Ricks (Evan Vucci/AP Images)

A P val­ue of 0.38? NE­JM re­sults raise new ques­tions for Eli Lil­ly's vaunt­ed Covid an­ti­body

Generally, a P value of 0.38 means your drug failed and by a fair margin. Depending on the company, the compound and the trial, it might mean the end of the program. It could trigger layoffs.

For Eli Lilly, though, it was part of the key endpoint on a trial that landed them a $1.2 billion deal with the US government to supply up to nearly 1 million Covid-19 antibodies.

So what does one make of that? Was the endpoint not so important, as Lilly maintains? Or did the US government promise a princely sum for a pedestrian drug?

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Noubar Afeyan, Flagship founder and CEO (Victor Boyko/Getty Images)

UP­DAT­ED: Flag­ship launch­es Sen­da Bio­sciences with $88M in back­ing, look­ing to pi­o­neer the field of 'In­ter­sys­tems Bi­ol­o­gy'

Flagship Pioneering has a fresh company out this week, one that aims to lay the groundwork for a whole new discipline.

Senda Biosciences launched Wednesday with $88 million in Flagship cash. The goal? Gain insights into the molecular connections between people and coevolved nonhuman species like plants and bacteria, paving the way for “Intersystems Biology.”

Guillaume Pfefer has been tapped to run the show, a 25-year biotech veteran who comes from GSK after leading the development of the company’s shingles vaccine.

Daphne Koller, Getty

Bris­tol My­er­s' Richard Har­g­reaves pays $70M to launch a neu­rode­gen­er­a­tion al­liance with a star play­er in the ma­chine learn­ing world

Bristol Myers Squibb is turning to one of the star upstarts in the machine learning world to go back to the drawing board and come up with the disease models needed to find drugs that can work against two of the toughest targets in the neuro world.

Daphne Koller’s well-funded insitro is getting $70 million in cash and near-term milestones to use their machine learning platform to create induced pluripotent stem cell-derived disease models for ALS and frontotemporal dementia.

Eli Lilly CEO David Ricks at the Rose Garden, May 26, 2020 (Evan Vucci/AP Images)

Eli Lil­ly lines up a block­buster deal for Covid-19 an­ti­body, right af­ter it failed a NI­AID tri­al

Two days after Eli Lilly conceded that its antibody bamlanivimab was a flop in hospitalized Covid-19 patients, the US government is preparing to make it a blockbuster.

The pharma giant reported early Wednesday that it struck a deal to supply the feds with 300,000 vials of the drug at a cost of $375 million — once it gets an EUA stamp from the FDA. And once that 2-month supply deal is done, the government has an option on another 650,000 doses on the same terms — which could potentially add another $812 million.

Q32 Bio grabs $60M to kick off hu­man stud­ies for next-gen com­ple­ment drugs — with some Covid-19 tweaks along the way

For a company that launched in the early months of the pandemic, Q32 Bio had its fair share of run-ins with the new normals under Covid-19.

The original plan, for instance, was to conduct first-in-human studies of the IL-7 receptor antibody it licensed from Bristol Myers Squibb in the Netherlands. But they realized shortly after that while the country was beginning to open up clinical trials, there were additional restrictions on drugs that tampered with immunological mechanisms.

Konstantin Poukalov

Per­cep­tive re­cruits A-list in­vestors to back its in-house Chi­na start­up with a mam­moth $310M raise

It took two years for Perceptive Advisors to conceive and boot up LianBio, its big bet on a new kind of in-licensing model for China, seeding it with enough cash to set up two anchoring deals with MyoKardia and BridgeBio. The result was a startup that was all ready to go, reaping $310 million just a little over two months after official launch.

Homegrown Chinese biotechs — many of them boasting of US ties and execs with overseas credentials — have been raking in mega-venture rounds in 2020, both from influential local backers and overseas VC firms that have been loading up new cash. As with IPOs, the deal flow might be slower but the amounts are often more staggering. LianBio’s latest round, unusually, is branded both a Series A and crossover.

Ar­cus and As­traZeneca part­ner on a high stakes an­ti-TIG­IT/PD-L1 PhI­II can­cer study, look­ing to im­prove on a stan­dard of care

For AstraZeneca, the PACIFIC trial in Stage III non-small cell lung cancer remains one of the big triumphs for AstraZeneca’s oncology R&D group. It not only made their PD-L1 Imfinzi a franchise player with a solid advance in a large niche of the lung cancer market, the study — which continues to offer data on the long-range efficacy of their drug — also helped salve the vicious sting of the failure of the CTLA-4 combo in the MYSTIC study.

No­var­tis buys a new gene ther­a­py for vi­sion loss, and this is one pre­clin­i­cal ven­ture that did­n't come cheap

Cyrus Mozayeni got excited when he began to explore the academic work of Ehud Isacoff and John G. Flannery at UC Berkeley.

Together, they were engaged in finding a gene therapy approach to pan-genotypic vision restoration in patients with photoreceptor-based blindness, potentially restoring the vision of a broad group of patients. And they did it by using a vector to deliver the genetic sequence for light sensing proteins.

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CMO Merdad Parsey (Gilead)

Gilead hits the brakes on a tri­fec­ta of mid- and late-stage stud­ies for their trou­bled fil­go­tinib pro­gram. It's up to the FDA now

Gilead $GILD execs haven’t decided exactly what to do with filgotinib in the wake of the slapdown at the FDA on their rheumatoid arthritis application, but they’re taking a time out for a slate of studies until they can gain some clarity from the agency. And without encouraging guidance, this drug could clearly be axed from the pipeline.

In their Q3 report out Wednesday afternoon, the company says researchers have “paused” a Phase III study for psoriatic arthritis along with a pair of Phase II trials for ankylosing spondylitis and uveitis. Late-stage studies for ulcerative colitis and Crohn’s are continuing, but you can see for yourself how big a hole this leaves in the inflammatory disease pipeline, with obvious implications if the company abandons filgo altogether.

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