Prax­is Pre­ci­sion Med­i­cines launch­es with $100M and bold sights on long-eva­sive neu­ro­log­i­cal dis­or­ders

It’s go­ing to be the era of neu­ro­science, Roche CEO Bill An­der­son de­clared on stage at the JP Mor­gan Health­care Con­fer­ence in Jan­u­ary. The field, he said, had “the po­ten­tial to be in the ‘20s what on­col­o­gy was for the last decade.”

Five months in­to that decade, a new biotech is emerg­ing from stealth mode with large in­vest­ments from Black­stone and two drugs al­ready in Phase II, one of them near­ing a piv­otal tri­al. Called Prax­is Pre­ci­sion Med­i­cines, since 2016 it’s raised $100 mil­lion — with No­vo Hold­ings, Vi­da Ven­tures and Even­tide al­so chip­ping in — to back a bet that, by find­ing the un­der­ly­ing cause of rare neu­ro­log­i­cal dis­eases, they could find and treat mech­a­nisms be­hind more com­mon ones.

For all An­der­son’s op­ti­mism about the fu­ture, though, Prax­is emerges at a tough time for neu­ro­science-fo­cused biotechs. Much of the rest of Big Phar­ma has all but aban­doned the field. The last ma­jor piece of neu­ro-news of the pre­vi­ous decade was the an­nounce­ment, in De­cem­ber, that Sage Ther­a­peu­tics’ vaunt­ed drug for ma­jor de­pres­sive dis­or­der had failed a large tri­al, a read­out that has since cost the com­pa­ny $6 bil­lion in mar­ket cap and in­duced them to cut more than half their staff. Prax­is’s lead drug goes af­ter the same in­di­ca­tion.

Mar­cio Souza

“It’s a chal­leng­ing field,” Prax­is CEO Mar­cio Souza ac­knowl­edged in an in­ter­view.

The com­pa­ny be­gan as an ef­fort to find de no­vo mu­ta­tions that caused epilep­sy — the spon­ta­neous ge­net­ic mal­func­tions that cause the dis­ease in pa­tients who did not in­her­it it from their par­ents. The search turned up, among oth­er things, a gene that af­fects cal­ci­um chan­nels in the brain. But in­stead of try­ing to fix that mu­ta­tion, they used that in­for­ma­tion to fig­ure out how that chan­nel — and how that chan­nel falling out of bal­ance, with neu­rons fir­ing too much or too lit­tle —played a role in oth­er dis­eases.

“What be­came quite clear is that when you were look­ing be­yond just the pure mu­ta­tions … we were all talk­ing about im­bal­ances in a giv­en part of the brain, and nor­mal­ly as it re­lates to a spe­cif­ic chan­nel,” Souza said. “A lot of peo­ple jumped in the past from mu­ta­tion — cor­rect­ing that mu­ta­tion, or cor­rect­ing the ge­net­ic de­fect. What we’re do­ing dif­fer­ent­ly is look­ing in­to how that man­i­fests and at­tempt­ing to cor­rect the ac­tu­al man­i­fes­ta­tion.”

The re­sult was PRAX-944, a T-type cal­ci­um chan­nel block­er that is in the ear­ly stages of de­vel­op­ment for rare forms of ge­net­ic epilep­sy, but which the com­pa­ny is more ag­gres­sive­ly tak­ing for­ward in es­sen­tial tremor, one of the more com­mon neu­ro­log­i­cal con­di­tions. They ex­pect to have Phase II proof-of-con­cept re­sults be­fore the end of the year.

The lead pro­gram, though, is PRAX-114, a de­pres­sion drug. The over­lap­ping in­di­ca­tions here are per­i­menopausal de­pres­sion, a rel­a­tive­ly rare con­di­tion com­pared with ma­jor de­pres­sive dis­or­der. De­spite the vast med­ical need and mar­ket, sci­en­tists have strug­gled for years lead­ing up to Sage’s flop to build bet­ter an­ti-de­pres­sants.

Prax­is hopes to have an an­swer on their drug soon, with plans to en­ter a piv­otal tri­al be­fore the end of the year. The drug works by al­loster­i­cal­ly tar­get­ing GABAa, the neu­ro­trans­mit­ter im­pli­cat­ed in a long list of dis­or­ders and tar­get­ed head-on by ben­zo­di­azepines. Souza said the new tri­al will try to track pa­tients as they would use it in their dai­ly lives, hop­ing to show what he says they saw in Phase II: a safe and quick drug.

“They have the right safe­ty pro­file,” Souza said. “Most of the is­sue is not on­ly with the ef­fi­ca­cy, but with the safe­ty of these com­pounds. And we think 114 has that bal­ance.”

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Adam Russell, ARPA-H's incoming acting deputy director

NI­H's new, in­de­pen­dent break­through drug ac­cel­er­a­tor ARPA-H gets its first em­ploy­ee

Despite the controversy of housing it in NIH, HHS Secretary Xavier Becerra on Wednesday afternoon formally announced the establishment of the Advanced Research Project Agency for Health (ARPA-H) as an independent entity within the NIH, as HHS had previously stipulated that “NIH may not subject ARPA-H to NIH policies.”

Becerra also announced the appointment of ARPA-H’s inaugural employee, Adam Russell, who will serve as acting deputy director.

Serhat Gumrukçu, Enochian BioSciences co-founder (Seraph Research Institute)

LA biotech founder ar­rest­ed, charged in mur­der-for-hire scheme be­hind 2018 death

A biotech founder has been arrested and charged for his role in a murder-for-hire scheme that resulted in the death of a man in Vermont back in 2018.

Serhat Gumrukçu, the co-founder of Enochian BioSciences, was arrested in Los Angeles, where the company is based, according to the Department of Justice. He was charged alongside Berk Eratay of Las Vegas, and a third person, Jerry Banks of Colorado, was previously arrested for kidnapping and allegedly murdering the victim, Gregory Davis.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,700+ biopharma pros reading Endpoints daily — and it's free.

ProFound Therapeutics founding team

Flag­ship's lat­est biotech could turn some of the thou­sands of new pro­teins it dis­cov­ered in­to ther­a­pies — and it has $75M to start

Flagship Pioneering, the incubator of Moderna and dozens of other biotechs, says it has landed upon tens of thousands of previously undiscovered human proteins. The VC shop wants to potentially turn them into therapeutics.

Like other drug developers that have turned proteins into therapeutics (think insulin for diabetes), Flagship’s latest creation, ProFound Therapeutics, wants to tap into this new trove of proteins as part of its mission to treat indications ranging from rare diseases to cancer to immunological diseases.

Richard Silverman, Akava Therapeutics founder and Northwestern professor

This time around, Lyri­ca's in­ven­tor is de­vel­op­ing his North­west­ern dis­cov­er­ies at his own biotech

Richard Silverman was left in the dark for the last five years of clinical development of the drug he discovered. The Northwestern University professor found out about the first approval of Lyrica, in the last few days of 2004, like most other people: in the newspaper.

What became one of Pfizer’s top-selling meds, at $5 billion in 2017 global sales before losing patent protection in 2019, started slipping out of his hands when Northwestern licensed it out to Parke-Davis, one of two biotechs that showed interest in developing the drug in the pre-email days, when the university’s two-person tech transfer team had to ship out letters to garner industry appetite.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,700+ biopharma pros reading Endpoints daily — and it's free.

Peter Thompson, Terremoto Biosciences interim CEO

For­mer Prin­cip­ia team looks to shake up co­va­lent small mol­e­cules again, this time at 'earthquake' scale

Terremoto Biosciences goes back a long ways, in a sense, to about a dozen years ago when Principia Biopharma was founded by UCSF professor Jack Taunton. Peter Thompson initially helmed the biotech.

The company helped expand covalent small molecule inhibitors beyond oncology and into autoimmune disease by targeting cystine. But that amino acid is uncommon in a lot of proteins, offering fewer drug targets than, say, lysine, which is present in most proteins of interest. So, over the years, Taunton went back to the drawing board to check out that second amino acid.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,700+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,700+ biopharma pros reading Endpoints daily — and it's free.

David Ricks, Eli Lilly CEO (David Paul Morris/Bloomberg via Getty Images)

Eli Lil­ly set to in­vest $2.1B in home state man­u­fac­tur­ing boost

Eli Lilly is looking to expand its footprint in its home Hoosier State by making a major investment in manufacturing.

The pharma is investing $2.1 billion in two new manufacturing sites at Indiana’s LEAP Lebanon Innovation and Research District in Boone County, northwest of Lilly’s headquarters in Indianapolis.

The two new facilities will expand Lilly’s manufacturing network for active ingredients and new therapeutic modalities, including genetic medicines, according to a press release.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 142,700+ biopharma pros reading Endpoints daily — and it's free.

Up­dat­ed: US sees spike in Paxlovid us­age as Mer­ck­'s mol­nupi­ravir and As­traZeneca's Evusheld are slow­er off the shelf

New data from HHS show that more than 162,000 courses of Pfizer’s Covid-19 antiviral Paxlovid were administered across the US over the past week, continuing a streak of increased usage of the pill, and signaling not only rising case numbers but more awareness of how to access it.

In comparison to this week, about 670,000 courses of the Pfizer pill have been administered across the first five months since Paxlovid has been on the US market, averaging about 33,000 courses administered per week in that time.