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.”

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.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; and more

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Usama Malik

Ex-Im­munomedics CFO charged with in­sid­er trad­ing, faces up to 20 years in prison af­ter al­leged­ly tip­ping off girl­friend and rel­a­tives of a PhI­II suc­cess

The former CFO of Immunomedics, who helped steer the company to its $21 billion buyout by Gilead last year, has been charged with insider trading, the Department of Justice announced Thursday.

Usama Malik tipped off his then-girlfriend and four others that a Phase III study for Trodelvy would be stopped early four days before Immunomedics publicly announced the result in April 2020, DoJ alleged in its complaint. The individuals then purchased Immunomedics shares, selling them after the news broke and Immunomedics’ stock price doubled.

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Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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Vas Narasimhan, Novartis CEO (Thibault Camus/Pool via AP Images)

With gener­ic com­pe­ti­tion heat­ing up, Vas Narasimhan out­lines No­var­tis' growth plans at R&D day

Thursday marks Novartis’ annual R&D day, and with it comes CEO Vas Narasimhan’s attempt to spotlight the company’s pipeline strategy and emerging stars.

The biggest question entering Thursday’s presentation dealt with how the big biopharma will make up revenues from upcoming generic competition — Novartis says within the next five years, generics will eat away roughly $9 billion in sales. To offset this, Narasimhan outlined a strategy for 4% growth or higher until 2026, focusing on six key medicines he believes will see multibillion dollar profits during this time.

Radek Spisek, Sotio CEO (Cellestia)

A qui­et Czech biotech bags $315M to dri­ve its blos­som­ing can­cer pipeline through the clin­ic

In the rather insular world of biotech, most innovation inevitably comes from a cluster of R&D hubs — Cambridge, San Francisco, etc. But sometimes success stories sprout from rocky soil, which is most certainly the case with Prague-based Sotio Biotech and its suddenly jam-packed pipeline of cancer drugs.

After years in quiet development, Sotio now has $315 million in new funds to play with from parent company PPF Group, an investment group founded in the Czech Republic, as the biotech looks to advance its growing pipeline through early- and mid-stage trials.

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In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

Reshma Kewalramani, Vertex CEO (Vertex via YouTube)

Bat­tling a line­up of skep­tics, Ver­tex claims an­oth­er ear­ly clin­i­cal win — this time in kid­ney dis­ease

Vertex claimed its second early-stage win of the fall Wednesday, announcing positive results in a small study on a genetically defined form of kidney disease.

The 16-patient, Phase II trial focused on patients with focal segmental glomerulosclerosis, a rare disease where kidneys are unable to filter blood properly. Over 13 weeks on an experimental pill, the level of protein in the patients’ urine fell by an average of 47.6%.