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.”

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

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

The slate of products we’re offering here at Endpoints is continuing to grow, and it’s not just limited to editorial. If you haven’t, do visit your reader profile to see if there are any other weekly newsletters you’re interested in — as each comes with its own exclusive content. And don’t miss the publisher’s note from Arsalan Arif on Endpoints Studio, our latest avenue for advertising on Endpoints.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,600+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,600+ biopharma pros reading Endpoints daily — and it's free.

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.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,600+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,600+ biopharma pros reading Endpoints daily — and it's free.

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.

Ab­b­Vie tacks on a new warn­ing to Rin­voq la­bel as safe­ty frets crimp JAK class

The safety problems that continue to plague the JAK class as new data highlight some severe side effects are casting a large shadow over AbbVie’s Rinvoq.

As a result of a recent readout highlighting major adverse cardiac events (MACE), malignancy, mortality and thrombosis with Xeljanz a couple of months ago, AbbVie put out a notice late Friday afternoon that it is adding the new class risks to its label for their rival drug.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,600+ biopharma pros reading Endpoints daily — and it's free.

Biospec­i­men M&A: Dis­cov­ery ac­quires Al­bert Li's he­pa­to­cyte project; PhI­II tri­al on Bay­er's Nube­qa reached pri­ma­ry end­point

Discovery Life Sciences has acquired what claims to be the Maryland-based host of the world’s largest hepatocyte inventory, known as IVAL, to help researchers select more effective and safer drug candidates in the future.

The combined companies will now serve a wider range of drug research and development scientists, according to Albert Li, who founded IVAL in 2004 and is set to join the Discovery leadership team as the CSO of pharmacology and toxicology.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,600+ biopharma pros reading Endpoints daily — and it's free.