A few months af­ter emerg­ing from stealth, Prax­is is hand­ed a clin­i­cal hold for lead de­pres­sion drug

Less than a month af­ter Prax­is Pre­ci­sion Med­i­cines hit Wall Street with an up­sized IPO, the FDA has slapped a clin­i­cal hold on its lead can­di­date for ma­jor de­pres­sive dis­or­der (MDD), send­ing shares spi­ral­ing.

With­out pro­vid­ing much in­for­ma­tion, the FDA said in an email that it re­viewed Prax­is’ IND for its Phase II/III tri­al, and is plac­ing a full clin­i­cal hold on the study. More de­tails should come in the next 30 days, ac­cord­ing to the email. Prax­is says it has reached out to the agency, which said it isn’t done fi­nal­iz­ing com­ments.

“The FDA has not pro­vid­ed any rea­son for the clin­i­cal hold,” Prax­is said in a state­ment. Ac­cord­ing to an S-1/A filed on Oct 15, the study would have sat­is­fied one of two reg­is­tra­tional tri­als re­quired by the FDA to sup­port clin­i­cal ef­fi­ca­cy. The biotech’s stock $PRAX plunged more than 26% af­ter the news broke on Tues­day.

Cowen an­a­lysts aren’t wor­ried, though. In a note to in­vestors on Tues­day, an­a­lysts Ritu Bar­al and Ly­la Youssef said they be­lieve the hold “should be re­solved in short or­der giv­en the con­tin­ued clean pre­clin­i­cal and clin­i­cal safe­ty pro­file.” The can­di­date, PRAX-114, is cur­rent­ly in an on­go­ing Phase IIa tri­al in Aus­tralia. Part A en­rolled 33 MDD pa­tients, and ear­ly da­ta sug­gest “rapid, marked im­prove­ments in de­pres­sion scores” af­ter two weeks of treat­ment, ac­cord­ing to the an­a­lysts. Af­ter one week, pa­tients showed LS mean Hamil­ton De­pres­sion Rat­ing Scale im­prove­ments of 15 to 19 points across three dose groups.

The can­di­date, GA­BA pos­i­tive al­losteric mod­u­la­tor, is al­so in Phase II tri­als for per­i­menopausal de­pres­sion. The field is a tough one, with sci­en­tists strug­gling for years to de­vel­op bet­ter an­ti-de­pres­sants. Last De­cem­ber, Sage Ther­a­peu­tics’ MDD drug flopped in a large tri­al, cost­ing the com­pa­ny $6 bil­lion in mar­ket cap and forc­ing it to cut half of em­ploy­ees.

If PRAX-114 gets ap­proved, Cowen pre­dicts its US sales po­ten­tial will  be in the $3 bil­lion range.

Prax­is emerged from stealth mode this spring, with $100 mil­lion and two drugs in Phase II. It ini­tial­ly set out to iden­ti­fy mu­ta­tions that cause epilep­sy in pa­tients who didn’t in­her­it the dis­ease, and is now ap­ply­ing those dis­cov­er­ies to oth­er CNS dis­eases, like de­pres­sion, move­ment dis­or­ders and pain syn­dromes. In Ju­ly, Prax­is bagged an­oth­er $110 mil­lion in a Se­ries C1 led by Even­tide As­set Man­age­ment.

Even­tide holds 9.4% of Prax­is’ stock, ac­cord­ing to the S-1/A. Black­stone, No­vo Hold­ings and Vi­da Ven­tures, which al­so kicked in on the round, have 23.1%, 6/6% and 7.3% re­spec­tive­ly.

Back in Au­gust, Prax­is filed for a $100 mil­lion IPO. It end­ed up bring­ing in $190 mil­lion, from $10 mil­lion shares priced at $19 apiece — a buck high­er than the $17 to $18 range. Be­tween $70 to $80 mil­lion was tagged for PRAX-114.

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His­toric drug pric­ing re­forms pass; Pfiz­er ac­quires GBT; The long search for non-opi­oid pain drugs; 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 Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

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Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

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Seagen interim CEO Roger Dansey and Daiichi Sankyo CEO Sunao Manabe

Paving the way for Mer­ck­'s buy­out, Seagen los­es ar­bi­tra­tion dis­pute with Dai­ichi over ADC tech

As Seagen awaits a final buyout offer from Merck that could be in the territory of $40 billion, Seagen revealed Friday afternoon that it lost an arbitration dispute with Daiichi Sankyo relating to the companies’ 2008 collaboration around the use of antibody-drug conjugate (ADC) technology.

But that loss likely won’t matter much when it comes to Merck’s deal.

After breaking off its pact with Daiichi in mid-2015, the two companies battled over “linker” tech — a chemical bridge between an ADC’s antibody component and the cytotoxic payload — that Seagen claims Daiichi would improve upon and implement in its current generation of ADCs.

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FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

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Elcin Barker Ergun, Menarini Group CEO

Amid Roche and Sanofi's oral SERD set­backs, Menar­i­ni gets speedy re­view at FDA

Menarini and Radius Health are getting a speedy review at the FDA for their oral SERD breast cancer drug months after the field opened up with competitors failing and fleeing.

It was a one-two-three punch in March, April and May as Sanofi flunked its first big test for its oral selective estrogen receptor degrader (SERD), Roche also flamed out in a Phase II and G1 Therapeutics ended its program after scoping out the data and potential partners.

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Carole Ben-Maimon, CEO of Larimar Therapeutics

Lari­mar to re­spond to FDA this quar­ter on 15-month clin­i­cal hold of PhI Friedre­ich's atax­ia drug

More than a year after the FDA clamped down on Larimar Therapeutics with a clinical hold on its lead drug, the biotech thinks it has a way to get that lifted — and an answer could come next quarter if all goes to plan.

Amid a 15-month setback, the biotech plans on filing a complete response to the FDA’s concerns over its drug, CTI-1601, sometime this quarter, which is supposed to elicit an answer from the agency within 30 days, per the regulator.

House pass­es his­toric drug pric­ing re­forms, lin­ing up decades-in-the-mak­ing win for Biden and De­moc­rats

The US House of Representatives today voted along party lines (all Dems voted for it), 220-207 to pass new, wide-ranging legislation that will allow Medicare drug price negotiations for the first time ever, and cap seniors’ drug expenses to $2,000 per year and seniors’ insulin costs at $35 per month.

Setting up a major victory for President Joe Biden, representatives returned from their summer recess to pass the Inflation Reduction Act, even as many noted the bill would only modestly reduce inflation.

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Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.