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.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; 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.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

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No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'


Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

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Roche's Tecen­triq cross­es the fin­ish line first in ad­ju­vant lung can­cer, po­ten­tial­ly kick­ing off gold rush

While falling behind the biggest PD-(L)1 drugs in terms of sales, Roche has looked to carve out a space for its Tecentriq with a growing expertise in lung cancer. The drug will now take an early lead in the sought-after adjuvant setting — but competitors are on the way.

The FDA on Friday approved Tecentriq as an adjuvant therapy for patients with Stage II-IIIA non small cell lung cancer with PD-(L)1 scores greater than or equal to 1, making it the first drug of its kind approved in an early setting that covers around 40% of all NSCLC patients.

Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

A star Stan­ford pro­fes­sor leaves his lab for a start­up out to re­make psy­chi­a­try

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.

FDA ad­comm votes unan­i­mous­ly in sup­port of a J&J Covid-19 boost­er two months af­ter one-dose shot

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Friday voted 19-0 in favor of authorizing a second shot of J&J’s Covid-19 vaccine to follow at least two months after the initial dose.

Regulators don’t have to follow VRBPAC’s recommendation, but they almost always do. Considering that the CDC’s advisory committee has already been set to review the expanded EUA, VRBPAC’s recommendation is likely to be adopted.

Susan Galbraith speaking at Endpoints News' virtual EUBIO21 summit

Imfinzi/treme­li­mum­ab com­bo scores As­traZeneca an­oth­er OS win — this time in liv­er can­cer

Is the tide turning on AstraZeneca’s battered PD-L1/CTLA4 combo?

A single priming dose of the experimental tremelimumab, followed by Imfinzi every four weeks, beat Nexavar (sorafenib) in helping a group of liver cancer patients live longer in a Phase III study, the company reported, meeting the primary endpoint.

Specifically, the two drugs extended overall survival for patients with unresectable hepatocellular carcinoma who had not received prior systemic therapy and were not eligible for localized treatment.

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FDA's vac­cine ad­comm unan­i­mous­ly sup­ports Mod­er­na's boost­er in same pop­u­la­tions as Pfiz­er's boost­er

The FDA’s vaccine advisory committee on Thursday voted 19-0 in support of expanding Moderna’s Covid-19 vaccine EUA for booster doses for certain high-risk individuals. FDA is expected to authorize the Moderna booster shortly.

Similarly to the Pfizer booster shot, Moderna’s will likely be authorized for those older than 65, adults at high risk of severe Covid-19, and adults whose frequent institutional or occupational exposure to SARS-CoV-2 puts them at high risk of serious complications of Covid-19. But unlike the Pfizer adcomm, where FDA had to scramble to get the committee to vote in favor of a booster, this committee was unanimous with the Moderna shot.