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.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

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

No­var­tis bol­sters Plu­vic­to's case in prostate can­cer with PhI­II re­sults

The prognosis is poor for metastatic castration-resistant prostate cancer (mCRPC) patients. Novartis wants to change that by making its recently approved Pluvicto available to patients earlier in their course of treatment.

The Swiss pharma giant unveiled Phase III results Monday suggesting that Pluvicto was able to halt disease progression in certain prostate cancer patients when administered after androgen-receptor pathway inhibitor (ARPI) therapy, but without prior taxane-based chemotherapy. The drug is currently approved for patients after they’ve received both ARPI and chemo.

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FDA grants or­phan drug des­ig­na­tion to Al­ger­non's ifen­prodil, while ex­clu­siv­i­ty re­mains un­clear

As the FDA remains silent on orphan drug exclusivity in the wake of a controversial court case, the agency continues to hand out new designations. The latest: Algernon Pharmaceuticals’ experimental lung disease drug ifenprodil.

The Vancouver-based company announced on Monday that ifenprodil received orphan designation in idiopathic pulmonary fibrosis (IPF), a chronic lung condition that results in scarring of the lungs.  Most IPF patients suffer with a dry cough, and breathing can become difficult.

Albert Bourla, Pfizer CEO (Efren Landaos/Sipa USA/Sipa via AP Images)

Pfiz­er makes an­oth­er bil­lion-dol­lar in­vest­ment in Eu­rope and ex­pands again in Michi­gan

Pfizer is continuing its run of manufacturing site expansions with two new large investments in the US and Europe.

The New York-based pharma giant’s site in Kalamazoo, MI, has seen a lot of attention over the past year. As a major piece of the manufacturing network for Covid-19 vaccines and antivirals, Pfizer is gearing up to place more money into the site. Pfizer announced it will place $750 million into the facility, mainly to establish “modular aseptic processing” (MAP) production and create around 300 jobs at the site.

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Sekar Kathiresan, Verve Therapeutics CEO

Verve re­veals let­ter from FDA that lays out con­di­tions to lift base edit­ing tri­al hold

We now know why Verve’s lead candidate was placed on hold last month by US regulators.

In an SEC filing, Verve laid out the FDA’s conditions for lifting the hold on its lead therapy, VERVE-101. That includes submitting preclinical data about potency differences in human versus non-human cells, risks of gene editing germline cells, and off-target analyses in non-hepatocyte cell types.

The FDA also wants clinical data from the ongoing Heart-1 trial, and to modify the trial protocol in the US to add additional contraceptive measures and increase the length of a staggering interval between the dosing of participants.

Nkarta CEO Paul Hastings at Endpoints' #BIO22 panel (J.T. MacMillan Photography for Endpoints News)

Nkar­ta un­der­scores safe­ty of CAR-NK, boasts ear­ly re­spons­es

The first generation of personalized CAR-T therapies made big waves in the treatment of lymphoma for their stunning efficacy. Nkarta is hoping its off-the-shelf natural killer cell approach will stand out on safety — while keeping some of those impressive numbers on responses.

In a new update from its Phase I dose escalation study, the South San Francisco-based biotech reported that seven out of 10 patients treated with the highest doses of its NK cell therapy, NKX019, achieved a complete response, translating to a complete response rate of 70%.

Pfiz­er-backed Me­di­ar Ther­a­peu­tics ropes in an­oth­er Big Phar­ma in­vestor

A biotech centered on treating fibrosis — born out of Mass General and Brigham and Women’s Hospital — has received a financial boost.

According to an SEC filing, the company has raised $31,761,186 in its latest funding round, which includes 17 investors. The filing lists six names attached to the company, including Meredith Fisher, a partner at Mass General Brigham Ventures and Mediar’s acting CEO.

Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

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