Paula Ragan, X4 Pharmaceuticals CEO

X4 slash­es pipeline, lays off staff just three weeks af­ter net­ting a $55M PIPE

Not even a PIPE fi­nanc­ing could save X4 Phar­ma­ceu­ti­cals from en­act­ing lay­offs and trim­ming its pipeline.

The Boston biotech re­vealed Wednes­day af­ter­noon that it would down­size its re­search to fo­cus on its lead pro­gram, ma­vorix­afor, and lay off about 20% of its staff. The move comes less than three weeks af­ter X4 pulled to­geth­er a $55 mil­lion PIPE and amend­ed a loan fa­cil­i­ty with Her­cules Cap­i­tal to re­duce cash burn.

“We be­lieve that com­mer­cial­iz­ing ma­vorix­afor and pro­vid­ing a new ther­a­peu­tic op­tion to in­di­vid­u­als with life-threat­en­ing CN dis­or­ders has the po­ten­tial to rev­o­lu­tion­ize the treat­ment land­scape,” CEO Paula Ra­gan said in a state­ment.

X4 is de­vel­op­ing ma­vorix­afor in a hand­ful of dif­fer­ent in­di­ca­tions, with the top pri­or­i­ty be­ing WHIM syn­drome (warts, hy­pogam­ma­glob­u­line­mia, in­fec­tions, and myelokathex­is). Da­ta from a Phase III study are ex­pect­ed in the fourth quar­ter this year, X4 said Wednes­day, and an FDA sub­mis­sion is ex­pect­ed in the sec­ond half of 2023.

That would al­so mark a de­lay from the com­pa­ny’s pre­vi­ous guid­ance of 2023’s first quar­ter. X4 said as re­cent­ly as its Q3 2021 re­port the NDA would come dur­ing that quar­ter, though its lan­guage shift­ed in a Q4 fil­ing to a “pos­si­ble” NDA in 2023’s lat­ter half. Re­searchers are al­so de­vel­op­ing the pro­gram for chron­ic neu­tropenic dis­or­ders, where Phase Ib da­ta are ex­pect­ed to in­form next reg­u­la­to­ry steps this quar­ter.

But with the re­or­ga­ni­za­tion, X4 now plans to part­ner out some of ma­vorix­afor’s de­vel­op­ment costs. The biotech is cur­rent­ly re­search­ing how the pro­gram can im­pact pa­tients with a rare form of non-Hodgkin lym­phoma known as Walden­ström’s macroglob­u­line­mia, but de­spite new Phase Ib da­ta ex­pect­ed next month, ex­ecs say new tri­als won’t go for­ward with­out a part­ner.

X4’s oth­er two an­nounced pro­grams, two small-mol­e­cule CX­CR4 an­tag­o­nists called X4P-003 and X4P-002, were placed es­sen­tial­ly in lim­bo. X4P-003’s next steps will de­pend on an ap­proval for ma­vorix­afor, the biotech said, while X4P-002 — which hasn’t reached an IND fil­ing — will on­ly ad­vance if X4 finds a part­ner.

Ma­vorix­afor came to X4 in a 2015 deal with Sanofi, as the com­pa­ny launched with cash from Gen­zyme founder Hen­ri Ter­meer and the pro­gram it picked up off the Big Phar­ma’s scrapheap. The com­pa­ny added an­oth­er $27 mil­lion in a 2017 Se­ries B be­fore re­verse-merg­ing on­to Nas­daq in late 2018, us­ing a sub­sidiary of the now-de­funct Ar­sa­nis.

UP­DAT­ED: In a fresh dis­ap­point­ment, Am­gen spot­lights a ma­jor safe­ty is­sue with KRAS com­bo

Amgen had hoped that its latest study matching its landmark KRAS G12C drug Lumakras with checkpoint inhibitors would open up its treatment horizons and expand its commercial potential. Instead, the combo spurred safety issues that blunted efficacy and forced the pharma giant to alter course on its treatment strategy, once again disappointing analysts who have been tracking the drug’s faltering sales and limited therapeutic reach.

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Ad­dress­ing the ‘Ca­pac­i­ty Crunch’ with a Scal­able Plat­form Process Ap­proach

The field of gene therapy has been diligently moving forward over the past several decades to bring potentially life-saving treatments to patients with genetic diseases. In addition to two approved adeno-associated viral (AAV) gene therapies, there are more than 250 AAV gene therapies in various clinical trial stages.1 AAV vectors remain the most frequently used vector for delivering therapeutic transgenes to target tissues due to their demonstrated and lasting clinical efficacy and extensive safety track record. As AAV therapies advance through clinical trials and into commercialization, many biotech companies are turning to contract development and manufacturing organizations (CDMOs) to prepare their programs for late-stage clinical and commercial scale manufacturing. Given the scope and scale of the manufacturing needs that will accompany regulatory approvals for these assets, CDMOs continue to expand their capacity to meet the needs of increasing prevalent patient populations. However, despite rapid growth, projected gene therapy manufacturing demands still outpace the collective capacity of the CDMO industry.

A $5B Pfiz­er buy­out? Am­gen, Gilead head­line M&A Thurs­day; Al­ny­lam's AT­TR sweep; An­drew Lo's rare dis­ease quest; 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.

One of the cool things about adding EndpointsPharma to the daily roster is that my colleagues can now dedicate time to tracking quarterly updates and tuning into calls with Big Pharma companies. Check out their dispatch from the Q2 earnings below.

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Bob Bradway, Amgen CEO (Justin Kase Conder/AP Images for Amgen)

UP­DAT­ED: Am­gen chief Brad­way nabs a rare dis­ease play­er in $4B buy­out as the M&A tem­po ac­cel­er­ates

Amgen CEO Bob Bradway is bellying up to the M&A table today, scooping up the newly anointed commercial biotech ChemoCentryx $CCXI and its recently approved rare disease drug for $3.7 billion out of the cash stockpile. The deal comes in at $52 a share — a hefty increase over the $24.11 close yesterday.

Bradway and the Amgen team get a drug called Tavneos (avacopan) in the deal, a complement factor C5a inhibitor OK’d to treat anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis, an autoimmune disease which can be lethal.

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Albert Bourla, Pfizer CEO (Laurent Gillieron/Keystone via AP)

Break­ing: Pfiz­er in hot pur­suit of a $5B buy­out of Glob­al Blood Ther­a­peu­tics — re­port

Pfizer CEO Albert Bourla has vowed to leave no stone unturned in the search for new biotech deals, and the BD team is not letting him down.

The Wall Street Journal reported today that Pfizer is in the final stages of acquiring Global Blood Therapeutics for $5 billion. According to the Journal report, though, Pfizer is not the only buyer at the deal table and while the pharma giant may be close to clinching it, there are no guarantees it will continue.

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George Yancopoulos, Regeneron president and CSO (Brendan McDermid/Reuters/Alamy)

George Yan­copou­los says he's on the trail of the holy grail: ‘This could rep­re­sent the next break­through for im­munother­a­py’

Two of the most outspoken — and successful — drug developers in biotech say they’ve collected early-stage clinical data that are pointing them down the trail to the holy grail in cancer immunotherapy R&D.

While analysts largely busied themselves today with chronicling the ongoing success of Regeneron’s two big cash cows — Dupixent and Eylea — chief scientist George Yancopoulos and CEO Len Schleifer used the Q2 call to spotlight their early success with a combination of the “homegrown” PSMAxCD28 costimulatory bispecific antibody REGN5678 in combination with their PD-1 checkpoint Libtayo. The presentation comes just weeks after Regeneron completed a deal to gather all rights to the PD-1 that had been in Sanofi’s hands. And the two top execs are unstinting in their praise of the potential of a whole set of costimulatory pipeline projects which they say may finally deliver the long-awaited next-level approach to broadening the immunotherapy field of drugs.

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(AP Photo/Richard Vogel, File)

US de­clares mon­key­pox a na­tion­al health emer­gency, as new drug­mak­ers con­sid­er en­ter­ing vac­cine race

Rising monkeypox cases have put the US on high alert as it announces a national health emergency, which grants the government more power in its response.

The news comes as Bavarian Nordic continues to fill orders for its Jynneos vaccine and other companies – including Moderna – consider jumping into the vaccine race. Meanwhile, the New York Times reports that the US has allowed around 20 million doses of smallpox vaccine in its stockpile to expire.

Al Sandrock, Voyager Therapeutics CEO

Al San­drock prunes his post-Bio­gen voy­age with sim­i­lar fo­cus on ALS

Al Sandrock is narrowing the focus of Voyager Therapeutics, concentrating on CNS diseases that were the hallmark of his time leading R&D at Biogen, including an emphasis on a familial form of ALS for which his former employer is getting a speedy review at the FDA.

Less than six months into his journey as CEO at Voyager, Sandrock is focusing the preclinical pipeline on Alzheimer’s disease, GBA1 Parkinson’s disease and SOD1 amyotrophic lateral sclerosis, the rare form of ALS for which the FDA will decide whether to approve Biogen’s tofersen by Jan. 25, 2023.

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David Hallal (L) and George Daley (Hallal photo: Bertrand Guay/AFP via Getty Images)

David Hal­lal's El­e­vate­Bio launch­es new com­pa­ny to 'dis­rup­t' off-the-shelf cell ther­a­py, but pro­vides few oth­er de­tails

David Hallal’s ElevateBio is launching a new company Thursday as it looks to continue making its mark in the cell and gene therapy spaces. But Hallal is also keeping his cards close to the vest, preferring to toe the line between bombast and mystique rather than going all-in in one direction.

The new company comes out of a partnership with Boston Children’s Hospital and research from George Daley, the dean of Harvard Medical School. The triumvirate claims to have found a way to design better off-the-shelf cell therapies using new methods discovered in Daley’s Boston Children’s Hospital lab (Harvard is not involved in the collaboration).

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