Gil Beyen, Erytech Pharma CEO

French pen­ny stock will no longer knock on FDA door for ap­proval in a cer­tain type of blood can­cer

Things are not look­ing up for the French biotech Ery­tech Phar­ma.

The com­pa­ny is knock­ing off its plan to get FDA ap­proval for its drug Gras­pa in hy­per­sen­si­tive acute lym­phoblas­tic leukemia (ALL), fol­low­ing feed­back from the reg­u­la­tors, it said in a press re­lease Wednes­day af­ter­noon. ALL is the com­mon type of child­hood can­cer di­ag­nosed in ap­prox­i­mate­ly 5,000 new cas­es each year in the Unit­ed States.

“We are ob­vi­ous­ly dis­ap­point­ed to stop the process of seek­ing ap­proval for Gras­pa in ALL af­ter all the work done and clin­i­cal promise ob­served in this in­di­ca­tion,” Ery­tech CEO Gil Beyen said in a press state­ment.

The com­pa­ny’s stocks $ERYP plunged more than 20% fol­low­ing the news.

Af­ter pos­i­tive re­sults of a Phase II tri­al, Ery­tech had been in dis­cus­sions with the FDA for the ap­proval of Gras­pa to treat ALL pa­tients who had pre­vi­ous­ly ex­pe­ri­enced hy­per­sen­si­tiv­i­ty re­ac­tions to pe­gy­lat­ed as­parag­i­nase ther­a­py.

How­ev­er, the chang­ing com­pet­i­tive land­scape and the FDA’s re­quest for more clin­i­cal da­ta made it too dif­fi­cult for Ery­tech, lead­ing to its de­ci­sion to with­draw its ap­pli­ca­tion, ac­cord­ing to the com­pa­ny’s press state­ment.

In 2017, the FDA ap­proved Kym­ri­ah (ti­s­agen­le­cleu­cel) — a CAR-T ther­a­py for cer­tain pe­di­atric and young adult pa­tients with a form of ALL that has be­come one of the go-to ther­a­pies for the con­di­tion.

Near­ly four years ago, Ery­tech cel­e­brat­ed its im­pres­sive Phase IIb da­ta for sec­ond-line metasta­t­ic pan­cre­at­ic can­cer, but that hype died when the drug failed the Phase III piv­otal tri­al. That led the stock to fall 28%.

Gras­pa is Ery­tech’s lead prod­uct can­di­date. It con­tains a chemother­a­peu­tic agent L-as­parag­i­nase en­cap­su­lat­ed in­side a donor-de­rived red blood cell. By en­cap­su­lat­ing the L-as­parag­i­nase in­to red blood cells, Gras­pa aims to en­large its ther­a­peu­tic win­dow.

Mean­while, the com­pa­ny has been un­der­go­ing a “strate­gic re­view” since at least April. Ery­tech sold its com­mer­cial-scale cell ther­a­py man­u­fac­tur­ing fa­cil­i­ty in Prince­ton, NJ to Catal­ent for $44.5 mil­lion, and 40 peo­ple from Ery­tech’s New Jer­sey lo­ca­tion would be of­fered em­ploy­ment at Catal­ent.

For now, the com­pa­ny will fo­cus on its oth­er pre­clin­i­cal pro­grams, “while pur­su­ing strate­gic part­ner­ing op­tions to max­i­mize val­ue for our share­hold­ers and em­ploy­ees,” Beyen said.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Who’s spend­ing and who’s cut­ting from Big Phar­ma’s $127B R&D bud­get? Here are the top 15 play­ers

A couple of the Big 15 biopharma companies in R&D hit the gas on research spending last year. Merck and Sanofi still have lots to prove in the pipeline, and they’re willing to gamble large sums to make a better future for themselves.

Doing nothing would be infinitely worse.

But collectively, the top players rang up a modest 2.4% increase in spending in 2022, which didn’t cover inflationary pressures. And that set the tone for an extraordinarily cautious year for the industry — even as it laid out about $127 billion to advance new drugs or up the ante on approved therapies.

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Jeff Bluestone (R), Sonoma Biotherapeutics CEO

Jef­frey Blue­stone brings his start­up haul to $400M+, join­ing forces with Re­gen­eron on cell ther­a­pies

These days, when Jeffrey Bluestone gets together with his contemporaries in science, the conversation often turns to retirement plans.

But a little more than three years ago, Bluestone reached a momentous turning point in his career, exiting a prestigious post at UCSF, where he had spent decades in the scientific pursuit of new therapies. And it had nothing to do with retirement anytime in the near future.

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Mihael Polymeropoulos, Vanda Pharmaceuticals CEO

Van­da wins court case against FDA over dis­clo­sure of CRL de­tails for sleep drug

DC District Court Judge Christopher Cooper today granted Vanda Pharma’s request to require the FDA to disclose more info on the complete response letter for its sleep disorder drug Hetlioz.

The melatonin receptor agonist is approved by the FDA to treat non-24-hour sleep-wake disorder, a circadian rhythm disorder. But in 2018 Vanda filed a supplemental application to market Hetlioz as a treatment for jet lag, which the FDA rejected in August 2019, with few details on what Vanda needed to correct course, according to the company.

Gun­ning for 2023 ap­proval, GSK de­tails PhI­II da­ta for Jem­per­li in front­line en­dome­tri­al can­cer

GSK has a new slate of data to offer on its PD-1 inhibitor, Jemperli — data that the pharma giant hopes will cement one of the four drug approvals it’s expecting this year.

While Jemperli (dostarlimab) is already approved for a subset of patients with second-line endometrial cancer, GSK set out in the Phase III RUBY trial to test it as an earlier line of treatment while also enrolling a broader group of patients. In an interim analysis, Jemperli was shown to extend progression-free survival for both the subset and the overall trial population when added to chemotherapy.

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Chaim Lebovits, BrainStorm Cell Therapeutics CEO

Brain­Storm gets FDA ad­comm for ALS drug af­ter failed tri­al and RTF

BrainStorm Cell Therapeutics has had a rocky road trying to get its ALS treatment through to the FDA, including a particularly difficult setback after the FDA slapped the company with a refusal to file (RTF) letter in November after it submitted its original BLA in September last year.

But there’s a glimmer of hope for BrainStorm’s ALS treatment, NurOwn – the FDA will hold an advisory committee meeting to discuss the company’s BLA for the treatment, the company announced today.

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Doug Williams, departing Codiak BioSciences CEO

Co­di­ak files for Chap­ter 11 bank­rupt­cy as most ex­ec­u­tives head for the ex­it

Codiak BioSciences has filed for Chapter 11 bankruptcy, spelling an end to the employment of most executives, including founder Doug Williams, as the biotech says it “expects to consummate a sale.”

The eight-year journey at Codiak is nearing an end with Williams; CFO Linda Bain; medical chief David Mauro; scientific head Sriram Sathyanarayanan; legal and compliance chief Yalonda Howze; and SVP of HR Nicole Barna all packing up their bags in the first few days of April. Chief technology officer Konstantin Konstantinov will stay.

Rep. Cathy McMorris Rodgers (R-WA) (Michael Brochstein/Sipa USA/Sipa via AP Images)

House com­mit­tee chair re­quests in­fo from FDA on fund­ing for tri­als in­volv­ing 'vi­ral ma­nip­u­la­tion'

Republican members of Congress want FDA Commissioner Robert Califf and his agency to provide more information on the research FDA has funded that manipulates certain viruses like the SARS-CoV-2 virus — after the FDA confirmed that certain studies it funded involved viral manipulation, but in standard ways that are common in molecular biology.

The three-page letter, signed Monday by House Energy and Commerce Committee Chair Cathy McMorris Rodgers (R-WA) and Subcommittee Chairs Brett Guthrie (R-KY) and Morgan Griffith (R-VA), kicked things off by saying that the committee was looking into FDA’s oversight of “FDA-funded research that may pose significant biosafety or biosecurity risks.”

FDA plans up­com­ing ad­comms for OTC con­tra­cep­tive, ep­i­neph­rine nasal spray, Intar­ci­a's last shot

The FDA has scheduled or planned for three advisory committee meetings in May: one for an over-the-counter birth control pill, one for a nasal spray version of the EpiPen, and another for  Intarcia Therapeutics, which has all but completely folded already as a company.

Perrigo will bring its daily contraceptive, Opill, to the joint Nonprescription Drugs Advisory Committee and the Obstetrics, Reproductive and Urologic Drugs Advisory Committee on May 9 and 10 for its over-the-counter birth control application.