Un­fazed by dis­rup­tions, Cowen's in­vest­ment arm backs AM-Phar­ma's $176M piv­otal plan around lethal con­di­tion

It may not be the best time to start up a late-stage study un­re­lat­ed to Covid-19, but AM-Phar­ma isn’t let­ting a slight de­lay de­rail its over­all plan.

Erik van den Berg

The Dutch biotech has been sin­gu­lar­ly fo­cused on re­cAP, an an­ti-in­flam­ma­to­ry re­com­bi­nant hu­man form of an en­zyme orig­i­nal­ly found in cows called al­ka­line phos­phatase.

The one Phase III it be­lieves it needs for ap­proval was sched­uled for this sum­mer, fund­ed by a $133 mil­lion round last Ju­ly. Hav­ing pushed study ini­ti­a­tion to af­ter the sum­mer, AM-Phar­ma is adding $52 mil­lion for the reg­u­la­to­ry work need­ed to pre­pare for launch, in­clud­ing CMC val­i­da­tion and com­mer­cial sup­ply.

When the pan­dem­ic will peak is im­pos­si­ble to pre­dict, said CEO Erik van den Berg, but giv­en the sever­i­ty of the dis­ease that AM-Phar­ma is tack­ling — sep­sis-as­so­ci­at­ed acute kid­ney in­jury — they want­ed to start as soon as pos­si­ble and broad­ly stick to the orig­i­nal time­line. Da­ta from the Phase III tri­al are ex­pect­ed in 2023.

The new €23 mil­lion eq­ui­ty in­vest­ment by Cowen Health­care In­vest­ments, its first US-based in­vestor in the syn­di­cate, along­side the €24 mil­lion loan from the Eu­ro­pean In­vest­ment Bank, has boost­ed his con­vic­tion.

Right now, with all of AM-Phar­ma’s 23 staffers work­ing from home in Utrecht, the Nether­lands, crit­i­cal ac­tiv­i­ties are still on­go­ing. Doc­u­ments still need to be pre­pared for re­view be­fore sites can be ini­ti­at­ed — a siz­able project when you are plan­ning a tri­al in­volv­ing 1,400 pa­tients in 12 coun­tries.

“Our work is most­ly project man­age­ment and co­or­di­na­tion,” van den Berg said. “We don’t have any labs to close in this pe­ri­od. All of our sup­pli­ers are still on­line and al­so moved as much as pos­si­ble to home-based work­ing.”

There will be chal­lenges when the tri­al ac­tu­al­ly be­gins, as the pa­tients that will be re­cruit­ed tend to be treat­ed in in­ten­sive care units, many of which could be over­whelmed by se­vere coro­n­avirus pa­tients. While he is plan­ning for dif­fer­ent pos­si­bil­i­ties, van den Berg is hope­ful hu­man cre­ativ­i­ty will save them from the worst-case sce­nario.

In a sense it’s char­ac­ter­is­tic of a team that has built a busi­ness around of­fer­ing hope where there is lit­tle, re­duc­ing the mor­tal­i­ty rate by 40% ver­sus place­bo in Phase II. And it wouldn’t be the first time it has had to con­tend with un­fore­seen cir­cum­stances: van den Berg hadn’t been plan­ning on tak­ing the piv­otal pro­gram and com­mer­cial ef­forts un­til Pfiz­er pulled out of a deal to ac­quire the com­pa­ny, leav­ing it to go it alone.

“Worst case sce­nario is we’re sit­ting in our home of­fices for years,” he said. ”But I don’t think that’s re­al­is­tic. I don’t see it that bleak.”

Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; 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.

I hope your weekend is off to a nice start, wherever you are reading this email. As for me, I’m trying to catch the tail of the Lunar New Year festivities.

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Pfiz­er lays off em­ploy­ees at Cal­i­for­nia and Con­necti­cut sites

Pfizer has laid off employees at its La Jolla, CA, and Groton, CT sites, according to multiple LinkedIn posts from former employees.

The Big Pharma confirmed to Endpoints News it has let go of some employees, but a spokesperson declined to specify how many workers were impacted and the exact locations affected. Earlier this month, the drug developer had confirmed to Endpoints it was sharpening its focus and doing away with some early research on areas such as rare disease, oncology and gene therapies.

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Rodney Rietze, iVexSol CEO

Bris­tol My­ers, Charles Riv­er join Se­ries A fund­ing for iVex­Sol

Massachusetts-based iVexSol has secured funding to the tune of $23.8 million in its latest Series A round. The new investors include Bristol Myers Squibb, manufacturer Charles River Laboratories and Asahi Kasei Medical.

iVexSol is a manufacturer of lentiviral vectors (LVV), used in making gene therapies, and this latest round of fundraising brings its total Series A total over $39 million, which will be used to recruit more employees and bolster its technology.

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Jake Van Naarden, Loxo@Lilly CEO

Lil­ly en­ters ripe BTK field with quick FDA nod in man­tle cell lym­phoma

Eli Lilly has succeeded in its attempt to get the first non-covalent version of Bruton’s tyrosine kinase, or BTK, inhibitors to market, pushing it past rival Merck.

The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

Filip Dubovsky, Novavax CMO

No­vavax gets ready to take an­oth­er shot at Covid vac­cine mar­ket with next sea­son plans

While mRNA took center stage at yesterday’s FDA vaccine advisory committee meeting, Novavax announced its plans to deliver an updated protein-based vaccine based on new guidance.

Vaccines and Related Biological Products Advisory Committee (VRBPAC) members voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all future vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

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Eliot Forster, F-star CEO (Rachel Kiki for Endpoints News)

F-star gets down to the wire with $161M sale to Chi­nese buy­er as na­tion­al se­cu­ri­ty con­cerns linger

With the clock ticking on F-star Therapeutics’ takeover by a Chinese buyer, the companies are still scrambling to remove a hold on the deal from the US government’s Committee on Foreign Investment in the United States.

F-star and invoX Pharma said they are “actively negotiating” with CFIUS “about the terms of a mitigation agreement to address CFIUS’s concerns regarding potential national security risks posed by the transaction.”

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CBER Director Peter Marks (Susan Walsh/AP Images)

FDA ad­vi­so­ry com­mit­tee votes unan­i­mous­ly in fa­vor of bi­va­lent Covid shots re­plac­ing pri­ma­ry se­ries

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all current vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

The vote marks an effort to clear up confusion around varying formulations and dosing schedules for current primary series and booster vaccines, as well as “get closer to the strains that are circulating,” according to committee member Paul Offit, professor of pediatrics at the Children’s Hospital of Philadelphia.

John Rim, Samsung Biologics CEO (Samsung/PR Newswire)

Sam­sung Bi­o­log­ics spells out ex­pan­sion plans in South Ko­rea and US

The CDMO arm of one of South Korea’s largest conglomerates has posted its year-end results and plans for 2023, which include new construction.

Samsung Biologics netted north of KRW 3 trillion ($2.4 billion) in 2022 revenue and an operating profit of KRW 983.6 billion ($799 million), which the company touted on Friday as “record-high earnings.” The revenue boost was 55% compared to 2021.

No­var­tis' ap­proved sick­le cell dis­ease drug fails to beat place­bo in PhI­II

Novartis’ sickle cell drug, approved in 2019 and branded as Adakveo, has failed an ongoing Phase III, according to preliminary results.

The Swiss pharma giant unveiled early data from the ongoing STAND Phase III study on Friday, saying that crizanlizumab showed no statistically significant difference between the drug at two different dose levels compared to placebo in annualized rates of vaso-occlusive crises that lead to a healthcare visit over the first year since being randomized into the trial.