Rahul Ballal, Imara

As sick­le cell pa­tients find new op­tions, NEA-found­ed Imara pitch­es mid-stage al­ter­na­tive for $86M IPO

No­vem­ber 2019 proved to be a fruit­ful month for pa­tients with blood dis­or­ders known as he­mo­glo­binopathies. With­in days, the FDA ush­ered two drugs for sick­le cell dis­ease and an­oth­er for be­ta tha­lassemia to the mar­ket — liven­ing up a bar­ren field.

Willem Scheele Imara

Imara, a rel­a­tive­ly young plow­er, is rid­ing on that en­thu­si­asm as it shoots for an $86.25 mil­lion IPO.

Imara emerged from New En­ter­prise As­so­ci­ates’ or­phan drug ac­cel­er­a­tor Cy­dan in 2016 as a sin­gle-prod­uct com­pa­ny. $77.3 mil­lion in pri­vate fi­nanc­ing lat­er IMR-687 re­mains the sole as­set in its pipeline; the dif­fer­ence is the drug is now in Phase II for sick­le cell dis­ease, with topline da­ta slat­ed for lat­er this year and two oth­er mid-stage be­ta tha­lassemia stud­ies lined up.

IMR-687 was one of a group of small mol­e­cule PDE9 in­hibitors that Imara had li­censed from Lund­beck for a song — $1.8 mil­lion to-date — af­ter ev­i­dence emerged that the class of ther­a­pies, orig­i­nal­ly de­signed for neu­ro­log­i­cal dis­eases, could help ad­dress ab­nor­mal­i­ties in red blood cells. Pfiz­er is al­so de­vel­op­ing a PDE9 in­hibitor for SCD af­ter dis­con­tin­u­ing a pro­gram in Alzheimer’s.

Michael Gray Imara

The the­o­ry is that block­ing PDE9 in­creas­es cyclic GMP lev­els, which is as­so­ci­at­ed with re­ac­ti­va­tion of fe­tal he­mo­glo­bin and thus re­store some func­tion­al­i­ty im­paired in blood dis­or­ders.

In con­trast, No­var­tis’ Adakveo is an in­jec­tion that blocks P-se­lectin in hopes of stop­ping va­so-oc­clu­sion, while Glob­al Blood Ther­a­peu­tics’ Oxbry­ta in­hibits he­mo­glo­bin S poly­mer­iza­tion to pre­vent red blood cells from form­ing the sick­le shape. Re­blozyl, mar­ket­ed by Cel­gene (now Bris­tol-My­ers Squibb) and Ac­celeron, is a fu­sion pro­tein that boosts red blood cell growth in ane­mic pa­tients by tar­get­ing TGF-be­ta pro­teins.

While Re­blozyl is on­ly ap­proved for pa­tients who re­quire reg­u­lar trans­fu­sions, Imara hopes to al­so ad­dress those who are not de­pen­dent on the pro­ce­dure.

While al­lo­gene­ic hematopoi­et­ic stem cell trans­plants can be cu­ra­tive for both sick­le cell dis­ease and be­ta tha­lassemia, Imara not­ed that it’s rarely used due to a cum­ber­some process. Sim­i­lar­ly, while gene ther­a­pies by blue­bird and gene edit­ing ap­proach­es be­ing ad­vanced by CRISPR Ther­a­peu­tics are promis­ing, they are “com­plex, cost­ly, dif­fi­cult to ad­min­is­ter and po­ten­tial­ly on­ly suit­able for a lim­it­ed sub­set of pa­tients,” ex­ecs wrote in the S-1.

David Mott NEA

CEO Rahul Bal­lal leads the team of 16. His com­pen­sa­tion pack­age last year to­taled $2 mil­lion, thanks in large part to op­tion awards. CFO Michael Gray re­ceived $1.2 mil­lion-plus while CMO Willem Scheele got a lit­tle over $1 mil­lion.

NEA, rep­re­sent­ed on the board by chair­man David Mott as well as Sara Nay­eem, re­mains the largest share­hold­er at 31.8%. Lund­beck­fond In­vest holds 16.3%; Pfiz­er is in for 11.2%, Or­biMed and Ar­ix 10.8%, Bay City Cap­i­tal 6.4% and RA Cap­i­tal 5.6%.

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.