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%.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data are messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data are exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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David Lockhart, ReCode Therapeutics CEO

Pfiz­er throws its weight be­hind LNP play­er eye­ing mR­NA treat­ments for CF, PCD

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

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René Russo, Xilio CEO (Alicia Petitti for Xilio)

Xilio Ther­a­peu­tics goes pub­lic as bio­phar­ma IPOs rum­ble for­ward in Q4

It’s been a busy fall on Wall Street — and this week, we’ve got another biotech going public after Ventyx yesterday.

Massachusetts solid-tumor biotech Xilio Therapeutics made its public debut today, joining 149 other biotechs that have gone public so far this year.

Back on October 1st, Xilio filed its S-1 with the SEC to make its IPO debut for $100 million — the next step in financing after completing a $95 million Series C back in February.

Luc Boblet, Egle Therapeutics CEO

A new Treg play­er emerges with $46M and back­ing from Take­da

In recent years, the chorus of biotechs and Big Pharma backers targeting regulatory T cells — also known as “Tregs” — for cancer and autoimmune diseases has only grown louder.

The newest voice is from Egle Therapeutics, which sang out a $46.4 million Series A round on Friday led by LSP and Bpifrance through their InnoBio 2 fund. Takeda’s venture arm also chipped in, about a year after the pharma struck a research pact with the Paris-based upstart.

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Marty Duvall, Oncopeptides CEO

On­copep­tides stock craters as it pulls can­cer drug Pepax­to from the mar­ket

Shares of Oncopeptides crashed more than 70% in early Friday trading after the company said it’s pulling its multiple myeloma drug Pepaxto (melphalan flufenamide) from the US market after failing a confirmatory trial. The move will force the company to close its US and EU business units and enact significant layoffs.

The FDA had scheduled an adcomm meeting next Thursday to discuss Pepaxto, which first won accelerated approval in February and costs about $19,000 per course of treatment. The committee was to weigh in on whether the confirmatory trial demonstrated a worse overall survival in the treatment arm compared to the control arm.

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Pfiz­er pitch­es its Covid-19 vac­cine for younger chil­dren ahead of ad­comm next week

Pfizer will present its case to the FDA’s vaccine adcomm next week, seeking authorization for a lower-dose version of its Covid-19 vaccine for kids ages 5 through 12, which the Biden administration said will likely begin rolling out early next month.

Two primary doses of the 10 µg vaccine (the dose for those ages 12 and up is 30 μg) given 3 weeks apart in this group of children “have shown a favorable safety and tolerability profile, robust immune responses against all variants of concern including Delta, and vaccine efficacy of 90.7% against laboratory-confirmed symptomatic COVID-19,” the company said in briefing documents ahead of next Tuesday’s meeting of the FDA’s Vaccines and Related Biological Products Advisory Committee.