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%.

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.

After pharma partner Novartis boosted its IPO with a $10 million private placement, Pliant Therapeutics has wrapped its journey to the Nasdaq on a high note.

Pliant had penciled in a $86 million raise back in May. But as has become the norm in recent months, that initial number has turned out to be a mere placeholder, making way for the final haul of $144 million.

The South San Francisco biotech did so by pricing at $16, the high end of the range, while bringing the number of shares offered up to 9 million.

For nearly a decade now, the low-profile Cambridge biotech Kymab has been battling in US, UK, Japanese and Australian courts with the biotech behemoth Regeneron.

Regeneron has turned itself into a $70 billion company off of a platform of transgenically humanized mice they can use to make antibodies for anything from Ebola to colorectal cancer. The technology took decades and billions to build, 20 years from the company’s founding to the first approved drug. And the company guards and touts it zealously, breaking their production process down into various branded components — Velocimmune, Velocigene, Velocimouse and four other Velocis — and sometimes suing would-be copycats. In 2014, most notably, they sued two Pfizer-backed entities for patent infringement.