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%.

Dan Skovronsky, Eli Lilly CSO

UP­DAT­ED: An­a­lysts are quick to pan Eli Lil­ly's puz­zling first cut of pos­i­tive clin­i­cal da­ta for its Covid-19 an­ti­body

Eli Lilly spotlighted a success for one of 3 doses of their closely-watched Covid-19 antibody drug Wednesday morning. But analysts quickly highlighted some obvious anomalies that could come back to haunt the pharma giant as it looks for an emergency use authorization to launch marketing efforts.

The pharma giant reported that LY-CoV555, developed in collaboration with AbCellera, significantly reduced the rate of hospitalization among patients who were treated with the antibody. The drug arm of the study had a 1.7% hospitalization rate, compared to 6% in the control group, marking a 72% drop in risk.

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Leen Kawas, Athira

Seat­tle-based Athi­ra, aim­ing for tough Alzheimer's and Parkin­son's tar­gets, rais­es $204M for IPO

Following an $85 million Series B round back in June, Athira Pharma is set to become the third biotech in the last three days to go public Friday.

Athira raised $204 million for its IPO after pricing shares at $17 apiece, the high-point of its expected range. Initially pegging $100 million in financing, Athira said it offered 12 million total shares and will trade under the ticker $ATHA.

Now that Athira plans to hit the Nasdaq, there have been more than 50 biotech IPOs this year, more than the total from last year. Combined, the companies have raised more than $11 billion. Over half of those biotechs, including Athira, raised at least $200 million each.

#ES­MO20: Alk­er­mes of­fers their first snap­shot of a ben­e­fit for their next-gen IL-2 drug. But why did 1 pa­tient starve to death?

Everyone in the cancer R&D arena is looking to build new franchises around better drugs and combos. And one busy pocket of that space is centered entirely on creating an IL-2 drug that can be as effective as the original without the toxicity that damned it to the sidelines.

Alkermes $ALKS formally tossed its hat into the ring of contenders at virtual ESMO today, highlighting the first glimpse of efficacy for their candidate, ALKS 4230, as both a monotherapy as well as in combination with Merck’s Keytruda.

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Albert Bourla (Photo by Steven Ferdman/Getty Images)

Pfiz­er match­es Mod­er­na with their full Covid-19 tri­al blue­print — As­traZeneca says it will un­veil its pro­to­col 'short­ly'

Yesterday, after sustained public pressure as Moderna released its Phase III Covid-19 trial blueprint, Pfizer released its own full trial design for their vaccine trials. The move was designed to boost transparency and shore up public trust in the vaccines, but it also revealed differences in how the two companies are approaching the much-watched studies while failing to satisfy the demands of the fiercest advocates for transparency.

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Albert Bourla, Pfizer CEO (Steven Ferdman/Getty Images)

Pfiz­er ex­ecs con­fi­dent­ly tap their top 10 block­busters-to-be. But what are the chances of sur­viv­ing PhI­II, let alone hit­ting these big peak sales es­ti­mates?

Pfizer’s top executive team doesn’t lack for confidence.

Where many Big Pharmas would be reluctant to put a peak sales figure on their late-stage drugs, Pfizer CEO Albert Bourla has shrugged off the usual diffidence to outline where the pharma giant expects to get $15 billion-plus.

The list, outlined this week during their investor presentations, is topped by 3 drugs in the $3 billion-plus peak sales category. They are:

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Stronger to­geth­er? Boehringer and Mi­rati team to put first KRAS-KRAS com­bo in the clin­ic

Researchers are still waiting to see how much any of the vaunted KRAS drugs now in the clinic can, after decades of preclinical research and some early human studies, help patients. But while they do, two of the leading developers will look to see whether a KRAS-KRAS combo might pose a better shot than any KRAS alone.

Boehringer Ingelheim and Mirati have signed a collaboration to combine Mirati’s closely-watched lead KRAS inhibitor, MRTX849, in a clinical trial with the pan-KRAS blocker that Boehringer has quietly developed with high expectations behind their flashier contenders.

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#ES­MO20: Re­gen­eron, Sanofi eye an­oth­er first for their PD-1 con­tender Lib­tayo with promis­ing da­ta for on­col­o­gy niche

Regeneron and Sanofi took another step forward in the long march towards a greatly expanded market for their late-bloomer PD-1 checkpoint Libtayo.

The two occasional allies posted an objective response rate of 31% for Libtayo among 84 patients suffering from advanced cases of basal cell carcinoma at virtual ESMO. That spotlights progress for 26 patients, 5 of whom had a complete response. The data also reflect a boost in the number of responses seen from the last cut of the numbers.

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One month af­ter $115M megaround, mus­cle dis­ease-fo­cused Dyne goes pub­lic with $233M IPO

After completing a financing megaround for $115 million just a little over a month ago, Dyne Therapeutics is ready to hit the Nasdaq on Thursday.

The biotech has raised $233 million for its public offering after pricing shares at $19 apiece, above its expected range of $16 to $18. That’s an offering that’s well-upsized — by about 33% — after Dyne initially filed its S-1 in late August, when it estimated only a $100 million raise. Dyne will trade under the ticker $DYN.

Sean Bo­hen's break from bio­phar­ma is over. The ex-As­traZeneca CMO has re­tired his Big Phar­ma jer­sey and is now — hap­pi­ly — run­ning a lit­tle biotech

The last I had heard about Sean Bohen, he had stepped out of his high-profile job as chief medical officer at AstraZeneca at the beginning of 2019 as CEO Pascal Soriot triggered a broad-ranging R&D shakeup. And then, earlier this week, I got a chance to catch up.

It turns out that Bohen decided at the time that he would not just jump into a new job in the booming biopharma business. As an oncologist, he had worked on the big programs at AstraZeneca, and before that he was at Genentech. That was good for a ticket to just about anyplace in the big biopharma world. But he felt it was time to stop and think things through.

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