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 in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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Vlad Coric, Biohaven CEO

Vlad Coric charts course for new Bio­haven with neu­ro­science push and Big Phar­ma vets on board

What’s Biohaven without its CGRP portfolio? That’s what CEO Vlad Coric is tasked with deciding as he maps out the new Biohaven post-Pfizer takeover.

Pfizer officially scooped up Biohaven’s CGRP assets on Monday, including blockbuster migraine drug Nurtec and the investigational zavegepant, for $11.6 billion. As a result, Coric spun the broader pipeline into an independent company on Tuesday — with the same R&D team behind Nurtec but about 1,000 fewer staffers and a renewed focus on neuroscience and rare disease.

In AstraZeneca's latest campaign, wild eosinophils called Phils personify the acting up often seen in uncontrolled asthma

As­traZeneca de­buts an­noy­ing pur­ple ‘Phil’ crea­tures, per­son­i­fied asth­ma eosinophils ‘be­hav­ing bad­ly’

There are some odd-looking purple creatures lurking around the halls of AstraZenca lately. The “Phil” character cutouts are purple, personified eosinophils with big buggy eyes and wide mouths, and they’re a part of AZ’s newest awareness effort to help people understand eosinophilic asthma.

The “Asthma Behaving Badly” characters aren’t only on the walls at AZ to show the new campaign to employees, however. The “Phils” are also showing up online on the campaign website, and in digital and social ads and posts on Facebook and Instagram.

Marc Dunoyer, Alexion CEO (AstraZeneca via YouTube)

Up­dat­ed: As­traZeneca nabs a small rare dis­ease gene ther­a­py play­er for 667% pre­mi­um

AstraZeneca is kicking off the fourth quarter with a little M&A Monday for a gene editing player recently overcoming a second clinical hold to its only program in human studies.

The Big Pharma and its subsidiary Alexion are buying out little LogicBio for $2.07 per share. That’s good for a massive 667% premium over its Friday closing price, when it headed into the weekend at 27 cents and just weeks after Nasdaq said LogicBio would have to delist, which has been put on hold as the biotech requests a hearing. It’s one of two biotech deals to commence October, alongside the news of Incyte buying a vitiligo-focused biotech.

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Dave Marek, Myovant CEO

My­ovant board balks as ma­jor­i­ty own­er Sum­it­o­mo swoops in with a $2.5B deal to buy them out

Three years after Sumitomo scooped up Roivant’s 46% stake in the publicly traded Myovant $MYOV as part of a 5-company, $3 billion deal, they’re coming back for the whole thing.

But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage.

Sumitomo is bidding $22.75 a share for the outstanding stock, which now represents 48% of the company after Sumitomo bumped its ownership since the original deal with Roivant. Myovant, however, created a special committee on the board, and they’re shaking their heads over the offer.

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Christophe Bourdon, Leo Pharma CEO

Leo Phar­ma looks 'be­yond the skin' in atopic der­mati­tis aware­ness cam­paign

As Leo Pharma aims to take on heavyweight champ Dupixent in atopic dermatitis, the company is launching “AD Days Around the World,” an awareness campaign documenting real patient stories across Europe.

The project, unveiled on Monday, spotlights four patients: Marjolaine, Laura, Julia and África from France, Italy, Germany and Spain, respectively, in short video clips on the challenges of living with AD, the most common form of eczema.

Mar­ket­ingRx roundup: No­var­tis re­cruits NFL coach for Leqvio cam­paign; Pfiz­er pro­motes ‘Sci­ence’ merch on so­cial me­dia

Novartis is turning to a winning coach to talk about Leqvio and the struggles of high cholesterol — including his own. Bruce Arians, the retired NFL head coach of the Arizona Cardinals and Super Bowl-winning Tampa Bay Buccaneers, is partnering with the pharma for its “Coaching Cholesterol” digital, social and public relations effort.

In the campaign, Arians talks about the potential for “great comebacks” in football and heart health. Once nicknamed a “quarterback whisperer,” he is now retired from fulltime coaching (although still a front-office consultant for Tampa Bay), and did a round of media interviews for Novartis, including one with People and Forbes.

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Amy West, Novo Nordisk head of US digital innovation and transformation (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Q&A: No­vo Nordisk dig­i­tal in­no­va­tion chief Amy West dis­cuss­es phar­ma pain points and a health­care 'easy but­ton’

Amy West joined Novo Nordisk more than a decade ago to oversee marketing strategies and campaigns for its US diabetes portfolio. However, her career path shifted into digital, and she hasn’t looked back. West went from leading Novo’s first digital health strategy in the US to now heading up digital innovation and transformation.

She’s currently leading the charge at Novo Nordisk to not only go beyond the pill with digital marketing and health tech, but also test, pilot and develop groundbreaking new strategies needed in today’s consumerized healthcare world.

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