Lee Rauch, miRagen

A small buy­out deal gives a pen­ny-stock biotech a boost and a sec­ond chance

Just over a month af­ter for­mer mi­Ra­gen Ther­a­peu­tics COO Lee Rauch stepped up to take the helm, the mi­cro­cap is scoop­ing up Virid­i­an Ther­a­peu­tics — and with it, a new lead can­di­date and $91 mil­lion boost.

mi­Ra­gen’s work­force was cut in half by a cost­ly re­struc­tur­ing plan ini­ti­at­ed last Au­gust, with 44 po­si­tions elim­i­nat­ed. Dis­cov­ery re­search was side­lined, while the mi­croR­NA-fo­cused biotech fo­cused on the four pro­grams it had. About a year lat­er, CEO William Mar­shall re­signed, leav­ing Rauch to pick up the pieces.

Now, the Boul­der, CO-based biotech is bet­ting its fu­ture on VRDN-001 — the thy­roid eye dis­ease (TED) can­di­date it picked up from Virid­i­an. The in­sulin-like growth fac­tor-1 re­cep­tor (IGF-1R) mon­o­clon­al an­ti­body was orig­i­nal­ly li­censed from Im­muno­Gen, which is still el­i­gi­ble for ad­di­tion­al de­vel­op­men­tal mile­stones and “mid-sin­gle-dig­it” roy­al­ty pay­ments, ac­cord­ing to mi­Ra­gen.

TED can lead to bulging eyes, dou­ble-vi­sion and po­ten­tial blind­ness. mi­Ra­gen plans on launch­ing VRDN-001 in­to a Phase II study for the in­di­ca­tion in 2021. The com­pa­ny is al­so ready­ing an IND ap­pli­ca­tion for VRDN-002: a pro­gram start­ed by Virid­i­an to im­prove IGF-1R-tar­get­ed an­ti­bod­ies by us­ing half-life ex­ten­sion tech­nol­o­gy to re­duce the dose re­quired for full ef­fi­ca­cy and en­hance sol­u­bil­i­ty.

The Virid­i­an buy­out was a stock-for-stock trans­ac­tion, mean­ing all of Virid­i­an’s out­stand­ing eq­ui­ty in­ter­ests were ex­changed for a com­bi­na­tion of com­mon stock and Se­ries A pre­ferred stock shares. In ad­di­tion, mi­Ra­gen en­tered a $91 mil­lion pri­vate place­ment with new and old in­vestors, who were is­sued Se­ries A pre­ferred stock at $465.96 per share.

The pri­vate place­ment, which clos­es at the end of this month, was led by Fair­mount Funds Man­age­ment with help from Ven­rock Health­care Cap­i­tal Part­ners, BVF Part­ners, Cor­morant As­set Man­age­ment, Per­cep­tive Ad­vi­sors, Welling­ton Man­age­ment, Al­ly Bridge Group, Lo­gos Cap­i­tal, Sur­vey­or Cap­i­tal, Com­modore Cap­i­tal and Ridge­back Cap­i­tal. Pro­ceeds are tagged for the de­vel­op­ment of  VRDN-001.

mi­Ra­gen’s stock $MGEN jumped more than 93% up­on news of the ac­qui­si­tion — though the share price is still hov­er­ing around $1 a piece. Yes­ter­day, shares closed at about $0.52 each.

“Af­ter a thor­ough eval­u­a­tion of strate­gic al­ter­na­tives, the Board of Di­rec­tors of mi­Ra­gen be­lieves this ac­qui­si­tion rep­re­sents the high­est-po­ten­tial val­ue cre­ation op­por­tu­ni­ty for mi­Ra­gen’s stock­hold­ers,” Jef­frey Hat­field, chair­man of mi­Ra­gen’s board of di­rec­tors, said in a state­ment. “We are ex­cit­ed by the po­ten­tial for VRDN-001 to be­come a mean­ing­ful treat­ment op­tion for pa­tients liv­ing with TED.”

Un­lock­ing ESG strate­gies for growth with Gilead Sci­ences

RBC Capital Markets explores what is material in ESG for biopharma companies with the ESG leads at Gilead Sciences. Gilead has long focused on sustainability but recognized a more robust framework was needed. Based on a materiality assessment, Gilead’s ESG strategy today focuses first on drug access and pricing, while also addressing D&I and climate change. Find out why Gilead’s board is “acutely aware” of the contribution that ESG makes to firm’s overall success.

What con­tro­ver­sy? Eli Lil­ly plots Alzheimer's BLA fil­ing lat­er this year as FDA taps more an­ti-amy­loid drugs as break­throughs

The FDA is keeping the good news coming for Alzheimer’s drug developers. And Eli Lilly is taking them up on it.

Amid continued controversy around whether Biogen’s new flagship drug, Aduhelm, should have been approved at all — and swelling, heated debates surrounding its $56,000 price tag — the agency had no issue handing them and their Japanese partner Eisai a breakthrough therapy designation for a second anti-amyloid beta antibody, lecanemab, late Wednesday.

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Hervé Hoppenot, Incyte CEO (Jeff Rumans)

ODAC echoes FDA con­cern over In­cyte PD-1, as Paz­dur sig­nals broad­er shift for ac­cel­er­at­ed ap­proval

After the FDA lambasted their PD-1 ahead of an adcomm earlier this week, Incyte ran into new trouble Thursday as ODAC panelists voted against an accelerated OK by a wide margin.

Members of the Oncologic Drugs Advisory Committee recommended with a 13-4 vote to defer a regulatory decision on Incyte’s retifanlimab until after more data can be collected from a placebo-controlled trial. The PD-1 therapy is due for a PDUFA date in late July after receiving priority review earlier this year.

From left: Rajul Jain, Stefan Vitorovic, Arjun Goyal, Arie Belldegrun, Jean-Philippe (JP) Kouakou-Zebouah, Helen Kim

Arie Bellde­grun's Vi­da Ven­tures goes back to the well with $825M mega­fund and its eyes set on more in­no­v­a­tive meds

Among the list of bright names in biopharma, few shine brighter than Kite founder and serial entrepreneur Arie Belldegrun, who has rattled off a remarkable run of success in recent years. Now, a Belldegrun investment team is locking up a massive third fund to keep chasing the cutting edge in therapeutics.

Vida Ventures closed its third investment fund at a whopping $825 million — its largest yet — as the ever-expanding VC firm hits 30 companies in its portfolio developing new routes to hard-to-treat diseases, the company said Thursday.

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New FDA doc­u­ments show in­ter­nal dis­sent on Aduhelm ap­proval

In a lengthy review document and a pair of memos from top officials, the FDA released on Tuesday night its most detailed argument yet for approving Biogen’s intensely controversial Alzheimer’s drug aducanumab.

The documents amount to an agency attempt to quench the firestorm their decision kindled, as outside advisors members resigned and experts warned that an unproven drug now could stretch Medicare’s budget to a breaking point. Ultimately, the documents show how CDER director Patrizia Cavazzoni and Office of New Drugs director Peter Stein both concurred with FDA neuroscience head Billy Dunn on the accelerated approval while the staff at FDA’s Office of Biostatistics did not think an approval was warranted.

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Karen Flynn, Catalent

Q&A: When the pan­dem­ic struck, Catal­en­t's CCO had just joined the team

Karen Flynn came aboard Catalent’s team just in time.

The company was going through a surge of changes, and she had been brought over from her role as CCO of West Pharmaceutical Services to serve in the same capacity for the New Jersey-based CDMO. Then a few months later, the pandemic was in full-force.

Since then, Catalent’s been in hyper-expansion mode. In early May, it acquired Promethera’s Hepatic Cell Therapy Support SA subsidiary and its 32,40-square-foot facility in Gosselies, Belgium. Prior to that, the company acquired Belgian CDMO Delphi Genetics, wrapped up the expansion of an already-existing site in Madison, WI and added an ultra-low temperature freezer partner in Sterling. As Emergent has botched millions of doses of AstraZeneca’s vaccine, the company has swooped in to move that production to its Maryland plant as well.

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Richard Pazdur (vis AACR)

FDA en­cour­ages in­clud­ing in­cur­able can­cer pa­tients in tri­als, re­gard­less of pri­or ther­a­pies

The FDA on Thursday called to include those with incurable cancers (when there is no potential for cure or for prolonged/near normal survival) in appropriate clinical trials, regardless of whether they have received existing alternative treatments.

Historically, many cancer clinical trials have required that participating patients previously received multiple therapies, according to Richard Pazdur, director of the FDA’s Oncology Center of Excellence.

On heels of Aduhelm ap­proval, Bris­tol My­ers jumps back in­to Alzheimer's race

Bristol Myers Squibb last put major resources behind an Alzheimer’s drug nearly a decade ago, when their own attempt at targeting amyloid flamed out in mid-stage studies. They invented another molecule, a Tau-targeted antibody, but jettisoned it to Biogen in 2017 as they dropped out of neuroscience altogether.

But on Thursday, the New York pharma announced they were getting back in the game. Bristol Myers exercised an $80 million option to bring a tau-targeted antibody from Prothena into a Phase I study. The opt-in, which Bristol Myers triggered ahead of analyst expectations, opens the door for another $1.7 billion in milestones down the road.

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James Peyer, Cambrian

Can a cell ther­a­py treat mus­cu­lar dy­s­tro­phy? A Ger­man bil­lion­aire's an­ti-ag­ing start­up is try­ing to find out

Gene therapy companies have faced huge hurdles trying to deliver healthy genes into muscular dystrophy patients’ muscle cells, so here’s an idea: Why don’t we just replace the muscle cells themselves?

Over the last two years, Vita Therapeutics has been exploring that possibility, building on early stem cell work from Johns Hopkins professor Peter Andersen. And on Tuesday they announced a $32 million Series A to begin to move their first therapy into the clinic, where they hope it will help rebuild muscle in patients with a type of dystrophy that afflicts the arms and legs.