Rain Ther­a­peu­tics head­lines newest slate of IPO-bound biotechs with $100M pen­ciled in for lead on­col­o­gy pro­gram

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A fresh slate of biotech IPOs has filed to go pub­lic as the cal­en­dar turns to­ward the sec­ond quar­ter.

Three com­pa­nies filed their reg­is­tra­tional pa­per­work over the hol­i­day week­end, with Rain Ther­a­peu­tics and Im­pel Neu­roPhar­ma sub­mit­ting S-1s on Fri­day and Aneb­u­lo Phar­ma­ceu­ti­cals do­ing so last Thurs­day. Rain leads the es­ti­mat­ed rais­es of the group with a $100 mil­lion tranche pen­ciled in, fol­lowed by Im­pel at $75 mil­lion and Aneb­u­lo at a mod­est $15 mil­lion.

The moves come af­ter a huge haul for the sec­tor in the first quar­ter, which saw 32 biotechs file or price their IPOs. Among the com­pa­nies that went pub­lic, the in­dus­try raised more than $4.6 bil­lion in the first three months of the year.

Should biotechs con­tin­ue at this pace, 2021 will eclipse 2020’s record raise by more than $2 bil­lion, ac­cord­ing to fig­ures from Nas­daq.

Rain cap­i­tal­izes on Dai­ichi Sankyo pro­gram li­censed last year

Rain Ther­a­peu­tics saw a show­er of good for­tune in 2020.

Around La­bor Day last year, the biotech tripled its pipeline in the span of a week, li­cens­ing a re­search pro­gram from Drex­el Uni­ver­si­ty and nab­bing a Phase II-ready drug from Dai­ichi Sankyo. Rain fol­lowed that up with a $63 mil­lion fundraise short­ly af­ter to push for­ward all three of its pro­grams.

The new­er can­di­dates pair up with the com­pa­ny’s orig­i­nal mis­sion, one which helped it launch with an $18 mil­lion Se­ries A back in 2018. Rain’s first ex­per­i­men­tal drug was tar­lox­o­tinib, a small mol­e­cule in­hibitor de­signed to tar­get low oxy­gen lev­els in tu­mors and there­by spar­ing healthy tis­sues.

Now, though, the Dai­ichi pro­gram has be­come Rain’s lead. Dubbed RAIN-32, the can­di­date will see the bulk of Rain’s IPO funds fun­neled to­ward it. Per the S-1, Rain plans to launch three stud­ies for RAIN-32, in­clud­ing a piv­otal Phase III study in an MDM2-am­pli­fied sub­type of li­posar­co­ma.

The raise will al­so help fund a Phase II tu­mor-ag­nos­tic bas­ket tri­al in cer­tain sol­id tu­mors and a Phase 2 tri­al in in­ti­mal sar­co­ma for the pro­gram, as well as help man­u­fac­tur­ing costs and fund fur­ther trans­la­tion­al stud­ies.

Rain plans to trade un­der the tick­er $RAIN.

Im­pel tak­ing its in­haled drugs pub­lic ahead of mi­graine PDU­FA

More than two years af­ter its crossover raise, Im­pel Neu­roPhar­ma is fi­nal­ly head­ing to Nas­daq.

The Seat­tle-based biotech has a unique ap­proach for get­ting CNS drugs de­liv­ered to the brain — through the nose. Im­pel’s tech is cen­tered around a de­liv­ery sys­tem of nasal dos­es of old and thor­ough­ly un­der­stood drugs, with re­search go­ing to­ward mi­graines, Parkin­son’s and ag­i­ta­tion re­lat­ed to autism.

Led by CEO Adri­an Adams, Im­pel hopes an en­hanced nasal de­liv­ery ap­proach can im­prove a drug’s per­for­mance, of­fer­ing an open­ing for an im­proved ther­a­peu­tic ef­fect with a liq­uid or dry for­mu­la­tion of an old drug. Their mi­graine pro­gram has com­plet­ed its piv­otal study, with an ex­pect­ed PDU­FA date of Sept. 6 lat­er this year.

As such, the ma­jor­i­ty of the planned IPO cash will go to­ward fund­ing the po­ten­tial com­mer­cial launch of the mi­graine treat­ment, which Im­pel plans to mar­ket as Trud­he­sa. The drug is an up­per nasal for­mu­la­tion of di­hy­droer­go­t­a­mine to treat acute mi­graines.

The re­main­der of the IPO funds will help the INP105 pro­gram, de­signed for acute treat­ment of ag­i­ta­tion and ag­gres­sion as­so­ci­at­ed with autism spec­trum dis­or­der. This can­di­date is a nasal re­for­mu­la­tion of olan­za­p­ine.

Im­pel plans to list un­der the tick­er $IM­PL.

Cannabi­noid over­dose biotech Aneb­u­lo looks to fund new stud­ies

Aneb­u­lo Phar­ma­ceu­ti­cals brings up the rear this week, pen­cil­ing in just $15 mil­lion for its IPO raise.

The Lake­way, TX-based com­pa­ny, found­ed just last year, is fo­cused on de­vel­op­ing treat­ment for cannabi­noid over­dose and sub­stance ad­dic­tion. Aneb­u­lo’s lead can­di­date, ANEB-001, is de­signed to re­verse the neg­a­tive ef­fects of cannabi­noid over­dose with­in one hour of ad­min­is­tra­tion.

Aneb­u­lo is hop­ing to ful­ly fi­nance a Phase II proof-of-con­cept study for the pro­gram with its IPO mon­ey. But this won’t be the last time Aneb­u­lo is look­ing to raise mon­ey — in their S-1, they not­ed that they’ll have more cap­i­tal in about 18 months to run the piv­otal safe­ty tri­als, launch the drug com­mer­cial­ly and make mile­stone pay­ments to Ver­nalis, from whom Aneb­u­lo li­censed its pro­gram.

The biotech plans to trade un­der the tick­er $ANEB.

BY­OD Best Prac­tices: How Mo­bile De­vice Strat­e­gy Leads to More Pa­tient-Cen­tric Clin­i­cal Tri­als

Some of the most time- and cost-consuming components of clinical research center on gathering, analyzing, and reporting data. To improve efficiency, many clinical trial sponsors have shifted to electronic clinical outcome assessments (eCOA), including electronic patient-reported outcome (ePRO) tools.

In most cases, patients enter data using apps installed on provisioned devices. At a time when 81% of Americans own a smartphone, why not use the device they rely on every day?

Chris Gibson (Photo By Vaughn Ridley/Sportsfile for Web Summit via Getty Images)

Re­cur­sion founders gin for­tunes as IPO back­ers show­er $436M on one of the biggest boasts in AI -- based on some very small deals

In the AI drug development world, boasting often comes with the territory. Yet few can rival Recursion when it comes to claiming the lead role in what company execs like to call the industrialization of drug development, with promises of continued exponential growth in the number of drugs it has in the pipeline.

On Friday, the Salt Lake City-based biotech translated its unicorn-sized boasts into a killer IPO, pricing more than 24 million shares at the high end of its range and bringing in $436 million — with a large chunk of that promised by some deep-pocket backers.

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Covid-19 vac­cine halt drags on, an FDA ap­point­ment at long last, the great CRO con­sol­i­da­tion, and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Conference season is upon us, and while we’d much prefer to be wandering down the hallways and presentation rooms in person, the team is ready to cover the most consequential data coming out of these scientific meetings. Get in touch early if you have news to share.

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Eli Lil­ly asks FDA to re­voke EUA for Covid-19 treat­ment

Eli Lilly on Friday requested that the FDA revoke the emergency authorization for its Covid-19 drug bamlanivimab, which is no longer as effective as a combo therapy because of a rise in coronavirus variants across the US.

“With the growing prevalence of variants in the U.S. that bamlanivimab alone may not fully neutralize, and with sufficient supply of etesevimab, we believe now is the right time to complete our planned transition and focus on the administration of these two neutralizing antibodies together,” Daniel Skovronsky, Lilly’s CSO, said in a statement.

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Osman Kibar (Samumed, now Biosplice)

Os­man Kibar lays down his hand at Sa­mumed, step­ping away from CEO role as his once-her­ald­ed an­ti-ag­ing biotech re­brands

Samumed made quite the entrance back in 2016, when it launched with some anti-aging programs and a whopping $12 billion valuation. That level of fanfare was nowhere to be found on Thursday, when the company added another $120 million to its coffers and quietly changed its name to Biosplice Therapeutics.

Why the sudden rebrand?

“We did that for obvious reasons,” CFO and CBO Erich Horsley told Endpoints News. “The name Biosplice echoes our science much more than Samumed does.”

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Ex­clu­sive in­ter­view: Pe­ter Marks on why full Covid-19 vac­cine ap­provals could be just months away

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, took time out of his busy schedule last Friday to discuss with Endpoints News all things related to his work regulating vaccines and the pandemic.

Marks, who quietly coined the name “Operation Warp Speed” before deciding to stick with his work regulating vaccines at the FDA rather than join the Trump-era program, has been the face of vaccine regulation for the FDA throughout the pandemic, and is usually spotted in Zoom meetings seated in front of his wife’s paintings.

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Near­ly a year af­ter Au­den­tes' gene ther­a­py deaths, the tri­al con­tin­ues. What hap­pened re­mains a mys­tery

Natalie Holles was five months into her tenure as Audentes CEO and working to smooth out a $3 billion merger when the world crashed in.

Holles and her team received word on the morning of May 5 that, hours before, a patient died in a trial for their lead gene therapy. They went into triage mode, alerting the FDA, calling trial investigators to begin to understand what happened, and, the next day, writing a letter to alert the patient community so they would be the first to know. “We wanted to be as forthright and transparent as possible,” Holles told me late last month.

The brief letter noted two other patients also suffered severe reactions after receiving a high dose of the therapy and were undergoing treatment. One died a month and a half later, at which point news of the deaths became public, jolting an emergent gene therapy field and raising questions about the safety of the high doses Audentes and others were now using. The third patient died in August.

“It was deeply saddening,” Holles said. “But I was — we were — resolute and determined to understand what happened and learn from it and get back on track.”

Eleven months have now passed since the first death and the therapy, a potential cure for a rare and fatal muscle-wasting disease called X-linked myotubular myopathy, is back on track, the FDA having cleared the company to resume dosing at a lower level. Audentes itself is no more; last month, Japanese pharma giant Astellas announced it had completed working out the kinks of the $3 billion merger and had restructured and rebranded the subsidiary as Astellas Gene Therapies. Holles, having successfully steered both efforts, departed.

Still, questions about precisely what led to the deaths of the 3 boys still linger. Trial investigators released key details about the case last August and December, pointing to a biological landmine that Audentes could not have seen coming — a moment of profound medical misfortune. In an emerging field that’s promised cures for devastating diseases but also seen its share of safety setbacks, the cases provided a cautionary tale.

Audentes “contributed in a positive way by giving a painful but important example for others to look at and learn from,” Terry Flotte, dean of the UMass School of Medicine and editor of the journal Human Gene Therapy, told me. “I can’t see anything they did wrong.”

Yet some researchers say they’re still waiting on Astellas to release more data. The company has yet to publish a full paper detailing what happened, nor have they indicated that they will. In the meantime, it remains unclear what triggered the events and how to prevent them in the future.

“Since Audentes was the first one and we don’t have additional information, we’re kind of in a holding pattern, flying around, waiting to figure out how to land our vehicles,” said Jude Samulski, professor of pharmacology at UNC’s Gene Therapy Center and CSO of the gene therapy biotech AskBio, now a subsidiary of Bayer.

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J&J faces CDC ad­vi­so­ry com­mit­tee again next week to weigh Covid-19 vac­cine risks

The CDC’s Advisory Committee on Immunization Practices punted earlier this week on deciding whether or not to recommend lifting a pause on the administration of J&J’s Covid-19 vaccine, but the committee will meet again in an emergency session next Friday to discuss the safety issues further.

The timing of the meeting likely means that the J&J vaccine will not return to the US market before the end of next week as the FDA looks to work hand-in-hand with the CDC to ensure the benefits of the vaccine still outweigh the risks for all age groups.

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Pascal Soriot (AstraZeneca via YouTube)

Af­ter be­ing goad­ed to sell the com­pa­ny, Alex­ion's CEO set some am­bi­tious new goals for in­vestors. Then Pas­cal So­ri­ot came call­ing

Back in the spring of 2020, Alexion $ALXN CEO Ludwig Hantson was under considerable pressure to perform and had been for months. Elliott Advisers had been applying some high public heat on the biotech’s numbers. And in reaching out to some major stockholders, one thread of advice came through loud and clear: Sell the company or do something dramatic to change the narrative.

In the words of the rather dry SEC filing that offers a detailed backgrounder on the buyout deal, Alexion stated: ‘During the summer and fall of 2020, Alexion also continued to engage with its stockholders, and in these interactions, several stockholders encouraged the company to explore strategic alternatives.’

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