Mike Sherman, Chimerix CEO

Chimerix places its bets on On­co­ceu­tic­s' dopamine an­tag­o­nist for rare brain can­cer in quest to sal­vage its im­age

In the clin­i­cal waste­land of ag­gres­sive brain can­cers, Philly’s On­co­ceu­tics has churned out promis­ing da­ta with a Phase II can­di­date on the verge of an FDA fil­ing. Chimerix, look­ing to turn around its rep­u­ta­tion — and share price — has jumped on board, putting half-a-bil­lion-dol­lar hopes in On­co­ceu­tics’ drug.

Chimerix will ac­quire pri­vate biotech On­co­ceu­tics and lead can­di­date ONC-201, an oral small mol­e­cule dopamine re­cep­tor D2 an­tag­o­nist and ca­seinolyt­ic pro­tease ag­o­nist cur­rent­ly be­ing test­ed in a Phase II tri­al against re­cur­rent glioma, a form of brain can­cer with a par­tic­u­lar­ly poor prog­no­sis, the com­pa­nies said Fri­day.

On­co­ceu­tics ex­pects piv­otal da­ta on the ONC-201 pro­gram some time this year for glioma pa­tients with the H3 K27M mu­ta­tion, a con­di­tion that comes with a four-month over­all sur­vival prog­no­sis. Along­side ONC-201, On­co­ceu­tics will bring an­oth­er four on­col­o­gy pro­grams in­to the fold for Chimerix, which is ad­vanc­ing its own can­di­date, a gly­cosamino­gly­can com­pound de­rived from porcine he­parin dubbed DSTAT, through a Phase III tri­al in acute myeloid leukemia.

A win with ONC-201 could open a $500 mil­lion mar­ket op­por­tu­ni­ty in re­cur­rent glioma, Chimerix said.

As part of the deal, Chimerix will pay out $78 mil­lion to On­co­ceu­tics in­vestors — half in stock and half in cash — with a po­ten­tial $360 mil­lion in de­vel­op­ment, reg­u­la­to­ry and sales mile­stones down the road. In terms of the $39 mil­lion in cash on the ta­ble, Chimerix will pay out $25 mil­lion at clos­ing and the re­main­ing $14 mil­lion on the first an­niver­sary of clos­ing.

In ad­di­tion, share­hold­ers will be due 15% roy­al­ties on com­bined sales of ONC-201 and an­oth­er pro­gram, ONC206, of up to $750 mil­lion per year and 20% roy­al­ties above $750 mil­lion in an­nu­al rev­enue.

ONC-201 has earned the FDA’s fast track des­ig­na­tion based on da­ta from three tri­als show­ing promis­ing re­sponse rates. The drug’s Phase II study is test­ing the mol­e­cule in 50 pa­tients above the age of 2 who have re­ceived ra­di­a­tion at least 90 days pri­or to en­roll­ment and dis­played ev­i­dence of pro­gres­sive dis­ease, and cer­tain oth­er cri­te­ria, Chimerix said.

The re­sults from that study are planned for a Blind­ed In­de­pen­dent Cen­tral Re­view com­mit­tee’s analy­sis some­time this year, which could form the ba­sis for a reg­u­la­to­ry fil­ing down the road, Chimerix said. Re­sults from the first 30 pa­tients in that study were al­ready re­viewed in a sep­a­rate BI­CR held in No­vem­ber. So far, in over 350 pa­tients treat­ed, ONC-201 has shown a “gen­er­al­ly well tol­er­at­ed” safe­ty pro­file with most com­mon side ef­fects of nau­sea/vom­it­ing, fa­tigue and de­creased lym­pho­cytes.

Ac­quir­ing a promis­ing can­di­date in ag­gres­sive brain can­cers could help turn around the North Car­oli­na biotech with a sketchy past. In mid-2020, Chimerix laid off half its staff af­ter piv­ot­ing work on its oral an­tivi­ral brin­cid­o­fovir to small­pox fol­low­ing a string of clin­i­cal fail­ures. Now, that small­pox ap­pli­ca­tion for brin­cid­o­fovir is up for a PDU­FA date April 7 in what could be the biotech’s first ap­proval. Shares of Chimerix ($CM­RX) were trad­ing up 16% be­fore the bell Fri­day at $5.79.

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?

Image: Shutterstock

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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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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As­traZeneca-Alex­ion merg­er slides through FTC re­view af­ter sup­posed M&A crack­down pos­es no bar­ri­ers

The AstraZeneca-Alexion megamerger received a good sign Friday, despite warning signs of the tides turning against large M&A pharma deals.

US regulators at the FTC have cleared the acquisition for approval, AstraZeneca announced, all but signing off on the deal to go through once it officially closes in the third quarter. AstraZeneca originally said it was planning to buy out Alexion back in December for $39 billion.

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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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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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Sanjiv Patel, Relay CEO

In one of their first ever ac­qui­si­tions, Re­lay bets $85M cash on a new AI-based screen­ing ap­proach

Although they’ve never been short for cash, Relay Therapeutics hasn’t been one for acquisitions in its 5-year history, focusing instead on developing its own tools to study how proteins move and advancing molecules off those insights.

On Friday, though, the Third Rock-spun biotech plunked down $85 million in cash and another $185 million in milestones to acquire the small, two-year-old, Google-partnered machine learning company ZebiAI. The deal will allow Relay to add a critical new technology to its early-stage discovery tools now that, with three candidates in the clinic, they’ve shown those tools can pay off, said CEO Sanjiv Patel.

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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