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.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Tillman Gerngross (Adagio)

Till­man Gern­gross on Omi­cron: 'It is a grim sit­u­a­tion...we’re go­ing to see a sig­nif­i­cant drop in vac­cine ef­fi­ca­cy'

Tillman Gerngross, the rarely shy Dartmouth professor, biotech entrepreneur and antibody expert, has been warning for over a year that the virus behind Covid-19 would likely continue to mutate, potentially in ways that avoid immunity from infection and the best defenses scientists developed. He spun out a company, Adagio, to build a universal antibody, one that could snuff out any potential mutation.

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In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

Jeff Albers, Blueprint Medicines CEO

Look­ing past Big Phar­ma ri­vals, Blue­print buys a pre­clin­i­cal biotech for $250M+

J&J’s Rybrevant scored the first approval back in May for a small group of lung cancer patients with a rare EGFR mutation. Despite a swarm of other biopharma companies angling for a piece of that market, Blueprint Medicines is betting nearly $500 million on a candidate it thinks will stand out.

Blueprint is putting down $250 million in cash and another $215 million in biobucks for Lengo Therapeutics and its preclinical non-small cell lung cancer program LNG-451. Though it hasn’t been tested in humans, Blueprint says the candidate was “highly brain-penetrant” in preclinical trials, and has the potential to inhibit all common EGFR exon 20 insertion variants — which are found in just 2% to 3% of NSCLC patients.

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Like the flu vac­cine every year, the FDA could move quick­ly on a vari­ant-tar­get­ed Covid vac­cine

In the same way that the FDA signs off on flu vaccines every year without requiring large clinical trials to measure their efficacy, the FDA may employ a similar strategy in authorizing variant-focused versions of the mRNA vaccines.

As the world braces for more data on the latest variant Omicron, which may reduce vaccine efficacy, top vaccine developers like Moderna and Pfizer-BioNTech have promised they can pull together a new vaccine targeted against a specific Covid variant in about 100 days. Since Omicron emerged last week, Pfizer-BioNTech, Moderna and J&J have all said they’ve begun work on Omicron-specific vaccines, if needed.

Thanks­giv­ing edi­tion: Top 15 End­points sto­ries of 2021; Can you name that vac­cine?; Mer­ck­'s Covid an­tivi­ral dis­ap­points; FDA nom­i­nee's in­dus­try ties; 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.

Happy Thanksgiving to all those who are celebrating it — although, if we are being honest, this week’s abbreviated edition is really for those who are not. Wherever you’re tuning in from, we appreciate your support, hope you find this recap helpful and we wish you a wonderful weekend.

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What were End­points read­ers tun­ing in­to this year? Here’s a look at our 15 most pop­u­lar re­ports of the year (so far)

At the beginning of this year, I laid out a basic objective for Endpoints News as we headed to our 5th anniversary. We’ve long been doing a fine job covering the breaking news in R&D — if I do say so myself — but we needed to expand our horizons on industry coverage, increase the staff and go much, much deeper when the stories demanded it.

In a phrase: broader and deeper.

It’s safe to say, based on our daily web traffic, that you all seemed to like this idea. We’ve doubled the staff — thanks to a growing group of paid subscribers — ramped up the daily report and now publish a regular slate of in-depth articles. And traffic — those clicks you always read about — have gone up in volume too. Monthly sessions are up 43%, to close to 1.5 million. Unique readers are up 63%, to 874,480 in October, after setting a record of close to a million the month before. Page views are running at 3 million-plus a month. And the overall number of subscribers has surged to 124,000.

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Ursula von der Leyen, President of the European Commission

Omi­cron: Re­searchers scram­ble as new coro­n­avirus mu­ta­tion takes flight around the globe — Pfiz­er/BioN­Tech, Mod­er­na vow swift re­sponse

As Americans were waking up for their Black Friday rituals, they were greeted with the news that a new mutation of the Covid-19 virus has appeared and been sequenced — after it caught an international flight to Hong Kong. And two of the leading Covid-19 vaccine developers promised delivery of a new vaccine “within 100 days” if necessary while a third spelled out its 3-prong strategy hours later.

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Nev­er too late: For­bion pitch­es $100M SPAC; Kro­nos Bio re­leas­es ear­ly in­ter­im da­ta on CDK9 in­hibitor

Dutch VC Forbion is hopping on the ever-lengthening SPAC train.

To be led by Jasper Bos, who joined Forbion Growth as a general partner back in May just after the fund closed at $428 million, Forbion European Acquisition will target late-stage opportunities in the life sciences industry in Europe to merge with and bring onto Nasdaq.

Cyril Lesser, senior controller at Forbion, will be the CFO while Bos serves as CEO.