Raul Rodriguez, CEO of Rigel Pharmaceuticals

Rigel churns out an­oth­er late-stage clin­i­cal fail­ure — this time in Covid-19

In the wan­ing days of the Covid-19 pan­dem­ic, one pen­ny stock biotech is re­port­ing a close miss for its sole ap­proved drug. But alas, a close miss is still a miss.

Rigel Phar­ma­ceu­ti­cals — a biotech with on­ly ty­ro­sine ki­nase in­hibitor fos­ta­ma­tinib in its ar­se­nal — re­port­ed topline re­sults Tues­day from a Phase III Covid-19 tri­al in pa­tients with­out res­pi­ra­to­ry fail­ure and who had cer­tain, “high-risk prog­nos­tic” fac­tors. The drug is on­ly cur­rent­ly in­di­cat­ed to treat chron­ic im­mune throm­bo­cy­tope­nia, a blood con­di­tion when platelets are low.

Pos­i­tive re­sults for the tri­al were mixed. The biotech said the tri­al ap­proached sta­tis­ti­cal sig­nif­i­cance on the pri­ma­ry ef­fi­ca­cy end­point, which was the num­ber of days on oxy­gen through Day 29. How­ev­er, the tri­al did not ac­tu­al­ly meet sta­tis­ti­cal sig­nif­i­cance — the p-val­ue came out to p=0.0603. For the most part, a sta­tis­ti­cal­ly sig­nif­i­cant p-val­ue is de­ter­mined as p=0.05 or low­er.

The study, which en­rolled 280 pa­tients, put 141 pa­tients on the drug (twice dai­ly for two weeks) plus stan­dard of care, and put the re­main­ing 139 on place­bo plus stan­dard of care. Per Rigel, the mean num­ber of days on oxy­gen through Day 29 in the fos­ta­ma­tinib treat­ment arm was 6.9 days, com­pared to 9 days in the place­bo arm.

The biotech added that pre-spec­i­fied sec­ondary end­points fa­vored the drug over place­bo, in­clud­ing time in the ICU, mor­tal­i­ty and time to sus­tained re­cov­ery. In terms of mor­tal­i­ty, there were four deaths in the treat­ment arm ver­sus eight in place­bo — yet the p-val­ue there was sub­stan­tial­ly high­er than on the pri­ma­ry end­point, at p=0.4521.

Rigel CEO Raul Ro­driguez said in a state­ment that the biotech is en­cour­aged by the re­sults of the da­ta and is cur­rent­ly eval­u­at­ing next steps with the US De­part­ment of De­fense, which had part­nered with Rigel on the study.

The biotech has been fa­mil­iar with set­backs — just back in June, Rigel re­port­ed an­oth­er Phase III fail for fos­ta­ma­tinib, telling in­vestors that the pri­ma­ry end­point did not achieve sta­tis­ti­cal sig­nif­i­cance when com­pared to place­bo. Rigel was look­ing to in­duce a durable he­mo­glo­bin re­sponse in pa­tients with warm au­toim­mune he­molyt­ic ane­mia, or wAI­HA, in a bid to ex­pand its drug in­to oth­er dis­or­ders.

Shares of $RIGL were trad­ing be­low 80 cents as of Wednes­day morn­ing.

Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; 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.

As you start planning for #JPM23, we hope you will consider joining Endpoints News for our live and virtual events. For those who are celebrating Thanksgiving, we hope you are enjoying the long weekend with loved ones. And if you’re not — we’ll see you next week!

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Paul Perreault, CSL Behring CEO

CSL lands FDA ap­proval for he­mo­phil­ia B gene ther­a­py, sets $3.5M list price

The FDA has approved the world’s first gene therapy for hemophilia B, ushering into the market a treatment that’s historic in both what it promises to do and how much it will cost.

CSL will be marketing the drug, Hemgenix, at a list price of $3.5 million — which sets a new record for the most expensive single-use gene therapy in the US.

In a statement provided to Endpoints News, the Australian company noted that the current costs of treating people with moderate to severe hemophilia B can be significant over a lifetime. By some estimates, healthcare systems could spend more than $20 million per person.

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Elon Musk (GDA via AP Images)

Biggest drug com­pa­nies halt­ed Twit­ter ad buys af­ter Lil­ly in­sulin spoof

Almost all of the drug industry’s biggest advertisers cut their spending on Twitter to zero or near-zero over the last two weeks amid worries about impersonation of their brands by pranksters and the future of the social media company.

Among 18 of the biggest pharmaceutical advertisers in the US market, 12 cut their Twitter ad spending to nothing for the week beginning Nov. 14, according to Pathmatics, which tracks data on prescription drug ad spending as well as general corporate advertising. The list of drugmakers cutting spending to zero includes Merck, AstraZeneca, Eli Lilly, Novartis, Pfizer and others.

J&J's Spra­va­to pulls a PhI­II win against Sero­quel XR in treat­ment-re­sis­tant de­pres­sion

A day before Thanksgiving, J&J’s Janssen has a new cut of Phase III Spravato data to be grateful for.

The pharma giant announced on Wednesday that its nasal spray, also known as esketamine, beat extended-release quetiapine, previously sold by AstraZeneca as Seroquel XR, in treatment-resistant depression (TRD). Of 676 adults, a significantly higher number of patients on Spravato were able to achieve remission and avoid relapse after 32 weeks, according to J&J.

Isao Teshirogi, Shionogi president and CEO (Kyodo via AP Images)

Sh­ionogi's Covid an­tivi­ral lands first ap­proval in Japan's new emer­gency ap­proval path­way

Japanese regulators on Tuesday signed off on Shionogi’s homegrown antiviral for Covid-19, known as Xocova (ensitrelvir), making it the first approval under Japan’s emergency regulatory approval system.

The emergency approval, following a back-and-forth with regulators since last February, is based on a safety profile with more than 2,000 patients who have accessed the pill, and clinical symptomatic efficacy for five typical Omicron-related symptoms (primary endpoint) and antiviral efficacy (key secondary endpoint) in patients with mild to moderate SARS-CoV-2 infection, regardless of risk factors or vaccination status, and during the Omicron-dominant phase of the pandemic.

Rob Davis, Merck CEO

Up­dat­ed: No Seagen here: 'Do more' means a small $1.35B pur­chase of Ima­go for Mer­ck

Merck is making an acquisition, the Big Pharma announced before Monday’s opening bell. No, Seagen is not entering the fold, as had been speculated for quarters.

Folding under Merck’s wings will be Pfizer-backed Imago BioSciences. For nearly a year, Merck CEO Rob Davis has been saying the pharma giant needs to “do more” on the business development front after its 2021 $11.5 billion acquisition of Acceleron.

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Stanley Erck, Novavax CEO (Andrew Harnik/AP Images)

No­vavax pulls out of Covid-19 vac­cine al­liance with Gavi

Novavax is pulling out of its Covid-19 vaccine deal with Gavi, the Vaccine Alliance, a global partnership tasked with ensuring vaccine access in lower-income countries, following an alleged contract violation.

The Maryland-based company claimed on Friday that Gavi failed to purchase at least 350 million doses of its protein-based vaccine Nuvaxovid by the end of the year, per an advanced purchase agreement. Gavi, the World Health Organization and the Coalition for Epidemic Preparedness Innovations (CEPI) are co-leaders of COVAX, an effort to ensure that all participating countries, regardless of income levels, have access to vaccines.

Alzheimer’s drug bites the dust; Re­struc­ture, re­struc­ture, re­struc­ture; Land­mark di­a­betes OK; 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.

Being in the news business can give one a warped sense of time — it feels like quite a while since we published some of these stories below. But next Saturday’s Endpoints Weekly will definitely be shorter, as we take off Thursday and Friday for Thanksgiving. We will still have the abbreviated edition in your inbox at the usual time.

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Image: Shutterstock

MIT re­searchers re­veal DNA "Paste" tech be­hind lat­est gene edit­ing start­up

MIT scientists have developed a tool that they say can insert large gene sequences where they want in the genome.

In a paper published Thursday in Nature Biotechnology, MIT fellows Omar Abudayyeh, Jonathan Gootenberg and colleagues detail a technology they call PASTE, which they say can potentially be used to insert long strands of DNA and treat genetic diseases caused by many different mutations, such as cystic fibrosis and Leber congenital amaurosis, a rare eye disorder that causes blindness.