Unum racks up yet an­oth­er FDA hold as it twists its way through a re­struc­tur­ing, look­ing for a fresh start with a pre­clin­i­cal drug

Chuck Wil­son Unum

The drum­beat of bad news at Unum Ther­a­peu­tics just won’t stop.

In a fil­ing with the SEC to­day, the trou­bled biotech re­port­ed that its ex­per­i­men­tal ther­a­py AC­TR707 — used in com­bi­na­tion with rit­ux­imab in pa­tients with CD20+ B cell non-Hodgkin lym­phoma — may have been re­spon­si­ble for a pos­si­ble new ma­lig­nan­cy. The FDA slapped a par­tial hold on the study March 4 while the agency and the biotech ex­plore what hap­pened to trig­ger the Grade 3 SAE.

Unum’s stock, which had al­ready sunk deep in­to pen­ny stock ter­rain, dropped 5% Mon­day evening af­ter an 8% drop over the day, leav­ing the stock at 46 cents. The com­pa­ny is helmed by Chuck Wil­son and chaired by At­las’ Bruce Booth. And they’ve had to deal with one set­back af­ter the next since fil­ing to go pub­lic 2 years ago.

The hold came ex­act­ly 2 days af­ter the biotech re­port­ed that it was re­struc­tur­ing the com­pa­ny, lay­ing off 60% of the staff, and re­fo­cus­ing ef­forts away from the trou­ble­some AC­TR707 in fa­vor of a pre­clin­i­cal sol­id tu­mor ef­fort in the pipeline. At the same time, CSO Seth Et­ten­berg re­signed af­ter 5 years with Unum.

Seth Et­ten­berg Unum

Last sum­mer the biotech re­port­ed that its then pre­mier drug — AC­TR087 — was linked to se­ri­ous ad­verse events that in­clud­ed grade 3 neu­ro­tox­i­c­i­ty and cy­tomegalovirus in­fec­tion as well as grade 4 res­pi­ra­to­ry dis­tress, trig­ger­ing a hold. That came af­ter the IPO, when Unum wait­ed un­til it filed the S-1 to re­port that their drug was linked to two deaths — with 2 cas­es of AC­TR087-re­lat­ed se­vere CRS while one pa­tient died from AC­TR087-re­lat­ed neu­ro­tox­i­c­i­ty.

That al­so trig­gered a hold which was lift­ed just ahead of the IPO.

Unum plans to file an IND ap­pli­ca­tion for BOXR1030 in late 2020 as it works to get back in­to the clin­ic. It has a long way to go, though, in restor­ing faith among in­vestors.

So­cial im­age: Chuck Wil­son, Unum Ther­a­peu­tics

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

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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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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.

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.

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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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.

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.

Karen Aiach, Lysogene CEO (RE(ACT) Discovery Institute)

Gene ther­a­py flunks PhII/III study, but for­mer Sarep­ta part­ner sees a path for­ward — if it can find the cash

The development path for Lysogene’s gene therapy for MPS IIIA has been a rocky one. After the FDA slapped a partial clinical hold on a Phase II/III study, a patient already dosed in the trial died, although it was deemed unrelated to treatment. Then earlier this year, Sarepta pulled out of their three-year partnership due to disagreements on who will handle commercial supply.

And now, Lysogene reported the trial has failed its primary endpoint.

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