Bio­gen, Ab­b­Vie yank MS drug Zin­bry­ta off the mar­ket in the wake of en­cephali­tis cas­es

The mar­ket­ing ef­fort for the MS drug Zin­bry­ta (da­clizum­ab) im­plod­ed to­day af­ter Eu­ro­pean reg­u­la­tors flagged a lethal safe­ty warn­ing over cas­es of in­flam­ma­to­ry en­cephali­tis and menin­goen­cephali­tis. Bio­gen $BI­IB and Ab­b­Vie $AB­BV, which had been look­ing to spend this year build­ing sales of the MS treat­ment, are now yank­ing it off the mar­ket in the EU and the US.

“Giv­en the na­ture and com­plex­i­ty of ad­verse events be­ing re­port­ed, char­ac­ter­iz­ing the evolv­ing ben­e­fit/risk pro­file of Zin­bry­ta will not be pos­si­ble go­ing for­ward giv­en the lim­it­ed num­ber of pa­tients be­ing treat­ed,” the com­pa­nies said in a state­ment. “There­fore, Bio­gen and Ab­b­Vie be­lieve it is in the best in­ter­est of pa­tients to vol­un­tar­i­ly with­draw world­wide mar­ket­ing au­tho­riza­tions for Zin­bry­ta.”

En­cephali­tis is char­ac­ter­ized by brain in­flam­ma­tion, usu­al­ly caused by a vi­ral in­fec­tion, which is rare and po­ten­tial­ly dead­ly.

Al San­drock, Bio­gen

The drug hit the US mar­ket in 2016 with a black box warn­ing over the threat of liv­er tox­i­c­i­ty af­ter the FDA ap­proved it for third-line MS cas­es. Reg­u­la­tors, though, have been will­ing to put up with con­sid­er­able safe­ty prob­lems with MS drugs so long as the dam­age to pa­tients is rare and the ben­e­fits con­sid­er­able. In this case, Bio­gen and Ab­b­Vie have yet to gain much trac­tion for a drug that an­a­lysts had felt would be lim­it­ed to peak sales of $500 mil­lion a year.

The with­draw­al won’t have much im­pact on Ab­b­Vie longterm, where the R&D group is in hot pur­suit of sev­er­al po­ten­tial block­busters to bol­ster its work around Hu­mi­ra. Bio­gen, though, won’t get off so easy. The big biotech made its rep in MS and has a thin pipeline dom­i­nat­ed by a high-risk Alzheimer’s ther­a­py. Bio­gen CMO Al San­drock man­aged to rat­tle the mar­ket re­cent­ly with news that they were adding pa­tients to their piv­otal tri­al for the Alzheimer’s drug to ac­count for some vari­abil­i­ty in the da­ta — which in turn trig­gered fresh jit­ters over its fu­ture.

“Bio­gen be­lieves the vol­un­tary world­wide with­draw­al of ZIN­BRY­TA, a treat­ment for re­laps­ing mul­ti­ple scle­ro­sis, is in the best in­ter­est of pa­tients,” said San­drock in a state­ment. “Bio­gen and Ab­b­Vie con­tin­ue to pri­or­i­tize pa­tient safe­ty and the care of mul­ti­ple scle­ro­sis pa­tients world­wide.”

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Albert Bourla, Pfizer CEO (Efren Landaos/Sipa USA/Sipa via AP Images)

Pfiz­er makes an­oth­er bil­lion-dol­lar in­vest­ment in Eu­rope and ex­pands again in Michi­gan

Pfizer is continuing its run of manufacturing site expansions with two new large investments in the US and Europe.

The New York-based pharma giant’s site in Kalamazoo, MI, has seen a lot of attention over the past year. As a major piece of the manufacturing network for Covid-19 vaccines and antivirals, Pfizer is gearing up to place more money into the site. Pfizer announced it will place $750 million into the facility, mainly to establish “modular aseptic processing” (MAP) production and create around 300 jobs at the site.

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Vas Narasimhan, Novartis CEO (Thibault Camus/AP Images, Pool)

No­var­tis bol­sters Plu­vic­to's case in prostate can­cer with PhI­II re­sults

The prognosis is poor for metastatic castration-resistant prostate cancer (mCRPC) patients. Novartis wants to change that by making its recently approved Pluvicto available to patients earlier in their course of treatment.

The Swiss pharma giant unveiled Phase III results Monday suggesting that Pluvicto was able to halt disease progression in certain prostate cancer patients when administered after androgen-receptor pathway inhibitor (ARPI) therapy, but without prior taxane-based chemotherapy. The drug is currently approved for patients after they’ve received both ARPI and chemo.

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Nkarta CEO Paul Hastings at Endpoints' #BIO22 panel (J.T. MacMillan Photography for Endpoints News)

Nkar­ta un­der­scores safe­ty of CAR-NK, boasts ear­ly re­spons­es

The first generation of personalized CAR-T therapies made big waves in the treatment of lymphoma for their stunning efficacy. Nkarta is hoping its off-the-shelf natural killer cell approach will stand out on safety — while keeping some of those impressive numbers on responses.

In a new update from its Phase I dose escalation study, the South San Francisco-based biotech reported that seven out of 10 patients treated with the highest doses of its NK cell therapy, NKX019, achieved a complete response, translating to a complete response rate of 70%.

Pfiz­er-backed Me­di­ar Ther­a­peu­tics ropes in an­oth­er Big Phar­ma in­vestor

A biotech centered on treating fibrosis — born out of Mass General and Brigham and Women’s Hospital — has received a financial boost.

According to an SEC filing, the company has raised $31,761,186 in its latest funding round, which includes 17 investors. The filing lists six names attached to the company, including Meredith Fisher, a partner at Mass General Brigham Ventures and Mediar’s acting CEO.

Sekar Kathiresan, Verve Therapeutics CEO

Verve re­veals let­ter from FDA that lays out con­di­tions to lift base edit­ing tri­al hold

We now know why Verve’s lead candidate was placed on hold last month by US regulators.

In an SEC filing, Verve laid out the FDA’s conditions for lifting the hold on its lead therapy, VERVE-101. That includes submitting preclinical data about potency differences in human versus non-human cells, risks of gene editing germline cells, and off-target analyses in non-hepatocyte cell types.

The FDA also wants clinical data from the ongoing Heart-1 trial, and to modify the trial protocol in the US to add additional contraceptive measures and increase the length of a staggering interval between the dosing of participants.

Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

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Af­ter M&A fell through, Ther­a­peu­tic­sMD sells hor­mone ther­a­py, con­tra­cep­tive ring for $140M cash plus roy­al­ties

TherapeuticsMD, a women’s health company whose one-time billion-dollar valuation seems a distant memory as its blockbuster aspirations petered out, is finally cashing out.

Australia’s Mayne Pharma is paying $140 million upfront to license essentially TherapeuticsMD’s whole portfolio, including two prescription drugs that treat conditions relating to menopause, a contraceptive vaginal ring as well as its prescription prenatal vitamin brands.

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