Fac­ing ques­tions on pa­tient deaths, Au­rinia shares crater in wake of lu­pus study

Au­rinia Phar­ma­ceu­ti­cals man­aged to kick up a brief spike in its share price this morn­ing af­ter re­leas­ing some up­beat num­bers from a Phase IIb study of its lu­pus drug vo­closporin. But it didn’t last long. The shares $AUPH quick­ly cratered, plung­ing by 48%, as ques­tions turned to a clus­ter of deaths tracked in its two dosage arms as well as the cost of the piv­otal study need­ed for an ap­proval.

At first blush, the num­bers in the 265-pa­tient study looked sol­id. The low dose of the drug hit the pri­ma­ry end­point on com­plete re­spons­es for the lu­pus pa­tients. But dur­ing the fol­lowup call, an­a­lysts ques­tioned why the low dose would beat out the high dose on CRs. They al­so ze­roed in on 13 deaths record­ed in the study – 10 in the low-dose arm, 2 in the high-dose arm and on­ly one in the con­trol group.

Forty per­cent of all the pa­tients in the study were en­rolled in Asia, com­pa­ny ex­ecs re­spond­ed, where most of the deaths oc­curred. The deaths were not re­lat­ed to the drug, they said in the call, and might be at­trib­uted to the kind of treat­ment stan­dards in dai­ly prac­tice in Asia. In the state­ment, the com­pa­ny con­ced­ed that:

The over­all rate of se­ri­ous ad­verse events (SAEs) was high­er in both vo­closporin groups but the na­ture of SAEs is con­sis­tent with high­ly ac­tive LN.

Mary Anne Doo­ley, the chief in­ves­ti­ga­tor for the study, main­tained that vo­closporin “could po­ten­tial­ly change the cur­rent treat­ment par­a­digm for LN.”

In­ves­ti­ga­tors com­bined vo­closporin to the cur­rent stan­dard of care of my­cophe­no­late mofetil, com­par­ing it to a com­bo us­ing a place­bo.

The drug, though, wasn’t al­ways that much bet­ter than the place­bo/SoC arm. In the study, 32.6% of pa­tients on low dose achieved CR, com­pared to 27.3% on high dose and 19.3% in the con­trol arm. Leerink’s Joseph Schwartz not­ed that the deaths will need to be stud­ied more, adding that the pos­i­tive re­sults al­so left some­thing to be de­sired. He added:

“At first glance, the ef­fi­ca­cy seen in the drug arms looks low­er than what we had ex­pect­ed, but still re­spectable. The study was 80% pow­ered to de­tect a re­mis­sion rate of 41% for the drug ver­sus 20% for place­bo based on a planned sam­ple size of 258 sub­jects. CR rates as low as 35% vs. 15% or as high as 47% ver­sus 25% would al­so main­tain 80% pow­er. The re­verse dose re­sponse was sur­pris­ing, par­tic­u­lar­ly since this would have been pulled down by ac­count­ing for the rel­a­tive­ly high num­ber of deaths in the drug arm.”

The Cana­di­an biotech not­ed that it had $12.1 mil­lion in cash at the end of H1, leav­ing it look­ing for fresh funds with a bat­tered stock price.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

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Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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Dif­fu­sion to hand Nas­daq spot to EIP Phar­ma for PhI­Ib de­men­tia study of ex-Ver­tex drug

One of the more than a dozen bidders for Diffusion Pharmaceuticals’ spot on Nasdaq has prevailed.

Boston biotech EIP Pharma will merge with Diffusion in an all-stock deal, with plans to start a Phase IIb clinical trial in the coming months in a common form of dementia with no approved treatments. The combined company will be renamed CervoMed.

The nine-year-old privately-held EIP is working on a former Vertex drug that it will test in a 160-person Phase IIb in patients with dementia with Lewy bodies, or DLB. The National Institute on Aging is expected to fund that trial with a $21 million grant. With the reverse merger, slated for closing in the middle of this year, EIP will be funded through that readout in the second half of 2024. EIP’s equity and debt holders will own about 77.25% of the combined company.

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Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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See­los Ther­a­peu­tics 'tem­porar­i­ly' stops study in rare neu­ro dis­or­der for busi­ness rea­sons

Microcap biotech Seelos Therapeutics is halting enrollment of its study in spinocerebellar ataxia type 3 (also known as Machado-Joseph disease) because of “financial considerations,” and in order to focus on other studies, the company said today, adding that the pause would be temporary.

The study will continue with the patients who have already enrolled, and the data from them will be used to decide whether to continue enrolling others in the future.

Alec­tor cuts 11% of work­force as it dou­bles down on late-stage neu­ro pro­grams part­nered with GSK, Ab­b­Vie

A month after revealing plans to concentrate on its late-stage immuno-neurology pipeline, Alector is trimming its headcount by 11%.

The layoffs will impact around 30 employees across the organization, the company disclosed in an SEC filing, adding that the plan will “better align the company’s resources” with the new strategy. With $712.9 million in cash, cash equivalents and investments as of the end of 2022, Alector believes the reserves will now get it through 2025.

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Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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J&J bows out of RSV vac­cine race, end­ing PhI­II study and ced­ing to Pfiz­er, GSK

Johnson & Johnson announced Wednesday morning it is ending development of its adult RSV vaccine that was in the middle of a 27,200-patient trial, giving up a big slice of what’s expected to be the next multibillion-dollar pharma market.

The decision came down to the shifting RSV “competitive landscape,” a company spokesperson tells Endpoints News, adding the “breadth of options” was much different than when J&J first started its pivotal study. The spokesperson declined to comment on the Phase III data, saying only the shot is undergoing an “ongoing assessment.”

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No longer ‘dead or just hi­ber­nat­ing,’ drug­mak­ers re­turn to heart med­i­cines

In 2015, now-FDA Commissioner Robert Califf joined industry, academic and regulatory representatives in Washington to discuss why more drugs weren’t in development for cardiovascular diseases, the leading US cause of death and once a mainstay of pharmaceutical industry blockbusters.

The group pointed to many reasons. Clinical trials could take years and testing was expensive. Wide availability of generic drugs made the commercial prospects uncertain. Their paper title summed up the mood: “Cardiovascular Drug Development: Is it Dead or Just Hibernating?”

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