A year af­ter scor­ing FDA ap­proval for di­a­betes drug, Zealand touts pos­i­tive PhI­II da­ta for new in­di­ca­tion

When Ze­ga­logue, al­so known as dasiglucagon, got ap­proved by the FDA ear­ly last year for se­vere hy­po­glycemia in di­a­betes pa­tients, Zealand Phar­ma said that wasn’t the end of its vi­sion for the drug. One of the oth­er in­di­ca­tions they planned to pur­sue came in pe­di­atric pa­tients who had con­gen­i­tal hy­per­in­sulin­ism, a ge­net­ic dis­or­der where the pan­creas pro­duces too much in­sulin that re­sults in low blood sug­ar.

The re­sults are now in from a Phase III tri­al, and ac­cord­ing to the biotech, the pri­ma­ry end­point has been met. The pep­tide spe­cial­ist out­fit an­nounced Thurs­day that top-line re­sults from the tri­al, which en­rolled 12 ba­bies with the ge­net­ic con­di­tion (rang­ing from 12 months all the way down to just one week old), showed a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion in pa­tients re­quir­ing in­tra­venous glu­cose be­tween treat­ment arms ver­sus place­bo.

In­vestors cheered the news, send­ing Zealand shares $ZEAL up 18% in Thurs­day morn­ing trad­ing.

In the study, Zealand re­port­ed the mean IV GIR, a mea­sure of how much glu­cose is re­quired to keep pa­tients’ blood sug­ar lev­els sta­ble, in pa­tients in the treat­ment arm and the place­bo arm. The base­line IV GIR was 15.7 mg/kg/minute. For pa­tients who took Ze­ga­logue, the lev­el was re­duced to 4.3 mg/kg/min, and re­duced to 9.4 mg/kg/min for the place­bo group, good for a 55% re­duc­tion.

That clocked in at a p-val­ue of p=0.0037. Ad­di­tion­al­ly, 11 of the 12 pa­tients in the study are mov­ing in­to an on­go­ing, safe­ty ex­ten­sion tri­al.

This study was the sec­ond Phase III that Zealand went with in CHI. The first study read out back in 2020 and looked at old­er chil­dren, en­rolling 32 pa­tients be­tween the ages of 3 months and 12 years old. The phar­ma re­port­ed at the time that the drug plus stan­dard of care did not sig­nif­i­cant­ly re­duce the rate of hy­po­glycemia com­pared to stan­dard of care alone when as­sessed by in­ter­mit­tent self-mea­sured plas­ma glu­cose.

How­ev­er, when com­pared to con­tin­u­ous glu­cose mon­i­tor­ing via an ex­plorato­ry analy­sis, hy­po­glycemia was re­duced by 40 to 50% with Ze­ga­logue as com­pared to stan­dard of care alone. The analy­sis led to 31 of those pa­tients to en­roll in the afore­men­tioned safe­ty ex­ten­sion tri­al.

The biotech added that now that the da­ta are in, it’s look­ing at meet­ing with the FDA and start to pur­sue an NDA sub­mis­sion for that in­di­ca­tion. Zealand said in a state­ment that it plans to have the sub­mis­sion done by year’s end.

“We are ex­treme­ly pleased with the top-line re­sults from our sec­ond Phase III study of dasiglucagon for the treat­ment of in­fants with CHI,” Zealand CEO Adam Steens­berg said in a state­ment, adding, “We look for­ward to en­gag­ing with the US FDA and mov­ing for­ward with our New Drug Ap­pli­ca­tion.”

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

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

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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Hugo Peris, Spiral Therapeutics CEO

Hear­ing-fo­cused biotech grabs trio of pro­grams from Oton­o­my's fire sale

Otonomy may be shutting down, but the lessons learned there will live on at another biotech working on new treatments for hearing loss.

San Francisco-based Spiral Therapeutics has bought certain assets related to three of Otonomy’s programs, ranging from data, patent rights, and know-how to inventory. That includes data around Otonomy’s twice-failed lead program, OTO-104 (Otividex), a sustained-exposure formulation of dexamethasone.

Jeff Bluestone (R), Sonoma Biotherapeutics CEO

Jef­frey Blue­stone brings his start­up haul to $400M+, join­ing forces with Re­gen­eron on cell ther­a­pies

These days, when Jeffrey Bluestone gets together with his contemporaries in science, the conversation often turns to retirement plans.

But a little more than three years ago, Bluestone reached a momentous turning point in his career, exiting a prestigious post at UCSF, where he had spent decades in the scientific pursuit of new therapies. And it had nothing to do with retirement anytime in the near future.

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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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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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Mihael Polymeropoulos, Vanda Pharmaceuticals CEO

Van­da wins court case against FDA over dis­clo­sure of CRL de­tails for sleep drug

DC District Court Judge Christopher Cooper today granted Vanda Pharma’s request to require the FDA to disclose more info on the complete response letter for its sleep disorder drug Hetlioz.

The melatonin receptor agonist is approved by the FDA to treat non-24-hour sleep-wake disorder, a circadian rhythm disorder. But in 2018 Vanda filed a supplemental application to market Hetlioz as a treatment for jet lag, which the FDA rejected in August 2019, with few details on what Vanda needed to correct course, according to the company.