Or­bimed part­ner in search of a cure for Parkin­son’s launch­es new gene ther­a­py play­er

Jonathan Sil­ver­stein is ac­tive­ly look­ing for a biotech mir­a­cle.

Jonathan Sil­ver­stein

The Or­bimed part­ner formed a non­prof­it group called the Sil­ver­stein Foun­da­tion af­ter he was di­ag­nosed with Parkin­son’s, and now he’s launch­ing a new biotech which he hopes can pro­vide just that.

The new biotech is called Pre­vail Ther­a­peu­tics. Based in New York, it will be run by Co­lum­bia Uni­ver­si­ty’s Asa Abe­liovich, look­ing to de­vel­op gene ther­a­pies that will try to cor­rect a dis­ease that has proven to be one of the tough­est tar­gets in drug de­vel­op­ment. Or­bimed and the foun­da­tion — which Sil­ver­stein is al­so fund­ing — are back­ing the start­up along with Re­genXBio, one of the lead­ers in AAV tech used in the gene ther­a­pies now in clin­i­cal de­vel­op­ment.

The key fo­cus at Pre­vail will be the GBA1 mu­ta­tion car­ried by about 1 in 10 Parkin­son’s pa­tients. Ac­cord­ing to the biotech, the link be­tween the GBA1 gene, glu­co­cere­brosi­dase (GCase) pro­tein pro­duc­tion, and clear­ing out an ac­cu­mu­la­tion of al­pha-synu­cle­in in the brain “may have im­pli­ca­tions for the broad­er Parkin­son’s pop­u­la­tion, as well as in oth­er neu­rode­gen­er­a­tive dis­ease.”

The ven­ture cap­i­tal­ist told CN­BC that he’s seen biotechs like Bio­gen de­liv­er mir­a­cles to pa­tients, adding: “And … now I’ve got to cre­ate an­oth­er mir­a­cle.”

Adds Sil­ver­stein:

We be­lieve Re­genXBio’s NAV Tech­nol­o­gy Plat­form pro­vides us with a promis­ing op­por­tu­ni­ty to de­vel­op po­ten­tial life-chang­ing ther­a­pies for pa­tients suf­fer­ing from Parkin­son’s Dis­ease and oth­er neu­rode­gen­er­a­tive dis­eases. Dr. Abe­liovich’s lead­er­ship and deep ex­per­tise in the mol­e­c­u­lar mech­a­nisms of neu­rode­gen­er­a­tion unique­ly po­si­tion Pre­vail to make a sig­nif­i­cant mark in de­vel­op­ing in­no­v­a­tive bi­o­log­ic ther­a­pies for Parkin­son’s Dis­ease and oth­er cen­tral ner­vous sys­tem dis­eases with huge un­met med­ical needs.

Lina Gugucheva, NewAmsterdam Pharma CBO

Phar­ma group bets up to $1B-plus on the PhI­II res­ur­rec­tion of a once dead-and-buried LDL drug

Close to 5 years after then-Amgen R&D chief Sean Harper tamped the last spade of dirt on the last broadly focused CETP cholesterol drug — burying their $300 million upfront and the few remaining hopes for the class with it — the therapy has been fully resurrected. And today, the NewAmsterdam Pharma crew that did the Lazarus treatment on obicetrapib is taking another big step on the comeback trail with a €1 billion-plus regional licensing deal, complete with close to $150 million in upfront cash.

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How pre­pared is bio­phar­ma for the cy­ber dooms­day?

One of the largest cyberattacks in history happened on a Friday, Eric Perakslis distinctly remembers.

Perakslis, who was head of Takeda’s R&D Data Sciences Institute and visiting faculty at Harvard Medical School at the time, had spent that morning completing a review on cybersecurity for the British Medical Journal. Moments after he turned it in, he heard back from the editor: “Have you heard what’s going on right now?”

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Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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Pearl Huang, Dunad Therapeutics CEO (Ken Richardson, PR Newswire)

Long­time biotech leader Pearl Huang takes the reins as CEO of No­var­tis-backed up­start

It has only been a few months since Pearl Huang exited the top seat at Cygnal Therapeutics, but now she’s back at the helm of another biotech.

After taking a few months off — passing an exam in that time to get her captain’s license from the US Coast Guard — she’s been named CEO of Dunad Therapeutics, a biotech focused on developing a small molecule covalent therapies that was founded in 2020. Huang told Endpoints News that two factors attracted her to going back to the c-suite: the company’s technology and its co-founders.

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Matt Gline, Roivant CEO (John Sciulli/Getty Images for GLG)

Roivant chops sick­le cell gene ther­a­py, der­ma­tol­ogy drugs to fo­cus on 'high­er val­ue pro­ject­s'

Roivant is sweeping a suite of drugs, including a gene therapy for sickle cell disease already in the clinic, out of its pipeline.

Six programs from four of its “vants” are being wound down as part of “a company-wide cost optimization and pipeline reprioritization initiative to reduce our expected operating expenses and prioritize our capital resources.”

When reached by Endpoints News, a spokesperson said, “We don’t anticipate a material reduction in headcount but we will likely reassign some folks to higher value projects as part of winding down specific programs.”

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Ben Zimmer, Priovant CEO

Roivant un­veils lat­est spin­out as Pfiz­er en­trusts JAK1/TYK2 to Pri­o­vant

In November, Pfizer disclosed it’s spun out the Phase II dual JAK1/TYK2 inhibitor to a startup formed in collaboration with an unnamed, experienced partner.

We now know who the partner is. And as Pfizer and Roivant officially take the wraps off Priovant Therapeutics, the companies reveal that they have started two registrational trials of the drug, brepocitinib, as part of a broader plan to develop a big, first-in-class franchise spanning multiple orphan and specialty autoimmune diseases.

Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.

Members of the G7 from left to right: Prime Minister of Italy Mario Draghi, European Commission President Ursula von der Leyen, President Joe Biden, German Chancellor Olaf Scholz, British Prime Minister Boris Johnson, Canadian Prime Minister Justin Trudeau, Prime Minister of Japan Fumio Kishida, French President Emmanuel Macron and European Council President Charles Michel (AP Photo/Susan Walsh)

Biden and G7 na­tions of­fer funds for vac­cine and med­ical prod­uct man­u­fac­tur­ing project in Sene­gal

Amidst recently broader vaccine manufacturing initiatives from the EU and European companies, the G7 summit in the mountains of Bavaria has brought about some positive news for closing vaccine and medical product manufacturing gaps around the globe.

According to a statement from the White House, the G7 leaders have formally launched the partnership for global infrastructure, PGII. The effort will aim to mobilize hundreds of billions of dollars to deliver infrastructure projects in several sectors including the medical and pharmaceutical manufacturing space.

Laurence Reid, Decibel CEO

Still in pre­clin­i­cal test­ing for ear gene ther­a­pies, Deci­bel touts small snap­shot of chemo-in­duced hear­ing loss drug

Though Decibel Therapeutics has largely pivoted toward gene therapies for the inner ear, its lead clinical candidate simply aims to protect cancer patients from chemotherapy-induced hearing loss. On Tuesday, the biotech presented its first efficacy data for the program, and execs like what they see.

Decibel reported interim results from a Phase Ib study showing the experimental drug, dubbed DB-020, largely protected a small group of patients from losing their hearing. Researchers used a particularly unique study design, administering the compound in one of each patients’ ears before they received cisplatin chemotherapy and placebo in the other.