Can­cer drug to treat dwarfism? Aban­doned No­var­tis as­set snagged by Bridge­Bio’s QED Ther­a­peu­tics

Bridge­Bio is snatch­ing a re­cent­ly-aban­doned can­cer pro­gram off No­var­tis’ shelves, hand­ing the as­set to a spin­out com­pa­ny with un­usu­al plans for the drug.

The new­ly-li­censed prod­uct — called in­fi­gra­tinib or BGJ398 — was in Phase II tri­als at No­var­tis for pa­tients with bile duct can­cer with FGFR fu­sions. Al­though the drug showed mean­ing­ful clin­i­cal ac­tiv­i­ty ear­ly in the tri­al, No­var­tis de­cid­ed to dump the drug last year.

Michael Hen­der­son

“They were orig­i­nal­ly de­vel­op­ing the FGFR in­hibitor for a much broad­er spec­trum of on­col­o­gy,” said Michael Hen­der­son, the guy in charge of find­ing new as­sets for Bridge­Bio. While two in­di­ca­tions — bile duct can­cer (cholan­gio­car­ci­no­ma) and urothe­lial car­ci­no­ma — saw an in­ter­est­ing re­sponse from the drug, it wasn’t enough to dri­ve No­var­tis for­ward.

It’s enough for Bridge­Bio, which is spin­ning out QED Ther­a­peu­tics to de­vel­op the drug with a gift of $65 mil­lion in seed mon­ey. A chunk of that cash will go straight back out to No­var­tis to pay for li­cens­ing fees, al­though de­tails of the deal weren’t dis­closed.

But weird­ly, in­fi­gra­tinib was orig­i­nal­ly on Bridge­Bio’s radar for oth­er rea­sons, which will play in­to where the drug goes from here.

Hen­der­son, who co-found­ed Pelle­Pharm while still in med­ical school at the ripe age of 22, now works at Bridge­Bio scout­ing for promis­ing as­sets. At 28, he tells me he spends his time scrap­ing PubMed, read­ing ab­stracts, and some­times pour­ing over Big Phar­ma’s fi­nan­cial state­ments look­ing for some­thing worth­while.

Just months ago, Hen­der­son be­came in­trigued with a pa­per about a form of ge­net­ic dwarfism called achon­dropla­sia. The con­di­tion can cause brain­stem com­pres­sion and oth­er com­pli­ca­tions as the bones fail to de­vel­op. In the pa­per that caught Hen­der­son’s eye, re­searchers showed in mouse mod­els that low dos­es of No­var­tis’ can­cer drug in­fi­gra­tinib — which in­hibits FGFR — could treat achon­dropla­sia at its source by di­rect­ly treat­ing the mu­ta­tion in FGFR3. In the­o­ry, this could mean big things for some pa­tients suf­fer­ing from the ge­net­ic dis­ease. A cor­rec­tion at the source could help bones to grow more nor­mal­ly, per­haps de­creas­ing se­ri­ous com­pli­ca­tions of achon­dropla­sia, such as cra­nial im­pinge­ment, which can re­quire surgery lat­er in life. It might al­so de­crease spinal steno­sis, ob­struc­tive sleep ap­nea and ear in­fec­tions.

Neil Ku­mar

“We’re re­al­ly in­ter­est­ed in the no­tion that tar­get­ed can­cer ther­a­pies can be used to ad­dress oth­er ge­net­ic con­di­tions,” said Bridge­Bio’s CEO Neil Ku­mar.

Ku­mar tells me peo­ple are of­ten ner­vous “and right­ful­ly so” to use ki­nase in­hibitors in pe­di­atric con­di­tions be­cause they tend to be tox­ic. But Ku­mar thinks low dos­es may have “pro­found ef­fects.”

So Hen­der­son de­cides Bridge­Bio should try to snag the in­fi­gra­tinib pro­gram, and dis­cov­ers (about 250 pages deep in a 500-page 10-K) that No­var­tis is al­ready look­ing for buy­ers. In fact, No­var­tis al­ready had term sheets from oth­er com­pa­nies hop­ing to snatch the drug.

“We sprint­ed to get [the bid] to­geth­er with­in a month, as­sem­bled the right team, and with­in two months we were mov­ing for­ward,” Hen­der­son said.

Bridge­Bio made an ap­peal­ing case: they would take over the Phase II can­cer pro­gram, pur­sue tri­als in cholan­gio­car­ci­no­ma and urothe­lial car­ci­no­ma (good da­ta per­mit­ting), and tack on some pre­clin­i­cal work in the brand new in­di­ca­tion of achon­dropla­sia.

The more pa­tients the drug can reach, the bet­ter. So No­var­tis took them up on the deal.

QED is on the hunt for a CEO, and is be­ing led in the in­ter­im by its R&D lead­ers and CMO Daniel Hoth, the for­mer chief of the In­ves­ti­ga­tion­al Drug Branch of the Na­tion­al Can­cer In­sti­tute (NCI).

“We are com­mit­ted to mov­ing this com­pound for­ward in late-stage de­vel­op­ment and fur­ther prov­ing the strong ef­fi­ca­cy in can­cer that has al­ready been demon­strat­ed across mul­ti­ple tri­als,” Hoth said.

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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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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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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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.

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.

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.

State bat­tles over mifepri­s­tone ac­cess could tie the FDA to any post-Roe cross­roads

As more than a dozen states are now readying so-called “trigger” laws to kick into effect immediate abortion bans following the overturning of Roe v. Wade on Friday, these laws, in the works for more than a decade in some states, will likely kick off even more legal battles as states seek to restrict the use of prescription drug-based abortions.

Since Friday’s SCOTUS opinion to overturn Americans’ constitutional right to an abortion after almost 50 years, reproductive rights lawyers at Planned Parenthood and other organizations have already challenged these trigger laws in Utah and Louisiana. According to the Guttmacher Institute, other states with trigger laws that could take effect include Arkansas, Idaho, Kentucky, Mississippi, Missouri, North Dakota, Oklahoma, South Dakota, Tennessee, Texas, and Wyoming.

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Deborah Dunsire, Lundbeck CEO

Af­ter a 5-year re­peat PhI­II so­journ, Lund­beck and Ot­su­ka say they're fi­nal­ly ready to pur­sue OK to use Rex­ul­ti against Alzheimer's ag­i­ta­tion

Five years after Lundbeck and their longtime collaborators at Otsuka turned up a mixed set of Phase III data for Rexulti as a treatment for Alzheimer’s dementia-related agitation, they’ve come through with a new pivotal trial success they believe will finally put them on the road to an approval at the FDA. And if they’re right, some analysts believe they’re a short step away from adding more than $500 million in annual sales for the drug, already approved in depression and schizophrenia.

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A Mer­ck part­ner is sucked in­to the fi­nan­cial quag­mire as key lender calls in a note

Another biotech standing on shaky financial legs has fallen victim to the bears.

Merck partner 4D Pharma has reported that a key lender, Oxford Finance, shoved the UK company into administration after calling in a $14 million loan they couldn’t immediately make good on. Trading in their stock was halted with a market cap that had fallen to a mere £30 million.

“Despite the very difficult prevailing market conditions,” 4D reported on Friday, the biotech had been making progress on finding some new financing and turned to Oxford with an alternative late on Thursday and then again Friday morning.