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.

Hal Barron, GSK

Break­ing the death spi­ral: Hal Bar­ron talks about trans­form­ing the mori­bund R&D cul­ture at GSK in a crit­i­cal year for the late-stage pipeline

Just ahead of GlaxoSmithKline’s Q2 update on Wednesday, science chief Hal Barron is making the rounds to talk up the pharma giant’s late-stage strategy as the top execs continue to woo back a deeply skeptical investor group while pushing through a whole new R&D culture.

And that’s not easy, Barron is quick to note. He told the Financial Times:

I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

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Aca­dia is mak­ing the best of it, but their lat­est PhI­II Nu­plazid study is a bust

Acadia’s late-stage program to widen the commercial prospects for Nuplazid has hit a wall. The biotech reported that their Phase III ENHANCE trial flat failed. And while they $ACAD did their best to cherry pick positive data wherever they can be found, this is a clear setback for the biotech.

With close to 400 patients enrolled, researchers said the drug flunked the primary endpoint as an adjunctive therapy for patients with an inadequate response to antipsychotic therapy. The p-value was an ugly 0.0940 on the Positive and Negative Syndrome Scale, which the company called out as a positive trend.

Their shares slid 12% on the news, good for a $426 million hit on a $3.7 billion market cap at close.

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Some Big Phar­mas stepped up their game on da­ta trans­paren­cy — but which flunked the test?

The nonprofit Bioethics International has come out with their latest scorecard on data transparency among the big biopharmas in the industry — flagging a few standouts while spotlighting some laggards who are continuing to underperform.

Now in its third year, the nonprofit created a new set of standards with Yale School of Medicine and Stanford Law School to evaluate the track record on trial registration, results reporting, publication and data-sharing practice.

Busy Gilead crew throws strug­gling biotech a life­line, with some cash up­front and hun­dreds of mil­lions in biobucks for HIV deal

Durect $DRRX got a badly needed shot in the arm Monday morning as Gilead’s busy BD team lined up access to its extended-release platform tech for HIV and hepatitis B.

Gilead, a leader in the HIV sector, is paying a modest $25 million in cash for the right to jump on the platform at Durect, which has been using its technology to come up with an extended-release version of bupivacaine. The FDA rejected that in 2014, but Durect has been working on a comeback.

In­tec blitzed by PhI­II flop as lead pro­gram fails to beat Mer­ck­'s stan­dard com­bo for Parkin­son’s

Intec Pharma’s $NTEC lead drug slammed into a brick wall Monday morning. The small-cap Israeli biotech reported that its lead program — coming off a platform designed to produce a safer, more effective oral drug for Parkinson’s — failed the Phase III at the primary endpoint.

Researchers at Intec, which has already seen its share price collapse over the past few months, says that its Accordion Pill-Carbidopa/Levodopa failed to prove superior to Sinemet in reducing daily ‘off’ time. 

Cel­gene racks up third Ote­zla ap­proval, heat­ing up talks about who Bris­tol-My­ers will sell to

Whoever is taking Otezla off Bristol-Myers Squibb’s hands will have one more revenue stream to boast.

The drug — a rising star in Celgene’s pipeline that generated global sales of $1.6 billion last year — is now OK’d to treat oral ulcers associated with Behçet’s disease, a common symptom for a rare inflammatory disorder. This marks the third FDA approval for the PDE4 inhibitor since 2014, when it was greenlighted for plaque psoriasis and psoriatic arthritis.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors.

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Apotex, though, has been a disaster ground. The manufacturer voluntarily yanked the ANDAs on 31 drugs — in late 2017 — after the FDA came across serious manufacturing deficiencies at their plants in India. A few days ago, the FDA made it official.

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