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.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Spe­cial re­port 2022: Meet 20 women blaz­ing trails in bio­phar­ma R&D

When you run a special report for a fourth year, it can start feeling a little bit like a ritual. You go through the motions — in our case opening up nominations for top women in biopharma R&D and reviewing more than 500 entries — you make your choices of inclusion and exclusion. You host a ceremony.

But then things happen that remind you why you do it in the first place. Perhaps a Supreme Court rules to overturn the constitutional right to abortion and a group of women biotech leaders makes it clear they strongly dissent; perhaps new data on gender diversity in the industry come out that look all too similar to the old ones, suggesting women are still dramatically underrepresented at the top; perhaps protests and conflicts around the world put in stark terms the struggles that many women still face in earning the most basic recognition.

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Kristen Hege, Bristol Myers Squibb SVP, early clinical development, oncology/hematology and cell therapy (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Q&A: Bris­tol My­er­s' Kris­ten Hege on cell ther­a­py, can­cer pa­tients and men­tor­ing the next gen­er­a­tion

Kristen Hege leads Bristol Myers Squibb’s early oncology discovery program carrying on from the same work at Celgene, which was acquired by BMS in 2019. She’s known for her early work in CAR-T, having pioneered the first CAR-T cell trial for solid tumors more than 25 years ago.

However, the eminent physician-scientist is more than just a drug developer mastermind. She’s also a practicing physician, mother to two young women, an avid backpacker and intersecting all those interests — a champion of young women and people of color in STEM and life sciences.

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Gossamer Bio CEO Faheem Hasnain at Endpoints' #BIO22 panel (J.T. MacMillan Photography for Endpoints News)

Gos­samer’s Fa­heem Has­nain de­fends a round of pos­i­tive PAH da­ta as a clear win. But can these PhII re­sults stand up to scruti­ny?

Gossamer Bio $GOSS posted a statistically significant improvement for its primary endpoint in the key Phase II TORREY trial for lead drug seralutinib on Tuesday morning. But CEO Faheem Hasnain has some explaining to do on the important secondary of the crucial six-minute walk distance test — which will be the primary endpoint in Phase III — as the data on both endpoints fell short of expectations, missing one analyst’s bar on even modest success.

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Prometheus moves to raise cash hours af­ter PhII da­ta leads to stock surge

After releasing better-than-anticipated data on two mid-stage studies Wednesday morning, Prometheus Biosciences’ CEO said the company would “take some time to assess” its next financing options.

It only needed about seven hours. Wednesday afternoon after the market closed, the biotech announced it would seek $250 million through an equity offering as the company looks to edge out anti-TL1A competitor Pfizer and its new partner Roivant.

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Piper Trelstad, head of CMC, Bill & Melinda Gates Medical Research Institute

Q&A with Gates leader: Women tak­ing on more roles in phar­ma man­u­fac­tur­ing, but still work to do

More and more women are driving innovation and taking leadership roles in biotech – as evidenced today in the release of Endpoints News’ list of the top 20 women in the R&D world – but those gains are beginning to extend across pharma sectors.

In pharma manufacturing in the US today, around 46% of all roles are occupied by women, according to the US Bureau of Labor Statistics for 2021. And according to a Bloomberg report, women’s roles across manufacturing roles had a massive boost after the start of the pandemic.

Phar­ma rep­u­ta­tion re­tains 'halo' even as pan­dem­ic me­dia cov­er­age re­cedes — sur­vey

The Covid-19 halo effect on the pharma industry is continuing, according to a new global study from Ipsos. The annual survey for the International Federation of Pharmaceutical Manufacturers and Associations (IFPMA) finds considerable goodwill from consumers across measures of trust, cooperation with governments, and advancing research and drug development.

“Despite the pandemic in many countries no longer being the top of mind concern generally – although it does remain the top concern as a health issue – the industry’s reputation has remained positive,” said Ipsos research director Thomas Fife-Schaw.

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FDA commissioner Robert Califf (Jose Luis Magana/AP Images)

FDA pulls On­copep­tides' Pepax­to in­di­ca­tion, open­ing the door for dan­gling ac­cel­er­at­ed ap­proval en­force­ment

In a move all but ensured after an overwhelmingly negative adcomm vote this September, the FDA is yanking Oncopeptides’ dangling accelerated approval. And there may be more to come.

In recent months, US regulators have honed in on reforming the accelerated approval pathway and preventing drugmakers from continuing to sell their medicines in the event of a confirmatory study flop. The moves come after commissioner Rob Califf has called for companies to do more to produce post-marketing evidence quickly earlier this year.

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Mark McKenna, Prometheus Biosciences chairman & CEO

With clear PhII win in IBD, Prometheus thwarts Pfiz­er com­par­isons as it fol­lows Hu­mi­ra 'play­book'

Prometheus Biosciences reported a clear Phase II win in two inflammatory bowel disease conditions in a clinical development race with Pfizer, planting the biotech’s flag in a field of antibodies attempting to go against black box-cornered JAK inhibitors and AbbVie’s Humira.

Shares $RXDX have soared since the summer — a small dip last week notwithstanding when rival Pfizer teamed up with Roivant on a new company for their competing anti-TL1A monoclonal antibody. And they skyrocketed once again Wednesday morning, climbing from $36 apiece to more than $100 on the back of two Phase II studies: one placebo-controlled in ulcerative colitis and the other an open-label trial in patients with Crohn’s disease.

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