AS­CO17: As­traZeneca pumps up ex­pec­ta­tions on near-term can­cer drug wins, look­ing to shed a rep for fail­ure

CHICA­GO — As­traZeneca’s R&D team ar­rived at AS­CO on a crit­i­cal mis­sion.

With its lead­ing PARP in­hibitor outscor­ing chemo in treat­ing breast can­cer,  mak­ing the spot­light round on Sun­day, As­traZeneca was able to of­fer some fresh, sol­id ev­i­dence of its con­tin­u­ing progress on the all-im­por­tant can­cer front. And with sev­er­al key cat­a­lysts lined up over the sum­mer, ex­ecs want­ed to send a clear mes­sage that this can­cer drug roll they’re on is just be­gin­ning.

As far as Leerink’s Sea­mus Fer­nan­dez is con­cerned, As­traZeneca’s in­vestor meet­ing in Chica­go was a rous­ing suc­cess, with some sug­ges­tions that the phar­ma gi­ant is on the verge of of­fer­ing some ‘prac­tice chang­ing’ ad­vances in the field.

Sean Bo­hen, As­traZeneca

As­traZeneca ex­ecs left the crowd with the clear im­pres­sion that Lyn­parza was on its way to a broad ap­proval for sec­ond-line main­te­nance ther­a­py in treat­ing ovar­i­an can­cer. R&D chief Sean Bo­hen al­so hint­ed that the full read­out on the PA­CIF­IC tri­al — Imfinzi monother­a­py fol­low­ing chemo and ra­di­a­tion in Stage 3 non-small cell lung can­cer — will be a hit. Fer­nan­dez not­ed:

Based on this and com­ments from pre­vi­ous­ly in­ter­viewed KOLs, we be­lieve the re­sults are like­ly to be both clin­i­cal­ly mean­ing­ful and prac­tice-chang­ing. AZN es­ti­mates a Stage III NSCLC in­ci­dence of ~100k per year, with about half be­ing un­re­sectable. With oth­er PD-1/L1 in­hibitors ~2-3 years be­hind in the set­ting, this rep­re­sents a large mar­ket-ex­pand­ing op­por­tu­ni­ty for Imfinzi.

Fer­nan­dez be­lieves that the phar­ma gi­ant has al­ready filed its BTK drug acal­abru­ti­nib for an ap­proval at the FDA and is just wait­ing for con­fir­ma­tion that the pack­age has been ac­cept­ed for re­view. Peak sales es­ti­mates for this drug scrape the $1 bil­lion mark— pos­si­bly mak­ing it one of the top 10 or­phan drugs in the late-stage pipeline — which would make it a valu­able ad­di­tion to the port­fo­lio.

And then there’s MYS­TIC, As­traZeneca’s com­bo of its check­point Imfinzi (dur­val­um­ab) and treme­li­mum­ab, a CT­LA-4 drug. Over the past few months there’s been a grow­ing sense that this com­bi­na­tion could well floun­der on CT­LA-4’s well-known tox­i­c­i­ty. But As­traZeneca’s team is keep­ing their smiles in place, try­ing to as­sure every­one that this pro­gram re­mains on track, with plen­ty of po­ten­tial to im­press.

The da­ta are ex­pect­ed in a mat­ter of weeks now, so this mys­tery won’t play long. A suc­cess here would make a huge dif­fer­ence for the multi­na­tion­al, which has been dogged by clin­i­cal set­backs for years now. Some as­sur­ance that CEO Pas­cal So­ri­ot’s big bet on can­cer is pay­ing off with a multi­bil­lion-dol­lar re­turn would go a long way to as­sur­ing an­a­lysts that the long-await­ed turn­around is near.

Fail­ure would like­ly blight the process once again.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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The End­points 11: They've got mad mon­ey and huge am­bi­tions. It's time to go big or go home

These days, selecting a group of private biotechs for the Endpoints 11 spotlight begins with a sprint to get ahead of IPOs and the M&A teams at Big Pharma. I’ve had a couple of face plants earlier this year, watching some of the biotechs on my short list choose a quick leap onto Nasdaq or into the arms of a buyer.

Vividion, you would have been a great pick for the Endpoints 11. I’m sorry I missed you.

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Dave Lennon, former president of Novartis Gene Therapies

So what hap­pened with No­var­tis Gene Ther­a­pies? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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FDA+ roundup: Bs­U­FA III ready for show­time, court tells FDA to re-work com­pound­ing plan, new guid­ance up­dates and more

The FDA has now spelled out what exactly will be included in the third iteration of Biosimilar User Fee Act (BsUFA) from 2023 through 2027, which similarly to the prescription drug deal, sets fees that industry has to pay for submitting applications, in exchange for firm timelines that the agency must meet.

This latest deal includes several sweeteners for the biosimilar industry, which has yet to make great strides in the US market, with shorter review timelines for safety labeling updates and updates to add or remove an indication that does not contain efficacy data.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Maureen Hillenmeyer, Hexagon Bio CEO

Hexa­gon Bio rais­es $61M to con­tin­ue ef­forts to turn fun­gi in­to drugs

A year after raising a $47 million launch round, the fungi-loving drug hunters at Hexagon Bio have more than doubled their coffers.

Hexagon announced today that it raised another $61 million for its efforts to design cancer and infectious disease drugs based on insights mined from the DNA in millions of species of fungi. The new financing brings Hexagon’s committed funding to over $108 million.

Blue­bird sends blood dis­or­der drug to FDA for ap­proval; CG On­col­o­gy en­ters col­lab­o­ra­tion with Roche for Tecen­triq

Bluebird bio announced it completed the rolling submission of its BLA to the FDA for betibeglogene autotemcel gene therapy.

The therapy, designed for patients with beta-thalassemia who require regular red blood cell transfusions, was previously granted breakthrough therapy designation for treating transfusion-dependent beta-thalassemia (TDT). If approved, beti-cel will be the first hematopoietic stem cell ex-vivo gene therapy for patients in the US.

Joshua Liang, Clover Biopharmaceuticals CEO

With world still in sore need of dos­es, Clover says its Covid-19 vac­cine is 67% ef­fec­tive in Phase III

With concerns about the Delta variant rising and much of the world still in desperate need of vaccine doses, a Chinese biotech announced Wednesday that a new shot has shown positive results in a large trial against Covid-19, including new variants.

Clover Biopharmaceuticals announced Wednesday that its vaccine candidate showed 79% efficacy against the Delta variant in a Phase II/III trial dubbed Spectra, and 67% effective against Covid-19 overall.