Van­da's PhII da­ta of­fer hope for gas­tro­pare­sis pa­tients who have seen no new treat­ments in decades

As one of the hand­ful of drugs-in-de­vel­op­ment for gas­tro­pare­sis, Van­da Phar­ma­ceu­ti­cals’ $VN­DA tradip­i­tant has set it­self apart for the con­di­tion that af­fects about 6 mil­lion in the Unit­ed States and has not seen an ap­proval in near­ly four decades. On Mon­day, the drug­mak­er re­leased mid-stage da­ta that im­pressed, show­ing the drug con­ferred a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in nau­sea symp­toms and nau­sea-free days.

Gas­tro­pare­sis is a chron­ic con­di­tion with few treat­ment op­tions that af­fects the nor­mal spon­ta­neous move­ment of the mus­cles in the stom­ach, which usu­al­ly pro­pel food through the di­ges­tive tract. In pa­tients af­flict­ed with the con­di­tion, these mus­cles work poor­ly or not at all, de­lay­ing or pre­vent­ing the stom­ach from emp­ty­ing, caus­ing nau­sea, vom­it­ing and is­sues with blood sug­ar lev­els and nu­tri­tion. The eti­ol­o­gy of gas­tro­pare­sis is un­clear, but the con­di­tion some­times presents as a com­pli­ca­tion of di­a­betes or surgery. The one FDA-ap­proved drug specif­i­cal­ly sanc­tioned for use in gas­tro­pare­sis is meto­clo­pramide, which car­ries a black box warn­ing that cau­tions against pre­scrib­ing for longer than 12 weeks.

Van­da’s drug, a neu­rokinin-1 re­cep­tor (NK-1R) an­tag­o­nist that was li­censed from Lil­ly $LLY in 2012, was test­ed against a place­bo in id­io­path­ic and di­a­bet­ic gas­tro­pare­sis pa­tients in the month long Phase II study. The drug met the main goal, as well as a num­ber of key sec­ondary end­points, and was well tol­er­at­ed with a safe­ty pro­file sim­i­lar to that of the place­bo.

“Tradip­i­tant could of­fer a much more tol­er­a­ble, chron­ic treat­ment, and giv­en the pauci­ty of pro­grams in de­vel­op­ment for this in­di­ca­tion (we count ~4 oth­ers), we be­lieve this as­set could gar­ner in­ter­est form po­ten­tial part­ners. We think this point will be­come a de­bate among in­vestors on whether VN­DA should part­ner this pro­gram based on the Phase 2 da­ta or pro­ceed in­to Phase 3, with the lat­ter be­ing the strat­e­gy cur­rent­ly be­ing pur­sued by the com­pa­ny,” Stifel an­a­lysts wrote in a note.

The tri­al hit the pri­ma­ry end­point of nau­sea im­prove­ment as mea­sured by pa­tient di­aries that rat­ed their symp­toms on a 0-5 scale. Pa­tients on tradip­i­tant re­port­ed a drop of 1.2 ver­sus a fall of 0.7 on place­bo (p=0.0099). For nau­sea-free days, an­oth­er crit­i­cal end­point, tradip­i­tant brought about a rough­ly 29% in­crease, com­pared to about 15% for pa­tients re­ceiv­ing place­bo. Im­prove­ments were al­so seen in most of the core gas­tro­pare­sis symp­toms in­clud­ing vom­it­ing, bloat­ing, and full­ness af­ter meals — most ef­fects were ap­par­ent by the sec­ond week of treat­ment al­though im­prove­ments con­tin­ued through the fourth and last week of treat­ment in the tradip­i­tant group, the com­pa­ny re­port­ed.

“Based on our pre­vi­ous KOL checks, a ≥1 im­prove­ment on nau­sea and ≥20% im­prove­ment on nau­sea free days was viewed as clin­i­cal­ly mean­ing­ful, and we be­lieve tradip­i­tant has met this hur­dle in the more se­vere pa­tients, which made up the ma­jor­i­ty of the study pop­u­la­tion. Im­por­tant­ly, there were im­prove­ments/no wors­en­ing on the oth­er core symp­toms of gas­tro­pare­sis, which is im­por­tant from an FDA per­spec­tive based on the agency’s 2015 draft guid­ance,” Stifel an­a­lysts added.

Tradip­i­tant is ex­pect­ed to rake in peak sales of 898 mil­lion, es­ti­mat­ed Jef­feries an­a­lysts. Mean­while, an­a­lysts at Stifel said they had raised their peak sales ex­pec­ta­tions from $535 mil­lion to $800 mil­lion, not­ing that their up­dat­ed fore­cast could still be con­ser­v­a­tive, “giv­en the size of the mar­ket and the fact over ~3-4 mil­lion scripts are writ­ten each year for meto­clo­pramide”.

The drug is cur­rent­ly al­so be­ing test­ed in a late-stage study in atopic der­mati­tis as­so­ci­at­ed chron­ic pru­ri­tus, af­ter a mid-stage study yield­ed mixed da­ta in the con­di­tion.

As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Ab­b­Vie wins an ap­proval in uter­ine fi­broid-as­so­ci­at­ed heavy bleed­ing. Are ri­vals My­ovant and Ob­sE­va far be­hind?

Women expel on average about 2 to 3 tablespoons of blood during their time of the month. But with uterine fibroids, heavy bleeding is typical — a third of a cup or more. Drugmakers have been working on oral therapies to try and stem the flow, and as expected, AbbVie and their partners at Neurocrine Biosciences are the first to make it across the finish line.

Known chemically as elagolix, the drug is already approved as a treatment for endometriosis under the brand name Orilissa. It targets the GnRH receptor to decrease the production of estrogen and progesterone.

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Fabrice Chouraqui, Cellarity CEO-partner (LinkedIn)

Drug de­vel­op­er, Big Phar­ma com­mer­cial ex­ec, now an up­start biotech chief — Fab­rice Chouraqui is ready to try some­thing new as a ‘CEO-part­ner’ at Flag­ship

Fabrice Chouraqui’s career has taken some big twists along his life journey. He got his PharmD at Université Paris Descartes and jumped into the drug development game for a bit. Then he took a sharp turn and went back to school to get his MBA at Insead before returning to pharma on the commercial side.

Twenty years later, after steadily rising through the ranks and journeying the globe to nab a top job as president of US pharma for the Basel-based Novartis, Chouraqui exited in another career switch. And now he’s headed into a hybrid position as a CEO-partner at Flagship, where he’ll take a shot at leading Cellarity — one of the VC’s latest paradigm-changing companies of the groundbreaking model that aspires to deliver a new platform to the world of drug R&D.

David Chang, Allogene CEO (Jeff Rumans)

Head­ed to PhII: Al­lo­gene CEO David Chang com­pletes a pos­i­tive ear­ly snap­shot of their off-the-shelf CAR-T pi­o­neer

Allogene CEO David Chang has completed the upbeat first portrait of the biotech’s off-the-shelf CAR-T contender ALLO-501 at virtual ASCO today, keeping all eyes on a drug that will now try to go on to replace the first-wave personalized pioneers he helped create.

The overall response rate outlined in Allogene’s abstract for treatment-resistant patients with non-Hodgkin lymphoma slipped a little from the leadup, but if you narrow the patient profile to treatment-naïve patients — removing the 3 who had previous CAR-T therapy who didn’t respond, leaving 16 — the ORR lands at 75% with a 44% complete response rate. And 9 of the 12 responders remained in response at the data cutoff, offering a glimpse on durability that still has a long way to go before it can be completely nailed down.

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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Bryan Roberts, Venrock

Ven­rock sur­vey shows grow­ing recog­ni­tion of coro­n­avirus toll, wan­ing con­fi­dence in ar­rival of vac­cines and treat­ments

When Venrock partner Bryan Roberts went to check the results from their annual survey of healthcare leaders, what he found was an imprint of the pandemic’s slow arrival in America.

The venture firm had sent their form out to hundreds of insurance and health tech executives, investors, officials and academics on February 24 and gave them two weeks to fill it out. No Americans had died at that point but the coronavirus had become enough of a global crisis that they included two questions about the virus, including “Total U.S. deaths in 2020 from the novel coronavirus will be:”.

As­traZeneca’s $7B ADC suc­ceeds where Roche failed, im­prov­ing sur­vival in gas­tric can­cer

Another day, another win for Enhertu.

The antibody-drug conjugate AstraZeneca promised up-to $7 billion to partner on has had a quite a few months, beginning with splashy results in a Phase II breast cancer trial, a rapid approval and, earlier this month, breakthrough designations in both non-small cell lung cancer and gastric cancer.

Now, at ASCO, the British pharma and their Japanese partner, Daiichi Sankyo, have shown off the data that led to the gastric cancer designation, which they’ll take back to the FDA. In a pivotal, 187-person Phase II trial, Enhertu shrunk tumors in 42.9% of third-line patients with HER2-positive stomach cancer, compared with 12.5% in a control arm where doctors prescribed their choice of therapy. Progression-free survival was 5.4 months for Enhertu compared to 3.5 months for the control.