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.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.

FDA asks why No­var­tis took two months to launch for­mal in­ter­nal probe, af­ter AveX­is flagged da­ta ma­nip­u­la­tion

And the plot thickens. Novartis $NVS officials are reportedly now scrambling to explain to the FDA why it took them two months to open an internal investigation into data discrepancies for their $2.1 million gene-therapy for spinal muscular dystrophy — the world’s most expensive drug.

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Build­ing on suc­cess­ful PD-1 pact, Eli Lil­ly li­cens­es di­a­betes drug to Chi­nese part­ners at In­novent

Eli Lilly is expanding its partnership with China’s Innovent in a deal involving a diabetes drug sitting in its Phase I reserves.

The two companies had jointly developed one of China’s first homegrown PD-1 agents, scoring an approval for Tyvyt (sintilimab) late last year for relapsed/refractory classical Hodgkin’s lymphoma. This time around, Lilly is out-licensing a piece of its diabetes pipeline, a leading franchise that has historically produced the top-selling Trulicity and Humalog.

Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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