Ver­tex claims suc­cess on its non-opi­oid pain drug, push­ing can­di­date in­to piv­otal tri­als

Ver­tex may soon have three irons in the fire in its bid to re­peat the sci­en­tif­ic and fi­nan­cial suc­cess of its block­buster cys­tic fi­bro­sis med­i­cines.

The com­pa­ny on Thurs­day re­leased pos­i­tive re­sults from a pair of Phase II tri­als for its non-opi­oid pro­gram, an­nounc­ing that the ex­per­i­men­tal pill sig­nif­i­cant­ly re­duced pa­tients’ pain com­pared to place­bo in two post-op­er­a­tive tri­als. One tri­al looked at pa­tients who had just re­ceived a bunionec­to­my, the oth­er in pa­tients who re­ceived ab­domino­plas­ty — the med­ical term for a “tum­my tuck.”

Now, Ver­tex ex­ec­u­tives said they will push the drug in­to piv­otal tri­als lat­er this year. That will like­ly make it the third pro­gram in the Ver­tex pipeline to move in­to Phase III tri­als, af­ter its sick­le cell and be­ta-tha­lassemia gene ther­a­py and a treat­ment for ge­net­i­cal­ly dri­ven kid­ney dis­ease.

Al­though none have quite the sales po­ten­tial of CF, Ver­tex ar­gues they can each be cu­ra­tive or dis­ease-mod­i­fy­ing in ar­eas — like non-opi­oid pain re­lief — where progress has his­tor­i­cal­ly been slow.

“Our high ex­pec­ta­tions of achiev­ing ther­a­peu­tic pain con­trol … have been met with these re­sults from the two acute pain stud­ies,” said Ver­tex CMO Car­men Boz­ic. “The re­mark­able con­sis­ten­cy in the safe­ty, tol­er­a­bil­i­ty and ef­fi­ca­cy re­sults in these two stud­ies demon­strates the po­ten­tial of VX-548 to be a first-in-class non-opi­oid treat­ment for acute pain.”

Pain, how­ev­er, is a no­to­ri­ous­ly dif­fi­cult area for drug de­vel­op­ment, in part be­cause of the pro­found place­bo re­sponse many pa­tients can ex­pe­ri­ence. And the Phase II tri­als left at least some ques­tion about how well it will per­form in Phase III tri­als.

The com­pa­ny said that with­in 48 hours of surgery, bunionec­to­my pa­tients who re­ceived the high dose of the Ver­tex pill ex­pe­ri­enced sig­nif­i­cant­ly less pain than pa­tients who re­ceived place­bo. The same was true on tum­my tuck pa­tients in the high and mid-lev­el dose, when com­pared to tum­my tuck pa­tients on place­bo.

But one clear sign re­searchers look for in in­ter­pret­ing ear­ly stud­ies is whether there’s a dose re­sponse: Do pa­tients ex­pe­ri­ence more ben­e­fit as the dose in­creas­es?

In the bunionec­to­my tri­al, there wasn’t. Bunionec­to­my pa­tients who re­ceived the mid-lev­el dose ac­tu­al­ly ex­pe­ri­enced more pain than those on place­bo. And bunionec­to­my pa­tients who re­ceived the low­est dose saw some im­prove­ment over place­bo, al­though it was not sta­tis­ti­cal­ly sig­nif­i­cant.

At the same time, few com­pa­nies have two sta­tis­ti­cal­ly sig­nif­i­cant tri­als be­fore en­ter­ing Phase III tri­als. And it marks a mile­stone in a more than decades-long in­dus­try-wide ef­fort to de­vel­op al­ter­na­tives to opi­oids by tar­get­ing nerve re­cep­tors called Nav1.8 or Nav1.7 — a place where Pfiz­er, Roche and Bio­gen have all failed.

While there are nu­ances that im­ply some out­stand­ing clin­i­cal risk (i.e. pain is hard, on­ly the high dose sep­a­rat­ed), ul­ti­mate­ly we feel con­fi­dent say­ing this mech­a­nism works,” Stifel’s Paul Mat­teis wrote in a note to in­vestors.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Dave Marek, Myovant CEO

My­ovant board balks as ma­jor­i­ty own­er Sum­it­o­mo swoops in with a $2.5B deal to buy them out

Three years after Sumitomo scooped up Roivant’s 46% stake in the publicly traded Myovant $MYOV as part of a 5-company, $3 billion deal, they’re coming back for the whole thing.

But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage.

Sumitomo is bidding $22.75 a share for the outstanding stock, which now represents 48% of the company after Sumitomo bumped its ownership since the original deal with Roivant. Myovant, however, created a special committee on the board, and they’re shaking their heads over the offer.

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Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Rob Etherington, Clene CEO

Star­tup's gold nanocrys­tal ALS drug flops a PhII tri­al, a re­minder of the dis­ease's ob­sta­cles de­spite Amy­lyx OK

Despite the FDA approving an ALS drug for the first time in five years last week, the disease continues to fluster researchers, and another biotech is feeling the pain of a mid-stage failure.

Clene Nanomedicine reported early Monday that its ALS program, which uses gold nanocrystals to try to catalyze intracellular reactions, did not achieve its Phase II primary or secondary endpoints. And in a press release, the company noted for the first time that it’s speaking with “potential strategic partners” about the program — language that typically indicates a biotech is preparing to sell off an asset.

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Albert Bourla, Pfizer CEO (Gian Ehrenzeller/Keystone via AP)

Can a smart­phone app de­tect Covid? Pfiz­er throws down $116M to find out

What can a cough say about a patient’s illness? Quite a bit, according to ResApp Health — and Pfizer’s listening.

The pharma giant is shelling out about $116 million ($179 million AUD) to scoop up the University of Queensland spinout and its smartphone technology that promises to diagnose Covid and other respiratory illnesses based on cough and breathing sounds, the university announced last week.

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Big Phar­ma heavy­weights seek tweaks to FDA's clin­i­cal out­come as­sess­ment guid­ance

Pfizer, GSK, Janssen, Regeneron, Boehringer Ingelheim and at least a half dozen other companies are calling on the FDA to provide significantly more clarity in its draft guidance from this summer on clinical outcome assessments, which are a type of patient experience.

The draft is the third in a series of four patient-focused drug development guidance documents that the FDA had to create as part of the 21st Century Cures Act, and they describe how stakeholders (patients, caregivers, researchers, medical product developers and others) can collect and submit patient experience data and other relevant information for medical product development and regulatory decision-making.

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Silviu Itescu, Mesoblast CEO

Mesoblast sends in im­proved po­ten­cy as­say, look­ing to re­sub­mit to FDA on acute graft-ver­sus-host dis­ease drug

In 2020, the FDA rejected Mesoblast’s remestemcel-L, or Ryoncil, its lead candidate for pediatric acute graft-versus-host disease (aGVHD) that didn’t respond to steroids. The FDA raised a number of concerns, first objecting to Mesoblast’s single arm, open-label trial, though regulators struggled to describe how a randomized trial would work, since pediatricians and parents were reluctant to put children with aGVHD in a placebo arm.

Johnny Stilou, Scandion Oncology acting CEO

Scan­dion's shares fall af­ter on­col­o­gy biotech re­ports PhII fail

Danish biotech Scandion Oncology posted some Phase II results on Friday, and investors were none too pleased.

The biotech reported topline results from the second part of an ongoing Phase II trial called CORIST. The study was investigating Scandion’s lead candidate SCO-101 in 25 patients as a combination treatment with FOLFIRI chemotherapy in metastatic colorectal cancer. And so far, the study did not meet the biotech’s primary endpoint: tumor reduction of at least 30%.

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Marc Dunoyer, Alexion CEO (AstraZeneca via YouTube)

Up­dat­ed: As­traZeneca nabs a small rare dis­ease gene ther­a­py play­er for 667% pre­mi­um

AstraZeneca is kicking off the fourth quarter with a little M&A Monday for a gene editing player recently overcoming a second clinical hold to its only program in human studies.

The Big Pharma and its subsidiary Alexion are buying out little LogicBio for $2.07 per share. That’s good for a massive 667% premium over its Friday closing price, when it headed into the weekend at 27 cents and just weeks after Nasdaq said LogicBio would have to delist, which has been put on hold as the biotech requests a hearing. It’s one of two biotech deals to commence October, alongside the news of Incyte buying a vitiligo-focused biotech.

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