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.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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Sen­ate Fi­nance Com­mit­tee lobs more bi­par­ti­san pres­sure on­to PBMs

Congress is honing in on how it wants to overhaul the rules of the road for pharmacy benefit managers, with a Senate Finance Committee hearing Thursday serving as the latest example of the Hill’s readiness to make changes to how pharma middlemen operate.

While pledging to ensure patients and pharmacies “don’t get a raw deal,” Finance Committee Chair Ron Wyden (D-OR) laid out the beginning of what looks like a major bipartisan effort — moves the PBM industry is likely to challenge vigorously.

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Nicklas Westerholm, Egetis Therapeutics CEO

Ac­qui­si­tion talks on­go­ing for Swedish rare dis­ease biotech Egetis, shares up al­most 40%

Shares of the Sweden-based rare disease biotech Egetis Therapeutics skyrocketed on Thursday afternoon as the company said it’s engaged in “ongoing discussion” with external parties regarding a “potential acquisition.”

Egetis confirmed rumors with a statement on Thursday while noting that there is no certainty that a takeover offer will be made.

Nonetheless, the possibility of an acquisition has shot up Egetis’ share price. By the afternoon on Thursday, its stock price was {$EGTX.ST} up over 38%. An Egetis spokesperson told Endpoints News in an email that it has no further comments.

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Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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J&J bows out of RSV vac­cine race, end­ing PhI­II study and ced­ing to Pfiz­er, GSK

Johnson & Johnson announced Wednesday morning it is ending development of its adult RSV vaccine that was in the middle of a 27,200-patient trial, giving up a big slice of what’s expected to be the next multibillion-dollar pharma market.

The decision came down to the shifting RSV “competitive landscape,” a company spokesperson tells Endpoints News, adding the “breadth of options” was much different than when J&J first started its pivotal study. The spokesperson declined to comment on the Phase III data, saying only the shot is undergoing an “ongoing assessment.”

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No longer ‘dead or just hi­ber­nat­ing,’ drug­mak­ers re­turn to heart med­i­cines

In 2015, now-FDA Commissioner Robert Califf joined industry, academic and regulatory representatives in Washington to discuss why more drugs weren’t in development for cardiovascular diseases, the leading US cause of death and once a mainstay of pharmaceutical industry blockbusters.

The group pointed to many reasons. Clinical trials could take years and testing was expensive. Wide availability of generic drugs made the commercial prospects uncertain. Their paper title summed up the mood: “Cardiovascular Drug Development: Is it Dead or Just Hibernating?”

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Lu­pus drug de­vel­op­ment mar­ket heat­ing up, while FDA links with ad­vo­ca­cy group to fur­ther ac­cel­er­ate re­search

The long-underserved systemic lupus erythematosus (SLE) market is suddenly buzzing with treatment possibilities. Less than two years after AstraZeneca’s approval for Saphnelo — the first new SLE drug in a decade and joining just one other approved in GSK’s Benlysta – the pipeline of potential drugs numbers in the dozens.

Although most are very early stage — Spherix Global Insights estimates five in Phase II/III — the pharma R&D enthusiasm is catching on among doctors, patients and advocacy groups. On Wednesday, the Lupus Research Alliance and the FDA formed a novel private-public partnership called Lupus Accelerating Breakthroughs Consortium (Lupus ABC) to help advance lupus clinical trial success.

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Af­ter safe­ty re­view, EMA mir­rors FDA with up­dat­ed rec­om­men­da­tions for JAK in­hibitors

The EMA released updated recommendations today for the use of JAK inhibitors (JAKi) after reviewing data from several clinical trials that showed increased incidents of issues in certain patients who have rheumatoid arthritis and other risk factors.

The EMA noted malignancy, major adverse cardiovascular events (MACE), serious infections, venous thromboembolism (VTE) and mortality in some patients.