Medexus re­sub­mits NDA as it still looks to re­pur­pose a decades-old drug

The Ger­man drug­mak­er medac’s US sub­sidiary is hop­ing the sec­ond time is the charm for the com­pa­ny, as it is of­fi­cial­ly ask­ing the FDA once again to ap­prove its can­cer drug.

Medexus Phar­ma­ceu­ti­cals re­sub­mit­ted its re­quest for ap­proval of tre­o­sul­fan to be tak­en in com­bi­na­tion with flu­dara­bine, the com­pa­ny said Mon­day. The treat­ment is de­signed as a reg­i­men for al­lo­gene­ic hematopoi­et­ic stem cell trans­plan­ta­tion.

Medexus’ sub­mis­sion in­cludes up­dat­ed da­ta and a re­ex­am­i­na­tion of its Phase III tri­al as per the FDA’s re­quest. The re­sub­mis­sion has been in the works by Medexus and medac since April.

Its Phase III da­ta pub­lished in The Lancet showed that tre­o­sul­fan was no worse than busul­fan. The tri­al even showed a small, al­beit not sta­tis­ti­cal­ly sig­nif­i­cant ben­e­fit for tre­o­sul­fan: 64% of pa­tients who re­ceived medac’s drug went two years with­out re­lapse or death, com­pared to 54% of pa­tients who re­ceived busul­fan.

Ken d’En­tremont

“We are en­cour­aged by the re­cent pub­li­ca­tion of the fi­nal study re­sults and analy­sis of the piv­otal phase 3 clin­i­cal tri­al of tre­o­sul­fan con­duct­ed by Medac, which met its pri­ma­ry end­point and key sec­ondary end­points. An FDA ap­proval with­in a two- to six-month pe­ri­od from the ac­cep­tance date would then pave the way for a com­mer­cial launch of tre­o­sul­fan in the Unit­ed States in the first half of cal­en­dar year 2023,” said Medexus CEO Ken d’En­tremont in a state­ment.

While the FDA did not specif­i­cal­ly dis­close its orig­i­nal ob­jec­tions, on­ly that more da­ta and analy­sis were need­ed, the hit was a sur­prise to the com­pa­ny. Tre­o­sul­fan was ap­proved by Health Cana­da last year, and com­mer­cial­ly launched lat­er the same year. The EMA al­so ap­proved the drug in 2019.

How­ev­er, the news did see its share price drop by 37% in 2021. The shares now hov­er above the pen­ny stock zone at $1.46 — a 30% drop since Jan­u­ary.

Tre­o­sul­fan has been around in some form for sev­er­al decades at this point. Re­search in­to the com­pound’s po­ten­tial to thwart can­cer be­gan al­most 60 years ago and test­ing in hu­mans be­gan in 1968. With tri­al fits and starts over the decades, the drug nev­er be­came used wide­ly but did win ap­proval to treat ovar­i­an can­cer.

In the 2000s, medac be­gan de­vel­op­ing the drug as a con­di­tion­ing agent, stat­ing that it could prove less tox­ic to pa­tients than the wide­ly-used con­di­tion­ing agent busul­fan.

UP­DAT­ED: In a fresh dis­ap­point­ment, Am­gen spot­lights a ma­jor safe­ty is­sue with KRAS com­bo

Amgen had hoped that its latest study matching its landmark KRAS G12C drug Lumakras with checkpoint inhibitors would open up its treatment horizons and expand its commercial potential. Instead, the combo spurred safety issues that blunted efficacy and forced the pharma giant to alter course on its treatment strategy, once again disappointing analysts who have been tracking the drug’s faltering sales and limited therapeutic reach.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,200+ biopharma pros reading Endpoints daily — and it's free.

Ad­dress­ing the ‘Ca­pac­i­ty Crunch’ with a Scal­able Plat­form Process Ap­proach

The field of gene therapy has been diligently moving forward over the past several decades to bring potentially life-saving treatments to patients with genetic diseases. In addition to two approved adeno-associated viral (AAV) gene therapies, there are more than 250 AAV gene therapies in various clinical trial stages.1 AAV vectors remain the most frequently used vector for delivering therapeutic transgenes to target tissues due to their demonstrated and lasting clinical efficacy and extensive safety track record. As AAV therapies advance through clinical trials and into commercialization, many biotech companies are turning to contract development and manufacturing organizations (CDMOs) to prepare their programs for late-stage clinical and commercial scale manufacturing. Given the scope and scale of the manufacturing needs that will accompany regulatory approvals for these assets, CDMOs continue to expand their capacity to meet the needs of increasing prevalent patient populations. However, despite rapid growth, projected gene therapy manufacturing demands still outpace the collective capacity of the CDMO industry.

A $5B Pfiz­er buy­out? Am­gen, Gilead head­line M&A Thurs­day; Al­ny­lam's AT­TR sweep; An­drew Lo's rare dis­ease quest; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

One of the cool things about adding EndpointsPharma to the daily roster is that my colleagues can now dedicate time to tracking quarterly updates and tuning into calls with Big Pharma companies. Check out their dispatch from the Q2 earnings below.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,200+ biopharma pros reading Endpoints daily — and it's free.

George Yancopoulos, Regeneron president and CSO (Brendan McDermid/Reuters/Alamy)

George Yan­copou­los says he's on the trail of the holy grail: ‘This could rep­re­sent the next break­through for im­munother­a­py’

Two of the most outspoken — and successful — drug developers in biotech say they’ve collected early-stage clinical data that are pointing them down the trail to the holy grail in cancer immunotherapy R&D.

While analysts largely busied themselves today with chronicling the ongoing success of Regeneron’s two big cash cows — Dupixent and Eylea — chief scientist George Yancopoulos and CEO Len Schleifer used the Q2 call to spotlight their early success with a combination of the “homegrown” PSMAxCD28 costimulatory bispecific antibody REGN5678 in combination with their PD-1 checkpoint Libtayo. The presentation comes just weeks after Regeneron completed a deal to gather all rights to the PD-1 that had been in Sanofi’s hands. And the two top execs are unstinting in their praise of the potential of a whole set of costimulatory pipeline projects which they say may finally deliver the long-awaited next-level approach to broadening the immunotherapy field of drugs.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Albert Bourla, Pfizer CEO (Laurent Gillieron/Keystone via AP)

Break­ing: Pfiz­er in hot pur­suit of a $5B buy­out of Glob­al Blood Ther­a­peu­tics — re­port

Pfizer CEO Albert Bourla has vowed to leave no stone unturned in the search for new biotech deals, and the BD team is not letting him down.

The Wall Street Journal reported today that Pfizer is in the final stages of acquiring Global Blood Therapeutics for $5 billion. According to the Journal report, though, Pfizer is not the only buyer at the deal table and while the pharma giant may be close to clinching it, there are no guarantees it will continue.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,200+ biopharma pros reading Endpoints daily — and it's free.

Bob Bradway, Amgen CEO (Justin Kase Conder/AP Images for Amgen)

UP­DAT­ED: Am­gen chief Brad­way nabs a rare dis­ease play­er in $4B buy­out as the M&A tem­po ac­cel­er­ates

Amgen CEO Bob Bradway is bellying up to the M&A table today, scooping up the newly anointed commercial biotech ChemoCentryx $CCXI and its recently approved rare disease drug for $3.7 billion out of the cash stockpile. The deal comes in at $52 a share — a hefty increase over the $24.11 close yesterday.

Bradway and the Amgen team get a drug called Tavneos (avacopan) in the deal, a complement factor C5a inhibitor OK’d to treat anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis, an autoimmune disease which can be lethal.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,200+ biopharma pros reading Endpoints daily — and it's free.

Sen. Kyrsten Sinema (D-AZ) (Tom Williams/CQ Roll Call via AP Images)

De­moc­rats se­cure sup­port from key sen­a­tor ahead of po­ten­tial drug pric­ing vote

Senate Democrats may have all the votes they need to pass major drug pricing reform, after Sen. Kyrsten Sinema (D-AZ) reportedly pledged her support on Thursday — but will they fit it in before recess?

Sinema said she has agreed to “move forward” with the reconciliation bill with some stipulations, including the removal of a carried tax provision, according to recent reports. The bill is still expected to reduce the deficit by $300 billion, and Sen. Chuck Schumer (D-NY) said that he now anticipates “support from the entire Senate Democratic conference,” the Washington Post reported. 

(AP Photo/Richard Vogel, File)

US de­clares mon­key­pox a na­tion­al health emer­gency, as new drug­mak­ers con­sid­er en­ter­ing vac­cine race

Rising monkeypox cases have put the US on high alert as it announces a national health emergency, which grants the government more power in its response.

The news comes as Bavarian Nordic continues to fill orders for its Jynneos vaccine and other companies – including Moderna – consider jumping into the vaccine race. Meanwhile, the New York Times reports that the US has allowed around 20 million doses of smallpox vaccine in its stockpile to expire.

Vlad Coric, Biohaven CEO

Bio­haven touts surge in Nurtec sales ahead of Pfiz­er takeover

Forget buyer’s remorse, Pfizer is likely feeling pretty good about its $11.6 billion Biohaven takeover deal following reports of a 57% sales boost for migraine med Nurtec.

Biohaven reported in Q2 results on Friday that it’s cleared the necessary antitrust hurdles to move forward with the sale of its calcitonin gene-related peptide (CGRP) assets to Pfizer. However, because the company is “focused on workstreams related to the closing” of the deal, it did not host a call with analysts and investors.