Silviu Itescu, Mesoblast CEO

Mesoblast shares tank af­ter No­var­tis aban­dons a part­ner­ship for the biotech's po­ten­tial Covid-19 treat­ment

No­var­tis of­fered up $25 mil­lion in cash last No­vem­ber for the rights to Mesoblast’s po­ten­tial cell ther­a­py for Covid-19. But af­ter a Phase III flunk, the Big Phar­ma’s now get­ting cold feet — and Mesoblast’s stock is suf­fer­ing big time.

Mesoblast shares $MESO tanked more than 17% on Tues­day morn­ing af­ter an­nounc­ing that No­var­tis has walked out on its part­ner­ship for remestem­cel-L. And be­cause the ter­mi­na­tion oc­curred be­fore clos­ing, Mesoblast missed out on up­front pay­ments of $25 mil­lion cash and a $25 mil­lion eq­ui­ty in­vest­ment.

The rea­son? Sub­par da­ta, ac­cord­ing to No­var­tis.

“Af­ter as­sess­ing ad­di­tion­al da­ta, the to­tal­i­ty of ev­i­dence from the Phase III tri­al is not suf­fi­cient to con­tin­ue with the col­lab­o­ra­tion agree­ment. No safe­ty con­cerns have been iden­ti­fied,” the com­pa­ny said in a state­ment to End­points News. 

No­var­tis was drawn to remestem­cel-L af­ter an open-la­bel com­pas­sion­ate use pro­gram sug­gest­ed an 83% sur­vival rate in pa­tients on ven­ti­la­tors who were treat­ed with the ex­per­i­men­tal cell ther­a­py last March. Then in De­cem­ber, Mesoblast cut a 300-per­son Phase III tri­al short af­ter a da­ta safe­ty mon­i­tor­ing board con­clud­ed the ther­a­py was “not like­ly” to reach its pri­ma­ry end­point, a 43% re­duc­tion in mor­tal­i­ty at 30 days.

That Oc­to­ber, the FDA is­sued a com­plete re­sponse let­ter for the same can­di­date in a dif­fer­ent in­di­ca­tion, pe­di­atric acute graft-ver­sus-host dis­ease, over is­sues with tri­al de­sign. Mesoblast had sub­mit­ted its ap­pli­ca­tion on the ba­sis of one sin­gle-arm, open-la­bel study rather than a ran­dom­ized tri­al, even though the drug demon­strat­ed a sta­tis­ti­cal­ly sig­nif­i­cant ben­e­fit in its pri­ma­ry end­point against the his­tor­i­cal con­trol rate.

The com­pa­ny al­so suf­fered a miss for its sec­ond can­di­date, rexleme­stro­cel-L, in heart fail­ure last De­cem­ber. While the ex­per­i­men­tal cell ther­a­py did not sig­nif­i­cant­ly re­duce hos­pi­tal­iza­tions over place­bo in in­di­vid­u­als with re­cur­ring heart fail­ure, Mesoblast says the pro­gram man­aged to hit sec­on­daries in mor­tal­i­ty-rate re­duc­tion for ear­li­er stages of the dis­ease.

US in­vestor group Surg­Cen­ter De­vel­op­ment threw Mesoblast a life­line in March, lead­ing a $110 mil­lion pri­vate place­ment.

De­spite the set­backs, Mesoblast still thinks there’s a path for­ward for remestem­cel-L in acute res­pi­ra­to­ry dis­tress syn­drome due to Covid-19.

“The ob­served mor­tal­i­ty re­duc­tion with remestem­cel-L in pa­tients aged un­der 65 in the com­plet­ed COVID ARDS tri­al, de­spite hav­ing missed the pri­ma­ry end­point, is con­sid­ered by Mesoblast to be a suf­fi­cient­ly strong sig­nal to sup­port pur­su­ing an emer­gency use au­tho­riza­tion (EUA), the most di­rect path to mar­ket,” the com­pa­ny said in a state­ment.

Mesoblast was un­avail­able for com­ment as of press time, but End­points will up­date the sto­ry as it de­vel­ops. The com­pa­ny said in a state­ment that it’s prepar­ing to ini­ti­ate an­oth­er Phase III tri­al that may sup­port an EUA in Covid-as­so­ci­at­ed ARDS.

“Vari­ants in­clud­ing Omi­cron present a grow­ing threat due to in­creased in­fec­tiv­i­ty and im­mune eva­sion from vac­cines and mon­o­clon­al an­ti­bod­ies, in­creas­ing the ur­gent need for ther­a­peu­tics to pre­vent the like­ly high mor­tal­i­ty of those pro­gress­ing to ICU and ARDS,” the com­pa­ny said.

For a look at all End­points News coro­n­avirus sto­ries, check out our spe­cial news chan­nel.

Jean-Paul Clozel, Idorsia CEO (Patrick Straub/Keystone via AP Images)

Idor­si­a's brain bleed drug flunks PhI­II tri­al, a decade af­ter pre­vi­ous flop

Idorsia’s long journey with clazosentan came to an abrupt “unexpected result” Monday morning with a Phase III flop.

The Swiss biopharma said the drug did not meet the main goal of the late-stage REACT study, conducted in the US, Canada and Europe since early 2019.

The 409-patient trial tested the intravenous drug’s ability to prevent complications due to delayed cerebral ischemia following aneurysmal subarachnoid hemorrhage (aSAH), in which blood vessels in the brain narrow and blood accumulates around the brain’s surface, which then dials up the pressure on the brain.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,600+ biopharma pros reading Endpoints daily — and it's free.

Kenji Yasukawa, Astellas CEO (Photographer: Akio Kon/Bloomberg via Getty Images)

Astel­las taps chief strat­e­gy of­fi­cer as next CEO to 'go on the ag­gres­sive'

Five years into its big R&D revamp, Astellas says it’s time for a changing of the guard.

Kenji Yasukawa, who took over as president and CEO in 2018, will step down to become chairman of the board in April, making room for Naoki Okamura to take over. Okamura joined the company in 1986 and has served in a variety of finance, business and strategy roles, including most recently as chief strategy officer.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,600+ biopharma pros reading Endpoints daily — and it's free.

Clin­i­cal tri­al di­ver­si­ty da­ta show mis­match be­tween en­roll­ment and dis­ease preva­lence, GSK says

A lack of diversity in clinical trials has persisted despite decades of initiatives to try to turn the tide.

In a recent review of 17 years of clinical trials, drugmaker GSK found that there were some mismatches between the demographics of its US-based trials and how prevalent diseases were in those populations.

The results, the company says, will help GSK and others design studies that better represent epidemiological rates within races and ethnicities.

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; 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.

With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,600+ biopharma pros reading Endpoints daily — and it's free.

Goldfinch Bio CEO Tony Johnson (L) and Karuna Therapeutics CEO Bill Meury

Karuna li­cens­es Goldfinch as­sets to com­pete with Boehringer In­gel­heim in neu­ro­science

Karuna Therapeutics is looking to compete with Boehringer Ingelheim on depression and anxiety with a new license to Goldfinch Bio’s assets, starting with $15 million to the shuttered biotech.

Karuna steps into an arena already being tested by Boehringer in multiple Phase II studies — the two are targeting transient receptor potential canonical 4 and 5, or TRPC4/5, which is thought to have a role in neuroscience indications. Goldfinch’s asset went through a Phase II in kidney diseases, but Karuna’s sights are set on mood and anxiety disorders for now.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,600+ biopharma pros reading Endpoints daily — and it's free.

Photo: Julia Weeks/AP Images

FDA ax­es re­quire­ment for pos­i­tive Covid test be­fore Paxlovid use

FDA announced today that doctors and pharmacists can now prescribe Paxlovid to patients without a positive test for Covid-19.

CDER Director Patrizia Cavazzoni reissued Paxlovid’s authorization letter Wednesday, saying it has revised the authorization to “no longer require positive results of direct SARS-CoV-2 viral testing.” The EUA now requires instead that adults and kids 12 years of age and older have a “current diagnosis of mild-to-moderate COVID-19.”

Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,600+ biopharma pros reading Endpoints daily — and it's free.

Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.

Raymond Stevens, Structure Therapeutics CEO

Be­hind Fri­day's $161M IPO: A star sci­en­tist, GPCR drug dis­cov­ery and a plan to chal­lenge phar­ma in di­a­betes

What does it take to pull off a $161 million biotech IPO these days?

In Structure Therapeutics’ case, it means having a star scientist co-founder paired with the computational drug discovery company Schrödinger, $198 million in private funding from blue-chip investors, almost six years of research work on G protein-coupled receptors and a slate of oral, small-molecule drugs, with an eye on the huge and growing diabetes and weight-loss market.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,600+ biopharma pros reading Endpoints daily — and it's free.