Roche, Io­n­is her­ald a big win in ear­ly-stage Hunt­ing­ton’s dis­ease tri­al, demon­strate dis­ease mod­i­fy­ing po­ten­tial

Io­n­is $IONS and its part­ners at Roche have un­veiled the bio­mark­er da­ta from a Phase I/II study that re­cent­ly drove the phar­ma gi­ant to fol­low through with a $45 mil­lion pay­ment to op­tion rights to IO­N­IS-HTT Rx (RG6042).

Re­searchers said Thurs­day evening they tracked a mean re­duc­tion of 40% in mu­tant hunt­ingtin pro­tein at the two high­est dos­es of the ther­a­py. Some pa­tients saw up to 60% re­duc­tions. And that, they add, is a first for any Hunt­ing­ton’s drug. It’s that tox­ic pro­tein that lays waste to neu­rons in the brain, caus­ing a se­vere neu­rode­gen­er­a­tive dis­ease marked by de­clin­ing mus­cle co­or­di­na­tion and cog­ni­tive abil­i­ties, which typ­i­cal­ly slow­ly kills pa­tients over a course of 20 years.

If they’re right, the de­vel­op­ers are on track to the first po­ten­tial drug that could change the course of the dis­ease, though some steep hur­dles on safe­ty and ef­fi­ca­cy re­main to be cleared.

The re­search team add the dose de­pen­dent re­sponse demon­strat­ed that mu­tant pro­tein lev­els were still on the de­cline af­ter the last read­out, rais­ing ex­pec­ta­tions that they can do even bet­ter. An open-la­bel ex­ten­sion of the study is un­der­way. Roche has now tak­en con­trol of the drug and is re­spon­si­ble for mount­ing a piv­otal tri­al.

Sarah Tabrizi

Roche first signed on to work with Io­n­is on this drug back in 2013.

Io­n­is has been on an up­swing re­cent­ly, buoyed by roy­al­ties from Spin­raza. The biotech al­so has two drugs un­der re­view, in­clud­ing in­ot­ersen, which is wide­ly ex­pect­ed to get trounced on the mar­ket by Al­ny­lam’s ri­val patisir­an. Still, no one was hold­ing back on the cel­e­bra­tion to­day.

“For near­ly twen­ty years, I have seen many fam­i­lies dev­as­tat­ed from loss­es to this pro­gres­sive neu­rode­gen­er­a­tive dis­ease.  With IO­N­IS-HT­TRx (RG6042), the HD com­mu­ni­ty has new hope for a ther­a­py that can re­duce the cause of HD, and there­fore, may slow the pro­gres­sion and po­ten­tial­ly pre­vent the dis­ease in fu­ture gen­er­a­tions, which is tru­ly ground­break­ing,” said Dr Sarah Tabrizi, pro­fes­sor of clin­i­cal neu­rol­o­gy, di­rec­tor of the Uni­ver­si­ty Col­lege Lon­don’s Hunt­ing­ton’s Dis­ease Cen­tre and the glob­al lead in­ves­ti­ga­tor on the study. “I look for­ward to a longer-term, larg­er study that can es­tab­lish the ben­e­fit of re­duc­ing the tox­ic mu­tant hunt­ingtin pro­tein in peo­ple with HD.”

FDA hands Mor­phoSys and In­cyte a quick OK on their po­ten­tial block­buster CAR-T al­ter­na­tive

Nearly three years after okaying the CAR-Ts Yescarta and Kymriah, the FDA has approved a new CD19 therapy.

MorphoSys’ Monjuvi, or tafasitamab-cxix, was cleared Friday for use in refractory diffuse large B-cell lymphoma (DBLCL). The approval sets up both MorphoSys and their commercial partner Incyte to compete with Gilead and Novartis in the ultra-competitive indication, where similar trial results and far easier delivery could allow them to cut a fair share of the market.

CymaBay flash­es pos­i­tive re­sults from the tri­al they have to re­launch

Two weeks after the FDA lifted its clinical hold on their lead drug, CymaBay said it showed positive results in an aborted Phase III trial.

The drug, a small molecule known as seladelpar, had been in development for three different liver conditions before an independent review of a NASH study last year showed that it might actually be damaging patient’s liver cells. The FDA slapped a clinical hold across all three trials, only lifting it last month when an FDA review determined that the drug hadn’t caused liver damage.

So Covid-19 leader BioN­Tech has a can­cer vac­cine in de­vel­op­ment? Yes, and Re­gen­eron just jumped in for the PhII com­bo study

Before the coronavirus global emergency stole the R&D show in biopharma, the leaders in the race to develop new mRNA therapies had a big interest in determining if their tech could be used to create an effective cancer vaccine after all the first-gen tries had failed to impress. So perhaps it’s not surprising that an early cut of the data at frontrunner BioNTech went largely unnoticed.

Unless you were at Regeneron.

Endpoints Premium

Premium subscription required

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

Sanofi un­der for­mal in­ves­ti­ga­tion for De­pakine al­le­ga­tions; Beam li­cens­es CAR-T tech from Ox­ford Bio­med­ica

Sanofi is facing a formal investigation on manslaughter charges, due to accusations that its epilepsy drug Depakine caused birth malfunctions and slow neurological development when taken during pregnancy.

The French pharma was formally charged in February, years after evidence surfaced that the drug, sodium valproate, posed neurodevelopmental risks. Sodium valproate first hit the market in 1967 for the treatment of epilepsy and bipolar disorder, and is currently prescribed in more than 100 countries.

Rich Heyman (ARCH)

Rich Hey­man joins PMV Phar­ma, a p53 biotech, as it adds $70 mil­lion in Se­ries D

Less than a year after pulling in an impressive $62 million Series C round, PMV Pharma is back at it again.

The Cranbury, NJ-based biotech announced Monday an additional $70 million in Series D financing as it seeks to develop cancer therapies targeting p53 mutations. Additionally, PMV also introduced longtime biotech entrepreneur Rich Heyman as chairman of the board of directors.

“This financing provides PMV Pharma with the resources to expand our pipeline and to potentially advance multiple p53 therapies into the clinic,” said PMV president and CEO David Mack in a statement.

Days af­ter seal­ing Sanofi pact, Kymera beats a path to the Nas­daq with $100M IPO pitch

Back in March, when Kymera Therapeutics closed $102 million in Series C funding led by Biotechnology Value Fund and Redmile Group, CEO Nello Mainolfi noted the protein degradation player was “at the cusp of transitioning” into a fully integrated R&D company. Five months and a major Sanofi pact later, he’s back asking for another little push to get there.

Kymera has penciled in $100 million in its first IPO pitch — although given the public market’s seemingly insatiable appetite for biotechs these days the final figure is anyone’s guess.

Covid-19 roundup: Eli Lil­ly retro­fits RVs for first-of-its-kind an­ti­body tri­al with NIH; Am­gen, Ab­b­Vie, Take­da team on a drug

Eli Lilly and the NIH are about to start a first-of-its-kind trial that researchers and developers have talked about for months as a way of providing temporary immunity to the most at-risk populations.

Lilly announced this morning that it will start a 2,400-person trial with the National Institute for Allergy and Infectious Diseases to test whether its experimental Covid-19 neutralizing antibody can prevent people in nursing homes and assisted living facilities from developing the disease. The idea, known as passive immunity, is that rather than waiting on a vaccine to induce people to develop antibodies, doctors can give them lab-grown antibodies. Ideally, those antibodies will either attack the new SARS-CoV-2 infection, if the patient has recently been exposed, or persist in the blood for several weeks and prevent infection or disease for that period.

Im­mu­nic's lead MS drug hits pri­ma­ry and key sec­ondary end­points in PhII, but ques­tions re­main

Just a week after its lead program began enrolling patients in a study to treat Covid-19, Immunic Therapeutics is making more waves.

This time, the biotech is providing a glimpse at topline data from a Phase II trial studying the efficacy of vidofludimus calcium, or IMU-838, in relapsing-remitting multiple sclerosis patients. Taken orally, the candidate met its primary endpoint in reducing the cumulative number of combined unique active MRI lesions after 24 weeks for a 45 mg dose compared to a placebo, as well as a key secondary endpoint in such reductions for the 30 mg dose.

Sev­en plucky di­ag­nos­tics com­pa­nies win a $249M round of con­tracts af­ter sur­viv­ing NI­H's Covid-19 'Shark Tank' com­pe­ti­tion

As US Covid-19 deaths creep past 150,000 and officials stress the importance of contact tracing, the NIH’s Rapid Acceleration of Diagnostics (RADx) program has inked contracts totaling $248.7 million to expand testing capabilities.

The seven contracts, which were chosen “Shark Tank”-style from a pool of 100 proposals, are part of an effort to bump daily testing capacity to 2% of the country’s population by late summer or fall. That would be about 6 million people per day, compared to the current 520,000 to 823,000 tests being administered daily.