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.”

Com­mu­ni­cat­ing the val­ue of pre­ci­sion med­i­cine

By Natasha Cowan, Content Marketing Manager at Blue Latitude Health.
Many stakeholders are confused by novel precision medicines, including patients and healthcare professionals. So, how can industry help them to navigate this complexity?

Precision medicine represents a new paradigm in healthcare. It embodies the shift from treating many patients with the same therapy, to having the tools to identify the best treatment for every patient.

Spe­cial re­port: Twen­ty ex­tra­or­di­nary women in bio­phar­ma R&D who worked their way to the top

What differentiates a woman leader in biopharma R&D from a man?

Not much, except there are fewer of them in senior posts. Data suggest women are not more risk-averse, family-oriented or less confident than their male counterparts — indeed the differences between the two sexes are negligible. But a glance at the top R&D positions in Big Pharma leaves little doubt that upward migration in the executive ranks of biopharma R&D is tough.

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The lat­est Cin­derel­la sto­ry in on­col­o­gy ends with a sud­den rout as up­dat­ed da­ta dis­play spooks in­vestors

NextCure’s turn as the Cinderella of cancer-focused biotechs was short-lived.
Just a few days after its shares $NXTC zoomed up more than 250% on some very early stage results in a SITC abstract, a more complete analysis over the weekend spiked the hype and left investors in high dudgeon as the stock price collapsed back towards earth Monday.
The focus at NextCure is centered on NC318, an antibody that is intended to shut down the immunosuppressive Siglec-15 — or S-15 — target. After adding a small group of patients to the readout, investigators circled 2 clinical responses, a complete and partial response, along with 4 stable disease cases in non-small cell lung cancer.

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Te­va spin­out rais­es $85M in IPO; No­var­tis beefs up gener­ics unit with $440M deal

→ After Teva spinout 89bio recently announced that its IPO was being held up, the company is back in the game offering 5,304,687 shares at a price of $16 per share. The company has raised $84.9 million IPO in gross proceeds and will be listed under the ticker symbol $ETNB. BofA Securities, SVB Leerink and RBC Capital Markets are the joint book-running managers for the offering. Oppenheimer & Co is the co-manager for the offering.
→ Looking to amp up its presence in Japan’s hospitals, Novartis has struck a deal to buy out Aspen’s portfolio of generics in the world’s third largest healthcare market. The pharma giant is paying $440 million for Aspen’s Japanese subsidiary.
→ Novartis said tropifexor, a non-bile acid FXR agonist, has scored on several key biomarkers of NASH in a Phase IIb trial, including reductions in hepatic fat, alanine aminotransferase and body weight compared to a placebo at 12 weeks.

Break­through sta­tus and promise of a speedy re­view ar­rives for Op­di­vo/Yer­voy com­bi­na­tion as Bris­tol-My­ers bites at Bay­er

Its frontline and single-agent aspirations have been set back, but Bristol-Myers Squibb just took a big step forward in its efforts to apply its checkpoint inhibitor Opdivo to liver cancer. The FDA has granted breakthrough status and priority review to a combination, second-line treatment.

The designation is for Opdivo (nivolumab) in combination with Yervoy (ipilimumab),  for treating advanced hepatocellular carcinoma (HCC), the most common form of liver cancer. The PD-L1 drug was already approved as a single-agent, second-line treatment for HCC. A PDUFA date was set for March 10, 2020 — just 4 months from now.

Third time un­lucky: Lipocine's lat­est quest to mar­ket their oral testos­terone drug snubbed again by FDA

Lipocine’s latest attempt at securing approval for its oral testosterone drug has fizzled yet again.

The Utah-based drug developer on Monday said the FDA has spurned its marketing application, indicating that some efficacy data on the drug, Tlando, was not up to scratch to treat male hypogonadism, a condition characterized by low production of the hormone testosterone, which is responsible for maintaining muscle bulk, bone growth, and sexual function.

UP­DAT­ED: De­cry­ing 'ar­bi­trary and capri­cious' ac­tion, Re­genxBio sues FDA over clin­i­cal holds on gene ther­a­py

When RegenxBio disclosed that the FDA had placed a partial clinical hold on one of its lead gene therapies, execs outlined several customary next steps: continuing assessment and monitoring, delaying a related IND filing, and working with the FDA to address the matter.

As it turned out, they were planning something much less mundane. Two days after announcing the hold in its Q3 update, RegenxBio filed a lawsuit seeking to set it aside, the FDA Law Blog noted.

Roche's SMA chal­lenge to Bio­gen's Spin­raza fran­chise looms larg­er with piv­otal win

Roche has just landed a crucial advance in scoring a come-from-behind win on the spinal muscular atrophy field, giving Novartis and Biogen a run for their money.

The update was brief, but Roche said risdiplam hit the primary endpoint in the placebo-controlled pivotal SUNFISH trial, meeting the threshold for change from baseline in the Motor Function Measure 32 (MFM-32) scale after one year of treatment. The results, which is the second, confirmatory portion of a two-part study, involved 180 patients with type 2 or 3 spinal muscular atrophy between 2 and 25 years old.

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Roche steers Gazy­va in­to a new PhI­II pro­gram af­ter com­bo shows promise in lu­pus nephri­tis study

Roche is working on putting together a late-stage study for its monoclonal antibody Gazyva in patients with severe kidney disease associated with lupus after a combination approach helped patients in a mid-stage study.

The 125-patient NOBILITY trial evaluated Gazyva, combined with standard-of-care treatment mycophenolate mofetil or mycophenolic acid and corticosteroids, versus standard treatment alone. The combo met the main goal of inducing a statistically superior complete renal response (CRR) of 40% at week 76, versus 18% in patients given standard treatment, Roche said.