Roche Hemlibra breaks into wider hemophilia market with blockbuster OK

Roche’s Hemlibra officially has the hemophilia indication it’s been waiting for—and that its competitors have been dreading.

On Thursday, the FDA approved the drug to treat hemophilia A patients without factor VIII inhibitors, putting a significantly larger group of patients in line for Hemlibra therapy. Previously, the drug was cleared only in patients with inhibitors, which develop as patients use factor VIII replacement drugs to prevent bleeding.

Now that the drug has a green light in all U.S. hemophilia A patients—who number between 10,000 and 12,000, according to estimates provided by Roche—analysts expect its sales to soar. Industry watchers predict Hemlibra could rack up $2 billion by 2025, though some analysts have said that number could climb far higher. Jefferies analysts, for their part, have pegged 2025 sales at $5 billion.

One reason? Efficacy. In a phase 3 study in hemophilia A patients without factor VIII inhibitors, Hemlibra reduced bleeding incidents that needed treatment by 96% compared with no preventive therapy, Roche said in May. Hemlibra also boasts a convenience advantage: It can be administered subcutaneously every week, two weeks or four weeks.

But Hemlibra’s prospects don’t bode well for Shire, which entered the hemophilia sphere with its $32 billion Baxalta deal back in 2016. The company’s shares have been taking a beating throughout Hemlibra’s rise, and Thursday’s new approval sent them south once again.

Investors concerned about Shire’s ability to keep up with Roche in the hemophilia field have also hounded Takeda, which inked a $62 billion buyout agreement for Shire back in May. But the Japanese drugmaker’s executives have worked to reassure investors that the company has already factored that next-generation competition into its financial forecasts.

https://www.fiercepharma.com/pharma/roche-s-hemlibra-breaks-into-wider-hemophilia-market-blockbuster-approval

Gilead strikes discount Yescarta deal with NHS in adults

Gilead Sciences has one-upped Novartis in England’s CAR-T market. The drugmaker reached a deal with NHS England on Yescarta, beating rival Kymriah to become the first CAR-T therapy available to adult blood cancer patients on England’s public health system, albeit via the Cancer Drugs Fund.

The National Institute for Health and Care Excellence (NICE) had in August rejected Yescarta because its price didn’t meet the agency’s cost-effectiveness metrics. But now, NICE has come around to back its use under NHS England’s Cancer Drugs Fund—after a confidential discount off the £300,000 full list price.

The fund, managed by NICE, which operates under NHS England, serves as an extra means for patients to access novel medicines early before a full NICE backing.

With this deal, an estimated 200 patients a year will be eligible for the treatment, according to NHS England. These are adult patients with diffuse large B-cell lymphoma (DLBCL) and primary mediastinal B-cell lymphoma (PMBCL) who have failed at least two prior therapies.

In the clinical trial that led to Yescarta’s EU approval in late August, 72% of patients responded to it, and half of them remained cancer-free six months after treatment.

However, almost at the same time Yescarta won EU clearance, NICE gave its thumbs-down to the drug, citing the lack of direct clinical data that compare Yescarta with standard salvage chemotherapy. Though the agency recognized Yescarta as “a step-change in treatment,” the cost police concluded that Yescarta’s price was too high to be considered cost-effective.

Whatever discount Gilead offered to get a deal now, it is obviously enough to win partial endorsement from NICE in adult patients, which rival Novartis’ Kymriah doesn’t have. After stiff-arming Yescarta, NICE in September approved Kymriah at a list price of £282,000, but only in children and young adults with acute lymphoblastic leukemia.

Then, Kymriah’s hope to reach England’s adult patients was also nixed by NICE for the same reason Yescarta faced—it’s too expensive because no data against salvage chemo is available to gauge the drug’s benefit.

Yescarta is currently leading Kymriah in sales. In the first half of 2018—when neither drug had won an EU nod—the Gilead drug sold $108 million in the U.S. But with Kymriah’s second FDA nod in DLBCL in May, the two drugs are now officially vying in the same indication.

https://www.fiercepharma.com/pharma/beat-you-to-it-kymriah-gilead-strikes-yescarta-deal-nhs-england-at-discount

GW Pharma looks to dispel cannabis stigma with Epidiolex launch

Set to roll out the U.S. market’s first prescription cannabidiol soon, GW Pharmaceutical will be looking to assure doctors that Epidiolex, approved to treat rare forms of epilepsy, is vastly different from off-the-shelf artisan oils at cannabis shops.

To that end, GW has hired 66 neurology sales representatives who will target some 5,000 epilepsy specialists in the U.S., Stephen Schultz, VP of investor relations, said. Their message? Trust the science and rigor behind the only FDA-approved CBD.

“We’re the leader in this area, we’re science-based, and we believe physicians and patients will desire medicines that have gone through the FDA review process. When a drug is prescribed that’s FDA approved, you know it’s exactly the same every time it’s prescribed and every time it’s taken,” Schultz said.

Epidiolex was initially approved in June, but it had to wait until last week for the Drug Enforcement Agency to figure out how to handle the plant-derived cannabidiol CBD. The good news for GW was a rescheduling from a Schedule I drug—others in that class include heroin and marijuana—to a Schedule V, the class considered to have the least potential for abuse. That class includes medicines such as Robitussin AC and Pfizer’s Lyrica.

Two websites, one directed at physicians and the other at patients, will back the launch, although Schultz characterized the marketing as a “medically oriented education commercialization effort” directed at professionals.

Along with the launch of Epidiolex to treat seizures associated with Lennox-Gastaut syndrome or Dravet syndrome epilepsies, GW plans to continue to trial the drug for other uses. It currently has a single phase 2 study, due to read out next year, testing the drug as a treatment for seizures in tuberous sclerosis complex.

Schultz said GW’s own clinical experience also suggests promise for Epidiolex for treatment of autism spectrum disorders, and the company plans to launch a new study in Rett Syndrome in the fourth quarter.

https://www.fiercepharma.com/marketing/gw-pharma-cannabidiol-epidiolex-market-rollout-looks-to-dispel-stigma-and-promote-product

Bausch’s Salix arms its GI salesforce with 2nd liver treatment in Dova co-promotion

Salix Pharmaceuticals continues to roll up partners in bid to broaden its reach. Its latest deal not only beefs up its own portfolio, but gives Dova Pharmaceuticals a co-promotional boost for its chronic liver disease drug Doptelet, which just won FDA approval.

The deal gives Dova access to Salix’s bigger sales force with established physician connections, while Salix gets another liver medicine to market to doctors. Salix’s Xifaxan, better known for its IBS-D indication, is also approved to treat hepatic encephalopathy, a complication of liver cirrhosis.

The GI arm of Bausch Health, Salix plans to deploy 100 of its gastroenterology sales reps already working in liver disease and add Doptelet by mid-October. Dova will continue its separate sales efforts, which specifically target hepatologists and interventional radiologists. Doptelet is approved to treat thrombocytopenia in adults with chronic liver disease who are scheduled for a medical or dental procedure.

Thrombocytopenia is a platelet deficiency that can increase risks for bleeding in chronic liver disease patients. Patients with CDL typically receive a platelet transfusion before procedures, but Doptelet is designed to circumvent that.

Mark McKenna, Salix’ senior VP and general manager, said adding first-in-class Doptelet makes Salix “more relevant to physicians.”

“Dova has started initial marketing efforts, and they’ll continue to own the marketing end of the collaboration; however, we’ll offer a lot of assistance in regard to day one access to physicians, and knowledge and know-how on the market access side, he said. “And on the medical side, to change the way physicians think about how they treat these patients.”

This summer, Salix struck a deal with US WorldMeds to co-promote its opioid withdrawal drug Lucemyra, boosting a pain medicine franchise built around opioid-induced constipation drug Relistor. It also announced in August a team-up with Cedars Sinai to research new therapies related to the gut microbiome. McKenna said the latter is a bid to invest in earlier-stage treatments to accelerate its pipeline.

While Bausch Health, formerly Valeant, once spent heavily to promote Xifaxan through direct-to-consumer ads, it lately has concentrated more on a sales-force drive. Beginning in 2016, it ramped up its sales force for both Xifaxan and Relistor, a big investment; those additional sales resources, along with increased promotional spending, cost more than $50 million in 2017, according to its most recent financial report. Those were “amounts well spent,” the report said, as sales increased 37% for Xifaxan and 48% for Relistor for the six months ended in June 2018 versus the same time period in 2017.

While Bausch Health doesn’t break out individual drug sales, Salix’ total sales for the first six months of 2018 were $863 million, a 25% increase from $688 million during the same period in 2017.

https://www.fiercepharma.com/marketing/salix-inks-deal-dova-for-2nd-liver-treatment-looks-to-development-and-research-deals-to

Biogen Neurology Presentations at Parkinson’s, Movement Disorders Meet

Biogen Inc. (Nasdaq: BIIB) announced it will present data from its movement disorders portfolio at the upcoming International Congress of Parkinson’sDisease and Movement Disorders (MDS) in Hong Kong (Oct. 5-9, 2018).

Data being presented are from Biogen’s research programs evaluating potential treatments for progressive supranuclear palsy (PSP) and Parkinson’s disease (PD) as part of its larger neurology pipeline.

‘The data being presented at MDS are a testament to Biogen’s commitment to its goal of delivering innovative therapies for people living with complex, neurodegenerative conditions like PSP and PD,’ said Kate Dawson, M.D., vice president of late-stage clinical development at Biogen. ‘PSP and PD both have significant unmet need, especially for disease modifying therapies, so we look forward to advancing the clinical development programs to help better understand these challenging conditions.’

Biogen PSP presentations will focus on safety data from the Phase 1 long-term extension study and baseline demographics from the Phase 2 PASSPORT study. The company’s BIIB092 program has recently met milestones, including recruitment completion for the Phase 2 PASSPORT study and fast track designation by the U.S. Food and Drug Administration for PSP. PSP is a rare neurodegenerative disease, considered to be a primary tauopathy, characterized by rapidly progressing physical impairments, such as difficulty speaking, swallowing and walking, as well as cognitive/behavioral impairments, such as apathy and dementia.

PD presentations will feature an overview of the design of the BIIB054 SPARK Phase 2 study, which is the most advanced program in Biogen’s PD pipeline. PD is the second most common neurodegenerative disease after Alzheimer’s disease. Motor symptoms of PD include bradykinesia, muscular rigidity, rest tremor and postural and gait impairment.1

https://www.marketscreener.com/BIOGEN-4853/news/BIOGEN-TO-PRESENT-DATA-FROM-ITS-NEUROLOGY-PIPELINE-AT-THE-2018-INTERNATIONAL-CONGRESS-OF-PARKINSON-27375480/

CymaBay: Additional Positive Results of Phase 2 Liver Disease Study

CymaBay Therapeutics, Inc. (NASDAQ: CBAY), today announced that two late-breaking presentations describing new long-term data from its ongoing Phase 2 study of seladelpar in patients with primary biliary cholangitis (PBC) will be featured on November 12, 2018 during The Liver Meeting hosted by the American Association for the Study of Liver Diseases (AASLD) in San Francisco, CA (November 9-13, 2018).

Seladelpar is an orally administered, potent and selective peroxisome proliferator-activated receptor delta (PPAR) agonist currently in development for PBC and nonalcoholic steatohepatitis (NASH).

The first presentation, abstract LB-3, titled ‘Efficacy and Safety of Seladelpar, a Selective Peroxisome Proliferator-Activated Receptor Delta Agonist, in Primary Biliary Cholangitis: 52-Week Analysis of an Ongoing International, Randomized, Dose Ranging Phase 2 Study’ will be delivered by Professor Chris Bowlus, MD, Division Chief of Gastroenterology and Hepatology, University of California at Davis Health. The 52-week analysis from this ongoing Phase 2 study may provide some insight into the primary efficacy outcome of the upcoming pivotal Phase 3 seladelpar PBC study. The Phase 3 primary efficacy outcome is a composite responder rate measured at 52 weeks that comprises an alkaline phosphatase

https://www.marketscreener.com/CYMABAY-THERAPEUTICS-INC-16725724/news/CymaBay-Therapeutics-Announces-Additional-Positive-Results-from-its-Ongoing-Phase-2-Study-of-Selad-27375488/

Omeros: Additional Positive Data for OMS721 in IgA Nephropathy

Omeros Corporation (NASDAQ: OMER) today announced positive data from the company’s ongoing Phase 2 clinical trial of OMS721 for the treatment of renal diseases.

The data are from study patients with immunoglobulin A (IgA) nephropathy, a progressively worsening kidney disease and the most common glomerulonephritis worldwide, responsible for 10 percent of all people on dialysis globally. Patients in this cohort had IgA nephropathy with high risk of progression but none received corticosteroid treatment immediately prior to or during the study – different than the cohort reported last year in which all patients initiated the study while on steroid therapy. Reductions in protein levels in urine collected over a 24-hour duration (the ‘gold-standard’ in assessing proteinuria) in this steroid-free OMS721-treated group were of similarly large magnitude to those seen in the earlier cohort. In IgA nephropathy, proteinuria is the most reliable prognostic factor for loss of kidney function – the greater the proteinuria reduction, the better the prognosis. OMS721 is Omeros’ lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the complement system’s lectin pathway. In addition to an ongoing Phase 3 clinical trial in IgA nephropathy, OMS721 is in Phase 3 clinical programs for hematopoietic stem cell-associated thrombotic microangiopathy (HSCT-TMA) and for atypical hemolytic uremic syndrome. OMS721 has been granted breakthrough therapy designations by FDA for both IgA nephropathy and HSCT-TMA.

‘We now have results of treatment with OMS721 in IgA nephropathy patients both with and without concomitant steroid therapy,’ stated Jonathan Barratt, Professor of Renal Medicine at the University of Leicester and Honorary Consultant Nephrologist at Leicester General Hospital. ‘In contrast to the previously reported cohort who were enrolled while on steroid therapy, the patients in this study were steroid-free, and yet the magnitude of proteinuria reduction across both groups is consistent and likely to indicate a significant renoprotective effect of OMS721 in IgAN, with a legacy effect again seen for months beyond cessation of OMS721 treatment. Notably, the current study’s patients are at very high risk of progressive renal disease and generally have long-standing, established disease with multiple comorbidities, representing a difficult-to-treat population. Despite that, almost all of the OMS721-treated patients responded to the first 12-week course of treatment. Collectively, these data further confirm the rationale for lectin pathway inhibition as a promising future option for the treatment of patients with IgAN.’

The current cohort study was designed to evaluate the safety and tolerability of once-weekly intravenous (IV) dosing of OMS721 and to describe the effect of the drug on 24-hour proteinuria in patients with IgA nephropathy. Twelve patients were enrolled. Per protocol, patients are required (i) to not receive corticosteroids during the study duration or for at least three months prior to screening and (ii) to be stable on an optimized dose of ACE inhibitors/ARBs (i.e., renin-angiotensin system [RAS] blockade) before enrollment. In a double-blind design, study patients were randomized 1:1 to receive either OMS721 or placebo for 12 weeks, and then both groups could receive extended OMS721 dosing at investigator discretion. Three patients were excluded from the analysis: two for unstable RAS blockade with at least a 50 percent change in dosing of RAS-blockade medication during the study, and one for untreated gastrointestinal disease that persisted throughout a substantial majority of the study duration. These conditions are known to affect proteinuria levels markedly, and results from these patients were determined by IgA nephropathy expert review to be unevaluable.

For the nine evaluable patients, median reductions in proteinuria following the initial 12-week course of treatment were 18.4 percent and 18.0 percent for the OMS721 and placebo groups, respectively. The one OMS721-treated patient whose proteinuria level remained elevated after the initial treatment course subsequently reached a 67.8-percent reduction from baseline following an additional course of OMS721 dosing.

After the initial 12-week treatment with OMS721 or placebo, all patients rolled into a dosing-extension period during which time a patient could receive, at investigator discretion, treatment with OMS721 if his/her respective 24-hour proteinuria level remained above 1 gram or the proteinuria response was less than a 50-percent reduction from baseline. Eight patients received OMS721 in this extension period, after either first receiving 12 weeks of OMS721 or placebo. Comparison with baseline of the proteinuria data after at least 18 weeks in the study (i.e., after the first course of OMS721 or placebo treatment and six-week follow-up) shows a median proteinuria reduction for this group of 55.7 percent. Four patients in this OMS721 dosing-extension period have reached between 9 and 12 months beyond baseline, and show reductions in proteinuria of 53.9 percent, 57.4 percent, 65.3 percent, and 67.8 percent. At most recent assessment, two of these four patients have continued to demonstrate sustained reductions in proteinuria for 2.5 and 5 months, respectively, after cessation of treatment with OMS721; the other two patients just recently completed treatment courses.

For the evaluable patients in the OMS721-treated and placebo groups, respectively, median ages at study start were 50 vs 33 years old; median time since diagnosis was 19 vs 9 years and median baseline proteinuria was 2.9 vs 2.5 g/day. Forty-four percent of these patients had nephrotic-range proteinuria at baseline, ranging from approximately 4.5 to 12 g/day, and most patients had multiple comorbidities.

Consistent with all other studies conducted with OMS721, the drug was well tolerated and no safety concerns were seen in this cohort of patients. Across all 12 patients, no treatment-related serious adverse events were observed, and most reported adverse events were mild or moderate.

The OMS721 treatment-extension period in this cohort study remains ongoing. Omeros plans to report additional data from this Phase 2 cohort at a future medical congress.

https://www.marketscreener.com/OMEROS-CORPORATION-5628248/news/Omeros-Corporation-Announces-Additional-Positive-Data-for-OMS721-in-IgA-Nephropathy-27375495/