Biogen’s new Spinraza data for earlier use ‘pretty amazing,’ analyst says

Biogen’s spinal muscular atrophy med Spinraza hit the market running after its launch, helping fuel growth at the drugmaker as its MS franchise faced a slowdown. Now, the company is touting an interim analysis showing that infants with SMA can benefit “dramatically” from the drug if they’re treated before symptoms develop.

The Nurture study is testing Spinraza in 25 patients genetically diagnosed with the disease and treated before symptoms developed. At an interim analysis in May 2018, all patients were alive, and none required tracheostomy or permanent ventilation. The patients were 14 months or older at the time of the analysis.

Plus, 88% percent of the patients were able to walk, either with assistance or independently, and all were able to sit without support. In a note, Evercore ISI analyst Josh Schimmer said the results were “pretty amazing.”

Wildon Farwell, M.D., Biogen senior medical director of clinical development, said in a statement the data show that “early diagnosis and treatment with Spinraza has the potential to dramatically change the course of SMA.”

“This is the longest available span of data on infants with SMA who began treatment in a presymptomatic period and indicates that children treated early with Spinraza can achieve motor milestones they would likely not attain without treatment,” Farwell added.

Biogen launched Spinraza after an approval in late 2016, and the drug quickly captured headlines for its price of $750,000 for the first year and $375,000 annually after that. Despite some concern about the price, the drug has far surpassed early expectations, leaping out to $884 million in sales during its first full year on the market.

As of this summer, the drug has won approval in at least 24 countries. More than 5,000 patients were taking Spinraza as of June, including those in an expanded access program, clinical trials and a postmarketing study, according to Biogen.

Biogen presented the new Spinraza data at the World Muscle Society’s annual congress in Mendoza, Argentina.

https://www.fiercepharma.com/pharma/data-show-spinraza-can-dramatically-help-presymptomatic-patients-improve-course-disease

FDA reviewers endorse Celltrion’s Rituxan biosimilar ahead of panel vote

Celltrion’s biosimilar to Roche’s blockbuster Rituxan has already been rejected at the FDA once for manufacturing problems, but in the company’s second attempt, agency staffers have reacted favorably to the med. In a review document (PDF) posted ahead of an FDA panel meeting set for Wednesday, internal reviewers found no “clinically meaningful differences … in terms of safety, purity and potency” between the biosim and Roche drug.

The FDA’s Oncologic Drugs Advisory Committee will meet on Wednesday to consider Celltrion’s application after the FDA rejected the biosimilar last year. If the biosim wins approval, Celltrion could potentially launch this year; Roche has disclosed that its U.S. patent protections for cancer and rheumatoid arthritis blockbuster Rituxan expire in 2018. For this review, Celltrion is only seeking approvals to treat certain patients with non-Hodgkin’s lymphoma.

Even if the FDA’s panel endorses Celltrion’s biosimilar, that doesn’t guarantee an approval. The agency typically follows committee votes, but it doesn’t have to.

Roche’s Rituxan brought in more than $4 billion in the U.S. last year, topping FiercePharma’s 2018 report of top drugs susceptible of biosim or generic competition. Earlier this year, the drugmaker caught a break when a Novartis biosimilar suffered an FDA rejection. At the time, a Deutsche Bank analyst wrote that Rituxan could dodge biosim competition until next year.

Celltrion already has an FDA-approved biosimilar to Johnson & Johnson’s Remicade, plus two other biosims approved in Europe. The company is now seeking nods for its biosimilars to Rituxan and Herceptin after manufacturing problems caused FDA rejections.

In January, the FDA issued a warning letter related to plant failures in South Korea, triggering the rejections. In May, Celltrion said it “has made progress addressing the concerns … and is committed to working with the agency to fully resolve all outstanding issues with the highest priority and urgency.” The company refiled the submissions and at the time said it expects both FDA approvals yet this year. Under a partnership, Teva is set to market Celltrion’s Rituxan biosimilar in the U.S.

For Roche, potential biosim competition to its top med comes as no surprise. The company has been bracing for competition to a trio of its top blockbusters that annually bring in more than $20 billion. Over the next few years, Rituxan, Herceptin and Avastin will come under increasing biosim competition.

https://www.fiercepharma.com/pharma/fda-reviewers-endorse-celltrion-s-rituxan-biosim-ahead-panel-vote

Novartis to Launch Innovation Labs Across the Globe, Including Silicon Valley

To spur innovation and bolster ties with tech giants, Swiss pharma giant Novartis plans to set up innovation labs across the globe to partner with these companies to spur the development of new therapies.

The project, called Novartis Biome, will begin in San Francisco with a partnership with Mekonos and Medable, two Silicon Valley companies, Bloomberg reported this morning. Mekonos develops cell and gene therapies and Medable is a digital health firm. Bertrand Bodson, Novartis’ chief digital officer, told Bloomberg that now is a time when technology, big data and science are merging. Novartis, like many other companies, is looking to harness the power of big data and machine learning to develop more precise treatments for diseases such as cancer.

In New York, Novartis Biome formed partnerships with digital health company Hitlab and is also working with companies like Veta Health and ConversationHealth on various medical projects, Bloomberg reported.

Last year, ahead of taking over the reins of Novartis, Vas Narasimhan stressed the need for more efficient use of digital technology. At the time, Narasimhan said he intended to partner with companies that are specialists in artificial intelligence and data analytics. The move was intended to streamline Novartis’s strong but scattered data science capability,” Narasimhan said at the time. Bodson, a veteran of Amazon and Sainsbury’s Argos, was brought onboard Novartis about the same time that Narasimhan was tapped as the replacement for now-former Chief Executive Officer Joseph Jimenez. When Bodson was hired in August 2017, Novartis said he was expected to “lead the digital transformation Novartis has embarked upon to improve the way the Company uses data in drug discovery and development, engages with patients, doctors and other stakeholders, as well as automating business processes.”

With Bodson aboard and the new mission, Novartis has also made significant investments in its new path. In July, Novartis formed a deal with SHYFT Analytics to harness that company’s intelligent platform for life sciences. Novartis intends to use the platform to support the commercialization of key therapies in Europe. Novartis said it will use Shyft’s Strata Data Platform to “aggregate and manage” third-party and proprietary commercial data sources, as well as its Lumen Insights Platform to “deliver intuitively-designed analytics” that will be used to “drive the next-best-actions for commercial teams.” Through the power of Shyft’s platforms, Novartis said the partnership will “simplify and accelerate the aggregation, assimilation, and analysis of scores of disparate data sets that inform commercial sales and medical education programs.”

Novartis also formed an alliance with Chinese tech firm Tencent Holdings as it looks to harness that company’s digital power to develop innovative therapies in the Chinese market.

https://www.biospace.com/article/novartis-to-launch-innovation-labs-across-the-globe-including-silicon-valley/

Stellar Phase III Data from Janssen’s Stelara

New late-stage data released by Janssen shows that a single dose of Stelara can induce clinical remission and response in adult patients with moderate to severe ulcerative colitis who have not had a positive response from previous treatment options.

The new findings from the Phase III UNIFI Study were presented today at the American College of Gastroenterology Annual Scientific Meeting 2018. Results from the trial showed that after eight weeks of taking a single intravenous dose of Stelara (ustekinumab), patients saw “clinical remission in a significantly greater proportion of UC patients.” In its announcement, Janssen noted that 15.6 percent of patients who received 130 mg of Stelara and 15.5 percent of patients who received ~6 mg/kg of Stelara saw remission in comparison to placebo patients. Only 5.3 percent of patients on placebo saw the same kind of remission response, the company said.

Ulcerative colitis is a debilitating inflammatory bowel disease in adults and children, with limited therapeutic management options for many patients. It is estimated that close to 1 million patients with ulcerative colitis live in the United States.

Janssen also said that at Week 8, the trial data showed Stelara hit some of its major secondary endpoints, which includes the proportion of patients in clinical response, endoscopic healing, as well as health-related quality of life scores.

In addition, Janssen said that at least 50 percent of study participants were considered biologically refractory, and 17 percent had a history of inadequate response or intolerance to any anti-TNF antibody and Takeda’s Entyvio (vedolizumab).

“The significant rates of remission observed in this refractory population through the 8-week induction, coupled with a safety profile that is well-documented through years of research and use in other immune diseases, demonstrate the potential for ustekinumab as an effective treatment for ulcerative colitis.” lead investigator Bruce E. Sands said in a statement.

There were other significant points of interest discovered at Week 8. Of the patients who received 130 mg of Stelara, 26.3 percent experienced endoscopic healing, compared to 13.8 percent of placebo patients. For the patients who received ~6 mg/kg of Stelara, 27 percent experienced endoscopic healing compared to 13.8 percent of placebo patients. Endoscopic healing was defined as a Mayo endoscopy subscore of 0 (normal mucosa or inactive disease) or 1 (mild disease activity), Janssen said.

Also, Janssen said that 51.3 percent of patients who received 130 mg of Stelara and 61.8 percent of patients receiving ~6 mg/kg of Stelara achieved a clinical response, compared to 31.3 percent of placebo patients. Clinical response, Janssen said, was defined as a decrease from baseline in the Mayo score by ≥30 percent and ≥3 points, with either a decrease from baseline in the rectal bleeding subscore ≥1 or a rectal bleeding subscore of 0 or 1.

Philippe Szapary, head of clinical development at Janssen Research and Development, said Stelara is the only biologic that targets interleukin (IL)-12 and IL-23 cytokines, which are believed to play a role in immune-mediated diseases, like ulcerative colitis.

“These induction data from the Phase III UNIFI study underscore the potential for this unique pathway in the treatment of ulcerative colitis, which may lead to a new effective and safe treatment option for ulcerative colitis patients in the future,” Szapary said in a statement.

In addition to the UNIFI study data, Janssen is also presenting results from the IM-UNITI open-label long-term extension (LTE) study for ustekinumab in the treatment of adults with moderate to severe Crohn’s disease.

https://www.biospace.com/article/stellar-phase-iii-data-from-janssen-s-stelara/

Tilray price target raised to $172 from $62 at Cowen

Cowen analyst Vivien Azer raised her price target for Tilray (TLRY) to $172 from $62 and keeps an Outperform rating on the shares. The analyst also raised her price target for Outperform-rated Canopy Growth (CGC) to C$82 from C$74.

https://thefly.com/landingPageNews.php?id=2801355

STAAR Surgical, Dr. Tobias Neuhann announces strategic alliance agreement

STAAR Surgical Company announced a multi-year Strategic Alliance Agreement with Dr. Tobias Neuhann of the Augenklinik am Marienplatz in Munich, Germany. The agreement serves as a global partnership model for smaller and single site refractive surgery eye clinics that aim to provide STAAR’s EVO Visian ICL intraocular lenses as a primary and premium option for patients seeking visual freedom from spectacles and contact lenses.

https://thefly.com/landingPageNews.php?id=2801383

Myriad and Pfizer Build Upon Companion Diagnostic Agreement

Myriad Genetics, Inc. MYGN, +0.40% a global leader in personalized medicine, today announced that it has signed a commercialization plan with Pfizer Inc. The plan is under an existing companion diagnostic agreement, in which Myriad is pursuing U.S. Food and Drug Administration (FDA) approval for its BRACAnalysis CDx [(R)] to be used as a companion diagnostic with Pfizer’s investigational PARP (poly ADP ribose polymerase) inhibitor, talazoparib.

Talazoparib and BRACAnalysis CDx currently are under FDA review, with New Drug Application and Supplementary Premarket Approval submissions based on results from the EMBRACA trial, which evaluated talazoparib versus physician’s choice chemotherapy in patients with germline (inherited) BRCA-mutated, HER2-negative locally advanced or metastatic breast cancer. Myriad anticipates a regulatory decision from the FDA by December 2018.

Under the commercialization plan, each company remains responsible for the commercialization of its respective product. However, the companies will collaborate on certain commercial activities intended to support the use of the BRACAnalysis CDx in identifying patients for potential treatment with talazoparib following FDA approval.

“We believe this commercial collaboration is another strong indication of Myriad’s global leadership in the field of companion diagnostics for PARP inhibitors and personalized medicine,” said Lloyd Sanders, president of Myriad Oncology. “We are excited to be working with Pfizer and towards ensuring patients have access to this class of drugs.”

Approximately one in eight women are diagnosed with breast cancer in the U.S., and one-third are diagnosed with or will progress to the metastatic stage of the disease.

https://www.marketwatch.com/press-release/myriad-and-pfizer-build-upon-a-previously-announced-companion-diagnostic-agreement-2018-10-09