Ultragenyx Pharmaceutical announced that the European Commission, EC, has approved the Marketing Authorization Application,MAA, for Mepsevii vestronidase alfa, for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII. Mepsevii is now approved for use in all 28 EU countries and in Iceland, Liechtenstein and Norway.Dr William Sly’s science for this treatment has been around for over 25 years and we are honored to be able to develop and finally make this medicine available to MPS VII patients and families in Europe, people who did not know whether a therapy would ever be available to them,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “This would not have been possible without the commitment and dedication of the patients, their families, and the physicians who participated in our clinical program, and I would like to thank them.”The approval follows a positive opinion adopted on June 28 by the European Committee for Medicinal Products for Human Use,CHMP, to recommend approval of Mepsevii under exceptional circumstances. The European Medicines Agency granted Orphan Drug designation to Mepsevii in March 2012. Mepsevii was approved by the U.S. Food and Drug Administration for the treatment of pediatric and adult patients with MPS VII in November 2017.
Monday, August 27, 2018
AnaptysBio initiated at Cantor Fitzgerald
AnaptysBio initiated with an Overweight at Cantor Fitzgerald. Cantor Fitzgerald analyst Eliana Merle initiated AnaptysBio with an Overweight rating and $124 price target.
Companies Continue to Take Aim at Glioblastoma
Long before Sen. John McCain was diagnosed with brain cancer, I got the chance to briefly meet the man who developed a reputation as a maverick in the Senate. It was in Raleigh, N.C. about 12 years ago, and he was in town pushing for support of public financing of judicial elections in the Tar Heel State.
I was a political reporter with The Wilson Daily Times (now The Wilson Times) and covered McCain’s push for the plan, something for which he was a proponent. After the presentation, I got to spend a few minutes with the senator. I don’t recall the details of the conversation, only the kindness and character-defining way in which he treated me and all the reporters in the room. Although I no longer write about politics, I (sort of) crossed paths with Sen. McCain last year after he revealed he had been diagnosed with brain cancer. McCain died Saturday after deciding to discontinue treatment for the cancer. He approached the end of his life in the same manner in which he approached me that day in Raleigh – with the strength of character.
When McCain revealed his brain cancer last year, BioSpace pointed to 10 companies that were in pursuit of treatments for glioblastoma, the most common form of brain cancer and one that is highly aggressive. The disease is somewhat rare, impacting only about three out of every 100,000 people in the United States.
Although it’s a rare disease, companies are still chasing after a potential treatment. Citing the National Institutes of Health (NIH), CNN reported there are currently 274 glioblastoma multiforme studies underway or recruiting across the United States.
One early-stage treatment has been receiving some press due, primarily, to one patient who has shown a remarkable response to the treatment. Bob Rulli, a retired airplane engine designer, was diagnosed with glioblastoma multiforme five years ago. He has been a clinical patient of startup Bexion Pharmaceuticals. Rulli has been undergoing treatment with BXQ-350, a formulation of a synthetically produced, human lysosomal protein, Saposin C (sphingolipid activator protein, or SapC), and the phospholipid dioleoylphosphatidylserine (DOPS). Average life expectancy is less than two years. Rulli has been on the Bexion treatment for 19 months. His cancer hasn’t been eliminated, but the tumor has proven to be stable during that time. BXQ-350 is currently in Phase Ia.
Earlier this month AiVita Biomedical initiated its first clinical site for the ROOT OF CANCER Phase II trial in patients with newly diagnosed glioblastoma. Patients will receive autologous dendritic cells loaded with autologous tumor antigens derived from self-renewing tumor cells as an adjunctive therapy following primary surgery plus concurrent chemoradiation, the company said.
In April, China-based CANBridge Life Sciences announced plans to initiate its Phase II/III glioblastoma multiforme trial following the nod from the China Food and Drug Administration. The company’s lead candidate, CAN008 is a “fully human fusion protein that inhibits the CD95 ligand, a member of the tumor necrosis factor (TNF) family,” according to company data. By blocking CD95, CAN008 “restores the immune system’s anti-tumor response and inhibits invasive cell growth,” the company said.
In December 2017, Virginia-based Diffusion Pharmaceuticals initiated a Phase III trial for its lead glioblastoma product, a small molecule trans sodium crocetinate (TSC). Phase II results showed that in an inoperable patient subgroup, the led candidate helped them achieve a “nearly four-fold increase in survival compared with historical controls when TSC was added to their treatment regimen,” the company said. Data showed that 40 percent of the patients were alive at two years vs. 10.4 percent.
In 2016, Celgene acquired marizomib, a novel, brain-penetrant proteasome inhibitor. The company is developing the therapy as a possible treatment for patients with newly diagnosed glioblastoma. The therapy is entering Phase III.
Last year Inovio and Regeneron partnered on a brain cancer study. Inovio will pair two of its investigative products, INO-5401 T cell activating immunotherapy encoding multiple antigens and INO-9012, with Regeneron’s PD-1 inhibitor, REGN2810.
There have also been some setbacks with glioblastoma research.
ZIOPHARM Oncology announced a delay this spring in its planned Phase III glioblastoma treatment, Ad-RTS-hIL-12 plus veledimex. The company is developing a gene therapy product designed to treat patients with recurrent glioblastoma (rGBM) as a monotherapy and in combination with an immune checkpoint inhibitor. In May the company announced it had to delay the trial in order to resolve technical requirements related to Chemistry and Manufacturing Control (CMC). This was the second delay for ZIOPHARM’s study.
Novartis farms out rare disease drug to LifeMax Labs
Novartis is licensing a treatment for Netherton syndrome, a rare genetic disorder, to LifeMax Laboratories, a biotech based in the San Francisco area looking to address diseases that have limited treatment options.
LifeMax Labs, a subsidiary of LifeMax Healthcare, picks up the global rights to develop, manufacture and market BPR277, a topical treatment in the clinic for atopic dermatitis and Netherton syndrome. In return, Novartis will receive an upfront payment, development and regulatory milestones and royalties on net sales. Financial details were not disclosed.
Netherton syndrome is caused by mutations in the SPINK5 gene, which codes for LEKT1, a protein that inhibits the activity of certain enzymes called serine peptidases. Patients with Netherton make a form of LEKT1 that cannot control serine peptidase activity, which causes various symptoms including short, brittle hair, a predisposition to allergy problems such as asthma and eczema and inflamed, red, scaly skin. There are no FDA-approved therapies specifically for Netherton. Treatment focuses on managing symptoms and avoiding skin infections and other complications.
“We are very excited to enter into this licensing agreement with Novartis and are committed to continuing the development of BPR277 which fits well with our corporate strategy of developing drugs for diseases with few or no therapeutic options,” said LifeMax CEO Larry Hsu in a statement. Hsu co-founded LifeMax and previously founded Impax Laboratories.
The company could potentially develop BPR277 for other diseases “with skin barrier impairment,” Hsu said.
LifeMax isn’t alone in trying to address Netherton syndrome. Pittsburgh-based Krystal Biotech filedin August 2017 to raise about $35 million in its IPO, funds that were earmarked to advance its lead asset, a gene therapy for dystrophic epidermolysis bullosa, but that would also push a gene therapy for Netherton into the IND stage by the second half of this year. And BridgeBio Pharma reeled in $135 million last September to support a clutch of early-stage programs, including transthyretin amyloidosis, pantothenate kinase-associated neurodegeneration, dystrophic epidermolysis bullosa and various cancers in addition to Netherton.
The LifeMax deal comes after Novartis out-licensed a handful of cancer drugs. These include the failed multi-kinase inhibitor dovitinib and a pair of oral pan-Akt kinase inhibitors picked up in its asset swap with GlaxoSmithKline back in 2014. The Swiss pharma licensed dovitinib—which did not beat Bayer’s Nexavar in phase 3—to Oncology Venture in another deal of undisclosed size. It handed off GSK-developed afuresertib (ASB183) and uprosertib (UPB795), which had entered the clinic for a variety of cancers, to Shanghai-based Laekna.
J&J hunts for silver lining as Xarelto bombs blood clot, heart failure studies
At last year’s European Society of Cardiology annual meeting, Johnson & Johnson was touting data that put its clot-busting Xarelto on track for a new approval worth $1 billion-plus. Not so this year.
Instead, J&J was forced to admit Xarelto had flopped—not once, but twice. The company saidMonday that in two separate studies, the anticoagulant drug had failed to top a dummy pill at fending off the potentially deadly blood clots it’s designed to treat.
In the first, dubbed Mariner, Xarelto didn’t pare down a composite endpoint of blood clots and blood-clot-related death in acutely ill patients—those suffering from conditions such as heart failure, infectious diseases or ischemic stroke—after their hospital stays. But J&J’s Janssen unit thinks it may still have a potential approval on its hands.
The reason? Janssen has already tested Xarelto in this population with a study called Magellan, which showed long-term Xarelto use topped long-term use of enoxaparin, known by brand name as Lovenox from Sanofi, at cutting down on clots. That trial’s results weren’t all rosy, either, though: They showed Xarelto increased bleeding risks, too.
Still, the combined results from Mariner and Magellan “tell a more complete story” about Xarelto’s role in staving off clots in “appropriate acute medically ill patients” both in and out of the hospital, Jim List, Janssen R&D’s head for cardiovascular and metabolism, said in a statement, adding that “we see a filing pathway towards approval with these combined findings and look forward to discussing them” with the FDA.
In the second study unveiled at ESC, adding Xarelto to standard-of-care treatment didn’t reduce the risk of heart attack, stroke or death among heart failure patients whose symptoms had suddenly worsened.
The failures aren’t the first for J&J, which has been aggressive in hunting down new Xarelto indications. Just last October, for instance, J&J’s partner Bayer stopped a Xarelto study in certain stroke patients because the drug wasn’t likely to beat aspirin at fending off a second stroke or embolism.
But the companies have also turned up data to support some big new uses—most recently, aspirin-topping artery disease results that analysts have predicted could help generate $1.5 billion in U.S. sales. And as Janssen’s List was quick to point out, the company has other Xarelto studies on the horizon that should be reading out soon.
Xarelto, which rival Eliquis from Pfizer and Bristol-Myers Squibb has booted from the top of the atrial fibrillation heap, could use some new green lights. Once a slow-launching drug at the bottom of its class, Eliquis has zipped to the front of the new-age oral anticoagulant pack to put Xarelto in second place.
Cowen business/cannabis analysts to hold an analyst/industry conference call
Tobacco and Cannabis Analyst Azer, along with Business Analyst Hadiaris provide expert views on the market structure for Cannabis on an Analyst/Industry conference call to be held on August 27 at 2:30 pm.
Endo higher after FDA proposes restrictions on compounded medications
The FDA today proposed restricting the ability of companies that produce compounded medications in bulk from using three substances that are active ingredients in approved drugs, Reuters reports. The three substances include vasopressin, the active ingredient in Endo International’s (ENDP) blood pressure drug Vasostrict, which has been the subject of a lawsuit targeting how the FDA regulates drug compounding, Reuters adds. Shares of Endo are up 6%, or $1.00, to $16.54 in midday trading.
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