BeiGene announced the presentation of long-term Phase 1 data and results of structural and binding mechanistic analyses on its investigational anti-PD1 inhibitor, tislelizumab, in posters at the American Association for Cancer Research, or AACR. The multi-center, open-label Phase 1 trial of tislelizumab as monotherapy in advanced solid tumors is being conducted in Australia, New Zealand, the United States, Taiwan, and South Korea and consists of dose escalation, schedule expansion, fixed dose expansion and indication expansion. This first-in-human, or FIH, trial is fully enrolled with over 450 patients. As of October 27, 2018, 65 patients received tislelizumab for more than 12 months and were included in this long-term exposure, or LTE, analysis. These 65 patients were from both the dose-escalation and dose-expansion phases. Most patients with LTE received tislelizumab at 5 mg/kg Q3W, while additional patients received 2 mg/kg Q3W, 2 mg/kg Q2W, 5 mg/kg Q2W and 200 mg Q3W. The most common tumor types were non-small cell lung cancer, hepatocellular cancer and bladder and ovarian cancers. With a median follow-up of 27.2 months, the objective response rate, or ORR, among patients with LTE was 68%. Four patients achieved complete response, including patients with cutaneous squamous cell carcinoma, endometrial, bladder and esophageal cancers. All four patients were PD-L1 positive. Partial responses and stable disease were observed in both PD-L1 positive and PD-L1 negative tumors. The median duration of response was 21.1 months in those with LTE. LTE to tislelizumab was generally well-tolerated when given for more than 12 months. As of the data cutoff, 52 of 65 patients experienced one or more treatment-related adverse event, or TRAE, most of which were mild to moderate in severity. Rash was the only TRAE reported in more than 15% of patients, and no rash event of grade 3 or higher occurred. Serious TRAEs occurred in three patients, including pyrexia and arthritis and all resolved. Three patients experienced AEs that eventually led to discontinuation. There were no fatal AEs.
Monday, April 1, 2019
Pharma spends big as Massachusetts lawmakers review drug-pricing bills
State legislators in Massachusetts are preparing to review a slew of bills aimed at state-level drug pricing, and the pharmaceutical industry is opening its checkbook to fight back.
The state’s Joint Committee on Health Care Financing on April 11 will examine 21 bills targeting price transparency and affordability for patients. State Sen. Cindy Friedman, the committee chair, told the Boston Herald that lawmakers will take a hard look at measures to open up drugmakers’ pricing secrets and lower sticker prices for patients.
“Drugs are a huge contributor to health care costs, and it’s becoming, as many other parts of health care, more and more of a crisis in terms of people’s ability to pay for their health care,” Friedman said.
But pharmaceutical companies aren’t taking that challenge sitting down.
In 2018, drugmakers spent more than $4 million lobbying the Massachusetts statehouse, the Herald reported. One of the biggest players in the lobbying effort, the Pharmaceutical Research Manufacturers of America (PhRMA), told FiercePharma that the state’s push to cap prices would keep patients from getting the drugs they need.
“We oppose government price-setting proposals in the states,” spokeswoman Priscilla VanderVeer said. “Government price controls limit access to medicines for patients and they stifle new innovation.”
PhRMA itself spent $292,836 lobbying Massachusetts lawmakers in 2018, the Herald said. VanderVeer declined to comment on the group’s lobbying strategy.
The push by Beacon Hill lawmakers is not the first attempt to give Massachusetts more control over drug pricing, just as states such as Nevada and California have done.
In 2017, Massachusetts pushed for power to use formulary negotiations to manage its drug costs, but was rebuffed by Tim Hill, then-acting director of the Center for Medicaid and CHIP Services. The state wanted to adopt tactics similar to those used by private insurers and pharmacy benefits managers, but Hill said the plan did not meet CMS requirements that prevent Medicaid programs from managing coverage.
State legislators also pushed for a pricing transparency bill in early 2016 that Jonathan Fleming, general partner of biotech investment firm Oxford Bioscience Partners, called “a nightmare” in a committee hearing. The bill later fizzled out in early 2017.
The state’s drug pricing battle comes as President Donald J. Trump and the pharmaceutical industry continue their impasse at the federal level.
Last May, Trump outlined a plan that would push companies to lower prices by stepping up negotiations and competition, and providing incentives to lower list prices and cut out-of-pocket costs for patients. Pharma responded with widespread price hikes in January, upping the cost of more than 250 drugs, Reuters said.
Despite that setback, federal lawmakers are pushing ahead with measures in both houses of Congress. Most recently, Sen. Rick Scott, R-Fla., announced a Transparent Drug Pricing Act that would cap U.S. prescription drugs at the same list price as other developed nations, among other measures.
Piper Jaffray sees Endologix’s updates as ‘positive’
Piper Jaffray analyst Matt O’Brien maintained a Neutral rating on Endologix, saying he wanted to see “more evidence of execution in the coming quarters.” The analyst added that the updates the company released this morning, including an equity raise, an exchange of convertible debt, a number of updates with the Deerfield agreement, and a reaffirmation of its FY19 revenue guidance, were a “positive,” as it showed the company has “increased financial flexibility lower debt load risk, and easier covenants.”
Celyad price target lowered to $41 from $51 at Piper Jaffray
Piper Jaffray analyst Edward Tenthoff reiterated an Overweight rating on Celyad, but lowered his price target on shares to $41 from $51 as he pushes out approvals based on clinical progress. The analyst noted that Celyad intends to initiate a potentially registrational Phase II AML study in the second half of 2019, has begun a Phase I study of allogeneic CYAD-101 in mCRC with data in the second half of 2019, and will file INDs on CYAD-211 BCMA and CYAD-221 CD-19 CAR-Ts in 2020.
Catalyst Biosciences receives orphan drug desgination for MarzAA in EU
Catalyst Biosciences announced that the European Commission has awarded orphan designation of its Factor VIIa variant marzeptacog alfa, or MarzAA, for the treatment of haemophilia B. Catalyst has also completed dosing in the Phase 2 portion of the Phase 2/3 subcutaneous trial of MarzAA for the treatment of hemophilia A or B with inhibitors. Nine subjects successfully completed dosing and top-line results will be presented in the third quarter of 2019. “Orphan designation is another important acknowledgement of the significant benefits of subcutaneous MarzAA and will complement our orphan drug designation already granted in the U.S. by the Food and Drug Administration,” said Nassim Usman, Ph.D., CEO of Catalyst. “We have completed dosing in the Phase 2 portion of the Phase 2/3 trial for the treatment of hemophilia A or B with inhibitors and have clearly demonstrated efficacy as measured by greater than 90% reduction in annualized bleed rate, as well as bleeding density. We expect to present top-line results from the study in the third quarter of 2019. We also plan to initiate a Phase 3 MarzAA registrational study in 2020 and believe that subcutaneous MarzAA has significant commercial potential in the $2.2B hemophilia inhibitor market.”
Novartis to face lawsuit over doctor kickbacks
A Manhattan district court judge has ruled that Novartis (NVS -0.3%) must face a U.S. lawsuit over alleged kickbacks to physicians aimed at boosting prescriptions for its drugs.
The case began in 2011 when a former sales rep filed a whistleblower suit.
In 2010, the company agreed to pay $422M to settle various civil and criminal charges, including paying kickbacks to doctors. In 2015, it agreed to pay $390M to settle claims that it paid rebates to specialty pharmacies to promote two of its drugs.
Novartis spokesman Eric Althoff says, “We are disappointed in today’s decision and look forward to presenting our case at trial. We continue to believe that the government has insufficient evidence to support its claims.”
Celldex presents data from its bispecific and TAM receptor programs at AACR
Celldex Therapeutics presented promising data from the company’s growing bispecific and TAM receptor programs during poster sessions at the American Association for Cancer Research, AACR, Annual Meeting 2019. “Our research and discovery efforts have yielded multiple promising candidates that address important needs in cancer immunotherapy and complement our pipeline,” said Tibor Keler, Ph.D., Executive Vice President and Chief Scientific Officer of Celldex Therapeutics. “CDX-527 joins two powerful pathways in the immune system, PD-1 blockade and CD27 costimulation. The data we presented at AACR demonstrate that this bispecific antibody candidate has greater activity than the combination of the two individual antibodies and support advancing the program into IND-enabling studies. In addition, we also presented data on our lead candidate antibodies targeting the TAM receptors, which act as checkpoints on myeloid cells. We have uncovered a unique mechanism for antibody-mediated activation of dendritic cells and macrophages and demonstrated that a surrogate MerTK mAb promotes anti-tumor immunity alone and enhances the activity of PD-1 blockade. We look forward to continuing to progress both CDX-527 and our TAM program over the course of the year,” concluded Keler.
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