Jazz Pharmaceuticals pushes to expand label on Xywav; FDA eyes October for decision on Seagen's latest ADC
Eight and a half months since its last OK for Xywav, Jazz Pharmaceuticals has won priority review for a new indication with the drug.
The FDA granted the expedited inspection Monday morning, Jazz announced, as the biotech looks to expand Xywav into adult idiopathic hypersomnia patients. Regulators have set a PDUFA date of Aug. 12.
Back in July 2020, Xywav notched its first approval to treat cataplexy and excessive daytime sleepiness associated with narcolepsy in patients older than 7. At the time, the drug joined Sunosi and Xyrem in the company’s sleeping disorder-related portfolio.
Jazz’s new application, which will be officially submitted to the FDA on Tuesday, is based on a Phase III trial that wrapped up in December. The study, looking at adults with idiopathic hypersomnia, met its primary endpoint of improvements in the Epworth Sleepiness Scale (ESS) at a p-value of p<0.0001.
Although Xyrem has been a blockbuster for the company, concerns arose due to the drug’s high sodium content as a sodium oxybate. Xywav is similarly an oxybate-based drug, but contains 92 percent less sodium than its predecessor. — Max Gelman
FDA eyes October for decision on Seagen’s latest ADC
About a year after getting a nod in advanced breast cancer, Seagen could be on its way to its fourth drug approval, this time in cervical cancer.
The FDA handed Seagen and Genmab an Oct. 10 PDUFA date for their antibody-drug conjugate (ADC) tisotumab vedotin, the companies said on Friday. They filed for a BLA back in February to treat recurrent or metastatic cervical cancer patients whose disease progressed on or after chemotherapy.
“It’s a very good step and another option for patients in a disease with a grim prognosis,” CEO Clay Siegall told Endpoints News back in September.
The submission was based on Phase II data read out at virtual ESMO 2020, which showed a 24% objective response rate lasting a median of 8.3 months. It was a single-arm trial, but Seagen compared the results to data suggesting existing therapies “typically” have a response rate of less than 15% and patients survive for 6 to 9.4 months. By contrast, median survival in the study was 12.1 months, with 79% of patients alive after 6.
Seagen’s last two approvals came in the span of four months. In December 2019, the FDA OK’d its bladder drug Padcev, which was developed with Japan-based Astellas. Then, the following April, it approved Tukysa, an oral kinase inhibitor.
Tisotumab vedotin came out of a pact Seagen initially inked with Genmab back about a decade ago. Seagen — then called Seattle Genetics — agreed to give Genmab rights to its ADC tech back in 2010. A year later, they struck a deal giving Seagen an opt-in on tisotumab vedotin. The early data were good enough to persuade Seagen to share the development costs and any profits 50/50. — Nicole DeFeudis
Treovir has sights set on PhII for pediatric glioblastoma candidate
Treovir is hurrying its glioblastoma candidate into Phase II after a Phase I trial showed the treatment more than doubled the typical survival rate in children with high-grade glioma, the company said on Monday.
“It is our goal that the Phase 2 study will support market approval of G207 to treat pediatric recurrent high-grade gliomas and our company’s mission is clear: We want to commercialize G207 and provide hope to the children and families who currently have no effective therapeutic options,” CEO Michael Christini said in a statement.
The Phase I dose-escalation study enrolled 12 patients between the ages of 7 and 18 with progressive high-grade glioma. Participants got the oncolytic HSV immunotherapy, dubbed G207, either alone or in combination with radiation.
No dose-limiting toxic effects or serious adverse events were noted, Treovir said, though 20 Grade 1 adverse events were “possibly related” to the candidate. Researchers noted radiographic, neuropathological or clinical responses in 11 of the 12 patients, and median overall survival was 12.2 months. About 36% of the patients survived longer than 18 months, according to the biotech.
Treovir plans to start a Phase II trial later this year. G207 is also being studied at the University of Alabama-Birmingham and Children’s of Alabama in recurrent pediatric brain tumors, including medulloblastoma. — Nicole DeFeudis
Mirum and Vivet collaborate on liver disease gene therapy programs
Mirum has picked up exclusive license options on a couple of preclinical gene therapy programs.
The Foster City, CA-based biotech is teaming up with French outfit Vivet Therapeutics to potentially develop and commercialize gene therapy programs for progressive familial intrahepatic cholestasis, subtypes 3 and 2, the pair announced Monday. No financial terms were disclosed.
Vivet will remain responsible for the preclinical studies for the two programs, known as VTX-803 and VTX-802. Once those have been completed, Mirum can exercise options to lead clinical trials and commercialization. Mirum is still providing funding for the programs until that time.
The big idea behind the gene therapies is to correct the defective MDR3 transporter and bile salt export pump (BSEP) functions for PFIC3 and PFIC2, respectively. That, in turn, may ultimately provide a cure for patients living with the rare liver diseases.
“We are encouraged by the expertise and dedication of the Vivet team to develop next-generation gene therapies that have the potential to transform the lives of patients and their families,” Mirum CEO Chris Peetz said in a statement. “There are clear synergies in our combined missions to help the people who need it the most.” — Max Gelman
VBI Vaccines reports early data from Brii-partnered Hep B vaccine
Hepatitis B vaccine maker VBI Vaccines has new data out for their chronic HBV shot, and though the sample size is small, the biotech is hoping they’re strong enough to keep pushing forward.
In a Phase Ib/IIa trial from 33 evaluable patients, VBI observed a restimulation of T cell activity in reacting to hepatitis B antigens ranging from 50% to 78% across all cohorts, compared to no detectable response in the control group. The figures include patients taking 20 µg or 40 µg doses, with or without an adjuvant. P-values were not reported.
T cell and antibody responses were also consistent in both dosing groups, VBI added. VBI is developing the candidate with Brii Biosciences. — Max Gelman
ICER launches pilot program to update past assessments using real-world data
ICER is revisiting its 2018 assessments of three prophylactic treatments for hereditary angioedema (HAE) using real-world evidence collected through a partnership with health care tech company Aetion.
The institute said on Monday that updates are coming in July for its assessments of Takeda’s Takhzyro and Cinryze, as well as CSL Behring’s Haegarda. Back in 2018, ICER said all three treatments exceeded the threshold for cost-effectiveness ($50,000 to $150,000 per quality-adjusted life year gained).
“As ICER noted throughout our earlier assessment of these HAE therapies, our economic models were very sensitive to small changes in assumptions about the frequency of attacks, the amount of on-demand treatment required, and the exact dosing regimens of prophylactic therapy,” Jon Campbell, ICER’s senior VP of health economics, said in a statement. Now, he said, they’re trying to use real-world data to close some of these “information gaps.”
“By defining the baseline demographics and utilization characteristics of patients who start HAE prophylaxis, evaluating HAE attack rates before initiation of prophylaxis, and updating our health cost assumptions accordingly, ICER aims to further clarify the overall value of these HAE therapies in real-world settings,” Campbell said. — Nicole DeFeudis