PRA052 is a monoclonal antibody blocking CD30 ligand that has a pleiotropic effect on both innate and adaptive immunity and targets a pathway that is distinct from TNF -
- PRA052 is the second clinical candidate identified using the Prometheus360™ platform with one of the strongest genetic associations to IBD and is linked to a broad range of immune-mediated diseases -
- Initiation of Phase 1 study of PRA052 expected in 4Q 2022 -
The FDA has a relatively light calendar for October with Theratechnologies' Trogarzo for HIV, Alnylam's Lumasiran for advanced primary hyperoxaluria, scPharmaceuticals' Furoscix for heart failure, and more.
Here's a look at the agency's schedule for the month.
Theratechnologies' Trogarzo for HIV
Theratechnologies has a target action date of Oct. 3 for its supplemental Biologics License Application (sBLA) for an IV Push formulation of Trogarzo for HIV. Trogarzo is a CD4-directed post-attachment HIV-1 inhibitor. The new formulation is more convenient. It can allow an infusion within 30 seconds without dilution compared to the 15-minute infusion time of the original IV formulation.
The submission was based on results from the TMB-302 Phase III trial, which Theratechnologies' partner, TaiMed Biologics, ran. The study hit the primary endpoint with a 90% confidence interval of the ratio of IV Push to IV Infusion with no serious adverse events. It also hit two secondary endpoints, demonstrating no difference in HIV-1 viral load from the change to IV Push and no detection of anti-Trogarzo antibodies.
Alnylam's Lumasiran for Advanced Primary Hyperoxaluria Type 1
Alnylam Pharmaceuticals has a target action date of Oct. 6 for its sNDA for lumasiran to reduce plasma oxalate for patients with advanced primary hyperoxaluria type 1 (PH1). Under the brand name Oxlumo, the drug was approved by the FDA in 2020 for treatment of PH1 to lower urinary oxalate levels in pediatric and adult patients.
The sNDA was based on positive six-month data from the ILLUMINATE-C trial, which demonstrated the drug substantially decreased plasma oxalate levels in patients with compromised renal function from PH1, including patients on hemodialysis.
scPharmaceuticals' Furoscix for Worsening Heart Failure
scPharmaceuticals has a target action date of Oct. 8 for the resubmission of its NDA for Furoscix. The drug is a proprietary formulation of furosemide delivered by an on-body infusor to treat congestion in patients with worsening heart failure. The drug is formulated to a neutral pH, which allows for subcutaneous infusion via a wearable pre-programmed on-body drug delivery system allowing for outpatient self-administration.
"The resubmission and acceptance of our Furoscix NDA was a significant achievement for our company and potentially gives us line-of-sight to transitioning to a commercial-stage company," stated John Tucker, CEO of scPharmaceuticals. "We believe the NDA addresses all concerns and questions raised in the Complete Response Letter that we received from the FDA in December 2020. As a result, we are preparing for a commercial launch of FUROSCIX, if approved, in the fourth quarter of this year."
Harrow's AMP-100 for Eye Anesthesia and Pain Management
Harrow Health had a target action date of Oct. 16 for its NDA for AMP-100. The drug was being considered for ocular surface anesthesia and intraoperative pain management during ocular surgery. On Sept. 27, the FDA approved it for that indication under the brand name Iheezo (chloroprocaine hydrochloride ophthalmic gel) 3%.
Harrow Health acquired the drug from Sintetica in July 2021. The NDA was based on data from a Phase III trial that successfully compared its efficacy and tolerability to current standards of care.
"We have always believed in the unique clinical value of Iheezo, and now that Iheezo is approved for use in the U.S. market, it has the potential to become an indispensable premium tool for eyecare professionals and their patients requiring ocular surface anesthesia," stated Mark L. Baum, chairman and CEO of Harrow.
Ipsen's Palovarotene for Heterotopic Ossification with Fibrodysplasia Ossificans Progressiva
The FDA's Endocrinologic and Metabolic Drugs Advisory Committee is meeting on Oct. 31 to discuss Ipsen Biopharmaceuticals' NDA for palovarotene capsules. The proposed indications are for the prevention of heterotopic ossification in adults and children, specifically females eight years and older and males 10 years and older, with fibrodysplasia ossificans progressiva (FOP).
FOP is an ultra-rare genetic disorder that results in bone forming where it shouldn't, such as outside of the skeleton and in the soft and connective tissues like the muscles, tendons and ligaments. Bones forming outside the skeleton are called heterotopic ossification.
The FDA has assigned a target action date of Dec. 29 for the application after the company resubmitted the NDA for Priority Review. The NDA was originally accepted on May 28, 2021; then the company withdrew it on Aug. 13, 2021.
TNX-601 ER, Tianeptine Hemioxalate, is an Extended-Release Tablet for Once-a-Day Dosing
Phase 2 Clinical Trial of TNX-601 ER Expected to Start First Quarter 2023
https://finance.yahoo.com/news/tonix-pharmaceuticals-announces-ind-clearance-110000875.html
Enrollment ongoing in Phase 2 study evaluating sapanisertib, a dual mTORC1/2 inhibitor, in patients with squamous non-small cell lung cancer harboring a NRF2 mutation
https://finance.yahoo.com/news/calithera-receives-fda-fast-track-110000872.html
The primary endpoint of adjusted ALSFRS-R and secondary endpoints of CAFS and SVC were not met at 24 weeks
Prespecified exploratory analyses of the secondary survival endpoint for the 30 mg dose demonstrated a >90% reduction in risk of death or risk of death/permanently assisted ventilation at 24 weeks
Survival signal consistent with prior results from the Phase 2 RESCUE-ALS trial
Clene will continue the open-label extension of CNM-Au8 in the Healey ALS Platform Trial and is in discussions with the Healey & AMG ALS Center to design and offer an Expanded Access Protocol (EAP) of CNM-Au8 30mg for eligible participants of closed regimens and others
Clene is pursuing multiple paths, including ongoing discussions with potential strategic partners, in its goal of marketing authorization
Clene to host investor call and webcast at 8:30 am EDT today
Clene will host a conference call and webcast at 8:30 am EDT to discuss the Healey ALS Platform trial topline results for CNM-Au8. Members of Clene’s executive team will lead the discussion.
Time and Date: 8:30 a.m. EDT on Oct. 3, 2022
Investors: 1 (888) 660-6179 (toll-free) or 1 (929) 203-1946 (toll)
Conference ID: 5318408
Press *1 to ask or withdraw a question, or *0 for operator assistance.
To access the live webcast, please register online at this link. Participants are requested to register at a minimum 15 minutes before the start of the call. A replay of the call will be available two hours after the call and archived on the same web page for six months. A live audio webcast of the call will be available on the Investors section of the Company’s website Events page. An archived webcast will be available on the Company’s website approximately two hours after the event.
https://finance.yahoo.com/news/clene-reports-topline-results-demonstrating-120000314.html
TRITON3 study evaluating Rubraca monotherapy versus chemotherapy or second-line androgen deprivation therapy in patients with metastatic castration-resistant prostate cancer (mCRPC) with mutations in BRCA or ATM achieved the primary endpoint of improved radiographic progression-free survival (rPFS) by independent radiology review (IRR)
– Median rPFS of 11.2 months for Rubraca vs 6.4 months for control group in the BRCA subgroup
– Median rPFS of 10.2 months for Rubraca vs 6.4 months for control group in the ITT population (inclusive of all patients with a BRCA or ATM mutation enrolled in TRITON3)
Safety profile of Rubraca observed in TRITON3 was consistent with Rubraca labelling
TRITON3 is the confirmatory study for Rubraca’s current US accelerated approval in mCRPC and will also serve as the basis of a supplemental New Drug Application (sNDA) for US label expansion to be submitted during Q1 2023
TRITON3 data have been submitted for presentation at the Prostate Cancer Foundation Annual Scientific Retreat later this month and will also be submitted for presentation at a 2023 medical meeting
PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing a growing pipeline of targeted immunotherapies for cancer and infectious disease, today announced that it has successfully completed an End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) for PDS0101 in combination with Merck’s anti-PD-1 therapy, KEYTRUDA® (pembrolizumab) for the treatment of unresectable, recurrent/metastatic human papilloma virus (HPV) 16-positive head and neck squamous cell carcinoma (HNSCC).
https://finance.yahoo.com/news/pds-biotech-announces-successful-end-120000206.html
