Notizia - Recent Pharma, Healthcare and Biotech Happenings
May 06, 2025
Table of Contents
The FDA approved Abeona Therapeutics’ ZEVASKYN (prademagene zamikeracel, or pz-cel), the first and only autologous cell-based gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB). ZEVASKYN is designed as a single-application surgical treatment using gene-corrected skin cell sheets to promote long-term healing in large, chronic wounds. The approval is based on data from the pivotal Phase III VIITAL study, which showed significant healing and pain reduction compared to standard care.
“This marks a pivotal moment for the RDEB community,” said Abeona CEO Vish Seshadri. “ZEVASKYN can offer long-term relief from severe, painful wounds after a single application.” Jean Tang, M.D., principal investigator of the VIITAL™ study, added, “We’ve seen years of healing from just one treatment. This is a meaningful step forward for patients.” Brett Kopelan, Executive Director of debra of America and a parent of a child with RDEB, emphasized that ZEVASKYN could transform the standard of care.
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ZEVASKYN will become available in Q3 2025 at Qualified Treatment Centers across the U.S. The therapy delivers functional collagen via genetically modified autologous keratinocytes and has shown long-term benefits in follow-up studies lasting up to 8 years. Abeona received a Rare Pediatric Disease Priority Review Voucher with the approval and plans to monetize it. The company also launched Abeona Assist™, a support program for patient access, insurance navigation, and logistical aid.
“This is a scientific triumph,” said Dr. Joyce Teng. “ZEVASKYN brings long-awaited innovation to patients with RDEB.” Dr. Marissa Perman from Children’s Hospital of Philadelphia noted, “It’s a milestone for helping these patients live fuller, less painful lives.” The broader EB community, including organizations like EB Research Partnership, praised Abeona for delivering a groundbreaking treatment and reaffirmed their commitment to supporting access and continued innovation.
AbbVie’s RINVOQ (upadacitinib) has become the first and only oral Janus Kinase (JAK) inhibitor approved by the FDA for treating adults with giant cell arteritis (GCA). The once-daily 15 mg tablet is now indicated for GCA following its recent marketing authorization by the European Commission. The approval is supported by results from the pivotal Phase III SELECT-GCA trial, where 46.4% of patients on RINVOQ with a 26-week steroid taper achieved sustained remission from week 12 to 52, compared to 29% on placebo with a 52-week taper (p=0.002). This marks RINVOQ’s ninth approved indication in the U.S., spanning rheumatology, gastroenterology, and dermatology.
“This FDA approval will now provide an alternative treatment option that can offer patients with GCA the possibility of tapering off steroids and achieving sustained remission,” said Roopal Thakkar, M.D., EVP of R&D and Chief Scientific Officer at AbbVie. “With this new indication, we continue our efforts to address unmet needs in immune-mediated diseases.”
GCA, also known as temporal arteritis, is the most common form of vasculitis in adults, particularly affecting women over 50, especially those aged 70 to 80. It is an autoimmune condition that inflames large and medium arteries, potentially leading to serious outcomes such as blindness, stroke, or aortic aneurysm if untreated. While glucocorticoids are standard therapy, they are associated with significant toxicity and frequent relapse. The safety profile of RINVOQ in the SELECT-GCA trial was generally consistent with its known risks, including serious infections, cardiovascular events, certain cancers, and blood clots, especially in adults over 50 with preexisting heart disease risk factors.
“Glucocorticoids remain a mainstay of GCA treatment, but the side effects and relapse rates are concerning,” said Peter A. Merkel, M.D., MPH, chief of rheumatology at the University of Pennsylvania and SELECT-GCA investigator. “The trial results demonstrate that upadacitinib gives patients a real chance at sustained remission with reduced steroid exposure.”
Lantern Pharma has received FDA clearance for its Investigational New Drug (IND) application for LP-184 in the treatment of triple-negative breast cancer (TNBC), enabling a Phase Ib/II clinical trial to commence. LP-184 is a novel small molecule developed using Lantern’s proprietary AI platform, RADR®. The trial will evaluate LP-184 both as a monotherapy and in combination with the PARP inhibitor olaparib in patients with recurrent, advanced-stage TNBC. The IND clearance builds on LP-184’s momentum, having already received FDA Orphan Drug Designation in 2023 and Fast Track Designation in 2024. With metastatic TNBC patients facing an average survival of just 10–18 months, LP-184 aims to address a high unmet need in a market valued at over $4 billion annually.
“This IND clearance for LP-184 in a Phase Ib/II study represents a pivotal advancement in our mission to bring precisely targeted, AI-developed medicines to patients with aggressive cancers and limited treatment options,” said Panna Sharma, CEO and President of Lantern Pharma. “The strategic design of our clinical program reflects both the compelling mechanistic rationale and the encouraging data supporting LP-184’s potential in TNBC.”
The clinical program will feature a dual-arm trial structure: a monotherapy arm to optimize dosage in around 30 advanced-stage TNBC patients, and a combination arm targeting BRCA-mutated TNBC patients in a second-line setting. LP-184 is designed to cause DNA double-strand breaks via bioactivation by PTGR1, a mechanism particularly suited for tumors with homologous recombination deficiency (HRD), which characterizes nearly 70% of TNBCs. Preclinical studies have demonstrated strong efficacy, including tumor regression in both PARP-sensitive and -resistant models. LP-184’s ability to penetrate the blood-brain barrier also highlights its potential for treating brain metastases, a frequent complication in TNBC patients.
“Treating patients earlier with a combination of LP-184 and a PARP inhibitor like olaparib could provide deeper, more durable responses, especially in BRCA-mutated and PARP-resistant TNBCs,” said Sharma. “Our goal is to extend survival and improve outcomes where current therapies fall short.”
Lantern’s global clinical strategy includes trial sites in the U.S., India, and Nigeria, regions with high TNBC incidence and clinical demand. Beyond TNBC, LP-184 is being evaluated for potential in multiple solid tumors with DNA damage repair deficiencies, including lung, bladder, ovarian, and pancreatic cancers. Lantern is also planning additional investigator-initiated trials to explore these avenues. With a strong preclinical foundation and regulatory momentum, LP-184 is positioned as a next-generation precision oncology candidate poised to impact several high-need cancer segments.
Ichnos Glenmark Innovation, a clinical-stage biotechnology company advancing novel multispecific antibody therapies, announced that the FDA has granted Fast Track Designation for its investigational trispecific T-cell engager, ISB 2001, for the treatment of adult patients with relapsed or refractory multiple myeloma (RRMM). This designation underscores the urgent need for innovative therapies in heavily pretreated patients who have exhausted prior lines of therapy, including proteasome inhibitors, immunomodulatory agents, and anti-CD38 monoclonal antibodies.
ISB 2001, which targets BCMA, CD38, and CD3, was developed using IGI’s proprietary BEAT protein platform. The molecule is designed to improve tumor targeting, enhance avidity at low antigen expression, and reduce off-tumor toxicity—a critical limitation of existing bispecifics.
The Phase I dose-escalation results, previously presented at ASH 2024, showed promising overall response rates (ORR) and a favorable safety profile in heavily pretreated RRMM patients. Full data will be presented at the 2025 ASCO Annual Meeting (Abstract #7514) on June 2, 2025, during a rapid oral session in the Hematologic Malignancies – Plasma Cell Dyscrasia track.
“This Fast Track designation represents a crucial regulatory milestone in our journey to deliver a first-in-class trispecific therapy to RRMM patients who urgently need new options,” said Dr. Cyril Konto, President and CEO of IGI. “ISB 2001’s innovative design allows us to target tumors more effectively while minimizing toxicity, particularly in patients who have failed prior T-cell–directed therapies.”
ISB 2001 was also granted Orphan Drug Designation by the FDA in July 2023. The dose-expansion phase of the ongoing Phase I clinical trial (NCT05862012) is now enrolling patients in the U.S. and Australia.
With nearly all RRMM patients facing eventual disease progression and no current cure, ISB 2001 is positioned to fill a critical therapeutic gap for patients who have previously received CAR T-cell therapies or bispecifics.
Rezolute, Inc., a late-stage biopharmaceutical company focused on advancing therapies for rare metabolic diseases, today announced that the FDA has granted Breakthrough Therapy Designation (BTD) to its investigational therapy, ersodetug, for the treatment of hypoglycemia caused by tumor-induced hyperinsulinism.
This second BTD follows a prior designation for congenital HI and was awarded based on compelling clinical trial data and real-world evidence from patients successfully treated under the Company’s Expanded Access Program (EAP). The designation highlights the potential of ersodetug to address persistent, treatment-resistant hypoglycemia, which remains a major barrier to managing tumor HI, especially in patients who are not candidates for surgery or other tumor-targeted therapies.
“This Breakthrough Therapy Designation from the FDA reaffirms ersodetug’s unique mechanism of action and its clinical promise in managing tumor HI, a serious and often intractable condition,” said Nevan Charles Elam, CEO and Founder of Rezolute. “It represents a significant milestone in our mission to bring meaningful innovation to patients suffering from rare and underserved forms of hyperinsulinism.”
The Company plans to initiate a registrational clinical trial for tumor HI in mid-2025, with topline results expected in the second half of 2026. Rezolute will also leverage its BTD status to engage with the FDA on study design and regulatory requirements for a Biologics License Application (BLA) that would potentially expand the indication for ersodetug.
Ersodetug, a first-in-class therapeutic targeting the underlying mechanisms of hyperinsulinism, is already in a Phase III trial for congenital HI, and its development program now positions it as a leading candidate for both congenital and tumor-related HI.
The Breakthrough Therapy Designation is granted to expedite the development and review of investigational drugs for serious conditions where preliminary clinical evidence suggests a substantial improvement over available therapies.
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