Notizia - Recent Pharma, Healthcare and Biotech Happenings
Nov 05, 2024
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Prolong Pharmaceuticals, LLC, a clinical-stage biopharmaceutical company, announced that its investigational therapy, PP-007 (PEGylated carboxyhemoglobin, bovine), has been granted Fast Track designation by the FDA for the treatment of acute ischemic stroke. This designation highlights the FDA’s recognition of PP-007’s potential to address critical needs in stroke treatment. The therapy is currently under evaluation in the ongoing HEMERA-1 clinical trial in the United States, focused on assessing its safety and efficacy.
With over 700,000 ischemic strokes occurring each year in the U.S. alone, the demand for innovative stroke therapies remains high, especially given that around 50% of patients continue to experience lasting disabilities despite existing treatments like thrombolytic agents (TNKase® and Activase®) and mechanical thrombectomy (MT). Prolong Pharmaceuticals aims to address these limitations with PP-007, a therapy that not only improves oxygen delivery to hypoxic neurons but also has anti-inflammatory effects and a 24-hour duration of action, distinguishing it from other treatments in development.
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Ronald Jubin, Ph.D., Vice President of Early Development at Prolong Pharmaceuticals, emphasized the promise of PP-007, stating, “Receiving Fast Track designation underscores the groundbreaking potential of PP-007, backed by years of research. We believe no other stroke drug in development offers this unique combination: (1) opening collateral vessels, (2) selectively delivering oxygen to hypoxic neurons, (3) reducing inflammation, and (4) sustaining effects for 24 hours, as shown in pharmacokinetic studies with acute stroke patients. These capabilities, demonstrated in multiple PP-007 studies, are driving advances in stroke care and addressing critical unmet needs in acute ischemic stroke.”
In the ongoing HEMERA-1 trial, Principal Investigator Dr. Italo Linfante from Baptist Hospital in Miami noted positive patient outcomes. “We are increasingly encouraged by the promising 90-day mRS outcome measures achieved with PP-007 treatment in combination with IVT and MT, particularly as we expand enrollment to include patients with ASPECT scores ranging from 3 to 5,” he shared. Reflecting on World Stroke Day, Dr. Kirsten Gruis, Chief Medical Officer at Prolong Pharmaceuticals, added, “We are excited to closely collaborate with the FDA to expedite the development of PP-007 to improve survival and quality-of-life outcomes in AIS patients.”
Shorla Oncology announced that the FDA has expanded the approval of JYLAMVO (methotrexate) to include treatment for pediatric patients with acute lymphoblastic leukemia and polyarticular juvenile idiopathic arthritis. This expanded approval makes JYLAMVO the only oral liquid methotrexate on the market approved for both adult and pediatric use, marking a significant step forward in treatment accessibility for young patients with oncology and autoimmune needs.
Originally approved by the FDA for adult use, JYLAMVO has been utilized to treat conditions such as acute lymphoblastic leukemia, mycosis fungoides, non-Hodgkin lymphoma, rheumatoid arthritis, and severe psoriasis. Its liquid form provides a practical alternative for those who struggle with pill-based medications, broadening treatment options for patients with chronic health needs. The approval builds on JYLAMVO’s established role in adult care, now offering similar benefits to younger patients.
“This approval follows JYLAMVO’s successful use in adult patients and represents a critical step forward in addressing the unmet needs of pediatric care in oncology and autoimmune diseases,” said Sharon Cunningham, CEO of Shorla Oncology. “We are pleased to offer a convenient, patient-friendly alternative for both adult and pediatric patients in the U.S. as we continue to develop innovative solutions for those with limited treatment options.”
Adding further details on JYLAMVO’s design, Orlaith Ryan, Shorla Oncology’s Chief Technical Officer and co-founder, shared, “For patients with chronic conditions including cancer, JYLAMVO offers a convenient, palatable option for patients who may have difficulty swallowing pills. At Shorla, we are committed to continuity of supply of age-appropriate formulations for patients in need.” Additionally, Rayna Herman, Chief Commercial Officer of Shorla Oncology, highlighted JYLAMVO’s three-month stability at room temperature after opening, adding, “In addition to its palatable formulation, JYLAMVO eliminates the need for refrigeration, which brings greater ease to patients and families.”
Corcept Therapeutics Incorporated reported strong third-quarter results for 2024, driven by increased patient and physician adoption of its flagship product, KORLYM, which treats hypercortisolism. The company’s revenue rose to $182.5 million in Q3, up from $123.6 million in the same period last year, while net income reached $47.2 million. Corcept attributes this growth to greater awareness among physicians of the effects of hypercortisolism and the importance of early screening. The company’s support systems for KORLYM continue to play a vital role in helping patients maximize the benefits of the treatment. The company also raised its 2024 revenue guidance to between $675 and $700 million.
Looking ahead, Corcept anticipates a significant fourth quarter, with plans to submit a New Drug Application (NDA) for relacorilant as a treatment for patients with Cushing’s syndrome and to release clinical trial data across multiple programs. By year-end, the company expects results from its CATALYST study for KORLYM in patients with hypercortisolism and type 2 diabetes, as well as from its ROSELLA study for relacorilant combined with nab-paclitaxel in ovarian cancer. Corcept will also release findings from DAZALS, its Phase II trial for dazucorilant in patients with ALS. These clinical developments signal Corcept’s commitment to advancing treatment options for complex conditions linked to cortisol modulation.
Corcept CEO Dr. Joseph Belanoff highlighted the company’s growing footprint in hypercortisolism care, saying, “In the third quarter, we added more KORLYM prescribers and more patients received KORLYM treatment than ever before. Physicians are increasingly aware of hypercortisolism’s true prevalence and the negative health outcomes for patients who go untreated. Screening is becoming more common, and the number of patients receiving appropriate care continues to increase.” He underscored the importance of Corcept’s support services in optimizing patient outcomes, noting, “For patients and physicians who choose KORLYM, our extensive system of support services is critical to maximizing the benefit of our medication.”
Chief Development Officer Bill Guyer, PharmD, expressed optimism about relacorilant’s clinical potential, stating, “The positive results from our GRADIENT and GRACE studies in patients with Cushing’s syndrome highlight relacorilant’s promise as a significant advancement in treating this challenging disease. The data confirm clinically meaningful improvements in a broad range of hypercortisolism signs and symptoms without serious adverse effects.” Dr. Guyer also discussed the upcoming results from the ROSELLA and DAZALS trials, noting, “If ROSELLA replicates the positive outcomes of our Phase II study, relacorilant could become a transformative treatment for patients with platinum-resistant ovarian cancer.”
ESSA Pharma Inc. has announced the termination of its Phase II trial investigating masofaniten combined with enzalutamide for patients with metastatic castration-resistant prostate cancer who had not previously received second-generation antiandrogens. The decision was based on an interim analysis indicating a higher PSA90 response rate in patients on enzalutamide alone, the standard of care, compared to those on the combination therapy. Additionally, there was no observed efficacy benefit from combining masofaniten with enzalutamide. As a result, the company concluded the combination therapy was unlikely to meet the study’s primary endpoint.
Dr. David Parkinson, CEO of ESSA, explained, “Our commitment to delivering a meaningful clinical benefit to patients remains our highest priority, along with ensuring a robust safety profile. This decision, although difficult, was made in the best interest of patients and aligns with our clinical standards.” Parkinson expressed gratitude to those involved, adding, “We are incredibly thankful to our partners, trial investigators, and especially the patients and families who participated in this study. Their contributions are invaluable as we continue to seek effective prostate cancer treatments.”
The trial enrolled 52 patients from the United States, Canada, Australia, and France, aiming to compare the effects of the masofaniten-enzalutamide combination to enzalutamide monotherapy. Of those enrolled, 41 patients completed at least three months of follow-up, allowing the interim analysis to capture an early look at PSA response data. The safety profile of the combination therapy was consistent with previous studies, showing no new safety signals. ESSA’s leadership emphasized the decision was necessary to focus resources on therapies with greater potential benefits for patients.
Chairman Richard Glickman reaffirmed ESSA’s focus on strategic growth, stating, “The board, alongside senior management, is committed to preserving capital and exploring a range of options to maximize shareholder value.” Glickman emphasized that the company would carefully consider the best next steps, saying, “We remain dedicated to leveraging our resources wisely to continue our work in prostate cancer treatment and other potential opportunities for ESSA.”
The FDA has granted accelerated approval to Novartis’ SCEMBLIX (asciminib) for adult patients newly diagnosed with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). This decision is based on results from the Phase III ASC4FIRST trial, which demonstrated superior major molecular response (MMR) rates at 48 weeks for SCEMBLIX compared to investigator-selected standard of care (SoC) tyrosine kinase inhibitors (TKIs) such as imatinib, nilotinib, dasatinib, and bosutinib. Continued approval for this indication may be contingent upon confirmatory results from further clinical evidence.
The ASC4FIRST study showed nearly 20% more patients on SCEMBLIX reached MMR at 48 weeks compared to those on other SoC TKIs, and almost 30% more patients achieved MMR compared to imatinib alone. SCEMBLIX also demonstrated a favorable safety and tolerability profile, with fewer treatment-related adverse reactions (ARs), dose reductions, and discontinuations than other TKIs.
With this expanded indication, SCEMBLIX now becomes an option for a broader group of newly diagnosed Ph+ CML-CP patients, potentially improving long-term disease control. “Approvals of new first-line treatments are crucial for patients,” said Lee Greenberger, Chief Scientific Officer at The Leukemia & Lymphoma Society. “This option could allow patients to better manage their disease while minimizing disruptive side effects.” The most common adverse reactions reported included musculoskeletal pain, rash, fatigue, and headache, with no new safety issues observed.
Victor Bulto, President of Novartis US, expressed pride in the company’s commitment to CML treatment innovation: “With SCEMBLIX, we offer newly diagnosed patients a treatment option that balances efficacy and tolerability, possibly transforming the treatment journey for many living with CML.” The ASC4FIRST trial continues, with additional data expected at the 96-week mark.
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