In the United States, KEYTRUDA has received additional approvals for cervical cancer treatment. It is authorized for use in combination with chemotherapy, with or without bevacizumab, to treat patients with persistent, recurrent, or metastatic cervical cancer whose tumors express PD-L1 (Combined Positive Score [CPS] ≥1) as determined by an FDA-approved test. Additionally, KEYTRUDA is approved as a standalone treatment for patients with recurrent or metastatic cervical cancer whose disease has progressed after chemotherapy, and whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test.
FDA Approves Merck’s KEYTRUDA Plus Chemoradiotherapy as Treatment for Patients With FIGO 2014 Stage III-IVA Cervical Cancer
Bayer AG and Asklepios BioPharmaceutical, Inc., a gene therapy company wholly owned by Bayer AG and functioning independently as its subsidiary, have announced the commencement of the GenePHIT (Gene PHosphatase Inhibition Therapy) Phase II trial for AB-1002 (also referred to as NAN-101) in treating congestive heart failure (CHF). This adaptive, double-blind, placebo-controlled, randomized, multicenter trial aims to assess the safety and effectiveness of a single intracoronary infusion of AB-1002 in adults with non-ischemic cardiomyopathy classified as New York Heart Association (NYHA) Class III Heart Failure, provided they have maintained medical stability for a minimum of 4 weeks. The progression of AB-1002 to Phase II represents a noteworthy milestone in advancing this innovative investigational gene therapy for CHF patients facing substantial unmet medical needs.
GenePHIT will encompass a cohort of 90 to 150 adults exhibiting a left ventricle ejection fraction ranging from 15% to 35%. These individuals persistently experience symptoms of heart failure despite adhering to therapy recommended by guidelines. The main measure of effectiveness, evaluated at the 52-week mark, will be a modified win ratio based on various clinically significant assessments.
Jude Samulski, PhD, Co-Founder and Chief Scientific Officer at AskBio, expressed enthusiasm about the launch of the GenePHIT initiative led by Roger Hajjar, MD, Scientific Chair CHF, and Lothar Roessig, MD, Integrated Product Team Lead CHF. According to Samulski, the trial holds promise in assessing the potential of AB-1002 as a treatment for congestive heart failure, a highly debilitating condition. The company anticipates gaining valuable insights into this investigational cardiac gene therapy and envisions a future where AB-1002 may offer hope to patients dealing with congestive heart failure.
AB-1002 is a gene therapy under investigation, lacking approval from any regulatory authority, and its effectiveness and safety remain unverified and not fully assessed. Viralgen Vector Core, a subsidiary entirely owned and independently operated by AskBio, produces AB-1002.
SELLAS Life Sciences Receives FDA Fast Track Designation for SLS009 for Treatment of Relapsed/Refractory Acute Myeloid Leukemia
SELLAS Life Sciences Group, Inc., a biopharmaceutical company in the late stages of clinical development with a focus on creating innovative treatments for various cancer types, has revealed today that the FDA has awarded Fast Track Designation to SLS009 (previously known as GFH009). This novel and exceptionally targeted CDK9 inhibitor is intended for the treatment of relapsed/refractory (r/r) acute myeloid leukemia (AML). The Fast Track Designation aims to streamline the development and evaluation process of drugs designed to address severe conditions and meet a medical need that is currently unfulfilled.
Securing Fast Track Designation for SLS009 in the context of refractory/relapsed Acute Myeloid Leukemia (r/r AML), alongside the recent Orphan Drug Designation for the same indication, emphasizes the promising prospects of SLS009 and underscores the urgent demand among AML patients facing a grim prognosis due to the disease’s advancing nature,” said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. “The initial positive findings from the Phase IIa study at the 45 mg (safety) dose level demonstrate that SLS009 when combined with venetoclax and azacitidine (aza/ven), exhibits anti-leukemic effects while maintaining a favorable safety profile in AML patients resistant to venetoclax combination therapies. Notably, as of the latest follow-up, eight out of the nine patients in the 45 mg cohort remain alive. The first patient enrolled in the study achieved a complete response (CR) and is continuing the study into the seventh month with full peripheral blood recovery. The second patient, enrolled six months ago, further underscores the potential benefits of integrating CDK9 inhibition into the aza/ven regimen. We have also enrolled multiple patients in the ongoing 60 mg dose cohort. Our team is dedicated to advancing the development of SLS009 with the objective of offering effective solutions to patients seeking viable treatment options.
The Phase IIa clinical trial for SLS009 is a multi-center study with an open-label, single-arm design. Its purpose is to assess the safety, tolerability, and effectiveness of SLS009 at two different dose levels, namely 45 mg and 60 mg when combined with aza/ven. Within the 60 mg dose group, patients will be randomly assigned to one of two subgroups: a fixed 60 mg dose administered once per week, or a fixed 30 mg dose given twice per week. Each subgroup is expected to enroll 5 to 10 patients. The primary focus, in addition to evaluating the safety and tolerability of SLS009 when used in conjunction with aza/ven, will be on the composite complete response rate (CRc) and the duration of response (DOR).
The company anticipates sharing further information from the entirely enrolled 45 mg (safety dose level) group and the initial data from the 60 mg (recommended Phase II dose level) group in the first quarter of 2024. Additionally, the analysis of the 60 mg cohort is projected to take place in the second quarter of 2024.
RemeGen’s RC88 Obtained FDA Fast Track Designation, Heralds New Hope for Ovarian Cancer Patients
On China, Jan. 12, 2024, RemeGen announced that the US FDA has granted Fast Track Designation (FTD) to its independently developed antibody-drug conjugate (ADC), RC88, targeting mesothelin (MSLN). This designation is for the treatment of platinum-resistant recurrent epithelial ovarian, fallopian tube, and primary peritoneal cancers. This achievement follows the FDA’s approval of RC88’s international multicenter Phase II clinical trials just last month, marking another significant milestone for the company.
Comprising a recombinant humanized anti-MSLN monoclonal antibody connected to the microtubule inhibitor monomethyl auristatin E (MMAE), RC88 functions as a potent microtubule inhibitor. Demonstrating a notable affinity for MSLN, it selectively binds to tissues overexpressing MSLN and effectively exhibits a pronounced inhibitory effect on tumor cells with diverse levels of MSLN expression.
The company is positioned to commence international multicenter Phase II clinical trials spanning the United States, China, the European Union, and other regions. The primary goal is to gain additional insights into the optimal dosage, efficacy, and safety profile of RC88 monotherapy through these studies.
Reflecting on this process, Dr. Jianmin Fang, CEO of RemeGen, said, “The FDA’s FTD accelerates the development and review process of RC88, which affirms our commitment to pioneering treatments that address the urgent needs of those facing challenging disease. Moving forward, RemeGen will continue to accelerate the development of its ADC products, with the aim of bringing more and better solutions to patients globally.”
Epithelial ovarian cancer (EOC) stands as the primary contributor to cancer-related fatalities in women, frequently diagnosed at advanced stages and susceptible to relapse within a two-year timeframe, progressing from platinum-sensitive to resistant states. As ovarian cancer advances to more advanced and aggressive stages, the expression rate of MSLN tends to escalate. Current therapeutic choices are constrained, emphasizing the urgent need for innovative solutions. RC88 stands out due to its precise targeting of MSLN, offering a groundbreaking approach to tackle the intricate nature of this formidable medical challenge.
The approval and introduction of innovative therapies like RC88 are poised to bring about a significant transformation in the landscape of ovarian cancer treatment. As these advancements continue to unfold, the potential impact on the prognosis, quality of life, and overall outcomes for patients battling ovarian cancer is highly anticipated. The introduction of novel treatments, exemplified by RC88, represents a promising stride towards more effective and tailored therapeutic options for those affected by this complex and challenging condition.
Shorla Oncology Announces FDA Filing Acceptance of New Drug Application for Novel Formulation to Treat Breast and Ovarian Cancer
Shorla Oncology announced on January 09, 2024, that the US FDA has accepted to review the company’s New Drug Application (NDA) for a groundbreaking formulation designed to address breast cancer and ovarian cancer. The FDA has set a Prescription Drug User Fee Act (“PDUFA”) action date of June 29th, 2024, signifying the anticipated timeline for the completion of the regulatory review process.
SH-105 represents a ready-to-dilute version of a long-established drug that has been available in freeze-dried powder form since the 1950s, occasionally experiencing supply shortages. SH-105 formulation is designed for the treatment of adenocarcinoma of the breast or ovary. The liquid form of SH-105 obviates the necessity for reconstituting powder, enhancing operational efficiency, and mitigating the risks associated with the intricate preparation process.
“This innovative drug (‘SH-105’) will offer hospital pharmacists and patients access to a differentiated, ready-to-administer, injectable product with unique characteristics that’s expected to facilitate rapid adoption once approved,” said Orlaith Ryan, Chief Technical Officer and Co-Founder of Shorla Oncology.”
“This is an important step in improving access to and administration of a drug that will help women suffering from breast and ovarian cancer,” said Sharon Cunningham, Chief Executive Officer and Co-Founder of Shorla Oncology. “It also marks a significant milestone regarding Shorla’s efforts to bring innovative oncology products to market.”
SH-105 stands among a range of oncology drugs in Shorla’s advanced pipeline. Following a successful Series B funding round that raised $35 million, Shorla is well-positioned to expedite the expansion of its oncology portfolio. In the previous year, the company introduced Nelarabine, designed for the treatment of T-cell Leukemia, and JYLAMVO, recognized as the inaugural oral methotrexate solution sanctioned in the United States for adult use in treating acute lymphoblastic leukemia and other applicable indications.
According to the American Cancer Society, it is estimated that over 350,000 women will receive a diagnosis of breast cancer in the United States in 2023. Additionally, approximately 19,710 women are expected to be diagnosed with ovarian cancer in the same time period. The ongoing therapeutic development is anticipated to immensely transform breast cancer and ovarian cancer in the upcoming years.
Tonix Pharmaceuticals Presents Additional Data Highlighting the Favorable Tolerability and Differentiated Side Effect Profile of TNX-102 SL in Second Positive Phase 3 Clinical Trial for the Management of Fibromyalgia
On January 9, 2024, Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company) announced the presentation of supplementary safety and tolerability data derived from RESILIENT, the second positive Phase 3 study assessing TNX-102 SL (cyclobenzaprine HCl sublingual tablets) for the treatment of Fibromyalgia. This presentation took place at Biotech Showcase™ 2024 in San Francisco from January 8 to 10.
TNX-102 SL stands as a patented sublingual tablet formulation of cyclobenzaprine hydrochloride, offering swift transmucosal absorption and reduced production of a long half-life active metabolite, norcyclobenzaprine, thanks to its bypass of first-pass hepatic metabolism. Serving as a multifunctional agent with potent binding and antagonist activities at the 5-HT2A-serotonergic, α1-adrenergic, H1-histaminergic, and M1-muscarinic cholinergic receptors, TNX-102 SL is currently undergoing development as a daily bedtime treatment for Fibromyalgia, Fibromyalgia-type Long COVID (formerly known as post-acute sequelae of COVID-19 [PASC]), alcohol use disorder, and agitation in Alzheimer’s disease.
In presenting more detailed data from the RESILIENT study, Seth Lederman, M.D., President and Chief Executive Officer of Tonix, said, “The results showed that TNX-102 SL treatment was not associated with increases in systolic or diastolic blood pressure or body weight, nor were there any reported sexual side effects. In fact, when systematically investigated using the Changes in Sexual Functioning Questionnaire short form (CSFQ-14), women who received study drug had a higher CSFQ-14 score relative to those who received placebo consistent with improved sexual function. These are important tolerability factors for Fibromyalgia patients on long-term therapies, particularly since weight gain is associated with gabapentinoids, negative sexual side effects are associated with serotonin-reuptake inhibiting medications, and increased blood pressure is associated with potent noradrenergic-reuptake inhibiting medications.”
Dr. Lederman added, “We believe that the data from our two positive Phase 3 studies, with clinically meaningful separation from placebo on pain, sleep, and fatigue, show that Fibromyalgia can be successfully treated by TNX-102 SL 5.6 mg and may provide the opportunity for Tonix to launch the first FDA-approved drug for Fibromyalgia in more than a decade.”
The RESILIENT study is a double-blind, randomized, placebo-controlled trial designed to assess the efficacy and safety of TNX-102 SL (cyclobenzaprine HCl sublingual tablets) for the management of Fibromyalgia. This two-arm trial involved the randomization of 457 participants across 33 sites in the U.S. The initial two weeks of treatment included a run-in period, during which participants were initiated on either TNX-102 SL 2.8 mg (1 tablet) or a placebo. Subsequently, all participants escalated their dosage to TNX-102 SL 5.6 mg (2 x 2.8 mg tablets) or two placebo tablets for the remaining 12 weeks.
As per the updates, RESILIENT successfully achieved its pre-specified primary endpoint by significantly reducing daily pain compared to the placebo (p=0.00005) in individuals with Fibromyalgia. Additionally, statistically significant and clinically meaningful outcomes were observed in all crucial secondary endpoints, addressing improvements in sleep quality, fatigue reduction, and overall enhancement of Fibromyalgia symptoms and function. Tonix has outlined plans to submit a New Drug Application (NDA) to the US FDA in the latter half of 2024 for TNX-102 SL, seeking approval for the management of Fibromyalgia.
The inaugural Phase 3 trial of TNX-102 SL 5.6 mg in Fibromyalgia, known as RELIEF, concluded in December 2020. It successfully achieved its pre-defined primary endpoint by significantly reducing daily pain compared to the placebo (p=0.010). Additionally, the trial demonstrated efficacy in key secondary endpoints, marking a positive outcome in the evaluation of TNX-102 SL for Fibromyalgia management.
Fibromyalgia is a persistent pain condition thought to stem from increased sensitivity and abnormal pain signaling in the central nervous system. It affects an estimated 6 million to 12 million adults in the United States, with a notably higher incidence among women. Current treatment options for Fibromyalgia often leave both healthcare providers and patients dissatisfied, highlighting the pressing need for more effective and tailored therapeutic approaches. However, several major pharma and biotech companies are actively working in the Fibromyalgia therapeutic landscape to immensely transform the treatment landscape.
