The FDA approval is granted based on encouraging findings from the Phase III OUtMATCH trial, which assessed the effectiveness of Xolair in individuals aged 1 to 55 with peanut allergies and two or more additional food allergies, such as milk, egg, wheat, cashew, hazelnut, and walnut. The OUtMATCH investigation is supported and financed by the National Institute of Allergy and Infectious Diseases (NIAID), a division of the National Institutes of Health (NIH). It is being carried out by the NIAID-backed Consortium for Food Allergy Research (CoFAR) at 10 medical centers throughout the United States. The initiative is led by the Johns Hopkins Children’s Center and jointly led by the Stanford School of Medicine. Detailed findings from the trial will be presented at a special session during the 2024 American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting on Sunday, February 25.
Iovance’s AMTAGVI Receives FDA Accelerated Approval for Advanced Melanoma
Iovance Biotherapeutics, Inc., a biotech firm dedicated to pioneering, developing, and providing innovative polyclonal tumor-infiltrating lymphocyte (TIL) cell therapies for individuals with cancer, has declared that the FDA has approved AMTAGVI (lifileucel) suspension for intravenous infusion. AMTAGVI is an autologous T cell immunotherapy derived from tumors, designed for the treatment of adult patients with unresectable or metastatic melanoma who have previously received treatment with a PD-1 blocking antibody, and if positive for BRAF V600 mutation, a BRAF inhibitor with or without a MEK inhibitor. This approval is granted under an accelerated pathway based on the overall response rate (ORR) and duration of response. Additionally, Iovance is currently conducting TILVANCE-301, a Phase III trial aimed at validating clinical efficacy.
AMTAGVI stands out as the pioneer and sole one-time, personalized T-cell therapy sanctioned by the FDA for treating solid tumor cancer. Its innovative approach hinges on leveraging patient-specific T cells known as TIL cells. These cells are naturally produced by the immune system in response to cancer, targeting and eliminating cancerous cells by identifying unique markers on their surfaces. However, when cancer progresses, the body’s native TIL cells become ineffective in combatting the disease.
AMTAGVI is manufactured through an exclusive method designed to harvest and multiply a patient’s distinct T cells extracted from a segment of their tumor. Afterward, billions of these T cells are reintroduced into the patient’s system to combat their cancer. Approved Treatment Centers (ATCs) will oversee the administration of AMTAGVI to patients within a treatment plan involving lymphodepletion and a brief period of high-dose PROLEUKIN® (aldesleukin).
Every year, around 8,000 individuals in the United States succumb to melanoma. Up to this point, there haven’t been any treatments endorsed by the FDA for individuals with advanced melanoma that have worsened after initial treatment with an immune checkpoint inhibitor and, if suitable, targeted therapy. The approval by the FDA is founded on the safety and effectiveness findings from the C-144-01 clinical trial. This trial, conducted globally across multiple centers, explores AMTAGVI in patients with advanced melanoma who were previously treated with anti-PD-1 therapy and targeted therapy, as appropriate.
Astellas and Kelonia Therapeutics Enter into Research and License Agreement to Develop Novel Immuno-Oncology Therapeutics
Astellas Pharma Inc. and Kelonia Therapeutics have jointly announced a partnership wherein Xyphos Biosciences, Inc. and Kelonia will collaborate under a research and licensing agreement to advance innovative Immuno-Oncology treatments. Kelonia, known for its pioneering work in genetic medicines, employs a groundbreaking in vivo gene placement system called iGPS®, utilizing advanced lentiviral particles to precisely deliver genetic material to specific target cells within patients. Meanwhile, Xyphos possesses a unique and patented ACCELTM technology platform, which integrates its convertibleCAR® (convertible Chimeric Antigen Receptor) onto immune cells for therapeutic purposes.
According to the agreement, Kelonia and Xyphos aim to merge Kelonia’s iGPS® with Xyphos’ ACCELTM technology for the advancement of innovative in vivo CAR-T Cell therapies, focusing on up to two initiatives. Xyphos will lead the development and commercialization of the resulting products from their joint research efforts. In return, Kelonia will receive an initial payment of US $40 million for the first program, with an additional US $35 million if Xyphos chooses to pursue the second program, along with potential milestone payments totaling nearly US $800 million. Kelonia will also receive research and development funding for its contributions to the collaboration and stands to gain tiered royalties on net sales, potentially reaching a double-digit percentage.
“Our iGPS platform holds promise for significantly advancing cancer treatment. By merging Kelonia’s in vivo gene delivery expertise with the ACCELTM convertible CAR technology, we are forming an optimal fusion of technologies to produce readily available, universally applicable CAR-T Cell therapies. We believe this approach holds revolutionary potential and are committed to collaborating closely with Astellas to maximize its benefits for patients in critical need.”
Kevin Friedman, Ph.D., CEO and Founder of Kelonia
Certa Therapeutics’ FT011 Granted US FDA Fast Track for the Treatment of Systemic Sclerosis
On February 19, 2024, Certa Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its pioneering therapy, FT011. This designation comes as a significant milestone in Certa’s journey toward treating systemic sclerosis (scleroderma), a challenging condition. Notably, the therapy had previously received an Orphan Drug Designation, underscoring its potential to address unmet medical needs in rare diseases.
The granting of Fast Track Designation stemmed from findings of the Phase 2 study, as previously disclosed, demonstrating that administering FT011 to scleroderma patients for 12 weeks led to a significant clinical enhancement. Specifically, the study revealed that 60% of patients receiving FT011 at a dosage of 400mg experienced meaningful improvement, while 20% of those in the FT011 200mg group showed similar benefits, compared to a mere 10% in the placebo cohort.
Professor Darren Kelly Certa Therapeutics CEO and founder said, “We are thrilled to have received Fast Track Designation which supports further acceleration of the FT011 clinical development program. It also provides validation of FT011’s potential to offer patients with scleroderma the first anti-fibrotic and disease-modifying treatment of this type. We know that this debilitating and life-threatening disease can severely impact the lives of patients and to date, existing treatments only focus on the relief and management of symptoms, whereas FT011 precisely targets the root cause of fibrosis and has the potential to offer treatment across multiple organs within these patients.”
FT011 represents a groundbreaking, pioneering oral medication designed to address chronic fibrosis across various organs. It focuses on targeting a crucial yet previously untargeted membrane GPCR receptor known as GPR68. Extensive research data support its efficacy in various fibrotic disease models, showcasing promising outcomes. Transcriptomic investigations have further substantiated its mechanism of action, illustrating that FT011 treatment leads to a reversal in the activation of genetic markers linked to fibrosis. This breakthrough holds significant promise for precision therapy in combating fibrotic conditions.
Certa is actively advancing its efforts in preparing for a pivotal clinical trial of FT011 for the treatment of scleroderma. With the backing of esteemed clinical experts worldwide, the company is working on finalizing the trial’s design and associated development strategies. These plans will be promptly presented to the FDA for discussion in early 2024. Additionally, Certa intends to seek complementary scientific advice from the European Medicines Agency (EMA) by mid-2024. The ultimate goal is to initiate the pivotal study by late 2024, marking a significant step forward in bringing FT011 closer to potential approval for treating scleroderma.
According to DelveInsight, the total number of diagnosed prevalent cases of systemic sclerosis in the 7MM was 150,000+ in 2022. The United States alone accounted for 47% of these cases. Within the EU4 and the UK, the combined diagnosed prevalent cases of systemic sclerosis surpassed 59,000. Notably, the United Kingdom led with over 15,000 diagnosed prevalent cases, while Germany was projected to have the fewest cases among the EU4 and the UK.
In response to the escalating number of cases and the urgent need for effective treatment options, numerous major pharmaceutical and biotechnology companies, including Certa Therapeutics, are actively engaged in the systemic sclerosis therapeutics domain. These companies are dedicating significant resources and efforts toward developing novel therapies and advancing research initiatives aimed at addressing the complex challenges posed by systemic sclerosis. Their collective endeavors underscore the industry’s commitment to meeting the unmet medical needs of patients affected by this debilitating condition.
Cardiol Therapeutics Granted Orphan Drug Designation for its Lead Drug Candidate for the Treatment of Pericarditis
Cardiol Therapeutics revealed on February 15, 2024, that the United States Food and Drug Administration (“FDA”) has granted Orphan Drug Designation (“ODD”) to the company’s forefront small molecule drug candidate, CardiolRx™ (cannabidiol) oral solution. This significant designation is a pivotal step forward in the journey to combat pericarditis, encompassing recurrent pericarditis, a condition characterized by inflammation of the pericardium surrounding the heart. Currently, undergoing Phase II clinical trials, CardiolRx™ demonstrates promising potential in addressing both recurrent pericarditis and acute myocarditis, thus marking a crucial milestone in advancing treatment options for these debilitating cardiac conditions.
The MAvERIC-Pilot trial (NCT05494788) constitutes a Phase II open-label pilot investigation aimed at examining the tolerance, safety, and impact of administering CardiolRx™ to patients grappling with recurrent pericarditis. Beyond standard safety evaluations, MAvERIC-Pilot has been meticulously designed to scrutinize enhancements in objective metrics associated with this rare ailment.
The primary efficacy endpoint is the change, from baseline to eight weeks, in patient-reported pericarditis pain using an 11-point numeric rating scale (“NRS”). The NRS serves as a validated clinical instrument utilized across various conditions involving acute and chronic pain, including prior examinations of recurrent pericarditis.
Secondary endpoints encompass the NRS score following 26 weeks of treatment, alongside fluctuations in circulating levels of C-reactive protein, a widely utilized clinical indicator of inflammation. Significantly, the trial aims to gauge the incidence of pericarditis recurrence, providing critical insights into the efficacy of the treatment regimen.
“The FDA’s decision was based on pre-clinical data combined with initial clinical data from the Company’s MAvERIC-Pilot Phase II study,” commented Dr. Andrew Hamer, Cardiol Therapeutics’ Chief Medical Officer and Head of Research & Development. “This designation reinforces the potential of CardiolRx™ to improve the lives of patients suffering with recurrent pericarditis, a debilitating heart disease associated with symptoms that adversely affect quality of life and physical activity.”
Recurrent pericarditis characterizes a recurrent inflammatory condition affecting the pericardium, the sac-like membrane encasing the heart, often arising subsequent to an initial episode, commonly precipitated by viral infections. This condition is marked by the potential for multiple recurrences, manifesting through distressing symptoms such as intense chest pain, breathlessness, and fatigue. These symptoms not only impose physical constraints but also significantly diminish the quality of life for afflicted individuals, often necessitating visits to emergency departments and leading to hospitalizations.
Furthermore, persistent inflammation can lead to the accumulation of pericardial fluid and scarring, which pose a grave risk of constricting the heart and potentially leading to life-threatening complications. Hence, recurrent pericarditis demands vigilant management and treatment to mitigate its debilitating effects and prevent severe cardiac complications.
The only FDA-approved treatment for recurrent pericarditis, introduced in 2021, is expensive and typically reserved as a third-line option. Annually, an estimated 38,000 patients in the United States are reckoned to have encountered at least one recurrence. Moreover, around 60% of individuals experiencing multiple recurrences (>1) endure symptoms persistently for over 2 years, with a third still affected even after 5 years.
In the pericarditis therapeutic landscape, companies like Cardiol Therapeutics and others spearhead the development of novel therapies and play a pivotal role. Their efforts hold the promise of providing more accessible, effective treatments that could alleviate the burden of recurrent pericarditis and improve long-term outcomes for patients.
Innovent Announces Primary Endpoint Met in the Phase 3 Clinical Trial of IBI311 in Treating Thyroid Eye Disease and Plans to Submit NDA to the NMPA
In a groundbreaking development unveiled on February 19, 2024, Innovent Biologics declared a significant milestone in their Phase 3 registrational trial, dubbed RESTORE-1, for IBI311. This trial, focused on assessing the efficacy of IBI311, a novel recombinant anti-insulin-like growth factor 1 receptor (IGF-1R) antibody, specifically targeted Chinese subjects suffering from Thyroid Eye Disease. The study reported the successful achievement of its primary endpoint, marking a pivotal advancement in the field of ophthalmology.
Encouraged by the promising results, Innovent Biologics now sets its sights on the next crucial step: the submission of a New Drug Application (NDA) for IBI311’s approval in treating Thyroid Eye Disease. The company intends to present this application to the esteemed Center for Drug Evaluation (CDE) under the purview of the National Medical Products Administration (NMPA). This submission heralds a new chapter in the quest to address the unmet medical needs of patients affected with Thyroid Eye Disease, offering hope for improved treatment outcomes and quality of life.
RESTORE-1 (CTR20223393) is a multicenter, randomized, double-masked, placebo-controlled Phase 2/3 clinical trial to evaluate the efficacy and safety of IBI311 in subjects with Thyroid Eye Disease. The Phase 3 results of RESTORE-1 showed that the primary endpoint was successfully met: at Week 24, the proptosis responder rate in the study eye (the percentage of subjects with a reduction in proptosis of ≥2 mm from baseline in the study eye without deterioration ≥ 2 mm increase of proptosis in the fellow eye) was significantly higher in subjects treated with IBI311 than in subjects treated with placebo: 85.8% vs. 3.8%, with a difference of 81.9% (95% CI: 69.8% to 93.9%, P < 0.0001).
In addition, the key secondary endpoints of the study such as overall response rate (the percentage of subjects with a reduction in proptosis of ≥2 mm from baseline and improvement in clinical activity score ≥2 in the study eye), percentage of subjects with a clinical activity score (CAS) of 0 or 1, and mean change in proptosis from baseline in the study eye were successfully met: IBI311 significantly improved all the above parameters as compared to the placebo.
Throughout the study, IBI311 exhibited a consistently favorable safety profile, with no occurrences of serious adverse events noted. The efficacy and safety profiles observed during the Phase 3 segment of the RESTORE-1 investigation remained consistent with the findings from its Phase 2 counterpart. Comprehensive results from the study will be disseminated in forthcoming medical conferences or publications.
Thyroid Eye Disease, characterized by autoimmune involvement of ocular tissues, exhibits an annual incidence estimated at 16/100,000 in women and 2.9/100,000 in men, with a prevalence ranging from 0.1% to 0.3% for clinically relevant cases. Presently, China lacks an approved targeted drug for treating Thyroid Eye Disease, despite multiple clinical guidelines endorsing the use of IGF-1R targeted antibodies.
In response to the pressing need for more effective treatment options, numerous companies, including Innovent, have embarked on proactive initiatives aimed at advancing diverse therapies for Thyroid Eye Disease. These endeavors underscore a collective commitment within the pharmaceutical industry to alleviate the treatment burden associated with this debilitating autoimmune condition. Leveraging cutting-edge research and innovative approaches, these companies are dedicated to developing novel therapeutic interventions that hold the promise of significantly improving patient outcomes and quality of life.
