Tepezza (teprotumumab), a treatment for the painful autoimmune condition thyroid eye disease, accounts for nearly half of Horizon’s €3.4 billion ($3.6 billion) in annual sales (TED). Krystexxa (peglticase) for chronic gout and Ravicti for inborn urea disorders are among its other top drugs. Tepezza and Krystexxa generated $490 million and $192 million in revenue in the third quarter, respectively.
Vertex Receives IND Clearance for mRNA Cystic Fibrosis Therapy
Vertex Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration has approved VX-522, a messenger ribonucleic acid (mRNA) therapy aimed at treating the underlying cause of cystic fibrosis (CF) lung disease for the approximately 5,000 people with Cystic Fibrosis who are unable to benefit from a cystic fibrosis transmembrane conductance regulator (CFTR) modulator. In the coming weeks, Vertex plans to begin a single ascending dose clinical trial for VX-522 in people with cystic fibrosis.
Cystic fibrosis (CF) is a rare, life-shortening genetic disease that affects over 83,000 people worldwide. CF is a multi-organ disease that affects the lungs, liver, pancreas, gastrointestinal tract, sinuses, sweat glands, and reproductive tract. CF is caused by a defective and/or missing CFTR protein as a result of CFTR gene mutations.
VX-522 is administered to the lungs via inhalation of CFTR mRNA encapsulated in a lipid nanoparticle. The mRNA is designed to produce functional copies of the CFTR protein once it reaches the target lung cells. VX-522 is the result of an exclusive research collaboration established with Moderna in 2016..
“It has been our longstanding goal to bring highly effective therapies to all people with CF. Clearance of the IND represents a pivotal turning point in reaching the remaining ~5,000 people with CF who are still waiting for a medicine to treat the underlying cause of their disease,” said Reshma Kewalramani, M.D., FASN, Chief Executive Officer and President, Vertex. “The partnership with Moderna that began more than five years ago has been instrumental in achieving this milestone, and we look forward to continuing our work together.”
“This partnership brings together Vertex’s scientific expertise and decades of experience in developing cystic fibrosis medicines with Moderna’s proven leadership in mRNA technologies,” said Moderna Chief Executive Officer Stéphane Bancel. “Moderna’s development of a proprietary inhalable lipid nanoparticle to deliver a functional cystic fibrosis treatment to the lungs could lead to a transformational medical achievement. We are excited by the progress that has been made with the upcoming advancement of VX-522 to the clinic and look forward to our ongoing collaboration to develop treatments for the underlying cause of cystic fibrosis.”
Moleculin Gets FDA Fast-Track Designation for WP1122 in Glioblastoma Multiforme
On Dec. 7, 2022, Moleculin Biotech announced that the US FDA has granted Fast Track designation to its therapy WP1122 for the treatment of Glioblastoma Multiforme (GBM). The Fast Track designation will expedite the Moleculin in developing WP1122, with potentially more frequent regulatory meetings and communications with the FDA. Upon receiving the FDA Fast-Track Designation to the therapy, Walter Klemp, Chairman and Chief Executive Officer of Moleculin has commented that “We believe that based on the promising animal model data that support GBM as one of many potential indications, the clearance of our IND for WP1122 in GBM, and Orphan Drug Designation previously received from the FDA, WP1122 is well-positioned to be a potential treatment option for this devastating disease.”
Moleculin is working on WP1122 as a 2-DG prodrug to provide a more favorable pharmacological profile, and it is found to have greater potency than 2-DG alone in preclinical models where tumor cells require higher glycolytic activity than normal cells. Although, activity in animals does not necessarily translate to humans. However, the preclinical studies in mice transplanted with human brain tumors showed that WP1122 outperformed the standard of care, temozolomide, and performed even better in combination with temozolomide. Moleculin has further stated that it is currently evaluating opportunities for collaboration in clinical development. Earlier, in September 2022, FDA granted the Orphan Drug Designation to the therapy, WP1122, for treating GBM. Moreover, based on preclinical data indicating the potential for WP1122 as a treatment for GBM, Moleculin received FDA clearance for its Investigational New Drug application to initiate a Phase 1 open-label, single-arm, dose-escalation study of the safety, pharmacokinetics, and efficacy of oral WP1122 in adult patients with GBM.
GBM is the most aggressive malignant primary brain tumour and remains an incurable tumor with a median survival of only 15 months. As per DelveInsight, the total Glioblastoma Multiforme diagnosed incident population in the 7 major markets was estimated to be 29,000+ in 2020. In the United States, the diagnosed incident cases were 15,400 in 2020, which is expected to increase by 2032. Globally, several pharma and biotech giants are actively working in the Glioblastoma Multiforme therapeutics market to provide a better treatment option. The ongoing clinical development activities and the approval and launch of emerging therapies like WP1122 are expected to transform the treatment outlook.
Brim Receives Orphan Drug Designation For Neurotrophic Keratitis Treatment
On 8th December 2022, BRIM Biotechnology, Inc. announced that the US FDA had granted Orphan Drug Designation to its under-development therapy, BRM424, for treating a rare degenerative eye disease, Neurotrophic Keratitis (NK). BRM424 is a novel, first-in-class, potential treatment for Neurotrophic Keratitis. It is now planning to finalize the Phase 2 study protocol and accelerate the development toward commercialization to bring this novel treatment to patients as early as possible. The current Neurotrophic Keratitis treatment scenario is complicated, and the therapeutic market lacks effective and affordable treatment options. The Orphan Drug Designation will encourage the development of BRM424, which includes eligibility for federal grants, research and development tax credits, a waiver of prescription drug user fees, and the potential for a 7-year market exclusivity period upon FDA approval.
BRIM has further updated that it plans to apply for a Phase 2 NK clinical trial of BRM424 in the US, intending to provide an affordable and effective new drug for treating Neurotrophic Keratitis patients. Using the NK rabbit model, BRM424 has demonstrated preclinical efficacy and safety in compliance with regulatory requirements. BRM424 uses the same active pharmaceutical ingredient (API) as BRM421, BRIM’s lead candidate for Dry Eye Disease, which is about to begin Phase 3 clinical trials.
Neurotrophic Keratitis is a rare degenerative and disabling eye disease with an incidence rate of less than five in 10,000. As per DelveInsight, the total Neurotrophic Keratitis prevalent cases in the 7MM was 137,500+ in 2021. The cases of Neurotrophic Keratitis in the 7MM are expected to increase in the coming years. In the United States, the prevalent cases of Neurotrophic Keratitis were reported to be 65,500 in 2021. Clinically, the treatment of Neurotrophic Keratitis is very complicated, and there is a lack of effective treatment options. Currently, only one drug, Oxervate (cenegermin), is approved for treating Neurotrophic Keratitis in the US and Europe. The further approval and launch of BRM424, are expected to improve the Neurotrophic Keratitis treatment scenario.
Eisai Presents Full Results of Lecanemab Phase 3 Confirmatory Clarity Ad Study for Early Alzheimer’s Disease
Eisai presented complete Phase III results of lecanemab, an anti-amyloid beta (Aβ) protofibril antibody for the treatment of mild cognitive impairment, majorly occuring due to Alzheimer’s disease and early Alzheimer’s disease. The amyloid pathology presence in the brain was previously confirmed and presented at the 2022 clinical trials on Alzheimer’s Disease conference (CTAD), held at San Fransisco, California. Similarly, the results were presented in the New England Journal of Medicine, which is one of the most renowned and prestigious peer-reviewed medical-based journals.
Almost 1,700 individuals in North America, Europe and Asia were indulged in the global confirmatory Phase III placebo-controlled, double-blinded, parallel-group randomized study.
The volunteers were randomised 1:1 to receive doses of placebo and lecanemab, each 10-mg/kg IV dose. The classification was further divided into clinical subgroups due to the existence or absence of concomitant approved AD symptomatic medication at baselines, such as acetylcholinesterase inhibitors, memantine, and many more. Mild cognitive impairment is when a person constantly faces trouble in remembering, recalling, learning, and concentrating on new things. Moreover, the mild cognitive impairment makes it difficult for the patient to make day-to-day decisions affecting the quality of life.
“We continue to relentlessly pursue research that provides useful insights for people living with breast cancer,” said Dr Takashi Owa, Chief Scientific Officer, Senior Vice President, Eisai Co., Ltd. “A big part of this commitment is the ongoing sharing of our preclinical and clinical data with eribulin.”
UCB Announces Positive Phase 3 Studies for Bimekizumab in Hidradenitis Suppurativa
UCB, a global biopharmaceutical, recently announced positive Phase III results, including BE HEARD I and BE HEARD II for the drug bimekizumab for the treatment of Hidradenitis Suppurativa.
The results obtained from the study will further provide the base for global regulatory license applications for the drug bimekizumab treatment starting in quarter three of 2023. Bimekizumab is an investigational drug whose safety and efficacy haven’t been established for any indication in the United States until now, and therefore it hasn’t been approved by US FDA.
The drug is currently being examined for utilization in the treatment of hidradenitis suppurativa. Hidradenitis suppurativa is a painful, long-term skin condition that is responsible for abscesses and skin scarring. The accurate cause behind hidradenitis suppurativa is still unknown. It is majorly seen near the hair follicles where there are sweat glands, usually around the groin, bottom, breasts and armpits.
“We are excited to announce positive pivotal Phase 3 outcomes in moderate to severe hidradenitis suppurativa which support our strong belief in bimekizumab and provide the first Phase 3 evidence suggesting that targeting IL-17F in addition to IL-17A may be a promising treatment approach. We look forward to bringing bimekizumab to people living with this chronic inflammatory disease as soon as possible,” said Emmanuel Caeymaex, Executive Vice President, of Immunology Solutions and Head of U.S., UCB.
