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7 hours ago
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Beam Therapeutics Announces U.S. FDA Orphan Drug Designation Granted to BEAM-302 for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD)
CAMBRIDGE, Mass., May 29, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the United States (U.S.) Food and Drug Administration (FDA) has granted orphan drug designation to BEAM-302, a liver-targeting lipid-nanoparticle (LNP) formulation of a guide RNA and an mRNA encoding a base editor designed to correct the disease-causing mutation in patients with alpha-1 antitrypsin deficiency (AATD). AATD is an inherited genetic disorder that affects the lungs and/or liver, leading to early onset emphysema and liver disease, and for which there is significant unmet need for effective therapies that can treat the entire spectrum of disease. 'Receiving orphan drug designation for BEAM-302 is an important milestone in our efforts to bring a transformative therapy to people living with AATD, many of whom currently lack effective long-term treatment options,' said Giuseppe Ciaramella, Ph.D., president of Beam Therapeutics. 'This recognition by the FDA, following the receipt of RMAT designation from the FDA just weeks ago, highlights the urgency of addressing this serious genetic disease and the potential of BEAM-302 to directly correct the DNA mutation, the underlying root cause of this illness. We are encouraged by the FDA's continued support of this program and are committed to its advancement with the goal of delivering a one-time, potentially curative treatment to patients as quickly and safely as possible.' The FDA's orphan drug designation is designed to support the development and evaluation of treatments for rare diseases affecting fewer than 200,000 people in the U.S. The designation comes with potential benefits for the sponsor company, including tax credits for qualified clinical trials, exemption from user fees, and a potential seven years of market exclusivity after approval. Positive initial safety and efficacy data from the ongoing Phase 1/2 trial of BEAM-302, previously reported in March, established clinical proof of concept as a potential treatment for AATD and in vivo base editing. Preliminary results from the first three single-ascending dose cohorts in Part A of the study demonstrated that BEAM-302 was well tolerated, with single doses of BEAM-302 leading to durable, dose-dependent correction of the disease-causing mutation and total AAT protein levels above the therapeutic threshold in the 60 mg dose cohort. Beam has initiated dosing in the fourth cohort of Part A, evaluating 75 mg of BEAM-302, and expects to report updated data at a medical conference in the second half of 2025. Additionally, the company plans to dose the first patient in Part B, which will include AATD patients with mild to moderate liver disease, in the second half of 2025. Beam previously announced the clearance of the U.S. investigational new drug (IND) application for BEAM-302 for the treatment of AATD in March 2025, as well as the granting of Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA to BEAM-302 in May 2025. About BEAM-302BEAM-302 is a liver-targeting lipid-nanoparticle (LNP) formulation of base editing reagents designed to correct the PiZ mutation. Patients homozygous for this mutation (PiZZ) represent the majority of patients living with severe AATD disease. A one-time A-to-G correction of the PiZ mutation with Beam's adenine base editor has the potential to simultaneously reduce the aggregation of mutant, misfolded AAT protein that causes toxicity to the liver (Z-AAT), generate therapeutic levels of corrected protein (M-AAT), and increase total and functional AAT in circulation, thereby addressing the underlying pathophysiology of both the liver and lung disease. In addition, the reduction in circulating PiZ aggregates (i.e., polymers) has the potential to further minimize lung inflammation and dysfunction. Importantly, because the native AAT gene would be corrected in its normal genetic location, AAT levels are anticipated to increase physiologically in response to inflammation or infection. This is a critical aspect of AAT's normal function to regulate the body's inflammatory response, which does not occur with currently approved protein replacement therapies. Correction of the PiZ mutation is expected to be durable based on preclinical and clinical evidence. About Alpha-1 Antitrypsin Deficiency (AATD)AATD is an inherited genetic disorder that can cause early onset emphysema and liver disease. The most severe form of AATD arises when a patient has a point mutation in both copies of the SERPINA1 gene at amino acid 342 position (E342K, also known as the PiZ mutation or the 'Z' allele). This point mutation causes alpha-1 antitrypsin, or AAT, to misfold, accumulating inside liver cells rather than being secreted, resulting in very low levels (10%-15%) of circulating AAT. In addition to resulting in lower levels, the PiZ AAT protein variant is also less enzymatically effective compared to wildtype AAT protein. As a consequence, the lung is left unprotected from neutrophil elastase, resulting in progressive, destructive changes in the lung, such as emphysema, which can result in the need for lung transplants. The mutant AAT protein also accumulates in the liver, causing liver inflammation and cirrhosis, which can ultimately cause liver failure or cancer requiring patients to undergo a liver transplant. It is estimated that approximately 100,000 individuals in the U.S. have two copies of the Z allele, known as the PiZZ genotype, although only about 10% of all patients are thought to have been diagnosed. There are currently no curative treatments approved for patients with AATD, and the only approved therapy in the U.S., intravenous AAT protein replacement, has not been shown to prevent ongoing lung function decline and destruction in patients. About Beam TherapeuticsBeam Therapeutics (Nasdaq: BEAM) is a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. To achieve this vision, Beam has assembled a platform with integrated gene editing, delivery and internal manufacturing capabilities. Beam's suite of gene editing technologies is anchored by base editing, a proprietary technology that is designed to enable precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This has the potential to enable a wide range of therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases. Cautionary Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Investors are cautioned not to place undue reliance on these forward-looking statements, including, but not limited to, statements related to: the therapeutic applications and potential of our technology, including with respect to AATD; our plans, and anticipated timing, to advance our BEAM-302 program, including the clinical trial designs and expectations for BEAM-302; our plans to present data at upcoming medical conferences; expectations regarding potential benefits of the orphan drug designation for BEAM-302; and our ability to develop life-long, curative, precision genetic medicines for patients through base editing. Each forward-looking statement is subject to important risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties related to: our ability to develop, obtain regulatory approval for, and commercialize our product candidates, which may take longer or cost more than planned; our ability to raise additional funding, which may not be available; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the uncertainty that our product candidates will receive regulatory approval necessary to advance human clinical trials; that preclinical testing of our product candidates and preliminary or interim data from preclinical studies and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that initiation and enrollment of, and anticipated timing to advance, our clinical trials may take longer than expected; that our product candidates or the delivery modalities we rely on to administer them may cause serious adverse events; that our product candidates may experience manufacturing or supply interruptions or failures; risks related to competitive products; our ability to recognize the potential benefits conferred by the orphan drug designation for BEAM-302; and the other risks and uncertainties identified under the headings 'Risk Factors Summary' and 'Risk Factors' in our Annual Report on Form 10-K for the year ended December 31, 2024, our Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law. Contacts: Investors:Holly ManningBeam Therapeuticshmanning@ Media:Josie Butler1ABjosie@ in to access your portfolio
Business Wire
17 hours ago
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iVeena Announces IND Submission to the U.S. FDA for a Phase 2 Clinical Trial for Pediatric Myopia
SALT LAKE CITY--(BUSINESS WIRE)-- iVeena Delivery Systems Inc. ('iVeena'), a biopharmaceutical company advancing IVMED-85 for the treatment of pediatric myopia, today announced the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for IVMED-85 for the treatment of pediatric myopia. 'Today's announcement is an important milestone for iVeena in our journey to offer patients a novel therapeutic option,' said President and Founder, Bala Ambati, M.D., Ph.D., MBA. 'I am extremely proud of the remarkable commitment of the team at iVeena and look forward to initiating our multinational Phase 2 study in the coming months.' 'This IND marks a major step toward meeting a significant unmet need for patients. IVMED-85's novel LOX-based mechanism offers a differentiated, non-atropine approach that could redefine how we treat pediatric myopia,' said Vance Thompson, M.D., Founder of Vance Thompson Vision Sioux Falls, South Dakota, and the Director of Refractive Surgery. An IND submission is a request submitted to the FDA seeking permission to test a new drug or therapeutic substance in humans. The submission includes detailed information about the drug, its composition, pharmacology, toxicology data from preclinical studies, proposed clinical trial protocols, and information on manufacturing and quality control. With the IND application submission now complete, the FDA is expected to provide its review within approximately 30 days. Pending approval, the company plans to initiate the multinational clinical trial in 2025. About IVMED-85 Lead program IVMED-85 is a new chemical entity (NCE), preservative-free prescription eye drop to prevent myopic progression. IVMED-85 is a non-surgical, non-invasive, non-atropine daily eye drop that strengthens scleral and corneal collagen crosslinks through LOX activation, potentially leading to improved refraction and a decrease in the rate of axial elongation. About iVeena iVeena Delivery Systems, Inc. is a privately held, clinical stage ophthalmology company developing disease-modifying pharmacologic innovations for refractive diseases. iVeena has licensed its lead asset to Glaukos Corporation, IVMED-80, an Orphan Drug Designated eye drop for keratoconus. iVeena is developing IVMED-85, a first in class, investigational eyedrop formulation for pediatric myopia. About Myopia Myopia, also known as nearsightedness, is a common eye condition where distant objects appear blurry while close objects can be seen clearly. It occurs when the eye grows too long from front to back, causing light to focus in front of the retina rather than directly on it. Myopia typically begins in childhood and tends to progress with age. If left unmanaged, it can increase the risk of serious eye complications later in life, including retinal detachment, glaucoma, and myopic macular degeneration. The global rise in myopia—particularly among children—has become a significant public health concern. * Lysyl oxidase (LOX) is a copper ion-dependent amino-acyl oxidase ** Clin Ophthalmol. 2018 Aug 29;12:1581–1587. doi:
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a day ago
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Kexing Biopharm's GB18 project received clearance of IND application from NMPA and FDA
SHENZHEN, China, May 28, 2025 /PRNewswire/ -- On May 26th, Kexing Biopharm ( announced that the National Medical Products Administration (NMPA) has approved the Investigational New Drug (IND) application for its independently developed innovative product, GB18. Moreover, GB18 has previously received the clearance from U.S. Food and Drug Administration (FDA) for IND application on May 21st. GB18 is an innovative biologic product developed for the treatment of cancer cachexia, a complication with a prevalence up to 40% to 70% among cancer patients. Currently, no specific biological therapies are available to address the condition, presenting vast market potential. Featuring a unique nanobody-Fc fusion molecular structure, the product demonstrates enhanced stability, bettered bioavailability, and significantly improved performance in inhibiting disease-associated signaling pathways. Benchmarking against globally leading pipelines with the same target (GDF-15) and indication, Kexing Biopharm's GB18 features a special nanobody-Fc fusion structure (VHH-Fc), with the patents granted or applied for the molecule globally already. Last October, a research article of GB18's preclinical study was published in the renowned international academic journal mAbs, which demonstrated that GB18 effectively alleviated weight loss in cancer cachexia models, showing superior weight recovery and improved muscle fibers in both quantity and size compared to the comparator. In cancer cachexia, the expression level of GDF-15 is significantly elevated, which is closely associated with tumor progression and the severity of cachexia. Therefore, targeting GDF-15 has emerged as a new strategy in addressing the clinical challenge of cancer cachexia. This recent FDA IND approval marks a significant milestone in the Company's globalization of innovative products. Approximately millions advanced cancer patients annually in the world are suffering from cachexia. There is a significant unmet clinical need for the indication of cancer Biopharm will remain steadfast in its mission to deliver "Precise Products, Predictable Effects, and Health Protection", with a focus on product quality and a commitment to scientific innovation, and improve the lives of patients worldwide. View original content: SOURCE Kexing Biopharm Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
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2 days ago
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Enveric Biosciences Reports Positive Preclinical Results for Lead Drug Candidate EB-003
Statistically significant improvements in a preclinical model of severe chronic depression and despair Support for safe, extended, daily oral administration CAMBRIDGE, Mass., May 28, 2025--(BUSINESS WIRE)--Enveric Biosciences (NASDAQ: ENVB) ("Enveric" or the "Company"), a biotechnology company dedicated to the development of novel neuroplastogenic small-molecule therapeutics for the treatment of psychiatric and neurological disorders, today announced positive preclinical results for its lead neuroplastogen drug candidate, EB-003, in the Open Space Forced Swim Test, a preclinical mouse model of severe depression and despair. The Open Space Forced Swim Test, a well-established psychiatric behavioral model, is designed to induce potent and enduring chronic depression-like states in mice, leading to reduced swim distance and increased periods of despair-induced immobility. In a study performed by a third-party lab, an oral dose of EB-003 at 30 mg/kg significantly reduced depression-like behavior within 30 minutes of administration. This was indicated by the enhanced distance travelled and reduced immobility of treated mice during forced swim sessions (statistical p-value < 0.01). These results were comparable to the therapeutic effects of Imipramine, an approved tricyclic antidepressant drug. A repeat study, conducted by the same third-party lab, produced consistent results, confirming 30 mg/kg as an efficacious oral dose for EB-003. The study also confirmed no adverse locomotor effects were observed at this dose. The study also performed a preliminary assessment of extended, daily dosing of EB-003 to determine any potential safety concerns for chronic therapeutic administration. Mice receiving a daily oral dose of EB-003 at 30 mg/kg for 22 days showed no adverse behavioral, physiological or neurological effects. These observations demonstrate acceptable tolerance to long-term systemic drug exposure and expand the range of EB-003 dosing strategies. "We are encouraged by these statistically significant and biologically meaningful results in a severe chronic depression animal model," said Dr. Joseph Tucker, CEO of Enveric Biosciences. "EB-003 has now demonstrated strong behavioral efficacy and a clean safety profile in several animal models that are relevant to chronic treatment of severe depression. These data points validate the compound's pharmacological design and provide strong support for the planned Investigational New Drug (IND) application" EB-003 is the lead compound from Enveric's proprietary EVM301 Series, a pipeline of next-generation neuroplastogens engineered to stimulate synaptic plasticity without incurring hallucinogenic liability. About Enveric Biosciences Enveric Biosciences (NASDAQ: ENVB) is a biotechnology company dedicated to the development of novel neuroplastogenic small-molecule therapeutics for the treatment of psychiatric and neurological disorders. Leveraging its unique discovery and development platform Psybrary™, which houses proprietary information on the use and development of existing and novel molecules for specific mental health indications, Enveric seeks to develop a robust intellectual property portfolio of novel drug candidates. Enveric's lead molecule, EB-003, is a potential first-in-class neuroplastogen designed to promote neuroplasticity, without inducing hallucinations, in patients suffering from difficult-to-address mental health disorders. Enveric is focused on advancing EB-003 towards clinical trials for the treatment of neuropsychiatric disorders while out-licensing other novel, patented Psybrary™ platform drug candidates to third-party licensees advancing non-competitive market strategies for patient care. Enveric is headquartered in Naples, FL with offices in Cambridge, MA and Calgary, AB Canada. For more information, please visit Forward-Looking Statements This press release contains forward-looking statements and forward-looking information within the meaning of applicable securities laws. These statements relate to future events or future performance. All statements other than statements of historical fact may be forward-looking statements or information. Generally, forward-looking statements and information may be identified by the use of forward-looking terminology such as "plans," "expects" or "does not expect," "proposes," "budgets," "explores," "schedules," "seeks," "estimates," "forecasts," "intends," "anticipates" or "does not anticipate," or "believes," or variations of such words and phrases, or by the use of words or phrases which state that certain actions, events or results may, could, should, would, or might occur or be achieved. Forward-looking statements may include statements regarding beliefs, plans, expectations, or intentions regarding the future and are based on the beliefs of management as well as assumptions made by and information currently available to management. Actual results could differ materially from those contemplated by the forward-looking statements as a result of certain factors, including, but not limited to, the ability of Enveric to: finalize and submit its IND filing to the U.S. Food and Drug Administration; carry out successful clinical programs; achieve the value creation contemplated by technical developments; avoid delays in planned clinical trials; establish that potential products are efficacious or safe in preclinical or clinical trials; establish or maintain collaborations for the development of therapeutic candidates; obtain appropriate or necessary governmental approvals to market potential products; obtain future funding for product development and working capital on commercially reasonable terms; scale-up manufacture of product candidates; respond to changes in the size and nature of competitors; hire and retain key executives and scientists; secure and enforce legal rights related to Enveric's products, including patent protection; identify and pursue alternative routes to capture value from its research and development pipeline assets; continue as a going concern; and manage its future growth effectively. A discussion of these and other factors, including risks and uncertainties with respect to Enveric, is set forth in Enveric's filings with the Securities and Exchange Commission, including Enveric's Annual Report on Form 10-K and its Quarterly Reports on Form 10-Q. Enveric disclaims any intention or obligation to revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. View source version on Contacts Investor RelationsTiberend Strategic Advisors, Inc. David Irish(231) 632-0002dirish@ Media RelationsTiberend Strategic Advisors, Inc. Casey McDonald(646) 577-8520cmcdonald@ Sign in to access your portfolio
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2 days ago
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EpiVax Appoints Dr. Vibha Jawa to Lead Scientific Strategy as CSO
PROVIDENCE, R.I., May 28, 2025 /PRNewswire/ -- EpiVax, Inc., a leader in preclinical immunogenicity assessment for biologic therapeutics and vaccines, is pleased to announce the appointment of Vibha Jawa, PhD, FAAPS as Chief Scientific Officer. Dr. Jawa succeeds EpiVax founder and Executive Chairwoman Annie De Groot, MD, who has transitioned from CSO to Chief Medical Officer. Dr. Jawa brings over two decades of leadership in biologics, vaccine, and gene therapy development and immunogenicity strategy to EpiVax. Most recently, she served as Executive Director of Translational Medicine at Bristol Myers Squibb, where she led bioanalytical strategy, supporting PK- and immunogenicity-related preclinical and clinical assessments across the development pipeline. She has held senior roles at Merck and Amgen where she led immunogenicity assessment efforts, supporting discovery to development. Dr. Jawa has contributed to more than 20 successful IND, BLA, and MAA filings across many therapeutic modalities, and was part of the team at the Institute of Human Gene Therapy (UPENN) that supported the first approved gene therapy product. A recognized thought leader in bioanalysis and immunogenicity from discovery through commercialization, Dr. Jawa has authored over 100 peer-reviewed publications and holds prominent roles in various renowned scientific societies and consortiums, including AAPS, IQ, SC, and EIP. She was named an AAPS fellow in 2022 for her significant contributions. "We're thrilled to welcome Vibha to the team," said Dr. Rich-Henry Schabowsky, CEO of EpiVax. "Her depth of experience in biologics, especially novel modalities, combined with her expertise in evaluating and mitigating immunogenic risk will be instrumental as we continue evolving our methods to meet the complex needs of the industry." Dr. Annie De Groot, EpiVax co-founder and CMO, added: "Dr. Jawa is the perfect scientific leader for EpiVax 2.0: the transformation of our company that began in 2024. She brings the exact energy that EpiVax needs at this juncture. I'm thrilled to be handing the baton to Dr. Jawa, who will ensure that EpiVax remains at the cutting edge of immunogenicity science— pushing boundaries, validating new ideas, building bridges between in vitro data, immunoinformatics and AI, and setting higher standards for translational immunology." Dr. Jawa will have outward and inward facing responsibilities as CSO. She will represent EpiVax at key meetings, while leading internal research and validation efforts. Her leadership will help expand the company's reach into new therapeutic areas and reinforce EpiVax as a trusted partner in biologic and vaccine development. "I'm thrilled to be joining the EpiVax team," said Dr. Jawa. "This is an extraordinary company with a long history of scientific leadership in immunogenicity assessment. I look forward to building on that legacy and continuing to refine the team's approaches, ensuring that we deliver the most accurate, insightful support." About EpiVax: EpiVax is a leader in preclinical immunogenicity assessment and sequence optimization for peptide therapeutics, biologic therapeutics, gene/cell therapies, and vaccines. EpiVax partners with a global roster of companies, agencies, and academics to accelerate immunogenicity risk assessment, immune modulation, and rapid vaccine design. Press Contact:Sarah MonizSmoniz@ View original content: SOURCE EpiVax, Inc. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
