Longeveron Inc. (NASDAQ: LGVN), a clinical stage biotechnology company developing regenerative medicines, today announced that it will participate in the Emerging Growth Virtual Conference taking place June 12-13, 2024.
Details for the Company's presentation:
| Date: | Wednesday, June 12, 2024 | |
| Time: | 1:45 p.m. ET |
The webcast for this conference presentation may be accessed at the " Events and Presentations " section of the Company's website. A replay of the webcast will be available on the Longeveron website for 180 days following the conference.
Questions may be submitted in advance to Questions@EmergingGrowth.com or asked during the event.
About Longeveron Inc.
Longeveron is a clinical stage biotechnology company developing regenerative medicines to address unmet medical needs. The Company's lead investigational product is Lomecel-Bâ„¢, an allogeneic medicinal signaling cell (MSC) therapy product isolated from the bone marrow of young, healthy adult donors. Lomecel-Bâ„¢ has multiple potential mechanisms of action encompassing pro-vascular, pro-regenerative, anti-inflammatory, and tissue repair and healing effects with broad potential applications across a spectrum of disease areas. Longeveron is currently pursuing three pipeline indications: hypoplastic left heart syndrome (HLHS), Alzheimer's disease, and Aging-related Frailty. For more information, visit www.longeveron.com or follow Longeveron on LinkedIn , X , and Instagram .
Investor Contact:
Derek Cole
Investor Relations Advisory Solutions
derek.cole@iradvisory.com
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Veru Inc. (NASDAQ: VERU), a late clinical stage biopharmaceutical company focused on developing innovative medicines for preserving muscle for high quality weight loss, oncology, and viral induced acute respiratory distress syndrome, today announced that the Company will present two presentations at the American Diabetes Association's 84 th Scientific Sessions, taking place June 21-24, 2024, in Orlando, Florida.
The presentations are:
Pooled Safety Analysis of Enobosarm from Phase 2 and Phase 3 Placebo-Controlled Clinical Trials
Date: Saturday, June 22, 2024
Start and end time: 12:30 PM-01:30 PM Eastern Daylight Time
Location: West Concourse A4-B2
Potential to Optimize Weight Loss with Enobosarm—Meta-analysis of Body Composition from Three Randomized Clinical Trials Support the Ability of Enobosarm to Preserve Muscle while Reducing Fat
Date: Saturday, June 22, 2024
Start and end time: 12:30 PM-01:30 PM Eastern Daylight Time
Location: West Concourse A4-B2
Additional information on the meeting can be found on the American Diabetes Association website: https://professional.diabetes.org/scientific-sessions
About the Enobosarm Phase 2b clinical trial
The Phase 2b, multicenter, double-blind, placebo-controlled, randomized, dose-finding clinical trial is designed to evaluate the safety and efficacy of enobosarm 3mg, enobosarm 6mg, or placebo as a treatment to preserve muscle and augment fat loss in approximately 150 patients with sarcopenic obesity or overweight elderly (>60 years of age) patients receiving semaglutide (Wegovy®). The primary endpoint is total lean body mass, and the key secondary endpoints are total body fat mass and physical function as measured by stair climb test at 16 weeks. The Phase 2b clinical trial is actively enrolling patients from up to 15 clinical sites in the United States. Topline clinical results from the trial are expected by the end of calendar year 2024.
After completing the efficacy dose-finding portion of the Phase 2b clinical trial, it is expected that participants will then continue in blinded fashion into a Phase 2b extension clinical trial where all patients will stop receiving a GLP-1 RA, but will continue taking placebo, enobosarm 3mg, or enobosarm 6mg for an additional 12 weeks. The Phase 2b extension clinical trial will evaluate whether enobosarm can maintain muscle and prevent the fat and weight gain that occurs after discontinuing a GLP-1 RA. The topline results of the separate blinded Phase 2b extension clinical study are expected in calendar Q2 2025.
About Sarcopenic Obesity
According to the CDC, 41.5% of older adults have obesity in the United States and could benefit from a weight loss medication. Up to 34.4% of these obese patients over the age of 60 have sarcopenic obesity. This large subpopulation of sarcopenic obese patients is especially at risk for taking GLP-1 drugs for weight loss as they already have critically low amount of muscle due to age-related muscle loss. Further loss of muscle mass when taking a GLP-1 RA medication may lead to muscle weakness leading to poor balance, decreased gait speed, mobility disability, loss of independence, falls, bone fractures and increased mortality which is a condition like age-related frailty. Because of the magnitude and speed of muscle loss while on GLP-1 RA therapy for weight loss, GLP-1 RA drugs may accelerate the development of frailty in older obese or overweight elderly patients.
About Enobosarm
Enobosarm (aka ostarine, MK-2866, GTx-024, and VERU-024), a novel oral daily selective androgen receptor modulator (SARM), has been previously studied in 5 clinical studies involving 968 older normal men and postmenopausal women as well as older patients who have muscle wasting because of advanced cancer. Advanced cancer simulates a "starvation state" where there is significant unintentional loss or wasting of both muscle and fat mass which is similar to what is observed with in patients taking GLP-1 RA drugs. We believe the totality of the clinical data from these previous five clinical trials demonstrates that enobosarm treatment leads to dose-dependent increases in muscle mass with improvements in physical function as well as significant dose-dependent reductions in fat mass. The patient data that were generated from these five enobosarm clinical trials in both elderly patients and in patients with a cancer induced starvation-like state provide strong clinical rationale for enobosarm. The expectation is that enobosarm in combination with a GLP-1 RA would potentially augment the fat reduction and total weight loss while preserving muscle mass.
Importantly, enobosarm has a large safety database, which includes 27 clinical trials involving 1581 men and women, some of which included patients dosed for up to 3 years. In this large safety database, enobosarm was generally well tolerated with no increases in gastrointestinal side effects. This is important as there are already significant and frequent gastrointestinal side effects with a GLP-1 RA treatment alone.
About Veru Inc.
Veru is a late clinical stage biopharmaceutical company focused on developing novel medicines for the treatment of metabolic diseases, oncology, and ARDS. The Company's drug development program includes two late-stage novel small molecules, enobosarm and sabizabulin.
Enobosarm, a selective androgen receptor modulator (SARM), is being developed for two indications: (i) Phase 2b clinical study of enobosarm as a treatment to augment fat loss and to prevent muscle loss in sarcopenic obese or overweight elderly patients receiving a GLP-1 RA who are at-risk for developing muscle atrophy and muscle weakness and (ii) subject to the availability of sufficient funding, Phase 3 ENABLAR-2 clinical trial of enobosarm and abemaciclib for the treatment of androgen receptor positive (AR+), estrogen receptor positive (ER+) and human epidermal growth factor receptor 2 negative (HER2-) metastatic breast cancer in the 2nd line setting.
Sabizabulin, a microtubule disruptor, is being developed as a Phase 3 clinical trial for the treatment of hospitalized patients with viral-induced ARDS. The Company does not intend to undertake further development of sabizabulin for the treatment of viral-induced ARDS until we obtain funding from government grants, pharmaceutical company partnerships, or other similar third-party external sources.
The Company also has an FDA-approved commercial product, the FC2 Female Condom® (Internal Condom), for the dual protection against unplanned pregnancy and sexually transmitted infections.
Forward-Looking Statements
This press release contains "forward-looking statements" as that term is defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, express or implied statements related to whether and when the phase 2b trial of enobosarm discussed above will produce topline data or patients will progress into the extension study, the planned design, number of sites, timing, endpoints, patient population and patient size of such trial and whether such trial will successfully meet any of its endpoints, whether enobosarm will enhance weight loss or preserve muscle in, or meet any unmet need for, obesity patients and whether it will enhance weight loss, whether the Company's scientific advisors will make valuable contributions to the Company's enobosarm program and whether the Company will be successful in its transformation into a late stage biopharmaceutical company focused on obesity and oncology. The words "anticipate," "believe," "could," "expect," "intend," "may," "opportunity," "plan," "predict," "potential," "estimate," "should," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based upon current plans and strategies of the Company and reflect the Company's current assessment of the risks and uncertainties related to its business and are made as of the date of this press release. The Company assumes no obligation to update any forward- looking statements contained in this press release because of new information or future events, developments or circumstances. Such forward-looking statements are subject to known and unknown risks, uncertainties and assumptions, and if any such risks or uncertainties materialize or if any of the assumptions prove incorrect, our actual results could differ materially from those expressed or implied by such statements. Factors that may cause actual results to differ materially from those contemplated by such forward-looking statements include, but are not limited to: the development of the Company's product portfolio and the results of clinical studies possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical studies and the ability to enroll subjects in accordance with planned schedules; the ability to fund planned clinical development as well as other operations of the Company; the timing of any submission to the FDA or any other regulatory authority and any determinations made by the FDA or any other regulatory authority; the Company's existing product, FC2, and any future products, if approved, possibly not being commercially successful; the ability of the Company to obtain sufficient financing on acceptable terms when needed to fund development and operations; demand for, market acceptance of, and competition against any of the Company's products or product candidates; new or existing competitors with greater resources and capabilities and new competitive product approvals and/or introductions; changes in regulatory practices or policies or government-driven healthcare reform efforts, including pricing pressures and insurance coverage and reimbursement changes; risks relating to the Company's development of its own dedicated direct to patient telehealth platform, including the Company's lack of experience in developing such a platform, potential regulatory complexity, development costs, and market awareness and acceptance of any telehealth platform we develop; risks relating to our ability to increase sales of FC2 after significant declines in recent periods due to telehealth industry consolidation and the bankruptcy of a large telehealth customer; the Company's ability to protect and enforce its intellectual property; the potential that delays in orders or shipments under government tenders or the Company's U.S. prescription business could cause significant quarter-to-quarter variations in the Company's operating results and adversely affect its net revenues and gross profit; the Company's reliance on its international partners and on the level of spending by country governments, global donors and other public health organizations in the global public sector; the concentration of accounts receivable with our largest customers and the collection of those receivables; the Company's production capacity, efficiency and supply constraints and interruptions, including potential disruption of production at the Company's and third party manufacturing facilities and/or of the Company's ability to timely supply product due to labor unrest or strikes, labor shortages, raw material shortages, physical damage to the Company's and third party facilities, product testing, transportation delays or regulatory actions; costs and other effects of litigation, including product liability claims and securities litigation; the Company's ability to identify, successfully negotiate and complete suitable acquisitions or other strategic initiatives; the Company's ability to successfully integrate acquired businesses, technologies or products; and other risks detailed from time to time in the Company's press releases, shareholder communications and Securities and Exchange Commission filings, including the Company's Form 10-K for the year ended September 30, 2023, as amended by the Form 10-K/A, and subsequent quarterly reports on Form 10-Q. These documents are available on the "SEC Filings" section of our website at www.verupharma.com/investors .
* Wegovy ® is a registered trademark of Novo Nordisk A/S
Investor and Media Contact:
Samuel Fisch
Executive Director, Investor Relations and Corporate Communications
Email: veruinvestor@verupharma.com
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CEL-SCI Corporation (NYSE American: CVM) today announced Dr. Giovanni Selvaggi, an oncology key opinion leader instrumental in successfully bringing several drugs to market has joined CEL-SCI as a Clinical Advisor. Dr. Selvaggi joins CEL-SCI as the Company recently received its go-ahead from the U.S. Food and Drug Administration (FDA) for its confirmatory Registration Study of Multikine* in the treatment of head and neck cancer.
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Multikine is the first investigational pre-surgical cancer drug in newly diagnosed head and neck cancer. Multikine is a copy of the pro-inflammatory cytokine immune response human bodies produce every day and is designed to empower a person's intact immune system cells to attack their own cancer. Patients are treated with Multikine right after diagnosis, before any other standard of care treatment (surgery and radiation therapy) since that is when the immune system is strongest. There is robust safety and efficacy data from 750 patients who have been treated with Multikine.
Based on the following efficacy data, the FDA has agreed to CEL-SCI's 212-person Registration Study to confirm the findings for regulatory approval:
Dr. Selvaggi is a US-based drug developer, cancer researcher, and strategic advisor to big pharma and early-to-late stage biotech companies. He is currently Chief Medical Officer at Xcovery Holdings, where he manages an ongoing New Drug Application with the FDA for ensartinib, an ALK-TKI for non-small cell lung cancer (NSCLC). He is also a Clinical Consultant to Tubulis GmBH for a first-in-class ADC program in solid tumors, and Clinical Strategy Senior Advisor to Alira Health. He formerly served as Medical Director, Cancer Immunotherapy, in the MAGE-A3 lung cancer vaccine program at GSK. Later, Dr. Selvaggi played an instrumental role in the successful development and approval of ceritinib (Zykadia) in ALK-translocated NSCLC at Novartis. At Oncolytics he was VP of Clinical Development. Most recently, Dr. Selvaggi was part of the immunotherapy team at Bristol-Myers Squibb, serving as a program lead in thoracic malignancies, with a focus on SCLC and mesothelioma, leading to the approval of nivolumab (Opdivo) in third line small cell lung cancer. Dr. Selvaggi received his medical degree at the University of Torino School of Medicine, in Torino, Italy, and served as staff physician of thoracic oncology at the University Hospital in Torino, participating in several clinical trials in lung cancer and mesothelioma over a span of 20 years.
Dr. Selvaggi commented, "Multikine is a first in class immunotherapy that has demonstrated to meaningfully and safely prolong survival in a selected orphan population of newly diagnosed head and neck cancer. The importance of tackling cancer at the earliest stages to guarantee the most relevant impact in patients' lives is undeniable, especially in head and neck cancer which has no approved therapeutic options before surgery. I am fully committed to supporting CEL-SCI leadership to bring this asset to patients through an expedited registrational path that has been agreed with regulatory authorities and that has a potential for cure."
"Dr. Selvaggi has a passion for bringing cancer drugs to market to save lives and lead clinical research toward a cure for cancer. We are excited to have him join CEL-SCI as a Clinical Advisor at this pivotal point for Multikine which is set to commence a confirmatory FDA Registration Study," stated CEL-SCI CEO, Geert Kersten. "The data on Multikine is excellent, and as we proceed with and wrap up this final study, Dr. Selvaggi's experience and successful track record in navigating late-stage development, clinical trials, and the regulatory approval process will be hugely valuable for us."
Dr. Selvaggi joins several other top-tier physician consultants and head and neck cancer key opinion leaders who are advisors to CEL-SCI.
About Cel-Sci Corporation
CEL-SCI believes that boosting a patient's immune system while it is still intact should provide the greatest possible impact on survival. Multikine is designed to help the immune system "target" the tumor at a time when the immune system is still relatively intact and thereby thought to be better able to mount an attack on the tumor.
Multikine (Leukocyte Interleukin, Injection), a true first-line cancer therapy, has been dosed in over 750 patients and received Orphan Drug designation from the FDA for neoadjuvant therapy in patients with squamous cell carcinoma (cancer) of the head and neck. Multikine significantly extended life in its target patient population demonstrating a 73% survival rate with Multikine vs. only 45% without at 5 years after treatment. Based on this very strong data, the FDA agreed to CEL-SCI's target patient selection criteria and gave the go-ahead to conduct a small, focused, confirmatory Registration Study which will enroll 212 patients. CEL-SCI will enroll newly diagnosed advanced primary head and neck cancer patients with no lymph node involvement (determined via PET scan) and with low PD-L1 tumor expression (determined via biopsy), representing over 100,000 patients annually.
The Company has operations in Vienna, Virginia, and near/in Baltimore, Maryland.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. When used in this press release, the words "intends," "believes," "anticipated," "plans" and "expects," and similar expressions, are intended to identify forward-looking statements. Such statements are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Such statements include, but are not limited to, statements about the terms, expected proceeds, use of proceeds and closing of the offering. Factors that could cause or contribute to such differences include an inability to duplicate the clinical results demonstrated in clinical studies, timely development of any potential products that can be shown to be safe and effective, receiving necessary regulatory approvals, difficulties in manufacturing any of the Company's potential products, inability to raise the necessary capital and the risk factors set forth from time to time in CEL-SCI's filings with the Securities and Exchange Commission, including but not limited to its report on Form 10-K for the year ended September 30, 2023. The Company undertakes no obligation to publicly release the result of any revision to these forward-looking statements which may be made to reflect the events or circumstances after the date hereof or to reflect the occurrence of unanticipated events.
* Multikine (Leukocyte Interleukin, Injection) is the trademark that CEL-SCI has registered for this investigational therapy. This proprietary name is subject to FDA review in connection with the Company's future anticipated regulatory submission for approval. Multikine has not been licensed or approved for sale, barter or exchange by the FDA or any other regulatory agency. Similarly, its safety or efficacy has not been established for any use.
View source version on businesswire.com: https://www.businesswire.com/news/home/20240606671425/en/
Gavin de Windt
Cel-Sci Corporation
(703) 506-9460
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VTX3232 demonstrated improvements in body weight, systemic inflammatory biomarkers and cardiometabolic parameters in diet-induced obesity (DIO) mice
Additive effects were observed for VTX3232 in combination with the GLP-1 receptor agonist semaglutide across key endpoints compared to semaglutide or VTX3232 alone
Ventyx Biosciences, Inc. (Nasdaq: VTYX) ("Ventyx"), a clinical-stage biopharmaceutical company focused on advancing novel oral therapies that address a broad range of inflammatory diseases with significant unmet medical need, today announced positive preclinical data for its CNS-penetrant NLRP3 inhibitor VTX3232 in murine diet-induced obesity models.
"We are excited by these data showing that in the diet-induced obesity mouse model, VTX3232 monotherapy demonstrated a reduction in body weight, body fat content, food consumption, liver triglycerides and liver fat deposits as well as improvements in insulin resistance, cardiometabolic parameters and biomarkers of systemic inflammation," said John Nuss, PhD, Chief Scientific Officer. "In addition, combining VTX3232 with the GLP-1 receptor agonist semaglutide demonstrated additive effects across these outcomes. These preclinical data increase our confidence in the role of NLRP3 in obesity and we look forward to initiating a Phase 2a trial of VTX3232 in participants with obesity and other cardiovascular risk factors during the second half of this year."
Two separate 28-day studies were conducted with VTX3232 in DIO mice. In DIO Study 1, VTX3232 and semaglutide were evaluated as monotherapies compared to standard diet and DIO vehicle (high fat diet) controls. DIO Study 2 included an additional treatment group evaluating VTX3232 in combination with semaglutide. Key findings are summarized below.
DIO Study 1 (VTX3232 monotherapy):
DIO Study 2 (VTX3232 in combination with semaglutide):
The Company intends to submit the comprehensive results of these studies for future publication or presentation in a scientific forum. Slides summarizing the results from the two DIO mouse studies can be found in the Investors section of the Company's website at www.ventyxbio.com .
About Ventyx Biosciences
Ventyx is a clinical-stage biopharmaceutical company focused on developing innovative oral medicines for patients living with autoimmune and inflammatory disorders. We believe our ability to efficiently discover and develop differentiated drug candidates will allow us to address important unmet medical need with novel oral therapies that can shift inflammation and immunology markets from injectable to oral drugs. Our current pipeline includes internally discovered clinical programs targeting NLRP3, S1P1R and TYK2, positioning us to become a leader in the development of oral immunology therapies for peripheral and neuroinflammatory diseases. Ventyx is headquartered in San Diego, California. For more information about Ventyx, please visit www.ventyxbio.com .
Forward-Looking Statements
Ventyx cautions you that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on Ventyx's current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding: the anticipated timing for the initiation of a Phase 2a trial of VTX3232 in participants with obesity and other cardiovascular risk factors in H2 2024; and the intention to submit for publication in a scientific journal the results of the studies described above. The inclusion of forward-looking statements should not be regarded as a representation by Ventyx that any of its plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Ventyx's business, including, without limitation: potential delays in the commencement, enrollment and completion of clinical trials; Ventyx's dependence on third parties in connection with product manufacturing, research and preclinical and clinical testing; disruptions in the supply chain, including raw materials needed for manufacturing and animals used in research, delays in site activations and enrollment of clinical trials; the results of preclinical studies and clinical trials not necessarily being predictive of future results; regulatory developments in the United States and foreign countries; unexpected adverse side effects or inadequate efficacy of Ventyx's product candidates that may limit their development, regulatory approval and/or commercialization, or may result in recalls or product liability claims; Ventyx's ability to obtain and maintain intellectual property protection for its product candidates; the use of capital resources by Ventyx sooner than expected; disruption to Ventyx's operations from the ongoing military conflicts in Ukraine and the Middle East, including clinical trial delays; and other risks described in Ventyx's prior press releases and Ventyx's filings with the Securities and Exchange Commission (SEC), including in Part II, Item 1A (Risk Factors) of Ventyx's Quarterly Report on Form 10-Q for the quarter ended March 31, 2024, filed on May 9, 2024, and any subsequent filings with the SEC. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Ventyx undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.
Investor Relations Contact
Patti Bank
Managing Director
ICR Westwicke
(415) 513-1284
IR@ventyxbio.com
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Longeveron Inc. (NASDAQ: LGVN), a clinical stage regenerative medicine biotechnology company developing cellular therapies for life-threatening and chronic aging-related conditions, today announced the launch of its contract development and manufacturing business at the Company's 15,000 square feet state-of-the-art Good Manufacturing Practice (GMP) facility. This facility contains 3,000 square feet of cleanroom space, including eight ISO 7 cleanrooms and ancillary areas, as well as 1,150 square feet of process development, quality control and warehousing space. The Company also announced the initiation of work under its first manufacturing services contract with Secretome Therapeutics a biotechnology company developing first-in-class therapeutics from neonatal mesenchymal stem cells (nMSC).
"We are delighted to partner with Secretome Therapeutics to advance their portfolio of therapeutics in this, our first contract manufacturing agreement in a new, revenue generating business line," said Wa'el Hashad , Chief Executive Officer of Longeveron. "With cellular therapy manufacturing expertise and capabilities in high demand, and Longeveron's strength in both, we see a significant opportunity to employ currently unused capacity in our state-of-the-art GMP facility. We have assembled a team of experts and proprietary technologies that enable us to take a systematic approach to rapidly develop improved cell therapies. Longeveron's manufacturing expertise, capabilities and facility provide other pharmaceutical organizations the ability to advance their development programs without building their own manufacturing facility. We believe this contract manufacturing opportunity can expand our team's experience and has the potential to generate approximately $4-5 million in annual revenues once it is up and running fully."
"Our platform of neonatal stem cell-based therapeutics has the potential to revolutionize treatment for a wide range of chronic, inflammatory diseases," said Vinny Jindal , President and Chief Executive Officer of Secretome Therapeutics. "As we move our lead product, STM-01, into clinical studies for HFpEF and dilated cardiomyopathy this year, we look forward to tapping into Longeveron's extensive cellular therapy knowledge and manufacturing expertise, which has supported the launch of multiple clinical studies."
Longeveron is primarily focused on advancing development of its lead investigational therapeutic candidate, Lomecel-B Â TM , a proprietary, scalable, allogeneic cellular therapy, across multiple indications, including hypoplastic left heart syndrome (HLHS) (Phase 2 on-going), Alzheimer's disease (Phase 2 completed), and Aging-related Frailty (Phase 2 completed).
About Longeveron Inc .
Longeveron is a clinical stage biotechnology company developing regenerative medicines to address unmet medical needs. The Company's lead investigational product is Lomecel-Bâ„¢, an allogeneic medicinal signaling cell (MSC) therapy product isolated from the bone marrow of young, healthy adult donors. Lomecel-Bâ„¢ has multiple potential mechanisms of action encompassing pro-vascular, pro-regenerative, anti-inflammatory, and tissue repair and healing effects with broad potential applications across a spectrum of disease areas. Longeveron is currently pursuing three pipeline indications: hypoplastic left heart syndrome (HLHS), Alzheimer's disease, and Aging-related Frailty. For more information, visit www.longeveron.com or follow Longeveron on LinkedIn , X , and Instagram .
Forward-Looking Statements
Certain statements in this press release that are not historical facts are forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, which reflect management's current expectations, assumptions, and estimates of future operations, performance and economic conditions, and involve risks and uncertainties that could cause actual results to differ materially from those anticipated by the statements made herein. Forward-looking statements are generally identifiable by the use of forward-looking terminology such as "believe," "expects," "may," "looks to," "will," "should," "plan," "intend," "on condition," "target," "see," "potential," "estimates," "preliminary," or "anticipates" or the negative thereof or comparable terminology, or by discussion of strategy or goals or other future events, circumstances, or effects and include, but are not limited to, the potential demand for Longeveron's contract manufacturing services and its ability to enter into additional service agreements. Factors that could cause actual results to differ materially from those expressed or implied in any forward-looking statements in this release include, but are not limited to, adverse global conditions, including macroeconomic uncertainty; inability to raise additional capital necessary to continue as a going concern; our history of losses and inability to achieve profitability going forward; the absence of FDA-approved allogenic, cell-based therapies for HLHS or other cardiac-related indications; ethical and other concerns surrounding the use of stem cell therapy or human tissue; our exposure to product liability claims arising from the use of our product candidates or future products in individuals, for which we may not be able to obtain adequate product liability insurance; the adequacy of our trade secret and patent position to protect our product candidates and their uses: others could compete against us more directly, which could harm our business and have a material adverse effect on our business, financial condition, and results of operations; if certain license agreements are terminated, our ability to continue clinical trials and commercially market products could be adversely affected; the inability to protect the confidentiality of our proprietary information, trade secrets, and know-how; third-party claims of intellectual property infringement may prevent or delay our product development efforts; the inability to successfully develop and commercialize our product candidates and obtain the necessary regulatory approvals; we cannot market and sell our product candidates in the U.S. or in other countries if we fail to obtain the necessary regulatory approvals; final marketing approval of our product candidates by the FDA or other regulatory authorities for commercial use may be delayed, limited, or denied, any of which could adversely affect our ability to generate operating revenues; we may not be able to secure and maintain research institutions to conduct our clinical trials; ongoing healthcare legislative and regulatory reform measures may have a material adverse effect on our business and results of operations; if we receive regulatory approval of Lomecel-Bâ„¢ or any of our other product candidates, we will be subject to ongoing regulatory requirements and continued regulatory review, which may result in significant additional expense; being subject to penalties if we fail to comply with regulatory requirements or experience unanticipated problems with our therapeutic candidates; reliance on third parties to conduct certain aspects of our preclinical studies and clinical trials; interim, "topline" and preliminary data from our clinical trials that we announce or publish from time to time may change as more data become available and are subject to audit and verification procedures that could result in material changes in the final data; provisions in our certificate of incorporation and bylaws and Delaware law might discourage, delay or prevent a change in control of our company or changes in our management and, therefore, depress the market price of our Class A common stock; we have never commercialized a product candidate before and may lack the necessary expertise, personnel and resources to successfully commercialize any products on our own or together with suitable collaborators; and in order to successfully implement our plans and strategies, we will need to grow our organization, and we may experience difficulties in managing this growth. Further information relating to factors that may impact the Company's results and forward-looking statements are disclosed in the Company's filings with the Securities and Exchange Commission, including Longeveron's Annual Report on Form 10-K for the year ended December 31, 2023, filed with the Securities and Exchange Commission on February 27, 2024, as amended by the Annual Report on Form 10-K/A filed March 11, 2024, its Quarterly Reports on Form 10-Q, and its Current Reports on Form 8-K. The forward-looking statements contained in this press release are made as of the date of this press release, and the Company disclaims any intention or obligation, other than imposed by law, to update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise.
Investor Contact:
Derek Cole
Investor Relations Advisory Solutions
derek.cole@iradvisory.com
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-- CB-010 allogeneic CAR-T cell therapy w ith partial HLA matching has potential to rival efficacy and safety profile of approved autologous CAR-T cell therapies --
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-- Plan to enroll ~20 additional 2L LBCL patients in ANTLER to confirm that partial HLA matching improves patient outcomes; initial data expected in H1 2025 --
-- Caribou expects to initiate a pivotal trial for CB-010 in H2 2025, upon confirmation of improved outcomes in partially HLA matched cohort --
-- Off-the-shelf CB-010 is partially HLA matched to patient within current screening timelines --
--Â KOL webcast discussion of data from 46 ANTLER patients scheduled for today at 7:00 pm CDT --
BERKELEY, Calif., June 02, 2024 (GLOBE NEWSWIRE) -- Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, today presented updated clinical data from the ongoing ANTLER Phase 1 trial that indicates a single dose of CB-010, a readily available, off-the-shelf anti-CD19 CAR-T cell therapy with a PD-1 knockout, has the potential to rival the safety, efficacy, and durability of approved autologous CAR-T cell therapies. The clinical results are being presented during a poster presentation at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting.
"The Phase 1 data from the ANTLER trial continues to be encouraging in terms of both safety and efficacy of an allogeneic CAR-T cell therapy," said Boyu Hu, MD, director of lymphoma and CLL in the division of hematology and hematologic malignancies at the University of Utah and an investigator on the ANTLER trial. "The partial human leukocyte antigen, or HLA, matching strategy is incredibly intriguing and further evaluation is supported by the ASCO data presentation. As many patients in ANTLER were enrolled due to rapid disease progression that prohibited waiting for an autologous CAR-T cell therapy, I look forward to enrolling patients who will receive partially HLA matched CB-010 in this ongoing trial."
In ANTLER, three dose levels of CB-010 were evaluated (40x10 6 , 80x10 6 , and 120x10 6 CAR-T cells) in a total of 46 patients. In dose escalation, 16 patients with multiple subtypes of aggressive relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL) were enrolled, and in dose expansion, 30 patients with second-line large B cell lymphoma (2L LBCL) were enrolled. As of an April 1, 2024 data cutoff date, results demonstrated:
"We are excited to see that patients who receive partially HLA matched CB-010 have improved efficacy and durability outcomes that are on par with approved autologous CAR-T cell therapies," said Rachel Haurwitz, PhD, Caribou's president and chief executive officer. "We next plan to prospectively evaluate this compelling observation by enrolling approximately 20 additional 2L LBCL patients, in either the inpatient or outpatient treatment setting, and we will ensure that they receive a partially matched (≥4 HLA matches) dose of CB-010. We are also excited to open the ANTLER study for the first time to patients who have relapsed following any prior CD19-targeted therapy in a proof-of-concept cohort for up to 10 patients. We expect to report initial data from both the 2L LBCL and CD19 relapsed cohorts in the first half of 2025 and, upon confirmation of improved outcomes in additional patients receiving a partially HLA matched dose of CB-010, we plan to initiate a pivotal Phase 3 clinical trial in 2L LBCL patients, including patients regardless of HLA type, in the second half of 2025."
ANTLER Phase 1 trial of CB-010 – median PFS analyses
A photo accompanying this announcement is available at: https://www.globenewswire.com/NewsRoom/AttachmentNg/893722aa-a457-4e0f-a27e-457bf8b2c0d3
CI: confidence interval; HLA: human leukocyte antigen; NE: not estimable; partial HLA matching: patient has ≥4 HLA alleles that match donor T cells used for CB-010 manufacturing
* Retrospective analysis of HLA allele matching for class I and class II antigens
ANTLER Phase 1 clinical trial as of April 1, 2024 cutoff date, data collection ongoing
ANTLER Phase 1 trial of CB-010 – response data
| Endpoints (N, %) | All patients ≤3 HLA matches (N=33) | All patients ≥4 HLA matches (N=13) | LBCL ≥4 HLA matches (N=11) |
| Overall response rate (ORR) | 23 (69%) | 12 (92%) | 10 (91%) |
| Duration of response (DoR), median months (range) | 2.0 (1-23+) | 13.5 (1-23+) | NR (1-15+) |
| Complete response (CR) rate | 15 (45%) | 6 (46%) | 4 (36%) |
| Duration of CR, median months (range) | 5.0 (1-23+) | NR (5-23+) | NR (5-15+) |
| 6-month PFS | 25% | 62% | 53% |
| PFS, median months (range) | 2.8 (1-24+) | 14.4 (2-24+) | NR (2-16+) |
HLA: human leukocyte antigen; NR: not reached; PFS: progression free survival
ANTLER Phase 1 clinical trial as of April 1, 2024 cutoff date, data collection ongoing
ANTLER Phase 1 trial of CB-010 – safety data
| All treated (N=46) | ||
| Any grade (n, %) | Grade ≥3 (n, %) | |
| Prolonged cytopenias | 9 (20) 1 | 9 (20) 1 |
| CRS | 26 (57) 2 | 0 (0) |
| Infections | 22 (47) 3 | 10 (22) 3 |
| ICANS | 10 (22) 4 | 3 (7) 5 |
| Hemophagocytic lymphohistiocytosis (HLH) | 1 (2) | 0 |
| GvHD | 0 | 0 |
CRS: cytokine release syndrome; GvHD: graft-versus-host disease; ICANS: immune effector cell-associated neurotoxicity syndrome
There were five patient deaths due to adverse events following CB-010 infusion; 4 were unrelated to CB-010 treatment and 1 death possibly related to CB-010 per investigator due to complications of a bladder perforation in the context of BK virus hemorrhagic cystitis
1 Prolonged cytopenias are defined as grade 3 or higher events lasting beyond 30 days following CB-010 infusion; 37/46 (80%) of patients recovered from cytopenias to grade ≤2 by day 35 post CB-010 treatment
2 Median time of onset was 3 days (range 0-22), and median duration was 3 days (range 1-19)
3 Infection events reported were on or after CB-010 infusion, with highest grade reported per patient; median onset 8 days (range 0-279) and median duration is 14 days (range 1-239)
4 Median time of onset was 7.5 days (range 6-34), and median duration was 2 days (range 1-27)
5 2 Grade 3 and 1 Grade 4; all resolved with supportive care. Median time of onset was 8 days and median duration was 2 days
ANTLER Phase 1 clinical trial as of April 1, 2024 cutoff date, data collection ongoing
Based on these encouraging data, Caribou plans to enroll approximately 20 additional 2L LBCL patients in ANTLER to prospectively confirm that partial HLA matching improves patient outcomes. The patient HLA allele typing occurs within the current screening timelines.
"Integrating the partial HLA matching into manufacturing for CB-010 is straightforward, enabling Caribou to deliver CB-010 as a readily available off-the-shelf CAR-T cell therapy that can serve a broad patient population," said Tim Kelly, Caribou's chief technology officer. "In our planned 2L LBCL pivotal Phase 3 trial, we will provide the best possible matched dose of CB-010 to each patient based on lot availability. With at least 13 manufacturing batches of CB-010 on hand, we expect that approximately 90% of all patients who could enroll in our trial would receive a dose of CB-010 with ≥4 matched alleles."
Webcast conference call Sunday, June 2, at 7:00 pm CDT
Caribou will host a live webcast on Sunday, June 2, at 7:00 pm CDT for a discussion with KOLs and management on the CB-010 ANTLER Phase 1 data presentation. The presenters will include:
Additional participants from Caribou Biosciences Include:
The listen-only webcast with an accompanying presentation will be accessible under Events in the Investors section of Caribou's website. The archived audio webcast will be available on the company's website following the call and will be available for 30 days.
ASCO poster presentation on Monday, June 3, 9:00 am-12:00 pm CDT
Details of the ANTLER poster presentation at the 2024 ASCO Annual Meeting are as follows:
Title: A CRISPR-edited allogeneic anti-CD19 CAR-T cell therapy with a PD-1 knockout (CB-010) in patients with relapsed/refractory B cell non-Hodgkin lymphoma (r/r B-NHL): Updated Phase 1 results from the ANTLER trial
Presenter: Boyu Hu, MD, assistant professor, director of lymphoma and CLL, division of hematology and hematologic malignancies, Huntsman Cancer Institute at the University of Utah
Date and time: Monday, June 3, 2024, 9:00 am-12:00 pm CDT
Session: Hematologic Malignancies – Lymphoma and CLL
Location: Hall A, Poster Board 8, McCormick Place, Chicago
Abstract number: 7025
About CB-010
CB-010 is the lead clinical-stage product candidate from Caribou's allogeneic CAR-T cell therapy platform, and it is being evaluated in patients with relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL) in the ongoing ANTLER Phase 1 clinical trial and will be evaluated in patients with lupus nephritis (LN) and extrarenal lupus (ERL) in the GALLOP Phase 1 clinical trial. In ANTLER, Caribou is enrolling second-line patients with large B cell lymphoma (LBCL) comprised of different subtypes of aggressive r/r B-NHL (DLBCL NOS, PMBCL, HGBL, tFL, and tMZL). To Caribou's knowledge, CB-010 is the first allogeneic CAR-T cell therapy in the clinic with a PD-1 knockout, a genome-editing strategy designed to improve activity against diseases by limiting premature CAR-T cell exhaustion. CB-010 is also, to Caribou's knowledge, the first anti-CD19 allogeneic CAR-T cell therapy to be evaluated in the second-line LBCL setting and, for r/r B-NHL, CB-010 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, and Orphan Drug designations by the FDA. Additional information on the ANTLER trial (NCT04637763) can be found at clinicaltrials.gov .
About Caribou's novel next-generation CRISPR platform
CRISPR genome editing uses easily designed, modular biological tools to make DNA changes in living cells. There are two basic components of Class 2 CRISPR systems: the nuclease protein that cuts DNA and the RNA molecule(s) that guide the nuclease to generate a site-specific, double-stranded break, leading to an edit at the targeted genomic site. CRISPR systems are capable of editing unintended genomic sites, known as off-target editing, which may lead to harmful effects on cellular function and phenotype. In response to this challenge, Caribou has developed CRISPR hybrid RNA-DNA guides (chRDNAs; pronounced "chardonnays") that direct substantially more precise genome editing compared to all-RNA guides. Caribou is deploying the power of its chRDNA technology to carry out high efficiency multiple edits, to develop CRISPR-edited therapies.
About Caribou Biosciences, Inc.
Caribou Biosciences is a clinical-stage CRISPR genome-editing biopharmaceutical company dedicated to developing transformative therapies for patients with devastating diseases. The company's genome-editing platform, including its Cas12a chRDNA technology, enables superior precision to develop cell therapies that are armored to potentially improve antitumor activity. Caribou is advancing a pipeline of clinical-stage off-the-shelf cell therapies from its CAR-T cell platform as readily available treatments for patients with hematologic malignancies and autoimmune diseases. Follow us @CaribouBio and visit www.cariboubio.com .
Forward-looking statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In some cases, you can identify forward-looking statements by terms such as "may," "will," "should," "expect," "plan," "anticipate," "could," "intend," "target," "project," "contemplate," "believe," "estimate," "predict," "potential," or "continue," or the negative of these terms or other similar expressions, although not all forward-looking statements contain these words. These forward-looking statements include, without limitation, statements related to Caribou's strategy, plans, and objectives, and expectations regarding the timing of status and updates from its ANTLER Phase 1 clinical trial for CB-010, including expectations regarding the enrollment of 20 additional 2L LBCL patients to further study partial HLA matching outcomes, the timing of reporting of initial data from both 2L LBCL and CD 19 relapsed cohorts, the timing of reporting additional dose expansion data from the ANTLER trial, and the timing of initiation of a pivotal Phase 3 clinical trial for CB-010 in 2L LBCL patients, including the conditions to meet that timeline. Management believes that these forward-looking statements are reasonable as and when made. However, such forward-looking statements are subject to risks and uncertainties, and actual results may differ materially from any future results expressed or implied by the forward-looking statements. Risks and uncertainties include, without limitation, risks inherent in the development of cell therapy products; uncertainties related to the initiation, cost, timing, progress, and results of Caribou's research and development programs, preclinical studies, and clinical trials; and the risk that initial, preliminary, or interim clinical trial data will not ultimately be predictive of the safety and efficacy of Caribou's product candidates or that clinical outcomes may differ as patient enrollment continues and as more patient data becomes available and is fully evaluated; the ability to obtain key regulatory input and approvals as well as other risk factors described from time to time in Caribou's filings with the Securities and Exchange Commission, including its Annual Report on Form 10-K for the year ended December 31, 2023 and subsequent filings. In light of the significant uncertainties in these forward-looking statements, you should not rely upon forward-looking statements as predictions of future events. Except as required by law, Caribou undertakes no obligation to update publicly any forward-looking statements for any reason.
Caution should be exercised when interpreting results from separate trials involving other CAR-T cell therapies. The results of other CAR-T cell therapies presented or referenced in this press release have been derived from publicly available reports of clinical trials not conducted by Caribou, and Caribou has not performed any head-to-head trials comparing any of these other CAR-T cell therapies with CB-010. As such, the results of these other clinical trials may not be comparable to clinical results for CB-010. The design of these other clinical trials varies in material ways from the design of the ANTLER clinical trial for CB-010, including with respect to patient populations, follow-up times, clinical trial phases, and subject characteristics. As a result, cross-trial comparisons may have no interpretive value on Caribou's existing or future clinical results. For further information and to understand these material differences, you should read the reports for the other CAR-T cell therapy clinical trials and the sources included in the webcast slide presentation.
Caribou Biosciences, Inc. contacts:
Investors:
Amy Figueroa, CFA
investor.relations@cariboubio.com
Media:
Peggy Vorwald, PhD
media@cariboubio.com
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