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Email: veruinvestor@verupharma.com
Veru Reports Fiscal 2025 Second Quarter Financial Results and Clinical Program Progress
Unblinded safety data from Phase 2b QUALITY study expected in the second quarter of calendar 2025
Topline efficacy and safety data for Phase 2b extension maintenance study expected in the second quarter of calendar 2025
With positive Phase 2b QUALITY study, Veru plans for end of Phase 2 meeting with FDA to discuss Phase 3 clinical program
Company to host conference call and webcast today at 8:00 a.m. ET
MIAMI, FL May 8, 2025 Veru Inc. (NASDAQ: VERU), a late clinical stage biopharmaceutical company focused on developing
innovative medicines for the treatment of cardiometabolic and inflammatory diseases, today announced financial results for its fiscal 2025 second quarter and provided an update on progress of its clinical development programs.
In addition to our previously disclosed robust and exciting topline efficacy data from the enobosarm Phase 2b QUALITY study, we are now looking forward
to the near-term Company catalysts this calendar quarter including the unblinded safety data from the enobosarm Phase 2b QUALITY study as well as the topline efficacy and safety data from the Phase 2b extension maintenance study. The Phase 2b
extension study should show us what happens to patients when they stop GLP-1 receptor agonist treatment but remain on enobosarm. The treatment objective is for patients to have a healthier option for choosing
to stop GLP-1s without the fear of fat regain, said Mitchell Steiner, M.D., Chairman, President, and Chief Executive Officer of Veru. We also expect to submit our End of Phase 2 meeting request to
FDA. We anticipate that the End of Phase 2 FDA meeting will provide regulatory clarity for the Phase 3 clinical program and should occur in the third quarter of calendar 2025.
Positive Phase 2b QUALITY clinical study: Enobosarm in combination with GLP-1 RA drugs makes weight reduction more
tissue selective for fat loss while preserving lean mass and physical function.
In January 2025, the Company announced positive topline results from
the Phase 2b QUALITY clinical study which is a multicenter, double-blind, placebo-controlled, randomized, dose-finding clinical trial designed to evaluate the safety and efficacy of enobosarm 3mg, enobosarm 6mg, or placebo as a treatment to augment
fat loss and to prevent muscle loss in older ( 60 years of age) patients receiving semaglutide (Wegovy ) for chronic weight management.
In the topline efficacy analysis, the trial met its prespecified primary endpoint with a statistically significant and a clinically meaningful benefit in all
patients receiving enobosarm + semaglutide versus placebo + semaglutide at 16 weeks in total lean mass (71% relative reduction in lean mass loss, p=0.002). Notably, the enobosarm 3mg + semaglutide was the best dose with a >99% mean relative
reduction in loss of lean mass (p <0.001). Enobosarm 6mg + semaglutide dose was not better than the Enobosarm 3mg + semaglutide dose on lean mass.
Topline Secondary Endpoints
Enobosarm + semaglutide
treatment resulted in dose dependent greater loss of fat mass compared to placebo + semaglutide with the enobosarm 6mg dose having a 46% greater relative loss of fat mass compared to placebo + semaglutide group at 16 weeks (p=0.014). Although
enobosarm + semaglutide significantly preserved lean mass, the additional loss of fat mass caused by enobosarm treatment was able to replace the lean mass preserved to allow a similar net mean weight loss with semaglutide at 16 weeks. Accordingly,
the tissue composition of the total weight loss shifted to greater and selective loss of fat with enobosarm treatment. The median percentage of total body weight loss in the placebo + semaglutide group that was due to lean mass was 32% and estimated
fat loss was 68%. In contrast, in the all enobosarm + semaglutide group, the total weight loss due to lean mass was 9.4% vs estimated fat loss of 90.6%, and for the enobosarm 3mg + semaglutide group, it was 0.9% lean mass vs 99.1% estimated fat
loss. Therefore, enobosarm + semaglutide improved changes in body composition resulting in more selective and greater loss of adiposity than in subjects receiving placebo + semaglutide.
Physical function was measured by the
Stair Climb Test. Climbing stairs is an activity of daily living, and the Stair Climb Test measures functional muscle strength, balance and agility. Declines in performance measured by Stair Climb Test predicts in older patients a higher risk for
mobility disabilities, gait difficulties, falls and bone fractures, hospitalizations, and mortality. As a point of reference, stair climb power declines by -1.38% annually with aging according to Van Roie E.
PLOS ONE 14:e0210653, 2019.
Topline Results Conclusion
Enobosarm represents a novel drug that improves GLP-1 RA therapy resulting in tissue SELECTIVE quality weight
reduction, that is, enobosarm + semaglutide improved changes in body composition which resulted in more selective and greater loss of adiposity (fat mass) than in subjects receiving placebo + semaglutide alone.
Phase 2b QUALITY Clinical Trial Safety
the Phase 2b QUALITY study remains blinded as the Phase 2b extension clinical study portion is ongoing. The unblinded Phase 2b QUALITY clinical trial safety will be available this quarter. However, to the Company s knowledge, the aggregate,
blinded safety data have to date not shown any significant differences compared to what is expected based on previous studies of enobosarm or GLP-1 RAs.
Phase 2b Extension Maintenance Study
the efficacy dose-finding portion of the Phase 2b QUALITY clinical trial, the participants continued into a Phase 2b extension trial where all patients have stopped treatment with semaglutide, but continued taking placebo, enobosarm 3mg, or
enobosarm 6mg in a blinded fashion for 12 weeks. The Phase 2b extension clinical trial is evaluating whether enobosarm can maintain muscle and prevent the fat regain that generally occurs after discontinuing a
GLP-1 RA. The topline efficacy and safety results for the Phase 2b extension clinical study are expected this quarter.
Regulatory Next Steps
As the Phase 2b QUALITY clinical
trial is a positive study, we plan to request an End of Phase 2 meeting with the FDA. During the preIND FDA meeting, FDA provided general comments about a regulatory path forward for enobosarm as a drug that improves body composition during weight
loss including input on Phase 3 clinical program design.
As a path forward, we plan to propose a Phase 3 clinical program that is similar to the positive
Phase 2b QUALITY clinical trial. The proposed Phase 3 clinical trial design is a double-blind, placebo-controlled study in older patients, greater than or equal to 60 years of age, who have obesity or who are overweight and who are eligible for
treatment with GLP-1 RA. The GLP-1 RA may be either WEGOVY (semaglutide) and/or Zepbound (tirzepatide).
Patients will be randomized to oral daily enobosarm or matching placebo. All subjects will start and receive GLP-1 RA during the study. The proposed primary objective will be the effect of enobosarm on
physical function measured by the Stair Climb Test at 24 weeks. Proposed key secondary objectives will be to assess the effect of enobosarm on total lean mass, total fat mass, HOMA-IR, and hemoglobin A1c at 24
After the Phase 3 clinical trial ends at 24 weeks of treatment, the plan is to continue to measure total lean mass, total body weight, stair
climb, total fat mass, bone mineral density, HOMA-IR, and hemoglobin A1c up to 68 weeks to capture the longer-term benefits of enobosarm improvements on body composition for greater loss of adiposity or fat,
weight reduction, and preservation of both lean mass and bone.
Novel Modified Release Oral Enobosarm Formulation is on Track to be Available for Phase
3 Clinical Studies and Commercialization
Veru is currently developing a novel, patentable, modified release oral formulation for enobosarm. The actual
formulation, pharmacokinetic release profile(s), and method of manufacturing will be the subjects of future patent applications. If issued, the expiry for the new modified release oral enobosarm formulation patent is expected to be 2045. The new
enobosarm formulation has completed animal trials and is anticipated to be in Phase 1 bioavailability clinical trials during the first half of calendar 2025. The expectation is that this novel modified release oral enobosarm formulation will be
available for the Phase 3 clinical studies and for commercialization.
Atherosclerosis Inflammation Program
Veru has evolved its drug development strategy for sabizabulin, a novel oral broad anti-inflammatory agent, and is exploring the possibility of its clinical
development for the treatment of inflammation in atherosclerotic cardiovascular disease.
About Atherosclerotic Coronary Artery Disease
Atherosclerotic coronary artery disease (CAD) remains the leading cause of mortality worldwide. Inflammation and high cholesterol jointly contribute to
atherosclerotic cardiovascular disease. It appears that the pathogenesis and progression of coronary artery disease, however, is largely driven by inflammation in response to atheromatous plaques containing cholesterol in the arterial wall. Even
with maximum cholesterol reduction therapies, there remains a major and largely untreated residual inflammatory risk. The realization that the combined use of aggressive lipid-lowering and inflammation-inhibiting therapies might be needed to further
reduce atherosclerotic risk has sparked the search for anti-inflammatory medications that could lower the risk of atherosclerotic events in patients with CAD.
An old drug, colchicine, inhibits tubulin polymerization to disrupt microtubules resulting in broad anti-inflammatory activity. Recent randomized controlled
trials assessing the role of low-dose colchicine to treat inflammation to reduce major adverse cardiovascular events had promising results demonstrating significant cardiovascular risk reduction. Colchicine
lowered major adverse cardiovascular events by 31% among those with stable CAD and by 23% in patients following a recent myocardial infarction. This magnitude of benefit is greater than what has been observed in contemporary trials of lipid lowering
medications including those with proprotein convertase subtilisin/kexin type 9 (PCSK-9) inhibitors. Data from these trials led the FDA in June 2023 to approve colchicine as the first anti-inflammatory drug for
reducing cardiovascular events in adults with established atherosclerotic cardiovascular disease.
However, while colchicine may be the first FDA approved
drug to treat atherosclerotic inflammation, unfortunately colchicine has significant safety concerns that may limit its expected widespread use. Colchicine has high potential for drug-drug interactions with commonly used cardiovascular drugs
including almost all statins (HMG-CoA reductase inhibitors). In contrast, Veru s sabizabulin is a new molecular entity, small molecule that targets the colchicine binding site on -tubulin. Like
colchicine, sabizabulin inhibits microtubule polymerization and has demonstrated the ability to reduce the most important inflammatory mediators that play a role in the initiation and progression of atherosclerotic CAD. In contrast to colchicine,
sabizabulin has stable pharmacokinetics and low potential for drug-drug interactions; thus, sabizabulin may be administered potentially more safely as a secondary therapy in combination with statin therapy for the reduction of inflammation to slow
the progression or promote regression of atherosclerotic cardiovascular disease. Overall preclinical data from in vitro and in vivo inflammation studies show that sabizabulin treatment suppressed all cytokines and chemokines tested. In Phase 2 and 3
pulmonary inflammation COVID-19 clinical studies, sabizabulin has demonstrated broad anti-inflammatory activity. The safety database consists of 266 dosed patients from the previous sabizabulin clinical
development programs.
The Company s decision to explore this major cardiometabolic indication was based on the significant unmet medical need to
treat inflammation in atherosclerotic cardiovascular disease, the large global market opportunity, current clinical and safety sabizabulin database of 266 patients, high probability of success given that sabizabulin drug s mechanism of action
is similar to colchicine, strong intellectual property position, and is consistent with Company s focus on cardiometabolic diseases. Furthermore, the Company believes sabizabulin may be evaluated in a small Phase 2 dose finding proof of concept
study to assess the progression of coronary atherosclerosis in patients using as the primary endpoint coronary plaque volume and composition measured by coronary CT angiography imaging. If the Company decides to pursue the Phase 2 clinical study,
the Company plans to partner with the Colorado Prevention Center, Aurora, Colorado and Lundquist Institute, Torrence, California.
Veru had a pre-IND meeting with the FDA Division of Cardiology and
Nephrology Center for Drug Evaluation and Research on December 26, 2024. The indication for discussion was the use of sabizabulin to slow progression or promote regression of atherosclerotic disease in patients with a history of coronary artery
disease. The FDA agreed that there remains an unmet medical need based on disease pathophysiology and concurred with the general design of the small Phase 2 study using coronary CT angiography imaging as primary endpoint. The FDA also requested that
the Company conduct chronic nonclinical toxicology animal studies to support the chronic use of sabizabulin for this indication. The chronic nonclinical toxicology studies are expected to be completed and a new IND for the proposed indication is
planned to be submitted by the first half calendar 2026. Veru currently has sufficient drug substance to supply the proposed Phase 2 clinical study.
Second Quarter Financial Summary: Fiscal 2025 vs Fiscal 2024
Year-to-Date Financial Summary: Fiscal 2025 vs
Balance Sheet Information
The audio webcast will be accessible under the Home page and Investors page of the Company s website at www.verupharma.com. To join the
conference call via telephone, please dial 1-800-341-1602 (domestic) or 1-412-902-6706 (international) and ask to join the Veru Inc. call. An archived version of the audio webcast will be available for replay on the Company s website
for approximately three months. A telephonic replay will be available at approximately 12:00 p.m. ET by dialing 1-877-344-7529