Aviptadil

PALO ALTO, Calif., April 19, 2023 (GLOBE NEWSWIRE) -- Vincerx Pharma Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, today presented a poster of VIP924 preclinical data at the 2023 American Association for Cancer Research (AACR) Annual Meeting. VIP924 consists of an anti-CXCR5 humanized monoclonal antibody with a novel linker containing a unique peptide sequence specifically cleaved by legumain—a tumor-associated lysosomal protease. Inside the tumor cell, upon legumain cleavage, the active payload, VIP716, is released. VIP716 is a novel highly potent and selective kinesin spindle protein inhibitor (KSPi), which has been modified with a hydrophilic CellTrapper™ moiety to reduce membrane permeability without having a negative impact on efficient target binding. This modification allows for intracellular accumulation of VIP716 in CXCR5+ tumor cells, providing greater efficacy, and prevents cellular penetration into healthy tissues, reducing toxicity. “We are thrilled to continue advancing our bioconjugation platform with our newest ADC, VIP924,” said Ahmed Hamdy M.D., Chief Executive Officer of Vincerx. “At AACR, VIP924 showed meaningful preclinical activity in a panel of lymphoma cell lines and in cell line-derived and patient-derived lymphoma mouse models. Most notably, mouse models implanted with tumor cells from lymphoma patients demonstrated significant tumor growth inhibition and prolonged survival after VIP924 treatment. Showing activity in mouse models implanted with tumor cells from patients, also known as PDX models, is particularly meaningful because PDX models are more representative of the complexity of human cancer than results seen with cell lines.” Dr. Hamdy continued, “Despite progress in the treatment of B-cell malignancies, there is still an unmet medical need, especially for patients with relapsed or refractory disease. The results presented at AACR, together with our previous efficacy findings of durable complete tumor regression in CXCR5-positive, ibrutinib-refractory lymphoma models, suggest that VIP924 has the potential to be a treatment option for relapsed and refractory patients. Looking at the totality of preclinical data we’ve generated, including the data we presented at ASH that showed how our next generation linker, payload, and CellTrapper™ technology significantly improved the safety and efficacy of the approved ADC, Mylotarg™, we can’t help but be excited about the future of our ADC programs. We look forward to filing the IND for VIP924 in mid-2024.” Key Presentation Highlights: Poster presentation, titled, CXCR5 is a very promising drug target for the development of antibody-drug conjugates to treat patients with lymphoma, presented by Tibor Schomber, Ph.D., Vincerx Pharma GmbH, Monheim, Germany, include: High expression of CXCR5 was observed by immunohistochemistry on naive and previously treated diffuse large B-cell lymphoma (DLBCL), follicular lymphoma (FL), mantle cell lymphoma (MCL), and chronic lymphocytic leukemia (CLL) samples; with limited expression in healthy tissues. VIP924 showed superior in vitro cytotoxicity across different non-Hodgkin lymphoma cell lines compared with other B-cell lymphoma targeting antibodies conjugated to the same effector chemistry including, CD19-KSPi, CD22-KSPi, and CD70-KSPi. VIP924 (10mg/kg) resulted in significant tumor growth inhibition in two DLBCL patient-derived tumor models: 68% in a low CXCR5-expressing model and 87% in a high CXCR5-expressing model. Prolonged survival of VIP924-treated animals was observed in both models. No effect on body weight or any adverse effects in the VIP924-treated mice were observed. To determine the efficacy of VIP924 in large, established tumors, a single VIP924 dose of 10mg/kg was administered in mice transplanted with the HBL-1 lymphoma cell line. Complete responses were observed in 2 out of 3 mice with no measurable tumors on treatment day 26. After 24 and 48 hours, accumulation of VIP716 (ie, payload; “metabolite”) was observed in the tumors but not in plasma. KSP inhibition resulted in mitotic arrest and the formation of characteristic monopolar spindles (monoasters). VIP924 treatment produced higher levels and longer duration of monoaster formation in a lymphoma cell line compared to the permeable, small molecule KSPi, ispinesib. The poster can be accessed on the presentations section of the Vincerx website. About Vincerx Pharma, Inc. Vincerx Pharma, Inc. (Vincerx) is a clinical-stage biopharmaceutical company committed to developing differentiated and novel therapies to address the unmet medical needs of patients with cancer. Vincerx has assembled a seasoned management team with a proven track record of successful oncology drug development, approvals, and value creation. The company’s diverse pipeline consists of enitociclib, currently in Phase 1, and a proprietary modular bioconjugation platform, which includes a small molecule drug-conjugate, VIP236, in Phase 1, and preclinical next-generation antibody drug conjugates, VIP943 and VIP924. Vincerx is based in Palo Alto, Calif., and has a research facility in Monheim, Germany. For more information, please visit . Cautionary Statement This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended, that are intended to be covered by the “safe harbor” created by those sections. Forward-looking statements, which are based on certain assumptions and describe future plans, strategies, expectations and events, can generally be identified by the use of forward-looking terms such as “believe,” “expect,” “may,” “will,” “should,” “would,” “could,” “suggest,” “seek,” “intend,” “plan,” “goal,” “potential,” “on-target,” “on track,” “project,” “estimate,” “anticipate,” or other comparable terms. All statements other than statements of historical facts included in this press release are forward-looking statements. Forward-looking statements include, but are not limited to: Vincerx’s business model, pipeline, strategy, timeline, product candidates and attributes, and preclinical and clinical development, timing, and results. Forward-looking statements are neither historical facts nor assurances of future performance or events. Instead, they are based only on current beliefs, expectations and assumptions regarding future business developments, future plans and strategies, projections, anticipated events and trends, the economy and other future conditions. Forward-looking statements are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict and many of which are outside of our control. Actual results, conditions and events may differ materially from those indicated in the forward-looking statements. Therefore, you should not rely on any of these forward-looking statements. Important factors that could cause actual results, conditions, and events to differ materially from those indicated in the forward-looking statements include, but are not limited to: general economic, financial, legal, political, and business conditions; the potential effects of health epidemics and pandemics, including COVID-19; risks associated with preclinical or clinical development and trials, including those conducted prior to Vincerx’s in-licensing; failure to realize the benefits of Vincerx’s license agreement with Bayer; risks related to the rollout of Vincerx’s business and the timing of expected business milestones; changes in the assumptions underlying Vincerx’s expectations regarding its future business or business model; Vincerx’s ability to develop and commercialize product candidates; Vincerx’s capital requirements and availability and uses of capital; and the risks and uncertainties set forth in Forms 10-K, 10-Q, and 8-K most recently filed with or furnished to the SEC by Vincerx. Forward-looking statements speak only as of the date hereof, and Vincerx disclaims any obligation to update any forward-looking statements. Vincerx and the Vincerx logo are our trademarks. This press release also contains trademarks and trade names that are the property of their respective owners. Contacts Gabriela Jairala Vincerx Pharma, Inc. gabriela.jairala@vincerx.com Bruce Mackle LifeSci Advisors, LLC 646-889-1200 bmackle@lifesciadvisors.com

Relief Therapeutics Announces Positive 12-Month Stability Data for Inhaled and Intravenous Preparations of RLF-100 ® (Aviptadil Acetate) Relief to revised provisional patent application for RLF-100 ® based on the new stability results GENEVA, SWITZERLAND / ACCESSWIRE / April 17, 2023 / RELIEF THERAPEUTICS Holding SA (SIX:RLF)(OTCQB:RLFTF)(OTCQB:RLFTY) (Relief Therapeutics, or Relief), a biopharmaceutical company committed to delivering innovative treatment options with the potential for transformative outcomes to benefit those suffering from select specialty and rare diseases, today announced positive 12-month stability data for the liquid and lyophilized preparations of RLF-100 ® , intended for intravenous (IV) and inhaled administration. RLF-100 ® is the company's proprietary, investigational formulation of aviptadil acetate. The data from the stability study showed that both inhaled and IV RLF-100 ® demonstrated high purity levels at 12 months at all temperatures tested, including refrigerated and room temperature environments. The results are consistent with prior data observed at three- and six-month intervals. The stability testing study will continue to determine the maximum shelf life of RLF-100 ® . Based on the latest results, Relief Therapeutics intends to amend its previously filed provisional patent application for RLF-100 ® with the new findings. If granted, this patent could provide exclusivity for RLF-100 ® at least until 2042, without considering Hatch-Waxman extensions or other patent term adjustments. The Hatch-Waxman Act permits a patent extension term of up to five years as compensation for patent term lost during the FDA regulatory review process. "The testing conducted to date has consistently shown that our novel, optimized composition of aviptadil acetate provides better stability results in both the liquid and lyophilized preparations. RLF-100 ® was shown to be shelf stable and active after one year at temperatures suitable for shipping and long-term storage. This is an important milestone toward commercialization to ensure that RLF-100 ® is safe and effective, no matter where in the world it is supplied." said Jack Weinstein, chief executive officer at Relief Therapeutics. "We believe this new, stable formulation has significant clinical and commercial value and our goal is to establish RLF-100 ® as the standard of care for the prevention and treatment of respiratory failure and its complications in both the acute and chronic applications." Stability testing of pharmaceutical products is mandatory for regulatory approvals. If a product fails to meet the standards prescribed by regulatory authorities, the product will not be granted approval for commercialization. Planning, execution and completion of studies in given timelines plays a major role in securing approval and ensuring a product reaches patients who need it. RLF-100 ® is under development for the potential treatment of acute and chronic lung diseases, including pulmonary sarcoidosis, infectious acute respiratory distress syndrome (ARDS), checkpoint inhibitor-induced pneumonitis (CIP) and chronic berylliosis. The U.S. Food and Drug Administration (FDA) granted RLF-100 ® Orphan Drug designation (ODD) to inhaled RLF-100 ® for the potential treatment of pulmonary sarcoidosis in August 2021. About Drug Stability Requirements The purpose of long-term and accelerated stability testing is to determine how long a drug product will maintain the properties and characteristics, such as purity, efficacy and structure, it possessed at the time of manufacture as well as the quality of a drug product over time while under the influence of environmental conditions such as temperature, humidity and light. Under long-term and accelerated stability storage conditions, the drug is evaluated by high performance liquid chromatography (HPLC) to separate, identify and quantify each chemical component to a very high degree of resolution to assess if any changes occur in the chemical composition. Stability studies are conducted during all phases of drug development to satisfy the regulatory requirements for clinical trials. Data from these studies is then used to establish recommended storage conditions, retest intervals and shelf life. Demonstrating long-term stability of a drug to be used in human clinical testing is essential to prove the potency and efficacy of the drug is not affected during prolonged shelf life. Long-term stability is completed over three years, with reporting at 0, 3, 6, 9, 12, 18, 24 and 36 months. Accelerated stability is completed over six months, with reporting at 0, 3 and 6 months. [1] , [2] About RLF-100 ® (aviptadil acetate) Aviptadil acetate is a synthetic form of vasoactive intestinal peptide (VIP) comprising 28 amino acids, which was first discovered in 1970. Although initially identified in the intestinal tract, human VIP is now known to be produced throughout the body and primarily concentrated in the lungs. VIP has shown a multimodal mechanism of action: decrease of inflammatory cytokines release leading to prevention of cytokine storm syndrome and viral replication, immunomodulating effect, vasodilating and bronchodilating effects and prevention of surfactant depletion. Seventy percent of VIP in the body is bound to a less common type of cell in the lung, the alveolar epithelial type II (AT2) cell, which is critical to the absorption of oxygen into the body. Aviptadil acetate has a 20-year history of safe use in humans in multiple human trials for sarcoidosis, idiopathic pulmonary fibrosis, asthma, pulmonary arterial hypertension and sepsis-induced acute respiratory distress syndrome. For example, a combination of aviptadil with phentolamine is approved for the treatment of erectile dysfunction by intra-cavernous injections in countries outside the U.S. In early 2022, an unrelated pharmaceutical company in India received emergency use approval for its own formulation of aviptadil for the treatment of COVID-19 from the Drugs Controller General of India. While their approval does not affect the application for approval of our formulation in other countries, it substantiates our original hypothesis that RLF-100 is a potentially viable treatment for COVID-19-related ARDS. About Relief Therapeutics Relief Therapeutics is a commercial-stage biopharmaceutical company committed to advancing treatment paradigms and delivering improvements in efficacy, safety and convenience to benefit the lives of patients living with select specialty and rare diseases and disorders. Relief Therapeutics' portfolio offers a balanced mix of marketed, revenue-generating products, our proprietary, globally patented Physiomimic™ and TEHCLO™ drug delivery platform technologies and a highly targeted clinical development pipeline consisting of risk-mitigated assets to address rare metabolic disorders, rare skin diseases and rare respiratory diseases. In addition, Relief Therapeutics is commercializing several legacy products via licensing and distribution partners. Our mission to provide therapeutic relief to those suffering from rare diseases and disorders is being advanced by an international team of well-established, experienced biopharma industry leaders with extensive research, development and rare disease expertise. Relief Therapeutics' headquarters are in Geneva, with additional offices in Balerna, Switzerland, Offenbach am Main, Germany and Rome. Relief Therapeutics is listed on the SIX Swiss Exchange under the symbol RLF and quoted in the U.S. on OTCQB under the symbols RLFTF and RLFTY. For more information, please visit our website at or follow Relief Therapeutics on LinkedIn and Twitter . FOR MEDIA/INVESTOR INQUIRIES CONTACT: RELIEF THERAPEUTICS Holding SA Catherine Day Vice President, IR & Communications contact@relieftherapeutics.com LifeSci Advisors Irina Koffler +1-917-734-7387 ikoffler@lifesciadvisors.com DISCLAIMER This press release contains forward-looking statements. Forward-looking statements involve known and unknown risks, uncertainties, and other factors, which could cause the actual results, financial condition, performance or achievements of Relief Therapeutics to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. A number of factors, including those described in Relief Therapeutics' filings with the SIX Swiss Exchange and the U.S. Securities and Exchange Commission (SEC), could adversely affect Relief Therapeutics. Copies of Relief Therapeutics' filings with the SEC are available on the SEC EDGAR database at Relief Therapeutics does not undertake any obligation to update the information contained herein, which speaks only as of this date. REFERENCES [1] International Council for Harmonisation (ICH). Technical Requirements for Pharmaceuticals for Human Use. Quality Guidelines: Q1A - Q1F Stability. Available at: . Accessed on April 14, 2023. [2] U.S. Food and Drug Administration, Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER). May 2021. Guidance For Industry: Pharmaceutical Quality/Chemistry, Manufacturing & Controls (PQ/CMC). Available at: . Accessed on April 14, 2023. SOURCE: Relief Therapeutics Holdings AG View source version on accesswire.com: