ASCO 2019 preview: Big Pharmas looking for cancer R&D revival as we hit next-gen crossroads

fiercebiotech | May 31, 2019

Its that time of year again, when the big and the small come together to shout about (or try to quietly hide) their latest cancer data at the American Society of Clinical Oncology (ASCO) annual meeting.The last few years, if were honest, have not created the most exciting sets of oncology data weve ever seen. Were at something of a crossroads: A few years back, we had the late-stage results from checkpoint inhibitors showing us their promise, and then we had the rise of cell therapies in the form of Gilead Sciences (nee Kite Pharma) and Novartis chimeric antigen receptor T-cell (CAR-T)therapies.

Spotlight

Glowing animals and plants seem to be all the rage these days. A biotechnology company based out of St. Louis recently unveiled a commercially available plant that is entirely auto luminescent.

Spotlight

Glowing animals and plants seem to be all the rage these days. A biotechnology company based out of St. Louis recently unveiled a commercially available plant that is entirely auto luminescent.

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CELL AND GENE THERAPY

Aeglea BioTherapeutics Announces Leadership Transition and Corporate Restructuring

Aeglea BioTherapeutics, Inc. | August 25, 2022

Aeglea BioTherapeutics, Inc. a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare metabolic diseases, today announced a leadership transition and corporate restructuring to focus resources on AGLE-177 in development for patients living with Homocystinuria. The restructuring has further reduced the headcount of the Company resulting in an approximately 25% reduction year-to-date. The Company also plans to realize additional cost savings by transitioning patients out of the ongoing extension studies of pegzilarginase in Arginase 1 Deficiency while it engages with the FDA on a regulatory path forward. The implementation of the restructuring plan will prioritize development of AGLE-177 and result in extending the Company's cash runway into the fourth quarter of 2023. "The restructuring announced today increases the operational focus and capital deployment towards success of the AGLE-177 program in Homocystinuria. We believe AGLE-177 has the potential to become the best-in-class treatment option for these patients and we believe focused execution of the current Phase 1/2 trial and its transition to late-stage development are key strategic priorities for Aeglea." Russell J. Cox, chair of the board of directors of Aeglea The Company announced that Anthony Quinn, M.B., Ch.B., Ph. D., president and chief executive officer, and member of Aeglea's board of directors, has stepped down from his current positions and transitioned to an advisory role effective immediately. The board of directors has appointed Jim Kastenmayer, Ph.D., J.D., the Company's current general counsel, as interim president and chief executive officer as the Company initiates a search for a permanent replacement. Additionally, the Company's chief commercial officer, Michael Hanley, has been appointed chief business officer with expanded responsibilities in internal business operations. Mr. Cox continued, "On behalf of the board of directors, I want to thank Anthony for his leadership and unwavering commitment to patients during his tenure. Under his leadership, Aeglea has made substantial progress over the last five years with programs for ARG1-D and homocystinuria in clinical development and a research pipeline of other human enzyme-based therapeutics. He has built a strong, capable team with the experience to advance AGLE-177 through clinical and commercial development. Aeglea will continue to benefit from Anthony's scientific and clinical expertise, and I look forward to working with Anthony in his new role as an advisor to the Company." "It has been an enormous privilege to have led the evolution of Aeglea from a research organization to a company with development, manufacturing and commercial capabilities," said Dr. Quinn. "I am honored to have worked with the incredible team at Aeglea and am very excited about Aeglea's upcoming milestones. I look forward to continuing my engagement with the company in an advisory role." "I and the rest of the organization are excited to continue our work and focus on our mission to change lives by bringing innovative therapies to underserved rare disease communities. Delivering data from our Phase 1/2 trial for AGLE-177 later this year is our near-term focus in advancing this mission," said Mr. Kastenmayer. About AGLE-177 in Homocystinuria AGLE-177 is a novel recombinant human enzyme, which is engineered to degrade the amino acid homocysteine and its dimer. AGLE-177 is currently being studied in a Phase 1/2 clinical trial for the treatment of patients with Classical Homocystinuria, a rare inherited disorder of methionine metabolism that results in elevated levels of total homocysteine. Homocysteine accumulation plays a key role in multiple progressive and serious disease-related complications, including thromboembolic vascular events, skeletal abnormalities (including severe osteoporosis), developmental delay, intellectual disability, lens dislocation and severe near sightedness. Preclinical data demonstrated that AGLE-177 improved important disease-related abnormalities and survival in a mouse model of Homocystinuria. AGLE-177 has received both U.S. and EU Orphan Drug Designation as well as U.S. Rare Pediatric Disease Designation. About Pegzilarginase in Arginase 1 Deficiency Pegzilarginase is a novel recombinant human enzyme engineered to degrade the amino acid arginine and has been shown to rapidly and sustainably lower levels of the amino acid arginine in plasma. Aeglea is developing pegzilarginase for the treatment of people with Arginase 1 Deficiency (ARG1-D), a rare debilitating and progressive disease characterized by the accumulation of arginine. ARG1-D presents in early childhood and patients experience spasticity, seizures, developmental delay, intellectual disability and early mortality. The PEACE Phase 3 clinical trial met its primary endpoint with a 76.7% reduction in mean plasma arginine compared to placebo. Additionally, 90.5% of pegzilarginase treated patients achieved normal plasma arginine levels. The arginine lowering was accompanied by a positive trend in Gross Motor Function Measure Part E, a measure of patient mobility. About Aeglea BioTherapeutics Aeglea BioTherapeutics is a clinical-stage biotechnology company redefining the potential of human enzyme therapeutics to benefit people with rare metabolic diseases with limited treatment options. Aeglea is investigating AGLE-177 in an ongoing Phase 1/2 clinical trial for the treatment of Homocystinuria. AGLE-177 has been granted Rare Pediatric Disease Designation. Aeglea's other clinical program, pegzilarginase, achieved the primary endpoint of arginine reduction in the PEACE Phase 3 clinical trial and has received both Rare Pediatric Disease and Breakthrough Therapy Designations. Aeglea has an active discovery platform focused on engineering small changes in human enzymes to have a big impact on the lives of patients and their families.

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INDUSTRIAL IMPACT

Enveric Biosciences Advances Drug Development for Mental Health Indications and Intellectual Property Portfolio

Enveric Biosciences, Inc | July 14, 2022

Enveric Biosciences, Inc. a neuroscience-focused biotechnology company developing next-generation, psychedelic-inspired mental health medicines, today revealed drug discovery and development progress targeting mental health indications and intellectual property advances achieved during the first half of 2022. “The first half of 2022 was foundationally successful for Enveric. We validated and executed on the corporate premise that we can deploy PsyAITM, our proprietary artificial intelligence tool, and PsybraryTM, our expanding proprietary portfolio of new chemical entities and drug candidates, to design and improve upon new drug molecules inspired by legacy psychedelic drugs,” Dr. Joseph Tucker, PhD, Enveric’s Chief Executive Officer “Enveric is pleased to advise that our predictions have materialized, and our platforms have successfully generated a large number of promising new molecules that have successfully passed primary screening and moved on to lead optimization, providing us with a strong slate of drug candidates. Our leading program, EVM-201, includes a number of new molecules currently in lead optimization that are geared towards providing improved characteristics and drug-like properties compared with psilocybin. The successful outcomes from our testing efforts completed in the first half of 2022 have positioned the company very well to advance our drug candidates towards the clinic,” continued Dr. Tucker. Clinical-Need Driven Based on extensive direct clinical understanding of the current unmet needs in anxiety, depression, PTSD, and other mental health indications, coupled with in-depth knowledge of the current treatment landscape, Enveric’s Clinical Team is well positioned to tackle the challenges ahead, while aiming to shepherd novel new treatments for a host of mental health indications. Enveric’s Clinical Team is led by a past Board-certified psychiatrist and clinical trialist Chief Medical Officer, Dr. Bob Dagher, MD. Dr. Dagher joined Enveric in December 2021, bringing a wealth of clinical trial management and new CNS-drug development experience from his past roles, including at WCG, Cadent, Covance, Sanofi and GSK, in addition to his first-hand experience in treating patients from his previous clinical practice in psychiatric medicine. Next-Generation Drug Discovery Enveric’s Discovery Team, spearheaded by Chief Innovation Officer Dr. Peter Facchini, PhD, an international leader in natural product biochemistry and biotechnology, completed the rational design of more than 300 Generation-2 (“Gen-2”) psilocin prodrug candidates using well-established design strategies, but adapted to the unique structure of psilocin. The 300 Gen-2 original concept designs, now housed within the PsybraryTM, were screened using PsyAITM. The assessment helped predict chemical structures with appropriate receptor binding, toxicology, and pharmacokinetic profiles that would trigger the desired mechanisms of action while reducing undesirable side effects, and the initial candidate evaluation included, among other metrics, prodrug-likeness, toxicity, ease of chemical synthesis and degree of IP-white space surrounding the molecule. Ranked scoring of these and other AI assessments, as well as commercial considerations (e.g., ease of procuring synthetic starting materials), prioritized 31 novel Gen-2 molecules that have been synthesized in sufficient quantities (~10 mg, 95% purity) for preliminary pharmacological screening. Enveric’s Chemistry Group, led by Dr. Kaveh Matinkhoo, PhD, completed these syntheses, 29 of which were deemed stable enough to advance. Deep-dive chemical structure searches, patentability, and freedom-to-operate reviews were performed on all 29 candidates, under the leadership of Lead Internal Counsel C. Michael Gegenheimer. All the patentability analyses indicated potential patentability results, and 25 of the 29 molecules presented minimal concern with freedom to operate. To strengthen the IP protection for the overall Gen-2 prodrug program, three provisional patent applications, covering 7 structurally diverse clinical classes and thousands of additional potential new drug candidates, have been filed so far in 2022, and additional provisional and PCT filings are expected to continue throughout the year. In other IP developments, an additional three of Enveric’s international patent applications protecting tryptamine derivatives have been published by the World Intellectual Property Organization, bringing the total published so far to seven. EVM-201 Drug Candidate Testing Following the initial Gen-2 ideation, patent filing, synthesis, and screening efforts, more extensive assays were performed on the advancing drug candidates. Numerous internal assays were performed under the leadership of Dr. Jill Hagel, PhD, Vice-President of Innovation and Dr. Sheetal Raithatha, PhD, Director of Research and Development. The internal results were then confirmed and validated using leading independent, third-party Contract Research Organizations (CROs), covering 122 Toxicology tests, 72 receptor-binding and potency tests, 31 pharmacokinetic tests and 24 animal-model tests to provide ample information to support candidate nomination. The synthesis and analysis of these comprehensive pharmacology and toxicology datasets, both internal and external, was led by Dr. Kevin Leach, PhD DABT, Vice President of Preclinical and Translational Research. Enveric is now moving towards lead-candidate nomination and, in preparation for IND filing, has initiated numerous IND-enabling studies on several of the leading candidates at third-party CROs. Recognizing the need for stable drugs with robust, reproducible, and cost-effective manufacturing, several Contract Development & Manufacturing Organizations (CDMOs) are currently engaged with Enveric’s Chemistry Team to determine how best to manufacture our molecules. Enveric has plans in place to produce material at commercial facilities and the CDMO personnel have worked with our chemists to validate that our processes will work at their facilities. This is another necessary step prior to filing the IND. Finally, preliminary formulation work is underway, with a particular focus on enabling improved routes of administration. “The first half of the year has indeed been busy and productive for the dedicated team here at Enveric. We are all very excited at how those efforts have begun to bear fruit. We look forward to advising our shareholders of further key developments, which we anticipate will continue to unfold during the remainder of 2022,” concluded Dr. Tucker. About Enveric Biosciences Enveric Biosciences, Inc. is a neuroscience-focused pharmaceutical company developing next-generation, psychedelic-inspired mental health medicines. Enveric's robust pipeline supports drug development from the clinic to commercialization aimed to help millions of patients in need around the world suffering from conditions that include cancer-related distress, PTSD and more.

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CELL AND GENE THERAPY

Sesen Bio and Carisma Therapeutics Announce Merger Agreement

Sesen Bio and Carisma Therapeutics | September 22, 2022

Sesen Bio, Inc. and Carisma Therapeutics Inc. a privately held, clinical stage biopharmaceutical company focused on discovering and developing innovative immunotherapies, announced that they have entered into a definitive merger agreement to combine the companies in an all-stock transaction. The combined company will focus on the advancement of Carisma’s proprietary cell therapy platform that utilizes engineered macrophages and monocytes to potentially transform the treatment of cancer and other serious disorders. Carisma is pioneering the development of chimeric antigen receptor macrophage therapies and is believed to be the only company developing CAR-M therapies with demonstrated proof of mechanism and safety data in clinical trials. The combined company is expected to operate under the name Carisma Therapeutics Inc. and trade on Nasdaq under the ticker symbol “CARM”. Carisma has also secured commitments from a syndicate of investors for a $30 million financing, including HealthCap, AbbVie, Wellington Partners, SymBiosis, Penn Medicine, TPG Biotech, MRL Ventures Fund, the therapeutics-focused corporate venture arm of Merck & Co., Agent Capital, Solasta, Livzon, Pictet Alternative Advisors and 4Bio, which is expected to close concurrently with the completion of the merger. With the cash expected from both companies at closing and the proceeds of the concurrent financing, the combined company is expected to have approximately $180 million in cash, cash equivalents and marketable securities. These cash resources are expected to be used to advance Carisma’s pipeline through multiple ongoing and planned key data readouts across several clinical trials and to fund operating expenses and capital expenditure requirements through 2024. The merger and related financing are expected to close in the next three to four months. “The proposed merger represents an exciting opportunity for shareholders of each company, and we believe it gets us one step closer to our goal of revolutionizing the field of immunotherapy. This transaction will provide us with financial strength to not only continue to develop our lead candidate CT-0508, but also allow us to accelerate the growth of our platform and pipeline within and outside of oncology and develop additional strong strategic partnerships beyond those we already have with Moderna and Novartis. Carisma is focused on delivering cutting-edge technology for patients in a way that has never been done before, and we look forward to advancing this important mission.” Steven Kelly, President and Chief Executive Officer of Carisma “This transaction represents the result of a thoughtful and careful review of strategic alternatives over the past four months, during which Carisma’s clinical programs, management team, and corporate strategy stood out amongst the 42 bids reviewed,” said Dr. Thomas Cannell, President and Chief Executive Officer of Sesen Bio. “Carisma is an exciting clinical-stage company with groundbreaking science and an impressive management team, which we believe makes them the optimal partner to provide value for our shareholders. Our mission at Sesen Bio has always been to save and improve the lives of patients with cancer, and we believe Carisma has the science and the unwavering patient focus required to make that mission a reality.” About the Proposed Merger Pre-merger Sesen Bio stockholders are expected to own approximately 41.7% and pre-merger Carisma stockholders are expected to own approximately 58.3% of the combined company, in each case before giving effect to the concurrent financing described above and the conversion of the outstanding Moderna convertible note. Under the terms of the merger agreement, stockholders of Carisma will receive newly issued shares of Sesen Bio common stock pursuant to an exchange ratio formula set forth in the merger agreement. The percentage of the combined company that Sesen Bio stockholders will own upon the closing of the merger is further subject to adjustment based on the amount of Sesen Bio’s net cash at the time of closing. Immediately prior to the closing of the proposed merger, Sesen Bio stockholders of record will be issued a contingent value right (CVR) for each outstanding share of Sesen Bio common stock held by such Sesen Bio stockholder as of such date, representing the right to receive certain cash payments from proceeds received by Sesen Bio related to the Roche Asset Purchase Agreement, if any, subject to customary deductions, including for expenses and taxes. SVB Securities is acting as exclusive financial advisor to Sesen Bio for the transaction and Hogan Lovells US LLP is serving as its legal counsel. Evercore Group LLC is serving as lead financial advisor to Carisma for the transaction and BofA Securities, Inc. is also serving as financial advisor to Carisma for the transaction. Wilmer Cutler Pickering Hale and Dorr LLP is serving as legal counsel to Carisma. BofA Securities, Inc. and Evercore Group L.L.C. are serving as co-placement agents for Carisma’s concurrent financing and Shearman & Sterling LLP is serving as the placement agents’ legal counsel. About Sesen Bio Sesen Bio, Inc. is a late-stage clinical company focused on targeted fusion protein therapeutics for the treatment of patients with cancer. Sesen Bio’s most advanced product candidate, Vicineum™, also known as VB4-845, is a locally-administered targeted fusion protein composed of an anti-epithelial cell adhesion molecule antibody fragment tethered to a truncated form of Pseudomonas exotoxin A for the treatment of non-muscle invasive bladder cancer. On July 15, 2022, Sesen Bio made the strategic decision to voluntarily pause further development of Vicineum in the US. The decision was based on a thorough reassessment of Vicineum, which included the incremental development timeline and associated costs for an additional Phase 3 clinical trial, following Sesen Bio’s discussions with the United States Food and Drug Administration. Sesen Bio has turned its primary focus to assessing potential strategic alternatives with the goal of maximizing shareholder value. Additionally, Sesen Bio intends to seek a partner for the further development of Vicineum. About Carisma Therapeutics Carisma Therapeutics Inc. is a biopharmaceutical company dedicated to developing a differentiated and proprietary cell therapy platform focused on engineered macrophages, cells that play a crucial role in both the innate and adaptive immune response. The first applications of the platform, developed in collaboration with the University of Pennsylvania*, are autologous chimeric antigen receptor (CAR)-macrophages for the treatment of solid tumors. Carisma Therapeutics is headquartered in Philadelphia, PA.

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