CELL AND GENE THERAPY
Tiziana Life Sciences Ltd. | September 16, 2022
Tiziana Life Sciences Ltd. a biotechnology company developing breakthrough immunomodulation therapies via novel routes of drug delivery, today announces that a Lawrence & Isabel Barnett Drug Development Program Grant will be awarded to the Ann Romney Center for Neurologic Diseases at the Brigham and Women’s Hospital by the ALS Association for the study of an intranasal anti-CD3 monoclonal antibody in an animal model of Amyotrophic Lateral Sclerosis (ALS).
Howard L. Weiner, M.D., Co-Director of the Ann Romney Center for Neurologic Diseases at BWH and Chairman of Tiziana's Scientific Advisory Board, stated, “This prestigious research grant will be used to further study the role of intranasal anti-CD3 mAb in dampening the microglial activation which amplifies ALS disease progression. This research follows our recently presented positive findings on intranasal anti-CD3 mAb in Alzheimer’s Disease preclinical models of neuroinflammation. Additionally, we are currently studying foralumab, the first entirely human anti-CD3 mAb, in patients with secondary progressive multiple sclerosis.”
Gabriele Cerrone, Executive Chairman and interim Chief Executive Officer of Tiziana, remarked, “Intranasal foralumab has demonstrated potential across multiple Central Nervous System (CNS) indications. We are encouraged by the preclinical research using an intranasal anti-CD3 mAb in the neuroinflammatory related diseases of ALS and Alzheimer’s, as well as the impressive clinical benefits we have already shown for foralumab in patients with multiple sclerosis. While our initial focus is on our ongoing MS program which will continue to generate clinical read-outs, we are excited by foralumab’s potential to help highly debilitated ALS patients with limited therapeutic options and high unmet need.”
“We have now seen the potential of intranasal foralumab to dampen microglial activation in three major neuroinflammatory-related diseases, which creates significant optionality for exploring its benefits in some of the most important and burdensome medical conditions of our time.”
Matthew W. Davis, M.D., RPh, Chief Medical Officer of Tiziana
About the Barnett Drug Development Grant
The ALS Association’s Barnett Drug Development grant program supports preclinical drug discovery and development of new or repurposed treatments for ALS. There is an urgent need for new and improved therapies for ALS, as there is still no cure. The Lawrence and Isabel Barnett Drug Development Program is open to industry and academic investigators proposing to develop novel or repositioning approaches for ALS. The Association seeks applications for the preclinical assessment of therapeutics for ALS that have a high probability of reaching the clinic within three years.
Foralumab the only entirely human anti-CD3 mAb, has shown reduced release of cytokines after IV administration in healthy volunteers and in patients with Crohn's disease. In a humanized mouse model it was shown that while targeting the T-cell receptor, orally administered foralumab modulates immune responses of the T-cells and enhances regulatory T-cells thereby providing therapeutic benefit in treating inflammatory and autoimmune diseases without the occurrence of potential adverse events usually associated with parenteral mAb therapy. Once a day treatment for 10 consecutive days with intranasal foralumab was both well tolerated and produced clinical responses in COVID-19 patients. Based on these studies, the intranasal and oral administration of Foralumab offers the potential to become a well-tolerated immunotherapy for autoimmune and inflammatory diseases by the induction of Tregs.
About Tiziana Life Sciences
Tiziana Life Sciences is a clinical-stage biopharmaceutical company developing breakthrough therapies using transformational drug delivery technologies to enable alternative routes of immunotherapy. Tiziana’s innovative nasal, oral and inhalation approaches in development have the potential to provide an improvement in efficacy as well as safety and tolerability compared to intravenous (IV) delivery. Tiziana’s two lead candidates, intranasal foralumab, the only fully human anti-CD3 mAb, and milciclib, a pan-CDK inhibitor, have both demonstrated a favorable safety profile and clinical response in patients in studies to date. Tiziana’s technology for alternative routes of immunotherapy has been patented with several applications pending and is expected to allow for broad pipeline applications.
CELL AND GENE THERAPY
Rocket Pharmaceuticals, Inc. | September 21, 2022
Rocket Pharmaceuticals, Inc. a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, and Renovacor, Inc. a biotechnology company focused on delivering innovative precision therapies to improve the lives of patients and families battling genetically-driven cardiovascular and mechanistically-related diseases, today announced a definitive agreement under which Rocket will acquire Renovacor in an all-stock transaction for an implied value of approximately $2.60 per share, based on the volume weighted average trading price of Rocket shares of $15.51 for the 30 trading days through and including Monday, September 19, 2022. The boards of directors of both companies have unanimously approved the transaction, which is currently expected to close by the first quarter of 2023.
“The acquisition of Renovacor aligns with our strategy to expand our leadership position in AAV-based gene therapy for cardiac disease and gives us a perfect opportunity to continue on our mission to transform the lives of heart failure patients through the power of gene therapy. Building on our success in Danon Disease to date, I am particularly excited to expand our cardiology focus and capabilities and address a clear unmet medical need in BAG3-associated dilated cardiomyopathy. By combining Renovacor’s compelling preclinical work with our joint clinical, regulatory and CMC expertise, we believe we will be well-positioned to bring the highest impact gene therapy with the best chance for success to these patients in the most productive and efficient manner possible.”
Gaurav Shah, M.D., Chief Executive Officer of Rocket
Dr. Shah continued, “Given the positive pediatric safety data previously announced from our Phase 1 RP-A501 Danon Disease program, and the upcoming pediatric efficacy data and longer-term adult cohort data we anticipate presenting at the Heart Failure Society of America (HFSA) Scientific Meeting at the end of this month, this strategic acquisition gives us what we believe is the broadest platform in the field to address these devastating rare cardiac diseases. Furthermore, the acquisition will bring to Rocket key personnel, namely a team of leading cardiology drug development experts, critical capabilities, and valuable IP to support continued development of the BAG3 as well as other potential cardiac programs, including a gene therapy research collaboration for arrhythmogenic cardiomyopathy.”
Renovacor’s most advanced program, REN-001, is an AAV-based gene therapy targeting BAG3-associated dilated cardiomyopathy (DCM), a severe form of heart failure. BAG3-DCM represents a significant unmet medical need in a patient population with rapidly progressive cardiac dysfunction in whom no treatments targeting the underlying mechanism of disease exist. Renovacor has deep technical expertise in the development of precision therapies that address genetically driven cardiac diseases. Further, Renovacor is supported by world-class scientific collaborators, a robust intellectual property portfolio and personnel with expertise in BAG3-DCM. These assets and capabilities, all together, represent tremendous value and will enhance Rocket’s leading position in cardiac AAV-based gene therapy.
“Renovacor has made tremendous progress in advancing targeted gene therapies to address the high unmet medical needs of patients living with genetically driven forms of heart disease,” said Magdalene Cook, MD, Chief Executive Officer of Renovacor. “Our experienced team is excited to join Rocket in a shared vision of broadening patient access to precision medicines for cardiovascular disease and addressing common barriers jointly. We look forward to combining the considerable resources and expertise of Renovacor and Rocket in creating a category leader in the precision cardiology field. As a result of this combination, we will be suspending current guidance regarding preclinical and clinical timelines for our programs as we evaluate these items with the Rocket team.”
It is currently anticipated that the transaction will close by the first quarter of 2023, subject to approval by Renovacor and Rocket shareholders, receipt of any required customary regulatory approvals and the satisfaction of other customary closing conditions. RTW Investments, LP, a significant shareholder of both Rocket and Renovacor, has entered into a voting agreement with Renovacor, pursuant to which they have agreed, among other things, and subject to the terms and conditions of the agreement, to vote in favor of the Renovacor acquisition as a Rocket stockholder.
SVB Securities is serving as exclusive financial advisor and Goodwin Procter LLP is serving as legal counsel to Rocket. Wells Fargo Securities is serving as exclusive financial advisor and Troutman Pepper Hamilton Sanders LLP is serving as legal counsel to Renovacor.
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The Company’s platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rocket’s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition.
Renovacor is a biotechnology company focused on delivering innovative precision therapies to improve the lives of patients and families battling genetically-driven cardiovascular and mechanistically-related diseases. The company’s lead program in BAG3-associated dilated cardiomyopathy (DCM) uses gene transfer technology to address the monogenic cause of this severe form of heart failure. Renovacor’s vision is to bring life-changing therapies to patients living with serious genetic cardiovascular and related diseases, by developing medicines that target the underlying cause of disease and provide a transformative benefit and significant improvement to quality of life.
Alloy Therapeutics, Inc. | October 04, 2022
Alloy Therapeutics, a biotechnology ecosystem company, has closed $42 million in Series D financing led by its existing investors 8VC and Mubadala Capital and joined by return investors Thiel Capital, Presight Capital, Founders Fund, and other unnamed family offices and sovereign wealth funds. Alloy will use proceeds from the Series D financing to further support the drug discovery community with new pre-competitive drug discovery technologies in new biologic modalities and new partnership models designed to fuel innovation.
Since Alloy’s Series C fundraise in March 2021, Alloy has expanded from its foundational antibody discovery technologies and discovery services into T-cell receptors with the launch of its Keyway TCR Discovery division, and genetic medicines through its collaboration with Dr. Sudhir Agrawal, the inventor of gapmer technology. Alloy has further expanded with three new research sites, in Basel, CH; Athens, GA; and San Francisco, CA.
“After three years of hard work and planning, we were proud to formally launch Keyway TCR Discovery in January 2022. We envision a future in 20 years where engineered TCRs and TCR mimics are as successful a modality as monoclonal antibodies are today. Keyway believes this is possible by providing unrestricted access to enabling technologies and discovery services without pharma having to spend billions of dollars to acquire companies for proprietary technologies and people.”
Dongxing Zha, CEO of Keyway and Alloy’s CTO for TCR Modalities
In response to growing engagement from partners, Alloy will further leverage its Series D funds to continue to invest in expanded offerings across all six of its modalities. These will support the drug discovery community in the pursuit of multi-modal therapeutics while reducing or eliminating stacked downstream payments.
Through its affiliated venture studio, 82VS, Alloy supports scientist-entrepreneurs with seamless access to its cutting-edge technologies and discovery services. 82VS companies are often the first customers of Alloy’s innovations, having full access to the Alloy suite of technologies and discovery services to advance the best drug candidates and invent new applications that can benefit the broader Alloy ecosystem and, ultimately, patients and their caregivers.
To best serve the vast discovery needs of pharmaceutical companies, Alloy is pioneering an Innovation Subscription offering that gives its collaborators access to all current and future Alloy technology platforms for one flat, annual fee with no milestones or royalties. Partners benefit from Alloy as an ever-growing source of pre-competitive, foundational discovery technology. Alloy also supports complementary CROs, CDMOs and other discovery and development service providers as a critical part of the collaborative ecosystem to empower pharma in its mission to cure disease. In this subscription model, Alloy provides a new home for promising pre-competitive technologies and academic breakthroughs that have traditionally been underfunded or unavailable to all.
“Our Innovation Subscription model is available now as an exciting next step in furthering our collaborative mission and unlocking the potential for sharing foundational, pre-competitive drug discovery technologies with the global drug discovery community,” said Alloy Therapeutics Chief Executive Officer and Founder Errik Anderson. “Including this financing, Alloy will have invested hundreds of millions of dollars in innovation that the largest pharma companies can access through a flat, annual subscription with no additional milestones or royalties. Emerging biotech and academics can access our tech through collaboration and more traditional and creative structures. Combined with the reinvestment of 100% of our revenue, including our subscription revenue, back into innovation and access to innovation, Alloy is a new for-profit business model that plays for the collective long game in a non-zero-sum way. The value of a subscription goes up every year, while the fees stay flat or even will decrease with scale. We price this so a single avoided royalty will pay for the entire platform access over a 20-plus-year period. We are grateful for our longstanding investors for supporting this vision of arming the drug discovery industry with the tools they need to discover and develop the best medicines for patients more quickly.”
Alloy launched in 2017 to democratize foundational, pre-competitive technologies and capabilities for the discovery of therapeutic biologics and has since grown with over 130 partners across academia, biotech, and large biopharma. Its first platform was the ATX-Gx™, a suite of transgenic mouse strains for human antibody discovery that was originally in-licensed from a major pharma company and made broadly available to the drug discovery ecosystem by Alloy, with open access to newly introduced strains. Through its DeepImmune™ Antibody Discovery services, Alloy helps partners find the best therapeutic antibody candidates against targets of interest, by deploying a bespoke blend of its proprietary in vivo, in vitro, and in silico technologies and workflows. In 2022, Alloy launched Keyway™ TCR Discovery as its second fully integrated technology and services offering for the discovery of therapeutic engineered TCRs and TCR mimics.
“Alloy has successfully expanded its flagship discovery offering in antibodies to multiple promising modalities to better serve the global drug discovery community,” said Alaa Halawa, Head of Mubadala Capital’s US Ventures business, which first backed Alloy as an investor in its Series C financing. “We are proud to partner with the company as it executes on its promise of enabling scientists through the development of transformative drug discovery technologies. We look forward to seeing how the partnership with large pharma partners and other venture firms will leverage Alloy’s platform to enable their therapeutic discovery teams and emerging startups to advance the best medicines.”
About Alloy Therapeutics
aAlloy Therapeutics is a biotechnology ecosystem company empowering the global scientific community to make better medicines together. Through a community of partners across academia, biotech, and the largest biopharma, Alloy democratizes access to pre-competitive tools, technologies, services, and company creation capabilities that are foundational for discovering and developing therapeutic biologics across six modalities, including antibodies, TCRs, genetic medicines, peptides, cell therapies, and drug delivery. Partners may access all current and future technologies for a flat annual fee through Alloy’s Innovation Subscriptions offering. As a reflection of Alloy’s relentless commitment to the scientific community, Alloy reinvests 100% of its revenue in innovation and access to innovation.
About Alloy’s Antibody Discovery Offering
Alloy’s first foundational platform, the ATX-Gx™ mice, is a suite of proprietary transgenic mice strains for human therapeutic antibody discovery, which can be accessed in your lab or through DeepImmune™ Antibody Discovery. This service integrates Alloy’s full complement of proprietary in vivo, in vitro, and in silico discovery and optimization technologies into one comprehensive service offering for fully human monoclonal and bispecific antibody discovery. The technologies and protocols can be replicated in a partner’s organization through a platform transfer and as part of an Alloy Innovation Subscription. Alloy is a leader in bispecific antibody discovery and engineering services, utilizing its proprietary ATX-CLC common light chain platform integrating novel transgenic mice and phage display.
About Keyway™ TCR Discovery
In 2022, Alloy launched Keyway™ TCR Discovery, Alloy's second fully integrated platform and service offering. Led by a team of TCR therapeutic pioneers and experts and leveraging the full resources of the Alloy ecosystem, Keyway provides comprehensive proprietary technology and capabilities for end-to-end discovery of TCR mimics and engineered TCRs, which includes production and quality control of MHC/peptide complexes, biophysical characterization, specificity testing, reformatting, and engineering, affinity maturation, and in vivo and in vitro preclinical testing. Partners may access Keyway's technology and capabilities through a standard or bespoke one-stop-shop discovery campaign, platform transfer, or Innovation Subscription.