Rocket Pharmaceuticals, Inc. | December 02, 2022
Rocket Pharmaceuticals, Inc. a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces that it has completed the previously announced acquisition of 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. Under the terms of the merger agreement entered into on September 19, 2022, Renovacor shareholders received, for each share of Renovacor common stock, 0.1763 shares of Rocket common stock.
The acquisition provides Rocket with Renovacor’s most advanced program, REN-001, 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. Additionally, Rocket gains access to world-class scientific collaborators, a robust intellectual property portfolio and personnel with expertise in BAG3-DCM. These assets and capabilities altogether further extend Rocket’s leadership position in AAV-based cardiac gene therapy and help advance the Company’s goal of pursuing gene therapy cures for patients living with rare and devastating diseases.
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. is advancing an integrated and sustainable pipeline of investigational genetic therapies designed to 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. Rocket also has a preclinical AAV-based gene therapy program in BAG3-associated dilated cardiomyopathy.
CELL AND GENE THERAPY, MEDICAL
Be The Match BioTherapies® | January 18, 2023
On January 17, 2023, Be The Match BioTherapies, a company that provides solutions for organizations that develop and commercialize cell and gene therapies, introduced its offering of CIBMTR® clinical research organization services (CRO services), which significantly reduces the time needed to launch and execute high-impact clinical trials.
These CRO services are the outcome of a collaboration between Be The Match BioTherapies and CIBMTR, resulting in expertise and access to unmatched research, sites, donors, partnerships, scientific and operational knowledge, and other resources.
CIBMTR CRO services offer unique capabilities such as
Established relationships with over 225 clinical locations domestically
Nearly 20 years of expertise in transplant and cellular therapy trials and study management, as well as clinically-trained staff
On-staff transplant physicians provide direct contact with patients and donors for clinical and research insights.
A link to the CIBMTR outcomes database for over 575,000 patients
The new CRO services offer turn-key solutions, including protocol development and approval oversight, site selection, project management, site start-up, research sample/laboratory coordination, data management, study monitoring, patient-reported outcomes, statistical analysis, and financial administration. For certain services, such as data analysis, surveys, site selection and administration, and sample management, businesses may instead rely on the expertise of CIBMTR CRO services.
About Be The Match BioTherapies®
Be The Match BioTherapies is the sole supplier of cell and gene therapy solutions with fully configurable services to assist the cell therapy supply chain. It is committed to accelerating patient access to life-saving cell and gene treatments by delivering the best-quality cellular source material from Be The Match Registry®, the most-varied registry of prospective blood stem cell donors in the world, with over 7 million potential blood stem cell donors. Through global collaborations with apheresis, bone marrow collection, and transplant centers, the firm creates, onboards, trains, and maintains extensive collection networks to advance cell treatments.
INDUSTRIAL IMPACT, MEDICAL
Bioasis Technologies Inc. | December 14, 2022
BIOASIS TECHNOLOGIES INC. a multi-asset rare and orphan disease biopharmaceutical company developing clinical stage programs based on epidermal growth factors and a differentiated, proprietary xB3™ platform for delivering therapeutics across the blood-brain barrier and the treatment of central nervous system disorders in areas of high unmet medical need, today announced that it has entered into a definitive agreement dated December 13, 2022 with Midatech Pharma plc a company focused on the research and development of medicines that would benefit from improved bio-delivery or bio-distributions using proprietary drug delivery technologies, pursuant to which Midatech will acquire 100% of the issued and outstanding common shares in the capital of Bioasis from Bioasis’ shareholders in exchange for ordinary shares of Midatech in the form of American depositary shares.
Midatech has also entered into securities purchase agreement with an institutional investor for (i) a registered direct offering of ADSs for gross proceeds of approximately US$400,000 that is expected to be completed on or about December 15, 2022 and a private placement equity financing for gross proceeds of approximately US$9.6M that will be completed concurrently with Midatech’s acquisition of Bioasis.
The combination of Bioasis and Midatech will create a multi-asset rare and orphan disease company that will be renamed Biodexa Pharmaceuticals PLC (“Biodexa”). Bioasis and its shareholders are expected to benefit from Biodexa’s capital markets profile in the United States as a NASDAQ-listed company, as well as increased trading liquidity and broadened appeal to global index and generalist investors relative to Bioasis’ status as a TSXV-listed company. Biodexa is expected to have strengthened balance sheet with pro forma cash of approximately C$17.9 million as at June 30, 2022. Biodexa is also expected to benefit from the collective scientific, technical, and operational expertise of both Midatech and Bioasis as well as cost synergies as a result of the elimination of duplicative salaries, administrative and regulatory costs and other public company expenses. Through their ownership of ADSs, current Bioasis securityholders will maintain exposure to the value that is expected to be unlocked as Bioasis and Midatech’s pipeline programs progress through clinical development and the drug delivery technologies secure additional partnerships.
Bioasis’ Executive Chair, Deborah Rathjen PhD, commented “As shareholders are aware, in December 2021 we indicated that Bioasis would seek value accretive strategic alternatives and pursue opportunities that have the potential to unlock value and liquidity for shareholders. With this transaction and through the formation of Biodexa, it is anticipated that Bioasis’ pipeline will achieve value accretive milestones whilst continuing to monetize the xB3 platform through additional partnerships. Once the transaction is closed, Bioasis shareholders will have access to the benefits of a NASDAQ listing through their ownership of Biodexa. We are very excited by the prospects for this transaction given the evident synergies between Bioasis and Midatech”.
Mr Stephen Stamp, Midatech CEO and incoming Biodexa CEO added “By combining the two groups to create Biodexa Pharmaceuticals, we have the opportunity to reposition the enlarged group as an emerging biotech company focused on the development of therapeutics for rare diseases, supported by Midatech and Bioasis’s enabling drug delivery platforms. We continue to believe there is substantial value to be unlocked from Midatech’s MTX110, particularly in glioblastoma, and to leverage our Q-Sphera technology. In combination with Bioasis’s promising development pipeline we have the opportunity to create a much stronger group.”
Bioasis Technologies Inc. is a multi-asset rare and orphan disease biopharmaceutical company developing clinical stage programs based on epidermal growth factors and the xB3™ platform, a proprietary technology for the delivery of therapeutics across the blood brain barrier and the treatment of CNS disorders in areas of high unmet medical need. The delivery of therapeutics across the blood-brain barrier represents the final frontier in treating neurological disorders. The in-house development programs at Bioasis are designed to develop symptomatic and disease-modifying treatments for brain-related diseases and disorders.
Midatech is a drug delivery technology company focused on improving the bio-delivery and biodistribution of medicines. Midatech combines approved and development medications with its proprietary and innovative drug delivery technologies to provide compelling products that have the potential to powerfully impact the lives of patients. Midatech has developed three in-house technology platforms, each with its own unique mechanism to improve delivery of medications to sites of disease. All of Midatech’s technologies have successfully entered human use in the clinic, providing important validation of the potential for each platform: (i) Q-Sphera™ platform: a disruptive micro-technology used to prolong and control the release of injectable therapeutics over an extended period of time (from weeks to months), (ii) MidaSolve™ platform: an innovative nanotechnology used to solubilize inherently insoluble drugs so that they can be administered in liquid form directly and locally into tumors and (iii) MidaCore™ platform: a gold nanotechnology used for targeting medications to sites of disease. The platform nature of the technologies offers the potential to develop multiple drug assets rather than being reliant on a limited number of programs. Midatech’s technologies are supported by 36 patent families including 120 granted patents and an additional 70 patent applications. Midatech's headquarters and R&D facility is in Cardiff, UK.