On December 16, 2021 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) and Skyline Therapeutics (formerly Geneception), a gene and cell therapy company focused on developing novel treatments for unmet medical needs, reported a multi-year global strategic collaboration for the discovery, development and commercialization of Adeno-Associated Virus (AAV) gene therapies to treat genetic cardiovascular diseases (Press release, BioMarin, DEC 16, 2021, View Source [SID1234597351]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The partnership will leverage Skyline Therapeutics’ integrated AAV gene therapy platform based on its proprietary vector engineering and design technology and manufacturing capability to develop innovative gene therapies with a focus on genetic dilated cardiomyopathies (DCM), a group of progressively advancing, devastating diseases with no targeted treatment options.

Under the agreement, BioMarin and Skyline Therapeutics will collaborate on discovery and research through to an Investigational New Drug Application (IND). BioMarin brings experience in gene therapy development, cardiovascular biology and insights into genetic basis of diseases, and Skyline contributes its expertise in developing gene therapy products including vector engineering and design technology and manufacturing capabilities to this collaboration. Each company will advance the programs through clinical development in their pre-defined territories.

In support of its R&D efforts for the collaborative projects, Skyline Therapeutics will receive an undisclosed payment associated with signing, comprising an upfront payment and an equity investment from BioMarin, and is eligible to receive pre-specified payments for R&D, regulatory and commercial milestones.

BioMarin will have the rights to commercialize therapeutic products resulting from the collaboration in its territories, including the United States, Europe, and Latin America, and Skyline Therapeutics will be responsible for commercialization in the Asia-Pacific region. In addition, Skyline Therapeutics will be eligible to receive royalty payments on future sales from BioMarin in its territories.

"We are thrilled to announce what we anticipate will be a fruitful collaboration at the interface between Skyline’s innovative approach to AAV vector engineering and design and our team’s proven expertise in creating and developing gene therapies," said Kevin Eggan, Group Vice President, Head of Research and Early Development, from BioMarin.

"We are excited to partner with Skyline Therapeutics to tackle these genetic forms of dilated cardiomyopathy. This collaboration strengthens our leadership in cardiac gene therapy and extends our R&D collaboration to Asia, where a large number of patients suffer from these devastating diseases," said Brinda Balakrishnan, Group Vice President, Corporate and Business Development at BioMarin. "We look forward to fostering this collaboration and bringing transformative medicines to patients worldwide."

"Dilated cardiomyopathy is a serious cardiac disorder in which structural or functional abnormalities of the heart muscle can lead to complications such as arrhythmia and heart failure, resulting in substantial morbidity and mortality. Mutations in many genes are associated with the development of DCM, among other etiologies for the disease," said Jay Hou, Chief Scientific Officer at Skyline Therapeutics. "Together with BioMarin’s team we have identified a number of critical genes associated with DCM. We are delighted to work closely with BioMarin and apply our AAV vector technology to interrogate these new targets and develop novel treatments for DCM patients."

"The collaboration with BioMarin leverages both companies’ capabilities in the development of gene therapies. With the BioMarin team, we share the goal of working in concert to develop therapies for genetic cardiovascular disease that address high unmet medical needs," said Amber Cai, CEO of Skyline Therapeutics. "Together, we will utilize gene therapy to tackle cardiovascular diseases with a disease modifying trailblazing approach that could change the treatment paradigm in these conditions."

About Dilated Cardiomyopathy (DCM)

DCM is a common cause of heart failure and end-stage DCM, which often leads to heart transplantation. Despite improvements in pharmacotherapy and care, the five-year survival rate of DCM is only about 50%. Hundreds of thousands of patients suffer from the genetic forms of DCM in U.S., EU, China, and Japan. More than 50 genes associated with DCM have been identified, accounting for 40-50% of familial DCM cases. Many of these genes encode proteins with important known functions in cardiomyocytes related to cytoskeletal, sarcomere and nuclear envelope biology. Our aim is to correct the pathways altered by these genetic contributors to DCM through AAV based gene therapy, in each case addressing the root cause of the disease.

On December 16, 2021 Sirona Biochem Corp. (TSX-V: SBM) (FSE: ZSB) (OTC: SRBCF) ("Sirona") reported that Linda Pullan of Pullan Consulting will be providing strategic direction with the negotiations and licensing of TFC-1067 (Press release, Sirona Biochem, DEC 16, 2021, View Source [SID1234597350]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Sirona is in the negotiation phase with a global top 10 pharmaceutical company for the rights to TFC-1067. Simultaneously, the company is completing the legal due diligence regarding contracts and patents, which is a straightforward task. The pharma company is advancing on the project as expected and has been transparent with next steps and timelines.

"We are thrilled to have Pullan Consulting on board to assist us with this task. Linda has successfully closed 65 pharma/biotech deals and has advised on many more. She is a renowned professional in the field," said Howard Verrico, CEO of Sirona Biochem. "We are dealing with a pharmaceutical company that is very experienced in licensing negotiations. The highest level of expertise is needed to obtain the best outcome. We see this level in Pullan Consulting and look forward to working with their team to close an optimal deal."

On December 16, 2021 VolitionRx Limited (NYSE AMERICAN: VNRX) ("Volition"), a multi-national epigenetics company developing simple, easy to use, cost effective blood tests to help diagnose and monitor a range of cancers and other life-altering diseases in both humans and animals, reported that it has entered into its first contract for the Nu.Q Vet Cancer Screening Test, a supply and licensing agreement with SAGE Healthcare (Press release, VolitionRX, DEC 16, 2021, View Source [SID1234597349]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"With the global veterinary market being so fragmented, we determined the best way to ensure our test is accessible worldwide is to have multiple agreements with both large multi-nationals and regional players," commented Dr. Tom Butera, Chief Executive Officer of Volition Veterinary Diagnostics Development, LLC. "I am delighted to announce this agreement for centralized labs in Asia, a large and rapidly expanding pet care market. I expect this will be the first of many agreements, including with the big multi-nationals, so that we can cover the whole world through centralized labs and also with point of care."

View Source

An interview with Dr. Tom Butera, Chief Executive Officer of Volition Veterinary Diagnostics Development LLC.

Dr. Jasmine Kway, Chief Executive Officer of Singapore Volition commented "With rapid urbanization and rising disposable income, pets have become an important part of Asian families. Over half of the Asian population have a companion animal , with almost a third of households owning a dog. We believe that the opportunity for the Nu.Q Vet Cancer Screening Test is huge. SAGE Healthcare is one of the strongest and most respected distributors of leading veterinary brands in Singapore. We are pleased to appoint SAGE Healthcare to offer the Nu.Q Vet Cancer Screening Test widely in Singapore and later avail the Nu.Q Vet Cancer Screening Test in other countries in Asia."

"We are delighted to be appointed as a licensee and distributor of the Nu.Q Vet Cancer Screening Test in Singapore and other countries in Asia. Adding the Nu.Q Vet Cancer Screening Test to the routine wellness check-up for older dogs and at-risk breeds could help detect cancer early, when treatment is more effective and affordable. We believe that this is a clear unmet need in the veterinary space," commented Ms. Irene Kum General Manager for SAGE Healthcare Private Limited. "We are excited to launch the test in Singapore in the first quarter of 2022 and with planned subsequent launches in other Asian markets thereafter."

On December 16, 2021 Argobio and GeneCode reported their collaboration to develop small molecule Glial cell line – Derived Neurotrophic Factor (GDNF) mimetics for the treatment of Parkinson’s Disease and other indications (Press release, Argobio, DEC 16, 2021, View Source [SID1234597348]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

In the venture studio model developed by Argobio, breakthrough technologies are embraced by a team of scientific experts with an entrepreneurial and industrial experience. Over the course of incubation, Argobio will fund the project development up to 3 ME and incorporate the start-up. After incubation, further venture funding will be provided by specialized VC investors.

"The launch of our first collaboration is a great achievement for Argobio. Argobio intends to incubate until series A at least five projects in the fields of rare diseases, neurosciences, and oncology over the next three years. With a committed capital of €50 million, and a team of pharma industry experts, Argobio expects to deliver strong projects, well-prepared for Series A funding," Thierry Laugel Argobio Chairman said.

GeneCode, a discovery-stage biotech company developing small molecule GDNF mimetics, in collaboration with the laboratory of Mart Saarma, Professor and Research Director of the Institute of Biotechnology, HI-LIFE, University of Helsinki, Finland, has identified and patented a series of small molecules activating the GDNF signalling receptor RET tyrosine kinase. Professor Saarma´s team has discovered GDNF receptors, clarified the GDNF-receptor structure and investigated the therapeutic potential of GDNF family members.

Professor Mart Saarma commented, "Argobio’s investment and participation in the development of effective GDNF mimetics is extremely encouraging news. GDNF mimetics can overcome the limitations of the GDNF protein and can open up a completely new page in the treatment of Parkinson’s disease."

GeneCode CEO Paavo Pilv added, "Our combined expertise will catapult this technology beyond what we would have been able to achieve alone. Considering the pivotal role of GDNF family ligands and RET signalling pathways for other groups of cells, applications of compounds developed by GeneCode are not limited only to Parkinson’s Disease but can be applied to treat other medical conditions."

"We have ambitious goals for the next two years," Argobio Entrepreneur in Residence Yves Ribeill, PhD said. "The whole is greater than the sum of the parts with the combined, diverse expertise of GeneCode, Argobio and Professor Saarma. I am confident that we will be able to demonstrate that the unique business model of funded partnerships initiated by Argobio in Europe will give new therapeutics a boost to succeed."

On December 16, 2021 Protocol First, a software solutions provider focused on accelerating clinical research,reported that it has joined Flatiron HealthⓇ as a wholly owned subsidiary, the two companies (Press release, Flatiron Health, DEC 16, 2021, https://www.businesswire.com/news/home/20211215006052/en/Protocol-First-Joins-Flatiron-Health [SID1234597346]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"This is a win-win for both companies," said Carolyn Starrett, Flatiron CEO. "Flatiron and Protocol First share a common vision in which technology and software increasingly bridge the gap between real-world care and clinical research."

Protocol First’s technology enhances research efficiency by breaking down silos between sites, study sponsors and contract research organizations (CROs). Effective Dec. 14, Protocol First’s employees and operations joined Flatiron’s Clinical Research business unit, which builds on Flatiron’s established leadership in real-world evidence with new technologies that better integrate research into everyday clinical care.

"Flatiron has deep expertise in unstructured-data processing, experience building software that integrates seamlessly into site workflows, and an engaged network of community and academic research sites," said Alex Deyle, General Manager, Clinical Research at Flatiron. "Protocol First’s technology expands and accelerates our ability to drive much-needed innovation in clinical research."

Protocol First aims to reduce the operational complexity of data collection and validation in clinical research by leveraging proven technologies and standards. The team, led by CEO Hugh Levaux and CTO Amit Shah, has developed several industry-defining software solutions that have been adopted by many leading CROs and biopharma companies. The technology is designed to handle the unique complexities of research in oncology as well as other disease areas. Plans are being developed to integrate the direct EHR-to-EDC connector application Clinical Pipe – already in use with major academic medical centers and health systems – with Flatiron’s OncoEMRⓇ, which is used by hundreds of community cancer centers across the U.S.

"We are very excited about this combination, which will take our technology to the next level, scaling and broadening its availability across the life sciences industry," said Levaux, who joins Flatiron as Vice President, Clinical Research. "The transition will be seamless for our customers, and the future belongs to the patients we will all serve with even better solutions."