PRNewswire | July 10, 2023
Critical Path Institute (C-Path) and the Center for Health + Technology (CHeT) at University of Rochester today announced the release of two seminal publications about digital health technologies for Parkinson's. These technologies, such as smartphones and wearable sensors, offer an opportunity for objective, frequent and remote assessment of people with Parkinson's.
With 10 million people living with Parkinson's worldwide, collaboration and data sharing are crucial for driving innovation in drug development for the fastest-growing degenerative neurological condition.
Research leaders at CHeT have joined forces with organizations and regulators through C-Path's Critical Path for Parkinson's (CPP) Consortium, a precompetitive public-private partnership. CPP aims to accelerate the regulatory endorsement of digital tools in Parkinson's clinical trials through its Digital Drug Development Tools (3DT) initiative.
In June 2023, the Journal of Parkinson's Disease published two open access manuscripts, representing companion papers from this partnership that detail steps in conducting interviews of people with Parkinson's who participated in a clinical research study using smartphones. The manuscripts report on relevant symptoms in early Parkinson's and assess the relevance of digital measures from a smartphone application and smartwatch from the patient perspective.
The study was led by Jamie Adams, M.D., Associate Professor of Neurology and Associate Director of CheT at the University of Rochester and Jennifer Mammen, Ph.D., Assistant Professor at the University of Rhode Island, with support from CPP.
"This project pioneers a new path to efficiently advance tools aligned with the patient voice by engaging with regulatory agencies early and often," said Diane Stephenson, Ph.D., CPP Executive Director. "C-Path remains committed to learning what matters most to people living with Parkinson's."
"These manuscripts describe a novel methodology that will help inform the use of digital health technologies in clinical trials for Parkinson's disease and support more objective and patient-centered measures. The goal is to enable efficient, data-driven, and patient-centric utilization of digital health technologies to speed therapeutic development," said Adams. "Sharing the results with the study participants and witnessing how this information helped them in new ways has been most rewarding."
John Crawford, a CPP advisor, Parkinson's advocate, and manuscript coauthor states, "Digital health technologies have the potential to significantly impact Parkinson's management and have already revolutionized clinical trial conduct. Their advancement expedites trials, enhances intelligence, reduces costs, and ultimately accelerates the delivery of new therapies and technologies to patients."
The new publications coincide with CPP's participation in the World Parkinson's Congress taking place in Barcelona, Spain July 4-7, 2023. CPP will present two posters, Recommendations for Parkinson's Clinical Trials using Digital Health Technologies and Worldwide Collaborative Framework for Optimizing New Parkinson's Treatment Trials with Patient Centric Outcome Measures, at the conference coauthored by people living with Parkinson's highlighting the importance of worldwide collaborations.
About Critical Path Institute
Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path's mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and hundreds of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona and C-Path Europe is headquartered in Amsterdam, Netherlands with additional staff in multiple other locations.
C-Path FDA Acknowledgement
The Center for Health + Technology (CHeT) is an academic research organization within the University of Rochester Medical Center. We are comprised of six units, with expertise in clinical trials coordination, clinical materials services, patient and caregiver-reported outcome measures, tech research and innovation, data modeling and predictive analysis, and health policy. Our skilled team of consultants are readily available to provide guidance to academic institutions, pharmaceutical companies, technology firms, not-for-profit foundations, advocacy groups, and the federal government.
Globenewswire | August 23, 2023
Vincerx Pharma, Inc. a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, today reported that it has received clearance for its Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA) to initiate a Phase 1 clinical trial for VIP943. Vincerx expects to enroll the first patient in the study early in the fourth quarter of 2023.
"The IND clearance by the FDA is a significant step forward for the VIP943 development program, our first candidate from our antibody-drug conjugate (ADC) platform to enter clinical trials," said Ahmed Hamdy, M.D., Chief Executive Officer of Vincerx. "Our next-generation platform is designed to improve efficacy and address safety challenges associated with many ADCs. It combines an internalizing antibody binding to CD123, a novel KSPi payload, a selective and stable linker, and exclusive CellTrapper™ technology. We believe VIP943 has the potential to become a much needed treatment option for patients with acute myeloid leukemia (AML). We're eager to start the clinical program and unlock the potential therapeutic benefits of this compound."
Dr. Hamdy concluded by saying, “2023 is shaping up to be a very positive year for Vincerx. Within the last eight months, we successfully filed two INDs (VIP236 and VIP943) and swiftly garnered clearance from the FDA within 30 days. We believe these accomplishments highlight the strength of our development, drug manufacturing, and regulatory expertise. Looking ahead, in addition to beginning our Phase 1 trial for VIP943, we also expect to provide preliminary results for VIP236 by late 2023 or early 2024. Collectively, these milestones support the potential for our strategy to develop small molecule and antibody-based conjugate therapies to improve the care of patients with cancer.”
VIP943 is a novel ADC developed using Vincerx’s next-generation ADC technology designed to enhance intracellular accumulation of the KSPi payload (kinesin spindle protein inhibitor), potentially leading to higher efficacy, while limiting unwanted side effects. VIP943 binds to CD123, a validated target in myeloid malignancies. Once inside of the cell, it releases its KSPi payload. Vincerx’s CellTrapper™ technology ensures that the KSPi payload accumulates in the target cells, limiting uptake into non-target, non-dividing cells.
VIP943 has demonstrated increased survival and reduced tumor burden in AML (acute myelogenous leukemia) cell lines and patient-derived tumor models. In addition, Vincerx presented preclinical data on VIP943 at ASH 2022 demonstrating superiority against Mylotarg™, an approved ADC, with significantly improved safety in monkeys and better efficacy in a mouse model of acute leukemia. Preclinical data suggest that VIP943 does not induce significant cytokine release and demonstrated favorable safety in monkeys with an optimal drug, metabolism and pharmacokinetics profile.
The Phase 1 trial of VIP943 is intended to assess safety, pharmacokinetics/phamacodynamics, and preliminary efficacy of VIP943 in patients with advanced AML and myelodysplastic syndrome (MDS).
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 the next-generation antibody conjugate, VIP943, on track to begin a Phase 1 study in early Q4 2023; small molecule drug-conjugate, VIP236, in Phase 1; preclinical antibody drug conjugate, VIP924; CDK9 inhibitor, enitociclib, currently in an NIH-sponsored Phase 1; and its next-generation modular bioconjugation platform.
PHARMACY MARKET, PHARMA TECH
Globenewswire | August 22, 2023
Timber Pharmaceuticals, Inc. announced that it has entered into a definitive agreement to be acquired by LEO US Holding, Inc. a wholly-owned subsidiary of LEO Pharma A/S, in a total transaction value of up to $36 million with (i) an initial upfront consideration of $14 million and (ii) up to an additional $22.0 million in contingent value rights (CVRs) payable upon achievement of certain milestones described below. All of the issued and outstanding shares of capital stock and other equity interests of Timber will be converted into the right to receive the initial upfront consideration, less the payments for certain outstanding warrants that contain a Black Scholes cash payout value. For example, based on a current estimate of the Black Scholes value of such warrants of approximately $5.1 million, subject to change based on the assumptions detailed below, Timber expects the initial amount per share to be paid to Timber stockholders to be approximately $2.62 based on approximately 3.4 million shares of Timber common stock and restricted stock issued and outstanding as of August 20, 2023.
The current estimated value to stockholders is based on an implied value assigned to certain outstanding warrants based on Black Scholes option pricing model as of August 18, 2023. This value will not be finalized until the closing of the merger and is subject to increase or decrease based on certain variables, including the actual trading price of Timber at the time of the merger and the trading volatility of Timber common stock prior to the merger.
The CVRs that Timber stockholders will receive provide for the payment of up to an additional $22 million with respect to specific milestones for TMB-001, of which up to $12 million is related to FDA approval of TMB-001 by October 1, 2025 for the treatment of congenital ichthyosis, and up to $10 million of which is related to the first achievement of TMB-001 net sales exceeding $100 million within four consecutive calendar quarters by December 31, 2028. As part of the transaction, LEO Pharma has agreed to provide Timber with a bridge loan of up to $3.0 million, subject to certain conditions. The payments of the CVRs are subject to certain deductions relating to the repayment of 50% of the bridge loan provided by LEO Pharma to Timber in connection with the merger.
John Koconis, Chairman and Chief Executive Officer of Timber, said, “We are very pleased to deliver a transaction that will maximize long term value for Timber’s shareholders. LEO Pharma is a leader in global dermatology with a mission that matches our own - a relentless pursuit to help patients suffering from skin diseases.
“LEO’s expertise and global footprint make it the best choice to advance and achieve the full potential of Timber’s portfolio of product candidates. We believe that LEO has the potential to establish TMB-001 as the standard of care in the treatment of congenital ichthyosis, a devastating, rare disease.
“Finally, I would like to sincerely thank our dedicated team at Timber for their tireless efforts, and the clinical investigators, medical professionals, patients and families whose personal contributions have been instrumental in shaping our understanding of TMB-001.”
The transaction has been unanimously approved by the Boards of Directors of both companies and is expected to close in the fourth quarter of 2023, subject to customary closing conditions, including approval by the holders of a majority of the shares of Timber’s common stock. Following completion of the transaction, Timber will become a privately held company and shares of Timber’s common stock will no longer be listed on any public market.
Timber will file a Current Report on Form 8-K with the Securities and Exchange Commission (“SEC”) that will include a copy of the merger agreement and the CVR agreement and will contain a more detailed description of the merger and the consideration to be received by Timber stockholders.
About LEO Pharma
LEO Pharma is a global company dedicated to advancing the standard of care for the benefit of people with skin conditions, their families and society. Founded in 1908 and majority owned by the LEO Foundation, LEO Pharma has devoted decades of research and development to advance the science of dermatology, and today, the company offers a wide range of therapies for all disease severities. LEO Pharma is headquartered in Denmark with a global team of 4,700 people, serving millions of patients across the world. In 2022, LEO Pharma generated net sales of DKK 10.6 billion.
About Timber Pharmaceuticals, Inc.
Timber Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of treatments for rare and orphan dermatologic diseases. The Company's investigational therapies have proven mechanisms-of-action backed by decades of clinical experience and well-established CMC (chemistry, manufacturing, and control) and safety profiles. Timber is focused on developing non-systemic treatments for rare dermatologic diseases including congenital ichthyosis (CI) and sclerotic skin diseases. For more information, visit www.timberpharma.com.