PRNewswire | August 10, 2023
The Project to Accelerate New Treatments for Tuberculosis (PAN-TB) collaboration announced today the start of a phase 2b/c clinical trial, sponsored by the Bill & Melinda Gates Medical Research Institute (Gates MRI). The trial will evaluate whether novel regimens that combine registered products and new chemical entities have the potential to effectively treat drug-sensitive TB (DS-TB) and inform the development of a "pan-TB" regimen capable of treating all forms of active pulmonary TB.
The regimens under evaluation are designed to explore shorter treatment durations compared to existing drug regimens, without the need for accompanying drug-resistance testing for individuals. The goal is to identify a candidate regimen suitable for phase 3 development.
Trial sites are being hosted in countries with a high burden of TB. The trial has started in South Africa with the first participant screened in the last week of July. Subsequent trial sites are expected to launch in the Philippines in August and in Peru in 2024, pending national approvals.
The collaboration is evaluating two novel drug regimens comprising five antibacterial agents—bedaquiline, delamanid, pretomanid, quabodepistat (formerly known as OPC-167832), and sutezolid
DBQS – delamanid, bedaquiline, quabodepistat and sutezolid
PBQS – pretomanid, bedaquiline, quabodepistat and sutezolid
The trial employs an innovative design that consists of two stages to assess the efficacy of the regimens and evaluate the potential to shorten treatment duration. In the initial stage, the trial will enroll approximately 129 participants. The safety, tolerability, efficacy, and pharmacokinetics of the complete DBQS and PBQS regimens, with each drug administered daily for four months (17 weeks), will be evaluated in participants 18 to 65 years old with DS pulmonary TB. Approximately 43 participants will receive the six-month (26 weeks) standard-of-care treatment for DS-TB1 in the countries where trial sites are located.2
If successful, the second stage will enroll approximately 400 participants to test the selected regimen at shorter treatment durations, ranging from two to four months, across five arms. An additional 30-40 participants will be enrolled in the standard-of-care arm for the second stage of this trial.
To support participants during their treatment, the trial integrates a Stop Treatment and Watch (STrAW) Concilium, a group made up of expert clinical consultants that will evaluate trial participants' treatment response, blinded to the specific regimen they receive, and advise trial investigators on patient management.
The trial will utilize the best available tools including regular engagement with treatment support workers, direct observation of therapy with video technology, and medication reminder monitors. A novel Biomarkers Strategy will inform promising new biomarkers with the aim of enabling faster access to data to monitor treatment outcomes for future trials.
The need for novel TB treatment regimens
Despite the introduction of new medicines in recent years, treatment remains a long, complex, and demanding process for people diagnosed with TB and can place significant costs on households and the health system. The most used drug regimen for the treatment of DS-TB requires people to take multiple drugs for at least six months, often with routine monitoring.
TB treatment can place unmanageable financial strain on individuals and their households. The World Health Organization (WHO) estimates that nearly half of TB-affected households face catastrophic total costs associated with treatment, including costs of travel to clinics, loss of income during treatment, and more, fueling cycles of poverty in vulnerable communities.
Further, accurate, rapid drug resistance testing is essential for early diagnosis of both drug-sensitive and drug-resistant TB, but access to testing can be limited due to high costs, technical challenges, and other barriers.
Emilio Emini, PhD, CEO, the Bill & Melinda Gates Medical Research Institute, said: "The Bill & Melinda Gates Medical Research Institute is committed to developing biomedical interventions that address global health concerns for those in the greatest need. The team at the Gates MRI looks forward to working with our partners across the PAN-TB collaboration, to evaluate the potential of these novel TB treatment regimens, in the hope that it may offer a potentially easier pathway for all people diagnosed with pulmonary TB by offering well-tolerated, shorter and simpler treatment options."
Masanori Kawasaki, Global TB Project Leader, Otsuka Pharmaceutical Co. Ltd, said: "We are immensely proud to be part of this remarkable, trailblazing, collaboration which is now launching a clinical trial for a new, and shorter, pan-TB treatment regimen. We are excited too to be working closely with key partners to bring new treatment options to patients. Otsuka is dedicated to life-saving research and development as demonstrated by our sustained hard work and investment in developing a new chemical entity called quabodepistat. We are most grateful to the Bill & Melinda Gates Medical Research Institute for bringing together leaders in the field of TB, towards the goal of ending TB for good."
Ruxandra Draghia-Akli, MD, PhD, Global Head of Global Public Health R&D, Janssen Research & Development, LLC, said: "Our medicine for multidrug-resistant TB has reshaped TB treatment regimens over the past decade, but more must be done to develop the even shorter, simpler regimens needed to treat all forms of the disease and end TB once and for all. Today's milestone is another step in this direction, and we look forward to seeing the results of this important study."
Mel Spigelman, MD, President and CEO of TB Alliance, said: "Sincere thanks and congratulations to all who have made this innovative and exciting new journey a reality. Bringing revolutionary, highly impactful new treatments to all TB patients around the world is a goal that deserves and requires all our concerted dedicated efforts."
About the Project to Accelerate New Treatments for Tuberculosis
The Project to Accelerate New Treatments for Tuberculosis (PAN-TB) is a first-of-its-kind collaboration among philanthropic, non-profit and private sectors that aims to accelerate the development of an investigational drug regimen capable of treating all forms of tuberculosis.
The PAN-TB collaboration leverages members' collective assets, resources and scientific expertise to identify and evaluate new drug regimens with an acceptable safety profile, that have the potential to treat both drug-sensitive and drug-resistant TB and are well-tolerated, shorter in duration and simpler to use than existing options. The collaboration focuses on advancing research through phase 2 clinical efficacy studies to identify promising regimens for further development.
The PAN-TB collaboration plans to work closely and transparently with the European Regimen Accelerator for Tuberculosis (ERA4TB), which was launched in January 2020. New molecular entities identified by ERA4TB that show promise in initial human studies could later be incorporated into the PAN-TB collaboration's later-stage, clinical research. Several organizations, including Evotec, GSK, TB Alliance and Janssen Pharmaceutica NV, are members of both projects, which will help to ensure coordination across collaborations toward the common goal of advancing TB drug and regimen development.
The members of the PAN-TB collaboration are Evotec, GSK, Janssen Pharmaceutica NV, Otsuka Pharmaceutical Co., Ltd., based in Japan, TB Alliance, the Bill & Melinda Gates Medical Research Institute and the Bill & Melinda Gates Foundation. Additional members may be announced in the future.
About Evotec SE
Evotec is a life science company with a unique business model that delivers on its mission to discover and develop highly effective therapeutics and make them available to the patients. The Company's multimodality platform comprises a unique combination of innovative technologies, data and science for the discovery, development, and production of first-in-class and best-in-class pharmaceutical products. Evotec leverages this "Data-driven R&D Autobahn to Cures" for proprietary projects and within a network of partners including all Top 20 Pharma and over 800 biotechnology companies, academic institutions, as well as other healthcare stakeholders. Evotec has strategic activities in a broad range of currently underserved therapeutic areas, including e.g., neurology, oncology, as well as metabolic and infectious diseases. Within these areas of expertise, Evotec aims to create the world-leading co-owned pipeline for innovative therapeutics and has to-date established a portfolio of more than 200 proprietary and co-owned R&D projects from early discovery to clinical development. Evotec operates globally with more than 4,900 highly qualified people.
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.
Globenewswire | July 03, 2023
Baudax Bio, Inc. a pharmaceutical company focused on innovative products for acute care and related settings, announced the acquisition of TeraImmune, a privately held a biotechnology company focused on discovery and development of novel Treg-based cell therapies for autoimmune diseases.
“This combination blends the world class scientific expertise of the TeraImmune team with the Baudax team’s proven ability to execute clinical development programs, which we believe is a win for the shareholders of both companies,” said Ms. Henwood. “This merger adds TeraImmune’s TI-168 asset to the Baudax portfolio—a promising next-generation, autologous FVIII TCR-Treg cell therapy candidate to eliminate clotting factor VIII (FVIII) inhibitors in Hemophilia A patients. Hemophilia A is a rare genetic bleeding disorder that is caused by a lack of FVIII, with an Investigational New Drug (IND) application already FDA-cleared. We believe this combination can enable, with a modest initial budget, activating the Phase 1/2a Clinical Trial of TI-168 for Treatment of HA. We believe this is an attractive therapeutic area, with established preclinical proof of concept in TI-168 through successes observed in hemophilic animal models. We believe this platform, with customization to the target condition, has potential for clinical application in management of Myasthenia Gravis, Pemphigus Vulgaris and combination therapies for other conditions such as Organ Transplantation, MS and other auto-immune disorders.”
“Concurrently, we intend to continue to progress the development of our existing Neuromuscular Blockade (NMB) portfolio at a prudent pace,” continued Ms. Henwood. “With the positive data obtained from our Phase 2 trial of BX1000, we continue to believe that when combined with our reversal agent BX3000 our NMB regimen may provide improved control of neuromuscular paralysis for surgical patients and deliver the first innovation in NMB in decades.”
“With IND clearance from the FDA already in hand for TI-168, this transaction permits the continued development of this promising asset,” said Yong Chan Kim, PhD, Co-Founder of TeraImmune. “We are looking forward to working with Gerri Henwood and the excellent clinical development team at Baudax to advance this asset to its full potential,” said Jihoon (Jay) Park, PhD, Co-Founder of TeraImmune. Daniel Chai, former Board Member of TeraImmune and managing partner of Turret Capital Management said, “We are very excited to see the results that will be driven by the combination of an impressive leadership team and how many lives that can be impacted by the development of this platform technology.”
Gerri Henwood, President and Chief Executive Officer of Baudax Bio, will continue as CEO of the combined entity. In conjunction with the transaction, Yong Chan Kim, Ph D, former Chief Executive Officer of TeraImmune, will be appointed to the Board of Directors of Baudax.
About the Transaction
The acquisition of TeraImmune was structured as a stock-for-stock transaction whereby all TeraImmune outstanding equity interests were exchanged for a combination of shares of Baudax common stock, shares of newly designated convertible Series X Non-Voting Convertible Preferred Stock. Subject to shareholder approval of the conversion, each share of Series X Non-Voting Convertible Preferred Stock will automatically convert into 1,000 shares of common stock, subject to certain beneficial ownership limitations set by each holder. On a pro forma basis and based upon the number of shares of Baudax common stock and preferred stock issued in the acquisition, Baudax equity holders immediately prior to the acquisition will own approximately 18% of the combined Company (on an as-converted, fully-diluted basis and excluding certain out-of-the-money warrants held by Baudax’ equity holders) immediately after these transactions. The acquisition was unanimously approved by the Board of Directors of Baudax and the Board of Directors of TeraImmune. The closing of the transaction was not subject to the approval of Baudax shareholders.
Nobel Capital provided a fairness opinion to the Baudax Board of Directors.
About Baudax Bio
Baudax Bio is a pharmaceutical company focused on innovative products for acute care and related settings. The Company has a pipeline of innovative pharmaceutical assets including two clinical-stage, novel neuromuscular blocking (NMBs) agents, one that recently completed a Phase II clinical trial and an additional unique NMB undergoing a dose escalation Phase I clinical trial, as well as a proprietary chemical reversal agent specific to these NMBs, which is currently undergoing nonclinical and manufacturing studies to prepare for an expected IND filing in the summer of 2023.