BUSINESS INSIGHTS

Spotlight Therapeutics Appoints Visionary Global Pharma Drug Developer Antoine Yver, M.D., M.Sc. as First Independent Board Member

Spotlight Therapeutics | July 18, 2022 | Read time : 00:19 min

Spotlight Therapeutics
Spotlight Therapeutics, Inc. a biotechnology company applying new insights to develop cell-targeted in vivo CRISPR gene editing biologics, announced the appointment of Antoine Yver, M.D., M.Sc. to the Spotlight Board of Directors. Trained as an oncologist and immunologist, Dr. Yver’s extensive industry experience and perceptive acumen will be extremely valuable as Spotlight continues to strengthen its highly differentiated TAGE platform and advance lead therapeutic candidates towards the clinic.

We are thrilled to welcome Antoine to Spotlight’s Board of Directors. Few people in our industry enjoy such a remarkable track record of successful drug development, exercising extreme rigor in following the science to deliver practice-changing medicines that serve patients with significant unmet medical needs.”

Mary Haak-Frendscho, Ph.D., President and Chief Executive Officer of Spotlight

As Executive Vice President and Global Head of Oncology R&D at Daiichi Sankyo, Dr. Yver played a key role in the development of the new breakthrough cancer biologic, Enhertu®, that is redefining breast cancer treatment and promises to set a new standard of care, as well as other Daiichi Sankyo DXd ADCs. From 2009 to 2016, Dr. Yver held executive leadership positions at AstraZeneca, including SVP & Global Head of Oncology Development. Under his leadership, Tagrisso® and Lynparza® were successfully developed and commercialized for patients. Prior to joining AstraZeneca, Dr. Yver held roles at Merck, Johnson & Johnson, Aventis and Rhone-Poulenc Rorer. Dr. Yver is Executive Vice President and Chairman of Development at Centessa Pharmaceuticals plc and currently serves as an Independent Director of the Board of Directors at Sanofi.

“I am delighted to join the Spotlight Board and support the mission to unlock the full potential of gene editing and enable effective single administration therapeutics for patients,” said Dr. Yver. “Spotlight’s biologics-based delivery approach has the potential to forge a new generation of cell-targeted in vivo CRISPR gene editing medicines across multiple therapeutic areas.”

“Antoine’s deep insights and vast global pharma experience will help propel Spotlight to becoming a clinical stage company,” said Craig Gordon, M.D., Spotlight board member, Founder, CEO, and CIO of GordonMD™ Global Investments. “We look forward to working together to impact the strategic decisions of the company that will ultimately provide benefit to patients.”

About Spotlight Therapeutics
Established in mid-2018, Spotlight Therapeutics is a privately held biotechnology company advancing a pipeline of cell-targeted in vivo CRISPR gene editing therapies. Spotlight's proprietary technology platform TAGE is a new class of biologics, CRISPR effectors engineered for direct delivery in vivo, to achieve cell-selective therapeutic genome editing. Spotlight's pipeline is advancing its modular programmable CRISPR effectors towards clinical studies in immuno-oncology, ophthalmic diseases and hemoglobinopathies. 

Spotlight

USP is exploring how its extensive expertise in developing quality standards for drug products and ingredients can contribute to pharmaceutical continuous manufacturing.


Other News
BUSINESS INSIGHTS

Lifespin Launches Commercial Access to its Metabolic Profiler Software and Database for Pharma and Biobank Services

Lifespin | September 16, 2022

Lifespin GmbH, based in Regensburg with offices in Boston, Massachusetts, announced that it has launched a new commercial service that, for the first time, will provide the pharmaceutical and biobanking industry with access to Lifespin’s proprietary metabolic database, along with its advanced interpretive software to assist in various phases of drug research, development, and manufacturing. Immediate applications of Lifespin’s commercial services will include quality control for synthetic/natural compounds, quantitative profiling of metabolites in liquid samples, monitoring of drug responses and organ-specific metabolic phenotyping, and precision nutrition to therapeutic drug monitoring and longitudinal treatment monitoring. “Making our technology platform accessible to the pharma and biobanking industry will provide the field with deeper clinical insights and improve stratification of patient data in clinical trials, enabling pharmaceutical companies and others in the field to perform a range of precision phenotyping of cell or animal models and patient cohorts. Our goal is to provide the pharma and biobank scientists with yet another powerful resource in their development toolbox, including our advanced algorithms and database, to enhance their earlier phase analysis and to help better identify the strengths and weaknesses of compounds in study during research through clinical trials.” Dr. Ali Tinazli, CEO of Lifespin GmbH Lifespin, a deep data company that maps human health based on snapshots of metabolic states, has built one of the largest and most comprehensive databases of metabolic health profiles across healthy and diseased individuals covering multiple age and gender groups as well as specific diseases in neurology, oncology, and inflammation. Utilizing proprietary technology, Lifespin is performing quantitative in-house measurements of metabolomes, digitizing metabolic profiles that include billions of metabolic relationships. These digital metabolic profiles allow systematic mapping across various health conditions and may enable differential diagnosis and early detection of health conditions, staging of diseases, monitoring of treatment success and personalized medicine. Lifespin’s advisory board consists of key opinion leaders such as James Rothman (Nobel Laureate in Physiology/Medicine, Sterling Professor of Cell Biology, Yale University, New Haven, CT USA) and other prominent leaders in the relevant fields of study.

Read More

BUSINESS INSIGHTS

Aquestive Therapeutics Receives FDA Tentative Approval for Libervant™ (diazepam) Buccal Film

Aquestive Therapeutics, Inc. | August 31, 2022

Aquestive Therapeutics, Inc. a pharmaceutical company advancing medicines to solve patients' problems with current standards of care and provide transformative products to improve their lives, announced today that the U.S. Food & Drug Administration has granted tentative approval for Libervant™ Buccal Film for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity that are distinct from a patient’s usual seizure pattern in patients with epilepsy 12 years of age and older. "Tentative approval" means the FDA has concluded that Libervant has met all required quality, safety, and efficacy standards for approval but, due to an existing FDA regulatory grant of orphan drug market exclusivity for Valtoco®, a diazepam nasal spray product, Libervant is not yet eligible for marketing in the United States. As a result of the FDA determination, the Agency cannot give final approval for Libervant until the expiration or inapplicability of the orphan drug market exclusivity, including, for example, by court order, a selective waiver of the orphan drug exclusivity, or a reversal of the FDA’s decision and determination that Libervant is “clinically superior” to Valtoco. “The FDA’s decision provides welcome clarity to our business. The tentative approval of Libervant is a significant achievement that brings us one step closer to bringing this important medicine to patients who are unable or choose not to use the current standards of care. Libervant has the potential to offer these patients a treatment option that is simple, portable, and precise. A significant unmet need exists for additional alternate delivery options given the ongoing shortage of diazepam rectal gel, which continues to represent a substantial portion of the current diazepam rescue market. This FDA action further validates our ability to gain FDA approval of our pipeline programs. We look forward to continuing the rapid progression of AQST-109 for the treatment of severe allergic reactions, including anaphylaxis, towards a filing with the FDA.” Daniel Barber, Chief Executive Officer of Aquestive “We continue to examine the FDA’s determination on Libervant market access and plan to request a meeting with the Agency as soon as possible. We disagree with the FDA’s determination on the various points presented by the Agency and believe that, particularly in the case of our submitted studies on the effect of food on the absorption of diazepam formulations, Libervant has the distinct advantage of being able to be readily administered when needed without regard to food, providing an important benefit to patients. This is important to patients as rapid and extensive drug absorption is critical in a rescue situation. We will continue to vigorously engage with the FDA on the need for these data to be carefully considered.” During the review process, the Company submitted to the FDA the results of a 2021 Aquestive sponsored randomized, open-label, two-sequence, two-period, two-treatment crossover study to evaluate the effect of food on the pharmacokinetics of Valtoco in healthy adult subjects. The results of this study indicated that, when Valtoco is administered after a high fat meal, the maximum drug concentration (Cmax) was reduced by 48% compared to Valtoco administered to subjects in a fasted state. The study also showed that the time to maximum drug concentration (Tmax) of Valtoco doubled from 2 hours to 4 hours when administered after a high fat meal. Aquestive provided the data along with a cross-study comparison to a similar study performed with Libervant, to FDA during the review process. The FDA’s decision concluded that the information Aquestive submitted was not sufficient to overturn the Agency's previous conclusion regarding the lack of food effect for Valtoco. Specifically, the FDA stated that “[a] cross-study comparison is not considered [by the reviewer] to be a suitable approach for making a quantitative comparison of plasma concentrations between different products because of the lack of a common reference standard between the two studies.” Aquestive will seek to gain alignment with the Agency on a reasonable path to appropriately characterize the food effect of Valtoco including potentially conducting a comparative study as indicated by the FDA. About Libervant Libervant™ is a buccally, or inside of the cheek, administered film formulation of diazepam, a benzodiazepine intended for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity that are distinct from a patient’s usual seizure pattern in patients with epilepsy 12 years of age and older. Aquestive developed Libervant as an alternative to the device-based products currently available for patients with refractory epilepsy, including a rectal gel and nasal spray products. Approximately 1.0 million patients with epilepsy suffer from uncontrolled refractory seizures, approximately 85% of whom will not interact with the available treatments. About Aquestive Therapeutics Aquestive Therapeutics, Inc. is a pharmaceutical company advancing medicines to solve patients’ problems with current standards of care and provide transformative products to improve their lives. We are developing orally administered products to deliver complex molecules, providing novel alternatives to invasive and inconvenient standard of care therapies. Aquestive has five commercialized products on the U.S. market, four licensed products and one stand-alone proprietary product to date, Sympazan® (clobazam) oral film for the treatment of seizures associated with Lennox-Gastaut syndrome. Our licensees market their products in the U.S. and around the world. The Company also collaborates with pharmaceutical companies to bring new molecules to market using proprietary, best-in-class technologies, like PharmFilm®, and has proven drug development and commercialization capabilities. Aquestive is advancing a late-stage proprietary product pipeline focused on treating diseases of the central nervous system, or CNS, and an earlier stage pipeline for the treatment of severe allergic reactions, including anaphylaxis.

Read More

BUSINESS INSIGHTS

Enable Injections Enhances Board with Addition of John DeFord, Ph.D.

Enable Injections, Inc. | August 12, 2022

Enable Injections, Inc. a company developing and manufacturing the enFuse® platform of investigational wearable drug delivery systems, is pleased to announce the appointment of John DeFord, Ph.D. to the company's Board of Directors, effective July 29, 2022. Enable Injections' enFuse is an innovative drug delivery technology designed to subcutaneously (SC) deliver large volumes of up to 50mL for a wide range of therapies and diseases, dedicated to improving the patient experience. Dr. DeFord brings over three decades of expertise as a global medical technology executive, most recently the executive vice president and chief technology officer for Becton, Dickinson and Company (BD) (BDX), a global medical technology company. Prior to BD, Dr. DeFord served in senior science, technology and leadership roles of increasing responsibility at organizations such as C.R. Bard and Cook Incorporated, with additional experience in venture capital as managing director of Early Stage Partners venture capital fund. He is currently also a member of the board of directors at Nordson Corporation (NDSN), NuVasive (NUVA), and Blue Spark, Inc. DeFord graduated from Purdue University with a bachelor's degree and master's degree in electrical engineering, and a Ph.D. in electrical/biomedical engineering, dedicating his career to innovative medical technologies to save lives, enhance quality of life and improve the patient experience. "Enable Injections is at the forefront of both simplifying the therapeutic delivery of critical medications and enhancing quality of life for the patient. I am excited to join the Board of Directors at this important time in the company's growth and development; and I look forward to working with the management team and Board to help accelerate translation of this important technology to the clinical setting." Dr. DeFord "We are pleased to welcome Dr. DeFord as a new director to Enable's Board," said Mike Hooven, President and CEO of Enable Injections. "We believe adding depth of experience in the global medical technology space such as Dr. DeFord's is key to the strong growth of our business." About Enable Injections Cincinnati-based Enable Injections is a global healthcare innovation company developing and manufacturing drug delivery systems designed to improve the patient experience. Enable's body-worn enFuse® delivers high-volume pharmaceutical and biologic therapeutics via subcutaneous administration, with the aim of improving convenience, supporting superior outcomes, and advancing healthcare system economics.

Read More

BUSINESS INSIGHTS

Hovione expands drug product offering with a new manufacturing line dedicated to Continuous Tableting

Hovione | September 07, 2022

Hovione, the leader in spray drying and particle engineering, announced that it has expanded its continuous manufacturing offering and services. A new state-of-the-art continuous manufacturing facility is now coming online at the Loures site in Portugal. In addition, to support Hovione's commitment to providing access and services for continuous tableting, Hovione has further strengthened its capabilities by establishing a highly experienced multi-disciplinary global team in continuous tableting and upgrading its labs with the tools needed to support the drug product lifecycle, thereby further guaranteeing competency and capacity from research & development to production. Continuous manufacturing is expected to change the landscape of drug product development and commercial production. The Food and Drug Administration (FDA) is partnering with the International Council for Harmonization and leading a global initiative to advance continuous manufacturing of small molecules and other pharmaceutical modalities. Hovione is offering customers a commercial continuous tableting platform prepared to support key control needs and deliver on operational excellence, mechanistic modeling, and appropriate process analytical technology (PAT). Additionally, Hovione´s quality system has been designed to support the release of continuous tableting products through automated in-process controls, deployment of real time release, and compliant digital infrastructure. This cutting-edge technology offers a distinct set of features and advantages including faster development of simpler processes, agile manufacturing for variable demand, robust control strategies and high process quality standards. "Hovione has been historically focused on innovative technologies that benefit our customers and patients. With this investment in continuous tableting, our customers can rely on our support to bring much needed medicines to the market as fast as possible with the highest quality and manufacturing standards. We are investing to accelerate the adoption of this technology as we continue to innovate and enhance our offering around our areas of expertise." Jean-Luc Herbeaux, Hovione´s CEO "This investment in continuous tableting from R&D to production further strengthens Hovione´s CDMO offering and responds to the growing customer interest." says Filipe Neves, Hovione´s Strategic Business Director. This latest investment in Continuous Tableting is part of Hovione´s broader strategy to build differentiated and enabling capabilities and assets to meet customers unique needs for oral dosage forms manufacturing. About Hovione Hovione is an international company with over 60 years of experience as a Contract Development and Manufacturing Organization with a fully integrated offering of services for drug substance, drug product intermediate and drug product. The company has four FDA inspected sites in the USA, Portugal, Ireland and China and development laboratories in Lisbon, Portugal and New Jersey, USA. Hovione provides pharmaceutical customers services for the development and compliant manufacture of innovative drugs, including highly potent compounds, and customized product solutions across the entire drug life cycle. In the inhalation area, Hovione offers a complete range of services, from API and formulation development to devices. Hovione´s culture is based on innovation, quality and delivery. Hovione was the first Chemical/ Pharmaceutical Company to become a Certified B Corp, is a member of Rx-360, EFCG and participates actively in industry quality improvement initiatives to lead new global industry standards.

Read More