Monarch Becomes the 1st FDA-Cleared Device for ADHD

mddionline | April 22, 2019

NeuroSigma’s Monarch eTNS might be the first device to treat ADHD, but it is one of a growing number of technologies that occupy the burgeoning bioelectronic medicine space. The Monarch external Trigeminal Nerve Stimulation (eTNS) System made history last week after it became the first device to win a nod from FDA to treat attention deficit hyperactivity disorder (ADHD). It should be noted the technology has had CE mark since 2015. Monarch was developed by NeuroSigma and is indicated to help treat 7 to 12-year-olds suffering from ADHD. What makes this de novo clearance from FDA so significant is that typically pharmaceutical solutions are used to treat patients suffering from the neurological disorder. Monarch is about the size of a cellphone. The device generates a low-level electrical pulse and connects via a wire to a small patch that adheres to a patient's forehead, just above the eyebrows, and should feel like a tingling sensation on the skin.

Spotlight

Since the advent of the commercial Internet, digital technologies have empowered consumers worldwide, leading them to play an active and demanding role with the companies with which they do business. The rules of engagement have changed: By using the Internet, consumers can be heard, and they can influence other consumers powerfully. The Internet permeates every aspect of people’s lives and has transformed the way we live and work. The confluence of the Internet, social media and ubiquitous mobile devices has inalterably redrawn the contours of consumer empowerment. This tectonic shift is impacting the traditional interactions of healthcare professionals (HCP) and pharmaceuticals companies, even amid the regulatory scrutiny that is endemic to this industry.


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KemPharm Announces Strategic Acquisition of Arimoclomol from Orphazyme, Expanding its Rare CNS Diseases Pipeline

KemPharm | May 16, 2022

Arimoclomol is an NDA-stage, revenue-generating investigational drug candidate being developed for the treatment of Niemann-Pick disease type C a rare progressive neurodegenerative disease KemPharm plans to refile the New Drug Application (NDA) for arimoclomol in NPC with the U.S. Food and Drug Administration as early as the First Quarter of 2023 CELEBRATION, Fla., May 15, 2022 KemPharm, Inc. a specialty pharmaceutical company focused on the discovery and development of novel treatments for rare central nervous system (CNS) diseases, announced a definitive agreement with Orphazyme A/S to acquire arimoclomol, an orally-delivered, first-in-class heat shock protein (HSP) amplifier being developed as a treatment for Niemann-Pick disease type C (NPC). NPC is a rare progressive neurodegenerative disease that impacts children, adolescents, and adults, and is characterized by an inability of the body to transport cholesterol and lipids inside of cells, which leads to the abnormal accumulation of these substances within various tissues of the body, including the brain. Arimoclomol is currently being made available to NPC patients in the U.S., France and Germany under Orphazyme’s Early Access Programs (EAP). Under the terms of the agreement, KemPharm will purchase substantially all of the assets and operations of Orphazyme, including arimoclomol, for a cash payment of USD $12.8 million. The Company expects to finance the cash payment with a revolving line of credit secured by KemPharm’s balance sheet. KemPharm intends to retain the majority of Orphazyme’s current employees. In addition, KemPharm has agreed to assume an estimated reserve liability equal to approximately USD $5.2 million, which is an estimated future rebate due to the French regulatory authorities based on the revenue generated from the EAP in France. For the year ending December 31, 2022, the EAP is expected to generate at least USD $12 million in revenue based upon enrollment in France as of March 2022. The EAP is expected to remain in place until arimoclomol becomes commercially available in each of the current EAP markets. The transaction is expected to close on or before June 1, 2022, subject to customary closing conditions and approval by Orphazyme’s creditors and the Danish bankruptcy court. Canaccord Genuity LLC acted as a strategic advisor to KemPharm for the transaction. “This strategic acquisition of arimoclomol is a transformative event that significantly expands our rare CNS disease development pipeline, bringing to KemPharm an NDA-stage, revenue-generating product upon which we intend to build commercial capabilities that allow KemPharm to create and retain value for the benefit of shareholders. Moreover, the financial structure of the acquisition combined with the revenue currently being generated by arimoclomol from the early access program in France affords us the opportunity to acquire the asset in a capital efficient manner that has the potential to create positive cash flow, while incurring no shareholder dilution.” Richard Pascoe, Executive Chairman of KemPharm Arimoclomol is administered orally and has been studied in ten Phase 1, four Phase 2, and three pivotal Phase 2/3 trials. Arimoclomol has received Orphan Drug Designation (ODD) for NPC in the United States and the European Union. Arimoclomol has received Fast-Track Designation (FTD), Breakthrough Therapy Designation (BTD), and Rare Pediatric Disease Designation (RPDD) from the FDA for NPC. If approved in the U.S., arimoclomol would also be eligible to receive a Pediatric Priority Review Voucher. On June 17, 2021, Orphazyme received a Complete Response Letter (CRL) from the FDA regarding its NDA for arimoclomol for the treatment of NPC. Orphazyme also withdrew its European Marketing Authorisation Application (MAA) for arimoclomol for the treatment of NPC ahead of a final vote and opinion by the Committee for Medicinal Products for Human Use. “The acquisition of arimoclomol aligns perfectly with our strategy to build KemPharm’s value via the advancement and commercialization of novel treatments that address rare CNS conditions, including our lead clinical candidate, KP1077 in idiopathic hypersomnia,” stated Travis Mickle, Ph.D., President and Chief Executive Officer of KemPharm. “We have carefully evaluated the CRL issued by the FDA and the minutes from the subsequent Type A meeting, as well as the data that has been generated from the development work performed to date. We believe the efficacy signal for arimoclomol in NPC is convincing and that there is a viable regulatory path that could enable a successful NDA resubmission. KemPharm has significant experience with challenging regulatory situations, having successfully led or participated in three FDA product approvals, two of which followed an initial CRL. We welcome the opportunity to work with the FDA on the resubmission of the NDA for arimoclomol in NPC, which we expect to file as early as the first quarter of 2023.” NPC is a rare progressive lysosomal storage disorder characterized by an inability of the body to transport cholesterol and lipids inside of cells. This leads to dysfunction in organs such as the brain, spleen and liver. NPC can range from a fatal disorder within the first few months after birth (neonatal period) to a late onset, chronic progressive disorder that remains undiagnosed well into adulthood. Disease progression is irreversible in all patients, and loss of neuro-cognitive function adversely impacts their daily life. The mean age of death is 13 years (Bianconi, 2019), and there are no approved treatments for NPC in the United States. “NPC is an ultra-rare, inherited neurodegenerative disease that affects people of all ages from infancy to adulthood, and leads to progressive impairment of mobility, cognition, speech, and swallowing, culminating in premature death,” said Marc Patterson, MD, Professor of Neurology, Pediatrics and Medical Genetics at Mayo Clinic. “Therapies to treat NPC are desperately needed, and there is hope that a treatment such as arimoclomol could provide a solution to patients around the world who are living daily with the disease. It is encouraging that there is an opportunity to continue the regulatory process for arimoclomol with the FDA.” About KemPharm KemPharm is a specialty pharmaceutical company focused on the discovery and development of novel treatments for rare central nervous system diseases through its proprietary LAT® platform technology. KemPharm utilizes its proprietary LAT® platform technology to generate improved prodrug versions of FDA-approved drugs as well as to generate prodrug versions of existing compounds that may have applications for new disease indications. KemPharm’s prodrug product candidate pipeline is focused on the high need areas of idiopathic hypersomnia (IH) and other CNS/rare diseases. In addition, the U.S. Food and Drug Administration (FDA) has approved AZSTARYS®, a new once-daily treatment for ADHD in patients age six years and older containing KemPharm’s prodrug, serdexmethylphenidate (SDX), which is being commercialized by Corium, Inc. in the U.S., and APADAZ®, an immediate-release combination product containing benzhydrocodone, KemPharm’s prodrug of hydrocodone, and acetaminophen, which is being commercialized by KVK-Tech, Inc. in the U.S.

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PCI Pharma Services Selects Thoughtworks for Digital Platform Transformation with Agile Approach

Thoughtworks | July 11, 2022

Thoughtworks a global technology consultancy that integrates strategy, design and engineering to drive digital innovation, today announced the signing of a multi-year agreement with PCI Pharma Services, a leading global contract development and manufacturing organization. Thoughtworks will help PCI in its journey to digitize its supply chain, with the goal of accelerating the time it takes to bring clients’ life-changing therapies to market for patients. By co-developing tools for digital business modernization, Thoughtworks will help PCI improve client visibility into its clinical and commercial drug supply chains for pharmaceutical and biotech companies of all sizes in 100+ countries. By digitizing the supply chain, Thoughtworks will help PCI deliver greater insights to clients to increase their overall efficiency, support their business goals and ultimately produce high quality treatments for patients. The agreement includes expanding and adapting pci | bridge, an industry first-of-its-kind, end-to-end data platform that provides real-time status of projects, automatically identifies risks, spots trends and provides business analytics insights. With this modern application programming interface (API), clients have the ability to seamlessly integrate their systems with PCI’s, creating greater transparency and a more simplified process for decision-making. “Digital transformation has been a key differentiating factor for PCI’s business strategy, and the evolution of pci | bridge will be critical to increasing client engagement and maximizing the value of data and analytics for PCI and our external partners. With Thoughtworks’ 28+ years of experience at the forefront of innovation, we look forward to making our clients’ lives easier through this partnership." Wayne Hull, Chief Digital and Technology Officer, PCI Pharma Services With a shared commitment to customer-centricity, the Thoughtworks and PCI product and engineering teams will create a new level of intuitive self-service solutions for clients. The partnership will also simplify the collection and analysis of information to reach business insights faster and streamline processes to develop useful new products for clients. The teams will do this by working side-by-side using agile development methods, including pair programming, test-driven development and continuous integration/continuous delivery (CI/CD) automation. “Recognized as a leader in global CDMO services, PCI Pharma Services is well on its digital transformation journey, providing clients with sophisticated supply chain solutions and a digitally integrated experience unlike anything else in the industry,” said Chris Murphy, Chief Executive Officer, Thoughtworks North America. “We are thrilled to partner with PCI Pharma Services as it empowers its clients to make even more powerful, life-impacting decisions through their expansive data and analytics ecosystems.” About PCI Pharma Services PCI is a leading global CDMO, providing clients with integrated end-to-end drug development, manufacturing and packaging capabilities that increase their products’ speed to market and opportunities for commercial success. PCI brings the proven experience that comes with more than 50 successful product launches each year and over five decades in the healthcare services business. We currently have 30 sites across seven countries and over 4,300 employees that work to bring life-changing therapies to patients. Leading technology and continued investment enable us to address global drug development needs throughout the entire product life cycle – from manufacturing capabilities through the clinical trial supply chain and into commercialization. Our clients view us as an extension of their business and a collaborative partner with the shared goal of improving patients’ lives. For more information, please visit pci.com About Thoughtworks Thoughtworks is a global technology consultancy that integrates strategy, design and engineering to drive digital innovation. We are 11,000+ people strong across 49 offices in 17 countries. Over the last 25+ years, we’ve delivered extraordinary impact together with our clients by helping them solve complex business problems with technology as the differentiator.

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Protect Pharmaceutical Corporation has acquired top notch software development company.

PROTECT PHARMACEUTICAL Corp | June 28, 2022

Protect Pharmaceutical Corporation has acquired the 100% of the shares of CJSC “Inaiapp”, a Belarusian developer of corporate software for business digitalization. With 8 years of blockchain development experience and list of long term clients, Inaiapp will bring its qualification and management processes to a new level. “Robotic process automation, AI systems and machine learning systems, blockchain-based solutions, Data Science tools for business growth - we closely follow global trends in IT technology and are now ready to create the most profitable solution for your business.” Nick Greb, President of PRTT PRTT offers range of services and software products, to solve painful problems. The company is using time-tested practices in the field of robotics, artificial intelligence, machine learning and blockchain, creating first-class solutions that meet current business needs. “Now we will combine the US market presence with extensive technical expertise that enable us to develop products that accelerate and streamline business processes through autonomy, transparency and security.” - Nick adds. About PRTT Protect Pharmaceutical Corporation is a full-cycle software development and delivery company that produces, releases, and supports digital products for the business-to-business and business-to-consumer markets. The company’s expertise lies in custom software development and consulting services in Business automation, Artificial Intelligence, Machine Learning, Data Science, Big Data, Image recognition, Blockchain development, and cloud services. Its domain focus includes Supply chain, Logistics, Healthcare, Finance, Real estate, Legal, Insurance, Advertising, and more.

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ClinChoice Raises $150 mm Series E Round Financing, Further Strengthening its Global Services Capabilities

ClinChoice | July 05, 2022

Recently, ClinChoice announced the successful completion of its $150 mm Series E round financing. The investment was led by Legend Capital, and co-invested by Taikang Life Insurance, Sherpa Healthcare Partners and existing shareholders, including Lilly Asia Ventures and Apricot Capital. Proceeds of the financing will be used to fund the Company's continued business expansion and innovative service offering worldwide. Recently, ClinChoice announced the successful completion of its $150 mm Series E round financing. The investment was led by Legend Capital, and co-invested by Taikang Life Insurance, Sherpa Healthcare Partners and existing shareholders, including Lilly Asia Ventures and Apricot Capital. Proceeds of the financing will be used to fund the Company's continued business expansion and innovative service offering worldwide. As a full service clinical-stage CRO, ClinChoice serves the global market by providing integrated one-stop clinical development services to emerging biopharmaceutical, medical device and consumer health clients around the world. ClinChoice has over 3000 employees worldwide covering most of drug development hotbeds in US, China, multiple countries in Europe and Southeast Asia. With over 25 years of high-quality services, ClinChoice has completed more than 1,000 Phase I-IV full-service clinical projects and supported over 100 innovative drug registrations, clinical design and development. ClinChoice strictly adheres to GCP and ICH standards and has developed robust SOPs recognized by more than 50 large multinational companies, all of which have passed multiple inspections and audits by major regulatory agencies around the world. "We thank the investors for their trust in ClinChoice. This round of capital investment fully validates ClinChoice's rapid growth and future potential in the field of clinical CRO. With the new round of financing, we will further enhance our innovative service offering and continue business expansion by organic growth and M&A, in order to better serve our clients worldwide." Global Chairman and CEO of ClinChoice, Mr. Ling ZHEN Dr. Hongbin ZHOU, Co-Chief Investment Officer of Legend Capital, said, "We would like to express our gratitude for trusting in Legend Capital and we are excited to be part of ClinChoice, together with the Management Team and the existing shareholders, including Lilly Asia Ventures, Goldman Sachs. We are very positive on the growth outlook of the CRO industry. As a rapidly growing CRO, ClinChoice has built an excellent platform combining global expertise with deep knowhow across key local markets, to support innovation. Going forward, we will work closely with the management team to improve its existing business, attract industry talent and launch new strategic initiatives." About ClinChoice ClinChoice is a global clinical CRO, providing high-quality Integrated One-stop Service Offerings for new bio-pharmaceutical and medical device clients worldwide. Our Service Offerings cover Clinical Operation, Project Management, Biostatistics, Data Management, Biostatistical Programming, Regulatory Affairs, Medical Affairs, Feasibility Research, HGRAC Submission, Clinical Safety Management, Site Management Organization, Nature Medicine/Traditional Chinese Medicine and Phase I Clinical Research, etc. Now, ClinChoice has over 3,000 employees worldwide covering most of drug development hotbeds in US, China, multiple countries in Europe and Southeast Asia. Since its inception, ClinChoice has completed multiple rounds of financing, with investments from renowned institutional investors including Goldman Sachs, Lilly Asia Ventures, and DFJ Dragon Fund. About Legend Capital In April 2001, Legend Capital was established as a fund manager under Legend Holdings, focusing on the early-stage VC and growth-stage PE investment. Since its establishment, Legend Capital has been adhereing to internationally accepted standards to create best practices in fund management and operation. With a proven track record of more than 20 years, it now has full experience in fund management and operation and has built an extensive partner ecosystem across the entire investment chain.

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Spotlight

Since the advent of the commercial Internet, digital technologies have empowered consumers worldwide, leading them to play an active and demanding role with the companies with which they do business. The rules of engagement have changed: By using the Internet, consumers can be heard, and they can influence other consumers powerfully. The Internet permeates every aspect of people’s lives and has transformed the way we live and work. The confluence of the Internet, social media and ubiquitous mobile devices has inalterably redrawn the contours of consumer empowerment. This tectonic shift is impacting the traditional interactions of healthcare professionals (HCP) and pharmaceuticals companies, even amid the regulatory scrutiny that is endemic to this industry.

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