Zolgensma wins FDA green light in child SMA

pharmatimes | May 28, 2019

The U.S. Food and Drug Administration (FDA) has approved Novartis’ Zolgensma (onasemnogene abeparvovec-xioi) at a cost of $2.1 milllion per treatment. The drug is the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA), which is the most severe form of SMA and a leading genetic cause of infant mortality.The price tag has garnered substantial criticism, as even the split payment structure adds up to $425,000 per year over five years. Novartis has announced that it will give ‘partial rebates’ if the treatment doesn't work.The approval marks a “milestone in the transformational power of gene and cell therapies to treat a wide range of diseases,” said acting FDA commissioner Ned Sharpless. He continued, “With each new approval, we see this exciting area of science continue to move beyond the concept phase into reality. The potential for gene therapy products to change the lives of those patients who may have faced a terminal condition, or worse, death, provides hope for the future.“The FDA will continue to support the progress in this field by helping to expedite the development of products for unmet medical needs through the use of review pathways designed to advance innovative, safe and effective treatment options.”The indication is for the treatment of children less than two years of age with SMA, which is caused by a mutation in the survival motor neuron 1 (SMN1) gene. The gene encodes the survival motor neuron (SMN) protein  a protein found throughout the body, which is critical for the maintenance and function of specialised nerve cells, called motor neurons. Motor neurons in the brain and spinal cord control muscle movement throughout the body. If there is not enough functional SMN protein, then the motor neurons die, leading to debilitating and often fatal muscle weakness that can lead to the child eventually not being able to eat or breathe.

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Sandoz targets growth opportunities in respiratory and complex generics through acquisition of respiratory device company Coalesce

Novartis Pharma AG | March 14, 2022

Sandoz, a Novartis division, announced that it has successfully acquired the UK-based medical and drug delivery device development company, Coalesce Product Development Limited. Through this deal, Sandoz has acquired the significant capabilities and assets of Coalesce, which will help it build on its existing portfolio of respiratory medicines and further improve patient access to these high-quality, complex therapies. “Respiratory and complex generics are areas of relatively high unmet medical need, due largely to their comparatively high technical complexity,” Sandoz CEO, Richard Saynor “At Sandoz, we have the experience and expertise to succeed in these fields and this acquisition offers us a significant new growth platform, particularly in the US and Europe, reinforcing our commitment to pioneer access for patients.” Respiratory diseases are leading causes of death and disability worldwide.1 Asthma is a major non-communicable disease affecting both children and adults, and in 2019 it affected an estimated 262 million people.2 In the same year, about 3.23 million deaths were attributed to chronic obstructive pulmonary disease (COPD).3 The resulting burden of respiratory diseases on healthcare systems and productivity amounted to more than USD 380 billion per year across 28 EU member states.4 With an existing portfolio of six in market-products and nearly twice as many more in the pipeline, Sandoz sees respiratory as a key pillar of its ambitious long-term growth strategy and intends to actively explore further opportunities both in-house and externally. More generally, Sandoz is determined to grow its portfolio in the complex generics space, including complex injectables. About Sandoz Sandoz, a Novartis division, is a global leader in generic pharmaceuticals and biosimilars. Our purpose is to pioneer access for patients by developing and commercializing novel, affordable approaches that address unmet medical needs. Our ambition is to be the world’s leading and most valued generics company. Our broad portfolio of high-quality medicines, covering major therapeutic areas, accounted for 2021 sales of USD 9.6 billion.

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PHARMACY MARKET

IAMA Therapeutics Partners with the Italian Institute of Technology to Embark on Landmark Research on Neuroscience Drug Discovery

IAMA Therapeutics | March 31, 2022

IAMA Therapeutics, a Series A preclinical-stage pharmaceutical company focused on the discovery, development, and commercialization of novel medicines to make a difference in the lives of individuals suffering from brain disorders, today announced a strategic collaboration with the IIT-Istituto Italiano di Tecnologia where they will design and profile new classes of compounds targeting cation cotransporters in the CNS area. This partnership consolidates a scientific collaboration between IAMA Therapeutics and IIT, which signed, together with the Telethon Foundation earlier in 2021, a license agreement, granting IAMA therapeutics an exclusive, worldwide license to research, develop, manufacture, and commercialize a novel class of modulators of intracellular chloride concentration. "We are pleased with and excited by this partnering opportunity with IAMA Therapeutics, a clear example of IIT technology transfer approach. We are setting the Italian way of producing science and tech-based innovation, where the collaboration among brilliant researchers, important founding agencies like Fondazione Telethon, and investors creates the right ecosystem enabling new businesses to emerge." Dr. Matteo Bonfanti, Director of Technology Transfer at Istituto Italiano di Tecnologia "We are very happy for the partnership between IAMA Therapeutics and IIT," said Francesca Pasinelli, General Manager of Fondazione Telethon. "This collaboration is based on innovative science, which was selected and nurtured earlier on by Fondazione Telethon. As part of our focus on fostering development, we funded the project and supported researchers through the technology transfer process together with IIT. Now, this promising research avenue is accessing the resources and competencies necessary to take and accelerate the long and expensive path to patients. It shows what can be accomplished through a timely and coordinated effort to maximize research impact". About IAMA Therapeutics IAMA Therapeutics is a pharmaceutical company that couples emerging advances in drug discovery and neurobiology to build a leading, next-generation neuroscience pipeline. We have developed a platform to identify and advance diverse classes of small molecules to selectively inhibit the sodium potassium chloride cotransporter and other therapeutic targets relevant to treating central nervous system disorders characterized by neuronal imbalance, including primary and secondary autism, epilepsy, and movement disorders. About Istituto Italiano di Tecnologia Istituto Italiano di Tecnologia is a foundation established jointly by the Italian Ministry of Education, Universities, Research, and the Ministry of Economic and Finance to promote excellence in basic and applied research and contribute to Italy's economic development. The primary goals of the IIT are the creation and dissemination of scientific knowledge and the strengthening of Italy's technological competitiveness. To achieve these two goals, the IIT cooperates with academic institutions and private organizations, fostering scientific development, technological advances, and training in high technology. About Fondazione Telethon Fondazione Telethon is a major biomedical charity in Italy whose mission is to advance biomedical research to cure rare genetic diseases. Throughout its 32 years of activity, Fondazione Telethon has invested almost €600 million in funding over 2,700 projects to study 580 diseases, involving more than 1,600 researchers in Italy. As part of its goal to foster development and maximize the therapeutic impact of research, Fondazione Telethon further pursues cooperation with academia, industry, and venture capital through the creation of partnerships and start-ups.

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BUSINESS INSIGHTS

OPTEL ACQUIRES PHARMA TRACK-AND-TRACE UNIT OF KÖRBER

OPTEL Group | January 12, 2022

OPTEL GROUP, already a world leader in supply chain traceability, is expanding its expertise and global reach with the recent acquisition of the track-and-trace unit of the Körber Business Area Pharma, formerly known as Traxeed, based in Germany. Körber is a leading international technology group. The acquisition, announced today, will allow OPTEL to leverage the unit's expertise in pharmaceutical and agrochemical track-and-trace technologies, provide the company with a second foothold in the European marketplace and contribute to its diversification plans. "Creating a strong presence in the German market is key to the success of our strategic objectives because Germany is a well-established manufacturing hub, with a strong history of developing innovative manufacturing efficiency solutions and Industry 4.0 technologies," Louis Roy, founder and president of OPTEL "We are pleased that we have found a solid and future-oriented new owner with a broad international market access, from whose global network and years of experience in the highly specialized track-and-trace sector customers will clearly benefit. We are furthermore convinced that the new strategic owner can open up attractive development opportunities for the team," said Dr. Jürgen Krebs, Chief Technology Officer and Chief Operating Officer in the Körber Business Area Pharma. The acquisition allows OPTEL to welcome highly skilled pharma track-and-trace experts from Körber, complementing OPTEL's existing strengths in traceability, vision technologies, software and hardware. Furthermore, it will solidify and expand the company's presence in the European market by providing a second base of operations after Limerick, Ireland. In addition to the acquisition, a close collaboration between OPTEL and Körber's Business Area Pharma is planned in the future. The two companies plan to sign a partnership agreement whereby OPTEL becomes Körber's preferred partner for track-and-trace solutions for the pharmaceutical industry. Since its founding in 1989, OPTEL has become the world's foremost provider of track-and-trace solutions to the pharmaceutical industry. Its acquisitions in recent years have positioned it as the only company with the ability to provide complete, full-stack supply chain traceability. The Canadian multinational has since diversified into other industries, including consumer-packaged goods, metals and minerals, and agrochemicals. The acquisition of the track-and-trace unit of the Körber Business Area Pharma is expected to accelerate further expansion. ABOUT OPTEL OPTEL is a leading global provider of traceability systems whose goal is to use its innovative technologies to build a sustainable world through the Intelligent Supply Chain. OPTEL is the only company with the ability to offer complete end-to-end traceability, providing granular data at every step of the supply chain – from raw materials to the consumer and beyond. Founded in 1989, OPTEL is a Certified B Corporation headquartered in Canada, with facilities in Ireland, India and Brazil, as well as employees worldwide. ABOUT KÖRBER We are Körber – an international technology group with about 10,000 employees, more than 100 locations worldwide and a common goal: We turn entrepreneurial thinking into customer success and shape the technological change. In the Business Areas Digital, Pharma, Supply Chain, Tissue and Tobacco, we offer products, solutions and services that inspire. We act fast to customer needs, we execute ideas seamlessly, and with our innovations we create added value for our customers. In doing so, we are increasingly building on ecosystems that solve the challenges of today and tomorrow. Körber AG is the holding company of the Körber Group.

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Dermaliq Therapeutics Signs USD 15 Million Series A Round to Advance Three Transformative Drug Therapies into Clinical Trials

Dermaliq Therapeutics | January 18, 2022

Novaliq GmbH, 3E Bioventures Capital and Beijing Whale Technology Corporation Ltd. today announced the establishment of Dermaliq Therapeutics, Inc (Dermaliq), a newly formed Delaware-based pharmaceutical development company, and the signing of a USD 15 million Series A financing round. 3E Bioventures Capital, a leading international life science investor, led the round and was joined by Beijing Whale Technology Corporation Ltd. Dermaliq's existing shareholder Novaliq has transferred, and licensed Intellectual Property (IP) related to the field of dermatology to Dermaliq. Skin diseases are the fourth leading cause of non-fatal disease burden worldwide, underscoring the role of dermatology in the growing field of global health. Skin conditions have significant impact on patients' well-being, mental health, and social participation. Although topical and local delivery of active ingredients to diseased skin is the preferred treatment route for the majority of patients, current therapies often lack satisfactory skin penetration and patient compliance. Dermaliq is developing the next generation of skin care therapies to enhance targeted penetration into skin tissues and to reduce unwanted side effects for millions of patients. The unique and proprietary technology now owned by Dermaliq, called hyliQ™, is designed to allow cutaneous drug delivery with unmatched bioavailability. The technology enables the development of superior, highly effective liquid drug products with exceptional cosmetic properties. The proceeds will be used to conduct three Phase 1/2a clinical dermatology programs with the goal to demonstrate safety and efficacy and to advance this novel skin care drug category to market to improve the lives of millions of patients: DLQ01 is a liquid drug candidate for the topical treatment of female and male androgenetic alopecia, targeting the prostamide E2 (PGE2) receptor in hair bulbs through specific follicular drug delivery. DLQ02 is a novel and liquid topical calcineurin inhibitor drug therapy for plaque psoriasis, particularly in difficult to treat areas. DLQ03 is a broad-spectrum liquid antibiotic drug candidate that targets topical treatment of bacterial skin and soft tissue infections, including the reduction of antibiotic-resistant strains. The Company has demonstrated proof-of-principle for enhanced topical drug delivery using hyliQ™ in nonclinical models for all three applications. "We are excited that 3E Bioventures Capital and Beijing Whale Technology Corporation have committed to support Dermaliq as new investors in the Series A round. Our spin-off from Novaliq and successful raising of significant funds provides us with the opportunity to build a leading specialty pharmaceutical company and expand the application of Novaliq's transformative drug delivery technology into dermatology." Dr. Frank Löscher, Chief Executive Officer of Dermaliq "3E is committed to investing in global leading life sciences and biomedical companies and help them grow with access to globalized resources and capabilities," said Karen Liu, Managing Director 3E Bioventures Capital. "Dermaliq is using a unique, and scientifically sound technology to achieve high efficiency drug delivery across the skin. The technology is already proven in ophthalmology and holds great promise for dermatology. We will be excited to explore additional drug pipelines in China and synergize with Dermaliq on both pre-clinical and clinical development." "Whale Technology values the solid science foundation of Dermaliq's proprietary hyliQ™ technology platform," said Ming Gao, founder and Chief Executive Officer of Whale Technology Co. Ltd. "Dermaliq's founding team demonstrates a great combination of science, clinical experience and commercialization strength. We see great potential of Dermaliq's pipeline in the field of dermatology after Novaliq's proven clinical and commercial success in ophthalmology." "Dermatology is the logical next step into using Novaliq's proprietary technology platform outside eye care," said Dr. Christian Roesky, Chief Executive Officer Novaliq. "The spin-off into an independent company allows Dermaliq to greatly benefit from our experience in ophthalmology while being fully committed to the multiple product development activities in dermatology, and likely to further broaden its pipeline to other skin conditions." To reflect the new shareholder structure, Karen Liu of 3E Bioventures Capital will join Dermaliq's Board of Directors. Dr. Löscher has also transitioned from his role as Chief Technology Officer at Novaliq to lead Dermaliq as its CEO & President. About Dermaliq Dermaliq Therapeutics, Inc. is a private company founded in 2021 through a spin off from Novaliq to reimagine topical dermatology. The Company is incorporated in Wilmington (DE), USA under Delaware law. The company has signed a USD 15 million series A round to advance three transformative skin care drug therapies through clinical trials. Key shareholders are Novaliq GmbH, 3E Bioventures Capital and Beijing Whale Technology Corporation Ltd. About 3E Bioventures Capital 3E Bioventures Capital is a healthcare venture capital firm, dedicated to investing in cutting-edge life sciences and biomedical technologies, with a focus on breakthrough first-in-class therapies and disruptive cross-disciplinary innovations in medical devices and diagnostics. 3E Bioventures takes on a science-driven, entrepreneur-friendly investment philosophy by working closely with companies and research institutions to develop drugs or products that have strong unmet medical needs. With offices in Beijing, Shanghai, and the San Francisco Bay Area, 3E Bioventures leverages its experience, capabilities, and network to help companies tap into markets and resources across the Pacific and advance with greater speed and capital efficiency. The motto of 3E Bioventures Capital is captured in its name 3E: Expertise, Efficiency, Execution. About Beijing Whale Technology Corporation Ltd Beijing Whale Technology Corporation Ltd. is a China-based pharmaceutical group committed to the long-term sustainable development in the fields of chemistry, pharmaceuticals and aesthetics. With more than 20 years of history, Whale Tech. has developed two core business units. On one hand, Whale Pharmaceutical Co. Ltd., Whale Chemistry Co. Ltd. and Whale Pharmatech Co. Ltd. are grouped as pharmaceutical and chemistry business unit. On the other hand, Whale Biotech is a research stage biotech company with the focus of developing aesthetics products. About Novaliq Novaliq is a biopharmaceutical company focusing on the development and commercialization of first- and best-in-class ocular therapeutics based on EyeSol®, the worldwide first water-free eyedrop technology. Novaliq offers an industry-leading portfolio addressing today's unmet medical needs of millions of patients with eye diseases. Novaliq GmbH is headquartered in Heidelberg, Germany and Novaliq Inc. has an office in Cambridge, MA, USA. The long-term shareholder is dievini Hopp BioTech holding GmbH & Co. KG, an active investor in Life and Health Sciences companies.

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