FDA Rejects Regulatory Filing For Zogenix Epilepsy Drug; GW Pharma Moves In Sympathy

Zogenix, Inc | April 09, 2019

Zogenix, Inc.,which develops therapies for rare diseases, said Monday afternoon the FDA issued a refusal to file letter regarding its NDA for Fintepla, chemically fenfluramine hydrochloride, which is being evaluated for the treatment of seizures associated with Dravet syndrome. The FDA determined the NDA wasn't sufficiently complete to permit a substantive review, primarily due to two reasons: non-submission of certain non-clinical studies for assessing chronic administration of the pipeline asset and an incorrect version of a clinical dataset, which prevented the completion of the review process required to support the filing. Fintepla, previously codenamed ZX008, is Zogenix's lead asset and was touted to be a multi-billion-dollar opportunity for the company. The adverse development suggests a serious setback to the small-cap biotech. Bank of America analyst Tazeen Ahmad said last September ZX008's worldwide, risk-adjusted peak sales is estimated to be $1.5 billion in 2029, comprising a $604-million opportunity in Dravet syndrome, $503 million in Lennox-Gastaut syndrome and $386 million in other pediatric epilepsy conditions.

Spotlight

Size of the market in EU-5 set to grow by 25% between 2017-2022 with a CAGR of 4.5% over this period EU-5 accounts for 69% of European pharma market in 2022 Germany is forecast to have highest increase in market value at €9.7bn.


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PHARMA TECH

ArisGlobal Accelerates Growth Announces Executive Evolution

ArisGlobal | September 08, 2022

ArisGlobal, the leading provider of life sciences software that automates core drug development functions with its end-to-end technology platform LifeSphere®, today announces an expansion to its C-suite. James Jarrett is the company's new Chief Financial Officer, Kathleen Turland joins as the first Chief Legal Officer, and Aman Wasan has stepped into the new role of Chief Commercial Officer. The three appointments are happening during a time of significant momentum for ArisGlobal, which has experienced annual recurring revenue growth of 34% in the first half of 2022 YoY. James Jarrett, Chief Financial Officer Jarrett brings over a decade of executive leadership experience driving revenue and cash flow growth. He has partnered with management teams and boards of directors of high-growth, highly leveraged companies undergoing rapid change. Prior to joining ArisGlobal, he served as the CFO for Emergence Health Holdings, Carewell Urgent Care, and ProCure Proton Therapy Center. Kathleen Turland, Chief Legal Officer Turland is a global strategic leader with more than 20 years of legal experience. She brings her broad range of legal and business skills to ArisGlobal, having worked with companies experiencing significant growth and expansion over the years, including General Electric, and FUSE Group. She started her legal career in New York, clerking for a Federal Court Judge, and working at two revered New York law firms. Aman Wasan, Chief Commercial Officer Wasan is an expert in general management and commercial leadership with a deep understanding and knowledge of drug safety and clinical research. Since joining ArisGlobal in 2018, Wasan has excelled in six different positions, most recently as Senior Vice President of Global Commercials, where he managed global enterprise business. Before joining the ArisGlobal team, Wasan worked for Bioclinica as the company's Global Head of Safety and Regulatory Services. "As we continue scaling our business and building new functions to better serve our customer base, expanding our C-suite will help us execute our mission and further drive our success in the life sciences industry. James, Kathleen, and Aman carry a wealth of industry knowledge instrumental in empowering ArisGlobal to further expand its customer pipeline, continue to develop innovative technology, and maintain a role as a trusted leader in the market." CEO Mike Gordon Turland's addition to the company also represents consistent and focused efforts by ArisGlobal to continually put women in positions of leadership. Turland joins ArisGlobal veterans Ritu Shrivastava, Vice President Corporate Development, and Heidi Hattendorf, Vice President of Marketing, on the executive team. Currently, ArisGlobal also has women in core leadership positions across business, product, quality, people, and technology functions. About ArisGlobal ArisGlobal is led by passionate individuals who support life sciences leaders in developing and monitoring breakthrough medicines and therapies. With more than 35 years of expertise in the life sciences industry, ArisGlobal develops technology products within the platform LifeSphere to power pharmaceutical and biotech research and development. Building on our commitment to corporate social responsibility and sustainability initiatives, we give back to the communities where we live and work – in the U.S, Europe, India, Japan, and China.

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BUSINESS INSIGHTS

Inversago Pharma Raises $95 million CAD in Series C Financing

Inversago Pharma Inc. | October 18, 2022

Inversago Pharma Inc. a clinical stage biotech company with a unique portfolio of peripherally-acting CB1 inverse agonists, announced the completion of a Series C funding of $95 million CAD led by New Enterprise Associates. Additional new investors Forbion’s Growth Opportunities Fund and Amgen Ventures joined current investors including Forbion’s Ventures Fund IV Fonds de solidarité FTQ, Genesys Capital, AmorChem, JDRF T1D Fund and adMare BioInnovations in the financing round. Proceeds from the Series C financing will be used to move Inversago’s lead program, INV-202, into a Phase 2 clinical trial in Diabetic Kidney Disease in Q4 2022. This follows the previously announced IND clearance from the FDA and the upcoming promising Phase 1b topline results in subjects with metabolic syndrome. Additionally, the funds will accelerate the Company’s other key programs currently in pre-clinical development, while establishing its leadership in the field of CB1 blockers. “We are privileged to have the support and commitment of such an impressive investor syndicate at this critical stage of the Company’s development. This new round of financing is a strong endorsement of the whole Inversago team, who have carried the vision to leverage the potential of the CB1 blockade in developing potent treatments for a growing number of patients suffering from metabolic conditions, such as Diabetic Kidney Diseases and other diabetes-related conditions”. François Ravenelle, PhD, Chief Executive Officer of Inversago Pharma Commenting on the investment, Ed Mathers, General Partner at NEA, said: “Inversago has demonstrated that peripherally-acting CB1 blockers have an untapped potential to create a new class of target therapies that serve diabetic patient populations. Today’s financing reflects our shared vision and collaboration with the Company’s management as they initiate a Phase 2 clinical trial with INV-202”. Nanna Lüneborg, General Partner at Forbion Growth, added: “We are delighted to join this Series C financing of Inversago, and support the Company to develop a pipeline of new therapies which promise to make a profound impact on the lives of patients with cardiometabolic diseases. We have been impressed by the terrific progress of Inversago since Forbion led the Company’s Series B round two years ago and are excited to further support the Company from both Forbion Growth and Forbion Ventures”. About NEA New Enterprise Associates, Inc. is a global venture capital firm focused on helping entrepreneurs build transformational businesses across multiple stages, sectors and geographies. With nearly $24 billion in cumulative committed capital since the firm's founding in 1977, NEA invests in technology and healthcare companies at all stages in a company's lifecycle, from seed stage through IPO. The firm's track record of investing includes more than 260 portfolio company IPOs and more than 440 mergers and acquisitions. About Forbion Forbion is a dedicated life sciences venture capital firm with offices in The Netherlands, Germany and Singapore. Forbion invests in life sciences companies that are active in the (bio-) pharmaceutical space. Forbion manages well over EUR 2 billion across multiple fund strategies that cover all stages of pharmaceutical drug development. Forbion’s current team consists of over 30 life sciences investment professionals that have built an impressive performance track record since the late nineties with investments in 92 companies. Besides financial objectives, Forbion selects investments that will positively affect the health and well-being of patients. The firm is a signatory to the United Nations Principles for Responsible Investment. Forbion operates a joint venture with BGV, the manager of seed and early-stage funds, especially focused on Benelux and Germany. About Amgen Ventures Amgen Ventures is Amgen's corporate venture capital fund, dedicated to investing in emerging companies and technologies to advance promising new medicines and solutions to healthcare’s biggest challenges. About Inversago Pharma Located in Montreal, Inversago Pharma is a privately owned Canadian biotech company at clinical stage, specialized in the development of new therapies focusing on CB1 blockade, based on first-in-class, peripherally-acting CB1 inverse agonists. Inversago aims to provide new treatment options that improve the lives of patients affected by metabolic conditions such as Diabetic Kidney Disease including Diabetic Nephropathy Type 1 and Type 2 Diabetes Non-Alcoholic Steatohepatitis complications of obesity and hypertriglyceridemia as well as fibrotic indications like Progressive Fibrosis-Interstitial Lung Disease including Idiopathic Pulmonary Fibrosis and other conditions.

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RESEARCH

Corium Announces Publication of ADLARITY® Clinical Trial Data: Drug Exposure Equivalent to Oral Donepezil With Favorable GI Side Effect Profile

Corium, Inc. | September 20, 2022

Corium, Inc., a fully-integrated biopharmaceutical company leading the development and commercialization of novel central nervous system therapies, announced publication of its phase 1 healthy volunteer study results for ADLARITY in the peer-reviewed Journal of Alzheimer's Disease. The article, "Comparison of Steady-State Pharmacokinetics of Donepezil Transdermal Delivery System with Oral Donepezil," reports that Corium's novel once-weekly Alzheimer's dementia treatment delivered drug exposure equivalent to oral donepezil while presenting lower gastrointestinal (GI) adverse events overall compared to oral donepezil. The article is currently available online and expected to be published in the hardcopy of the Journal of Alzheimer's Disease, Volume 90, Issue 1, on October 25, 2022. "Transdermal delivery offers meaningful potential benefits over oral administration, including ease of use, maintenance of steady concentrations of drugs, reduced gastrointestinal adverse effects, and better treatment compliance. The availability of a transdermal formulation of donepezil gives clinicians, patients, and their caregivers an important new option to consider when treating dementia of the Alzheimer's type," said study co-author Pierre N. Tariot, M.D., director of the Banner Alzheimer's Institute in Phoenix, AZ. "The study demonstrated the equivalent exposure of Adlarity to oral donepezil and supports the use of ADLARITY as a compliant and safe once-weekly dosing regimen for treatment of patients with dementia of the Alzheimer type. The trial also shows Corium's commitment to addressing the unmet needs of patients with CNS disorders." Charles Oh, M.D., Chief Medical Officer of Corium ADLARITY Transdermal System: Equivalent to Oral Donepezil Exposure, Fewer GI Adverse Events Overall In the trial, investigators compared the extent of donepezil exposure from the once-weekly ADLARITY to a once-daily oral donepezil formulation in 60 healthy adults (NCT04617782). The trial included three treatment periods of 5 weeks each. In the first period, all the participants received ADLARITY weekly, which provided 5 mg/day of donepezil. In the second period, participants were randomized to receive either 10 mg/day once-weekly ADLARITY or 10 mg/day daily oral donepezil, followed by switching to the alternative treatment, ADLARITY or oral donepezil, in the third period. All the participants knew which treatment they received in this open-label trial. Investigators examined the amount of donepezil in the participants' blood, including the maximum plasma concentration (Cmax) and the total amount of drug exposure (area under the curve or AUC). About Alzheimer's Disease Alzheimer's disease is a progressive and irreversible brain disorder. It involves changes in brain tissue including abnormal buildup of proteins as well as loss of neuron function. The resulting damage leads to the loss of remembering, reasoning, and thinking abilities. The related behavioral changes include the loss of independence in activities of daily living and self-care. Dementia ranges in severity from mild, when it is just beginning to affect a person's functioning, to moderate, to severe, when the person must depend on others for the basic activities of day-to-day life. Patients with advanced Alzheimer's disease may be unable to chew and swallow easily. About Corium Corium, Inc., is a fully-integrated biopharmaceutical company that is leading the development and commercialization of CNS therapies that provide physicians with innovative treatment options for patients, their families, and their caregivers. Corium is commercializing two U.S. FDA approved products, ADLARITY and AZSTARYS. About Gurnet Point Capital Gurnet Point Capital is a unique healthcare investment platform within the B-Flexion group and led by a team with deep expertise in an industry for which they share a passion, both as investors and senior executives. GPC invests long-term capital and supports entrepreneurs in building a new generation of companies that deliver outsized returns through active ownership. Based in Cambridge, MA, its remit encompasses life sciences and health care focused businesses, with a particular emphasis on businesses that have high growth potential in the product development and commercialization stages of their evolution. With its strategy of driving best in class operational transformation for these businesses, to create social impact while generating significant economic value, GPC is able to deliver differentiated results for its investors and partners.

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BUSINESS INSIGHTS

BioMarin Announces Advancements in FDA Review of ROCTAVIAN™ (Valoctocogene Roxaparvovec) for Adults with Severe Hemophilia A

BioMarin Pharmaceutical Inc. | November 24, 2022

BioMarin Pharmaceutical Inc. announced advancements in the U.S. Food and Drug Administration review of the Biologics License Application of ROCTAVIAN™ for adults with severe hemophilia A. The Company was recently notified by the FDA that after further consideration, at this time, the Agency no longer plans to hold an advisory committee meeting to discuss the BLA for ROCTAVIAN that is currently under review. Previously, the FDA communicated to the Company that it did intend to hold an advisory committee meeting but did not specify a date. The Company also remains on track to host the scheduled FDA Pre-Licensure Inspection (PLI) of BioMarin's gene therapy manufacturing facility located in Novato, CA. "The review of a BLA is a dynamic process, and we appreciate FDA's ongoing engagement as we work toward delivering a potentially transformative treatment choice to those patients with severe hemophilia A. We look forward to further dialogue with the Agency as it reviews our application." Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin About valoctocogene roxaparvovec The FDA granted Regenerative Medicine Advanced Therapy designation to valoctocogene roxaparvovec in March 2021. RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are expected to address an unmet medical need in patients with serious conditions. The RMAT designation is complementary to Breakthrough Therapy Designation, which the Company received for valoctocogene roxaparvovec in 2017. In addition to the RMAT Designation and Breakthrough Therapy Designation, BioMarin's valoctocogene roxaparvovec also received orphan drug designation from the EMA and FDA for the treatment of severe hemophilia A. Orphan drug designation is reserved for medicines treating rare, life-threatening, or chronically debilitating diseases. The European Commission (EC) granted conditional marketing authorization to valoctocogene roxaparvovec gene therapy under the brand name ROCTAVIAN™ on August 24, 2022. Robust Clinical Program BioMarin has multiple clinical studies underway in its comprehensive gene therapy program for the treatment of severe hemophilia A. In addition to the global Phase 3 study GENEr8-1 and the ongoing Phase 1/2 dose escalation study, the Company is also conducting a Phase 3, single arm, open-label study to evaluate the efficacy and safety of valoctocogene roxaparvovec at a dose of 6e13 vg/kg with prophylactic corticosteroids in people with severe hemophilia A. Also ongoing is a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with pre-existing AAV5 antibodies (Study 270-203) and a Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with active or prior Factor VIII inhibitors. About Hemophilia A People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A often experience painful, spontaneous bleeds into their muscles or joints. Individuals with the most severe form of hemophilia A make up approximately 50 percent of the hemophilia A population. People with hemophilia A with moderate or mild disease show a much-reduced propensity to bleed. Individuals with severe hemophilia A are treated with a prophylactic regimen of intravenous Factor VIII infusions administered 2-3 times per week or a bispecific monoclonal antibody that mimics the activity of Factor VIII administered 1-4 times per month. Despite these regimens, many people continue to experience breakthrough bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life. Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have hemophilia A. About BioMarin BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening genetic diseases and medical conditions. The Company selects product candidates for diseases and conditions that represent a significant unmet medical need, have well-understood biology and provide an opportunity to be first-to-market or offer a significant benefit over existing products. The Company's portfolio consists of eight commercial products and multiple clinical and preclinical product candidates for the treatment of various diseases.

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Spotlight

Size of the market in EU-5 set to grow by 25% between 2017-2022 with a CAGR of 4.5% over this period EU-5 accounts for 69% of European pharma market in 2022 Germany is forecast to have highest increase in market value at €9.7bn.

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