DiNAQOR AG

DiNAMIQS focuses on the development of viral vectors for cell and gene therapies. Credit: ArtemisDiana via Shutterstock.com. Switzerland-based Siegfried has purchased a 95% stake in DiNAMIQS, a biotechnology firm that focuses on the development and production of viral vectors for cell and gene therapies. Siegfried will leverage DiNAMIQS’ capabilities to commercial scale by constructing a facility that complies with good manufacturing practice standards. The facility will be situated with DiNAMIQS’ existing operations at Bio-Technopark in Zurich-Schlieren, Switzerland, and is anticipated to become operational in 2025. DiNAMIQS’ parent company, DiNAQOR, will own the remaining 5% stake in the firm. The deal includes an option for Siegfried to purchase this remaining 5% five years after the acquisition. The move represents Siegfried’s strategy to expand its presence in biologics, and provides important growth opportunities in the mid to long term. DiNAMIQS will initially continue to run under its own name and as a mostly independent venture within Siegfried. DiNAMIQS and its clients will take advantage of the integration of its drug substances production activities into Siegfried’s capabilities, as well as the capacities for the aseptic filling and completion of complex biological entities at its facilities in Hameln, Germany, and Irvine in the US. Siegfried CEO Dr Wolfgang Wienand stated: “With this acquisition, we are entering the rapidly growing space of cell and gene therapies where viral vector-based technologies support the majority of clinical candidates in a rich development pipeline. “In our view, this approach of an initial small-scale acquisition of a highly capable technology platform followed by flexible bolt-on investments into capabilities and capacities in line with actual market needs, provides significant value upside at reasonable entry costs. “This is perfectly in line with our EVOLVE strategy and our track record of disciplined execution of value-adding mergers and acquisitions – and a further step towards our vision of creating a leading contract development and managing organisation in our space.” Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Optimise your cell therapy process: a guide to cell thawing Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer. This guide addresses how cell thawing has historically developed into the new techniques used today, along with the physical and biological implications of key metrics and components such as warming rate and ice structure. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies. By Cytiva Thematic By downloading this case study, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

Siegfried (SIX: SFZN), a leading global Contract Development and Manufacturing Organization (CDMO) for the pharmaceutical industry headquartered in Zofingen (Switzerland), announces the acquisition of a majority stake in DINAMIQS, a Swiss-based company focused on the development and manufacturing of viral vectors for cell and gene therapies. Siegfried will take DINAMIQS’ capabilities to commercial scale by building a state-of-the-art GMP compliant facility with flexible capacities of up to 500 liters. As the current operations of DINAMIQS, the new manufacturing facility will be located at the Bio-Technopark in Zurich-Schlieren and is expected to be operational in 2025. Siegfried will acquire 95% of DINAMIQS, while 5% will remain with DiNAQOR, an innovative life sciences platform company specialized in early-stage drug development and organ-specific delivery of genetic medicines and DINAMIQS’ current parent company. The total investment of Siegfried, including the establishment of the new GMP facility, will be in the mid double-digit million Swiss Francs and will be funded from existing cash and available financing arrangements. Five years following the closing of the transaction, Siegfried has the option to acquire the remaining 5% from DiNAQOR. Today, DINAMIQS employs a team of experienced industry experts and cutting-edge scientists in fully equipped R&D labs with high-end pilot-scale equipment and provides clinical viral vector development, analytical and manufacturing services for the gene therapy market. The company is led by Dr. Eduard Ayuso, a renowned expert in viral vectors, and operates in the Bio-Technopark Zurich-Schlieren, a prime ecosystem for biotech innovation and venturing in Switzerland with close links to leading universities in the space. DINAMIQS already has ongoing projects with DiNAQOR as well as biotech and large pharma customers. During the initial phase of scaling up its manufacturing activities, DINAMIQS will continue to operate under its name and as a largely independent venture within the Siegfried group in order to benefit from its high level of agility and flexibility while at the same time getting all necessary support from Siegfried’s global organization and in-house experts as well as access to the whole pharmaceutical customer universe. Furthermore, DINAMIQS and its customers will benefit from the forward integration of its drug substances manufacturing activities into Siegfried’s capabilities and capacities for the aseptic filling and finishing of complex biological entities at its commercial sites in Hameln (Germany) and Irvine (US). In line with recent investments in biologic drug product manufacturing including vaccines, the acquisition is a continuation of Siegfried’s strategy to further expand in the field of biologics and is expected to deliver significant growth opportunities in the mid to long term. Dr. Wolfgang Wienand, CEO of Siegfried: “With this acquisition, we are entering the rapidly growing space of cell and gene therapies where viral vector-based technologies support the majority of clinical candidates in a rich development pipeline. In our view, this approach of an initial small-scale acquisition of a highly capable technology platform followed by flexible bolt-on investments into capabilities and capacities in line with actual market needs, provides significant value upside at reasonable entry costs. This is perfectly in line with our strategy EVOLVE and our track record of disciplined execution of value-adding M&A – and a further step towards our vision of creating a leading CDMO in our space.” Dr. Johannes Holzmeister, M.D., Founder, Chairman and CEO of DiNAQOR: “Siegfried is our partner of choice for DiNAQOR’s gene therapy pipeline and will bring extensive CDMO experience as well as profound expertise in GMP manufacturing for the benefit of all current and future customers of DINAMIQS. This combination provides the perfect environment to scale up and fully exploit our excellent know-how and strong technology platform and create a leading CDMO for cell and gene therapies. I am happy to remain part of the success story of DINAMIQS as a member of the board of directors and look forward to rely on DINAMIQS as the development and manufacturing backbone for DiNAQOR also in the future.” The acquisition will have a limited impact on Siegfried’s financial performance in the short term, with expected additional annual revenues of low single-digit million Swiss Francs and negligible impact on reported profits. At the same time, the Siegfried group expects its half-year 2023 results to be well on track to support full-year results at the upper end of the current guidance for revenue growth of low to mid-single digit percentages (in LC) and a Core EBITDA margin of 20% or higher. About Siegfried The Siegfried Group is a global life sciences company with sites in Switzerland, Germany, Spain, France, Malta, the USA and China. In 2022, the company achieved sales of 1.229 billion Swiss francs and employed on 31.12.2022 more than 3’600 people at eleven sites on three continents. Siegfried Holding AG is publicly listed on SIX Swiss Exchange (SIX: SFZN). Siegfried is active in manufacturing pharmaceutical APIs (and their intermediates) as well as drug products (tablets, capsules, sterile vials, ampoules, cartridges and ointments) for the pharmaceutical industry and provides development services.

VOXZOGO® $48 Million Contribution in the Quarter Results in BioMarin Raising Full-year 2022 VOXZOGO Net Product Revenue Guidance to Between $140 Million and $170 Million U.S. Biologics Application for ROCTAVIAN™ Accepted by U.S. Food and Drug Administration (FDA); Prescription Drug User Fee Act (PDUFA) Target Action Date is March 31, 2023; Commercial Launch of ROCTAVIAN Underway in the European Union (EU) Following August 24, 2022 Approval SAN RAFAEL, Calif., Oct. 26, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) today announced financial results for the third quarter ended September 30, 2022. Continue Reading "As anticipated, BioMarin is on-track to deliver double-digit revenue growth and profitability for the full-year 2022, underscored by our record year-to-date operating results. VOXZOGO demand is driving our financial performance and we expect additional launches in Japan and other global markets to further accelerate sales of this innovative product," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "Our third quarter and year-to-date performance not only showcased the continuing success of our VOXZOGO commercial launch, but also the European regulatory approval of ROCTAVIAN, the world's first gene therapy approved for the treatment of severe hemophilia A. The launch in the EU is underway and, in the United States, the BLA for ROCTAVIAN was accepted by the FDA with an assigned PDUFA target action date of March 31, 2023. With two key product approvals and commercial launches over the past 12-months, the foundation of our 5-year strategic plan is in place." BioMarin Announces Third Quarter 2022 Year-over-year Total Revenue Growth of 24% (31% Excluding KUVAN) Tweet this Financial Highlights : Total Revenues for the third quarter of 2022 were $505.3 million, an increase of 24% compared to the same period in 2021 despite continued erosion of the U.S. KUVAN market, and incremental foreign exchange headwinds. The increase in Total Revenues was primarily attributed to the following: Higher VOXZOGO commercial sales due to new patients initiating therapy globally following regulatory approvals by the European Medicines Agency (EMA) and the FDA in the third and fourth quarters of 2021, respectively and Higher NAGLAZYME and VIMIZIM product revenues primarily driven by the timing of orders in countries that place large government orders, particularly in Europe and Latin America and new patients initiating therapy in Europe and the Middle East; partially offset by Lower KUVAN product revenues primarily due to generic competition as a result of the loss of exclusivity in the U.S., consistent with expectations. GAAP Net Loss decreased to $6.7 million for the third quarter of 2022 compared to GAAP Net Loss of $36.5 million for the same period in 2021. The decrease was primarily related to higher gross profit driven by increased sales volume, partially offset by higher selling, general and administrative (SG&A) expenses and a higher tax provision. The increase in SG&A expenses was largely due to higher costs to support the commercial launch of VOXZOGO and ROCTAVIAN, higher foreign currency exchange losses and severance costs associated with the Company's organizational redesign announced in October 2022. The increase to the tax provision was primarily attributed to higher year-to-date income driven by increased gross profits and the net gain on the sale of the Priority Review Voucher during the first quarter of 2022. Non-GAAP Income increased to $82.7 million for the third quarter of 2022 compared to Non-GAAP Income of $33.5 million for the same period in 2021 driven by higher gross profit due to increased sales volume partially offset by higher SG&A expenses largely driven by higher costs to support the commercial launch of VOXZOGO and ROCTAVIAN and higher foreign currency losses. New Product Approvals and Launches (ROCTAVIAN and VOXZOGO) Following EMA approval in the quarter, the commercial launch of ROCTAVIAN is now underway. It is estimated that approximately 20,000 adults are affected by severe hemophilia A across more than 70 countries in Europe, the Middle East, and Africa. Of the 8,000 adults with severe hemophilia A in the 24 countries within BioMarin's footprint covered by the EMA approval, there are an estimated 3,200 patients who are indicated for ROCTAVIAN based on the current label. To determine eligibility for ROCTAVIAN, treating physicians in countries covered by the EMA approval can use a companion diagnostic (CDx) test to ensure that patients do not have pre-existing antibodies to AAV5. The CDx test is CE-marked and designed to ensure the highest safety standards for use in determining patient eligibility for treatment with ROCTAVIAN. On October 12, 2022, BioMarin's resubmission of the BLA for ROCTAVIAN was accepted by the FDA with a PDUFA target action date of March 31, 2023. The FDA recently communicated plans to hold an advisory committee meeting but has yet to provide a date. If approved, ROCTAVIAN would be the first gene therapy in the U.S. for the treatment of severe hemophilia A. At present, in the U.S. the Premarket Approval (PMA) application is under review at the Center for Devices and Radiological Health to support contemporaneous approval of a CDx along with the ROCTAVIAN BLA. The global expansion of VOXZOGO is actively underway, with market access and reimbursement progressing as anticipated. As of September 30, 2022, there were 29 active markets contributing to VOXZOGO sales with an estimated 713 children being treated, as compared to an estimated 446 children as of June 30, 2022. In the quarter, VOXZOGO became commercially available in Japan resulting in meaningful contributions from the early launch. Japan accounts for approximately half of the 1,500 patient opportunity in the Asia-Pacific region. Mid-stage Product Life Cycle Expansion Opportunities (VOXZOGO and ROCTAVIAN) During the quarter, the Company held discussions with global regulatory health authorities regarding the favorable results from the Phase 2 randomized, double-blind, placebo-controlled VOXZOGO study in infants and young children up to five years of age with achondroplasia. Based on these interactions, BioMarin intends to submit supplemental marketing applications by the end of 2022 in the U.S. and EU to expand access to VOXZOGO treatment for this younger age group. Product expansion opportunities with ROCTAVIAN are supported by a number of clinical studies currently underway. The Phase 3b study to evaluate ROCTAVIAN with prophylactic corticosteroids has completed enrollment and is expected to read-out in early 2023. Two additional studies, one investigating ROCTAVIAN treatment in those with active or prior inhibitors, as well as one study investigating ROCTAVIAN in people with pre-existing antibodies against AAV5. Earlier-stage Development Portfolio (BMN 255, BMN 331, BMN 351, BMN 349, BMN 293 (DiNA-001)) BMN 255 for primary hyperoxaluria, a prognostic factor for chronic renal disease: The Company is proceeding with the multi-ascending dose phase of the First-in-Human study with BMN 255. BioMarin believes the availability of a potent, orally bioavailable, small molecule like BMN 255 may be able to significantly reduce disease and treatment burden in certain people with chronic renal disease. BMN 331 gene therapy product candidate for Hereditary Angioedema (HAE): Dosing continues in the Phase 1/2 HAERMONY study to evaluate BMN 331, an investigational AAV5-mediated gene therapy for people living with HAE, including dose escalation to the 6e13vg/kg dose, which our non-clinical studies project to provide therapeutic levels of C1-inhibitor. BMN 351 for Duchenne Muscular Dystrophy (DMD): Investigational New Drug application (IND)-enabling studies continue with BMN 351, an antisense oligonucleotide therapy for individuals with exon 51-skip-amenable DMD. BMN 351 was developed using familiar chemistry and superior biology, by targeting a novel, upstream, splice enhancer site demonstrating improved binding affinity and tolerability in preclinical models. Preclinical data suggest that restored expression of near-full-length dystrophin protein at levels of up to 40% will convert phenotypes from rapid loss to durable preservation of strength and ambulation. The IND is expected to be activated in early 2023 to enable initiation of the clinical phase of development. BMN 349 for alpha-1 antitrypsin deficiency: Preclinical studies have demonstrated that BMN 349 is an orally bioavailable, small molecule that is titratable with rapid onset and high potency and efficacy. Preclinical results have strong implications for potential improvement of current management, particularly for severe liver disease requiring rapid action. IND enabling studies are well-underway and BioMarin's goal is to file an IND for BMN 349 in the second half of 2023. BMN 293 (formerly DiNA-001) for MYBPC3 hypertrophic cardiomyopathy (HCM): Preclinical studies are underway with BMN 293 following a collaboration announced in 2020 with DiNAQOR, a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies. Mutations in MYBPC3 are the most common cause of inherited HCM. Early investigations suggest that gene therapy-mediated gene transfer can lead to widespread expression of the gene product, cardiac myosin-binding protein C (MyBP-C), in cardiac tissue, which can normalize cardiac hypertrophy, improve relaxation kinetics and potentially alleviate functional deficits in individuals suffering from cardiomyopathy. BioMarin's goal is to file an IND for BMN 293 in 2023. BioMarin will host a conference call and webcast to discuss third quarter and year to date 2022 financial results today, Wednesday, October 26, 2022 at 4:30 p.m. ET. This event can be accessed through this link or on the investor section of the BioMarin website at . About BioMarin BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare 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 seven commercial products and multiple clinical and preclinical product candidates for the treatment of various diseases. For additional information, please visit . Forward-Looking Statements This press release and the associated conference call and webcast contain forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the expectations of Total Revenues, Net Product Revenues, Research and Development Expense, Selling, General and Administrative Expense, Cost of Sales, GAAP Net Loss, Non-GAAP Income, and other specified income statement guidance for the full-year 2022; cash flows from operating activities; the timing of orders for commercial products; the timing of BioMarin's clinical development and commercial prospects, including announcements of data from clinical studies and trials; the clinical development and commercialization of BioMarin's product candidates and commercial products, including (i) the outcome of BioMarin's BLA resubmission for ROCTAVIAN to the FDA (as well as the outcome of the PMA application submitted for contemporaneous approval of the related CDx test), (ii) the results from clinical studies regarding product expansion opportunities for ROCTAVIAN, (iii) BioMarin's plans to submit supplemental marketing applications in the U.S. and EU to expand access to VOXZOGO for infants and young children up to five years of age with achondroplasia by the end of 2022, (v) BioMarin's anticipated IND activation for BMN 351 in early 2023, (vi) BioMarin's anticipated IND submission for BMN 349 in the second half of 2023, and (vii) BioMarin's goal to file an IND for BMN 293 in 2023; the potential approval and commercialization of BioMarin's product candidates, including ROCTAVIAN for the treatment of severe hemophilia A, and the timing of such approval decisions and product launches, including (i) the anticipated start of commercial sales of VOXZOGO in additional countries, and (ii) the duration of the FDA's review procedure of our BLA resubmission for ROCTAVIAN, including the possibility that the FDA determines more review time is necessary based on the number of data read-outs that will emerge during the procedure; and the expected benefits and availability of BioMarin's product candidates; the anticipated benefits of BioMarin's organizational redesign plan announced in October 2022; and potential growth opportunities and trends, including that BioMarin expects (i) double-digit growth in revenues and profitability in 2022, and (ii) increasing access to VOXZOGO as the product launch continues in future quarters, including BioMarin's expectation that the addition of Japan and other global markets will increase the pace of uptake in demand for VOXZOGO. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: BioMarin's success in the commercialization of its commercial products, including BioMarin's projected impact of the COVID-19 pandemic on its global revenue sources, including due to demand interruptions such as missed patient infusions and delayed treatment starts for new patients; results and timing of current and planned preclinical studies and clinical trials, as well as the potential impact of the COVID-19 pandemic on (i) BioMarin's ability to continue such preclinical studies and clinical trials and (ii) the timing of such preclinical studies and clinical trials, and the release of data from those trials; BioMarin's ability to successfully manufacture its commercial products and product candidates; the content and timing of decisions by the FDA, the European Commission and other regulatory authorities concerning each of the described products and product candidates, including the potential impact of the COVID-19 pandemic on the regulatory authorities' abilities to issue such decisions and the timing of such decisions; the market for each of these products; actual sales of BioMarin's commercial products and the impact that the COVID-19 pandemic may have on such sales; the introduction of generic versions of BioMarin's commercial products, in particular generic versions of KUVAN; successful implementation of BioMarin's organizational redesign plan announced in October 2022; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2022 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise. BioMarin®, BRI NEURA®, KUVAN®, NAGLAZYME®, PALYNZIQ®, VIMIZIM® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. ROCTAVIANTM is a trademark of BioMarin Pharmaceutical Inc. ALDURAZYME® is a registered trademark of BioMarin/Genzyme LLC. All other brand names and service marks, trademarks and other trade names appearing in this release are the property of their respective owners. Non-GAAP Information The results presented in this press release include both GAAP information and Non-GAAP information. As used in this release, Non-GAAP Income is defined by the Company as GAAP Net Income/Loss excluding net interest expense, provision for income taxes, depreciation expense, amortization expense, stock-based compensation expense, contingent consideration expense and, in certain periods, certain other specified items, as detailed below when applicable. In addition, BioMarin includes in this press release the effects of these adjustments on certain components of GAAP Net Income/Loss for each of the periods presented. In this regard, Non-GAAP Income and its components, including Non-GAAP Cost of Sales, Non-GAAP Research and Development expenses, Non-GAAP Selling, General and Administrative expense, Non-GAAP Intangible Asset Amortization and Contingent Consideration, Non-GAAP Gain on the Sale of Intangible Asset and Non-GAAP Benefit From Income Taxes are statement of operations line items prepared on the same basis as, and therefore components of, the overall Non-GAAP measures. BioMarin regularly uses both GAAP and Non-GAAP results and expectations internally to assess its financial operating performance and evaluate key business decisions related to its principal business activities: the discovery, development, manufacture, marketing and sale of innovative biologic therapies. Because Non-GAAP Income and its components are important internal measurements for BioMarin, the Company believes that providing this information in conjunction with BioMarin's GAAP information enhances investors' and analysts' ability to meaningfully compare the Company's results from period to period and to its forward-looking guidance, and to identify operating trends in the Company's principal business. BioMarin also uses Non-GAAP Income internally to understand, manage and evaluate its business and to make operating decisions, and compensation of executives is based in part on this measure. Non-GAAP Income and its components are not meant to be considered in isolation, as a substitute for, or superior to comparable GAAP measures and should be read in conjunction with the consolidated financial information prepared in accordance with GAAP. Investors should note that the Non-GAAP information is not prepared under any comprehensive set of accounting rules or principles and does not reflect all of the amounts associated with the Company's results of operations as determined in accordance with GAAP. Investors should also note that these Non-GAAP measures have no standardized meaning prescribed by GAAP and, therefore, have limits in their usefulness to investors. In addition, from time to time in the future there may be other items that the Company may exclude for purposes of its Non-GAAP measures; likewise, the Company may in the future cease to exclude items that it has historically excluded for purposes of its Non-GAAP measures. Because of the non-standardized definitions, the Non-GAAP measure as used by BioMarin in this press release and the accompanying tables may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies. The following table presents the reconciliation of GAAP Net Income to Non-GAAP Income: The following reconciliation of the GAAP reported to the Non-GAAP information provides the details of the effects of the Non-GAAP adjustments on certain components of the Company's operating results for each of the periods presented. SOURCE BioMarin Pharmaceutical Inc.