Leadiant Biosciences, Inc.

DUBLIN, Oct. 2, 2023 /PRNewswire/ -- The "Enzyme Replacement Therapy Global Market Report 2023" report has been added to ResearchAndMarkets.com's offering. The global enzyme replacement therapy market is expected to grow from $10.03 billion in 2022 to $10.85 billion in 2023 at a compound annual growth rate (CAGR) of 8.1%. The market is expected to reach $14.79 billion in 2027 at a CAGR of 8.1%. In the forefront of medical advancement, the enzyme replacement therapy (ERT) market stands tall, offering vital treatment avenues for individuals grappling with chronic disorders arising from enzyme deficiencies. The research report delves deep into the nuances of the ERT sector, highlighting market statistics, regional shares, and key players commanding significant market share. ERT's pivotal role in normalizing urinary GAG levels and promoting liver and spleen health is becoming increasingly recognized. Primary enzymes like agalsidase alfa - a critical tool in the combat against Fabry's disease - are reshaping the therapeutic landscape. Our comprehensive report also categorizes other prominent enzymes used, shedding light on their applications in conditions such as Gaucher disease, Pompe disease, and more. A spotlight trend in the ERT realm is the surge in product approvals. For instance, the recent FDA nod to Lamzede (velmanase alfa) in February 2023 marked a significant milestone. As the inaugural enzyme replacement therapy greenlit in the U.S for tackling the non-central neurological manifestations of alpha-mannosidosis, Lamzede offers a promising beacon of hope. Moreover, with the escalating prevalence of rare, chronic, and genetic disorders, the ERT market trajectory looks set for robust growth. The U.S National Library of Medicine projects a near-doubling of individuals aged 50 and above with a chronic ailment by 2050, further emphasizing the critical need for effective enzyme replacement therapies. The report also touches upon the market revenues, detailing contributions from key treatments like Sebelipase alfa, Elosulfase alfa, and more. Our exhaustive coverage ensures businesses and stakeholders are well-armed with insights to navigate the dynamic ERT ecosystem. In a geographical context, while North America reigned supreme in the ERT domain in 2022, the forecasted period paints a promising picture for the Asia-Pacific region. Our study covers a broad spectrum of nations from Australia to the USA, offering a global perspective on this burgeoning market. Harness the power of data-driven insights, industry trends, and strategic opportunities with our comprehensive report, and be poised at the cusp of ERT innovations and advancements. Major players in the enzyme replacement therapy market are Biomarin Pharmaceutical Inc. Leadiant Biosciences Inc. Pfizer Inc. Sanofi S.A. AbbVie Inc. Takeda Pharmaceutical Company Limited Protalix BioTherapeutics Inc. Amicus Therapeutics Inc. Johnson & Johnson Services Inc. Digestive Care Inc. Nestle Health Science Alexion Pharmaceuticals Inc. Allergan Plc. Actelion Pharmaceuticals Ltd. CANbridge Life Sciences Ltd. Chiesi Farmaceutici S.P.A. Astrazeneca Plc. Genzyme Corporation JCR Pharmaceutical Co. Ltd. TEIJIN LIMITED Horizon Therapeutics Plc. Perseo Pharma AG SmartPharm Therapeutics Inc. Key Topics Covered: 1. Executive Summary 2. Enzyme Replacement Therapy Market Characteristics 3. Enzyme Replacement Therapy Market Trends And Strategies 4. Enzyme Replacement Therapy Market - Macro Economic Scenario 4.1. COVID-19 Impact On Enzyme Replacement Therapy Market 4.2. Ukraine-Russia War Impact On Enzyme Replacement Therapy Market 4.3. Impact Of High Inflation On Enzyme Replacement Therapy Market 5. Enzyme Replacement Therapy Market Size And Growth 5.1. Global Enzyme Replacement Therapy Historic Market, 2017-2022, $ Billion 5.1.1. Drivers Of The Market 5.1.2. Restraints On The Market 5.2. Global Enzyme Replacement Therapy Forecast Market, 2022-2027F, 2032F, $ Billion 5.2.1. Drivers Of The Market 5.2.2. Restraints On the Market 6. Enzyme Replacement Therapy Market Segmentation 6.1. Global Enzyme Replacement Therapy Market, Segmentation By Enzyme Type, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion Agalsidase Alfa Agalsidase Beta Galsulfase Other Enzyme Types 6.2. Global Enzyme Replacement Therapy Market, Segmentation By Application, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion Gaucher Disease Pompe Disease Fabry Disease Other Applications 6.3. Global Enzyme Replacement Therapy Market, Segmentation By Route Of Administration, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion Oral Parenteral 6.4. Global Enzyme Replacement Therapy Market, Segmentation By End-Users, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion Hospitals Infusion Centers Other End-Users 7. Enzyme Replacement Therapy Market Regional And Country Analysis 7.1. Global Enzyme Replacement Therapy Market, Split By Region, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion 7.2. Global Enzyme Replacement Therapy Market, Split By Country, Historic and Forecast, 2017-2022, 2022-2027F, 2032F, $ Billion For more information about this report visit About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. Media Contact: Research and Markets Laura Wood, Senior Manager [email protected] For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900 U.S. Fax: 646-607-1904 Fax (outside U.S.): +353-1-481-1716 Logo: SOURCE Research and Markets

LA JOLLA, Calif., May 23, 2023 (GLOBE NEWSWIRE) -- CalciMedica Inc. (“CalciMedica”) (OTCQB: CALC), a clinical-stage biopharmaceutical company focused on developing therapies for life-threatening inflammatory diseases with high unmet need, today announced the appointments of Raven Jaeger, M.S., as Chief Regulatory Officer and Andrew Cunningham, M.D., MRCPI, as Senior Vice President of Clinical Development. Both Mrs. Jaeger and Dr. Cunningham bring extensive expertise to their positions on the CalciMedica team. Mrs. Jaeger has over 20 years of experience in the pharmaceutical and biotech industries as a Regulatory Affairs professional in both investigative and commercialized drugs and biologics across a variety of therapeutic areas. Most recently, she served as the Chief Regulatory Officer at Diffusion Pharmaceuticals, Inc. where she supported the product development program and various business initiatives. Prior to this, Mrs. Jaeger held Regulatory Affairs positions at other leading pharmaceutical companies including BridgeBio Pharma, Leadiant BioSciences, Inc. and Nabi Biopharmaceuticals. While at BridgeBio Pharma, she led her team to the first FDA approved New Drug Application across all subsidiaries of the organization. “I am excited to join the CalciMedica team and to build on the clinical progress of Auxora in multiple indications by spearheading regulatory strategy,” said Mrs. Jaeger. “Throughout my career, I have been fortunate to play important roles in the advancement of multiple therapies through the clinic and to regulatory approval, and I look forward to applying my knowledge and experience to the CalciMedica pipeline.” Dr. Cunningham has over 10 years of clinical development experience in the pharmaceutical industry. Prior to this position, Dr. Cunningham served as a consultant for CalciMedica since December 2022. He has valuable leadership and clinical development experience, having served as Head of the Neurodegenerative Franchise at Taysha Gene Therapies and Associate Vice President of Clinical Development at Reata Pharmaceuticals. In his prior Medical Director positions at Takeda Pharmaceutical Company, Sanofi Genzyme and Reata Pharmaceuticals, Dr. Cunningham helped advance drug candidates through all stages of clinical development as well as participated in the filing of New Drug Applications. “I was impressed with Auxora’s potential to treat inflammatory diseases which currently have no approved therapies,” said Dr. Cunningham. “I look forward to applying my skills and experience in clinical development as we continue progress in the Phase 2b CARPO trial for acute pancreatitis and advance our pipeline by expanding the Phase 1/2 CRSPA trial in asparaginase-associated pancreatitis, further investigating Auxora in acute kidney injury and exploring new oral agents for chronic pancreatitis and other chronic inflammatory indications.” “Following a successful merger, these appointments underscore CalciMedica’s commitment to grow and expand our clinical programs. We are delighted to welcome Raven and Andrew to the CalciMedica team,” said Rachel Leheny, Ph.D., Chief Executive Officer of CalciMedica. “In this new phase as a public company, CalciMedica is equipped to advance our mission to develop therapies for critically ill patients whose needs are unmet by the current therapies.” About CalciMedica CalciMedica is a clinical-stage biopharmaceutical company focused on developing therapies for life-threatening inflammatory diseases with high unmet need. CalciMedica’s proprietary technology targets the inhibition of CRAC channels designed to modulate the immune response and protect against tissue cell injury, with the potential to provide therapeutic benefits in life-threatening inflammatory diseases for which there are currently no approved therapies. CalciMedica’s lead product candidate Auxora, a proprietary, intravenous-formulated CRAC channel inhibitor, has demonstrated positive and consistent clinical results in four completed efficacy clinical trials. Auxora is in development for acute pancreatitis with systemic inflammatory response syndrome and asparaginase-associated pancreatitis. CalciMedica was founded by scientists from Torrey Pines Therapeutics and the Harvard CBR Institute for Biomedical Research, and is headquartered in La Jolla, CA. For more information, please visit . Forward-Looking Statements This communication contains forward-looking statements which include, but are not limited to, statements regarding the design and potential benefits of Auxora; CalciMedica’s ongoing and planned clinical trials; and the development and outcomes of CARPO and CRSPA trial programs. These forward-looking statements are subject to the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. CalciMedica’s expectations and beliefs regarding these matters may not materialize. Actual outcomes and results may differ materially from those contemplated by these forward-looking statements as a result of uncertainties, risks, and changes in circumstances, including but not limited to risks and uncertainties related to: the impact of fluctuations in global financial markets on CalciMedica’s business and the actions it may take in response thereto; CalciMedica’s ability to execute its plans and strategies; the ability to obtain and maintain regulatory approval for Auxora; results from clinical trials may not be indicative of results that may be observed in the future; potential safety and other complications from Auxora; the scope progress and expansion of developing and commercializing Auxora; the size and growth of the market therefor and the rate and degree of market acceptance thereof; economic, business, competitive, and/or regulatory factors affecting the business of CalciMedica generally; CalciMedica’s ability to protect its intellectual property position; and the impact of government laws and regulations. Additional risks and uncertainties that could cause actual outcomes and results to differ materially from those contemplated by the forward-looking statements are included under the caption “Risk Factors” in CalciMedica’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2023 and elsewhere in CalciMedica’s subsequent reports on Form 10-K, Form 10-Q or Form 8-K filed with the SEC from time to time and available at These documents can be accessed on CalciMedica’s web page at ir.calcimedica.com/financials-filings/sec-filings. CalciMedica Contact: Investors and Media Argot Partners Sarah Sutton/Kevin Murphy calcimedica@argotpartners.com (212) 600-1902

A company that has been under the microscope for drug pricing before has found itself fined by Spanish regulators. Leadiant Biosciences has been fined €10.3 million ($10.6 million) by the Spanish regulators Comisión Nacional de Los Mercados y la Competencia (CNMC), after a massive price hike of its chenodeoxycholic acid (CDCA) compound, which is used to treat the rare disease cerebrotendinous xanthomatosis (CTX). According to the CNMC, there are around 200 to 250 patients diagnosed with the disease in Europe, around 50 of whom live in Spain. Leadiant has also been the sole supplier of medicines with CDCA in the country since 2010, first with a drug named Xenbilox and then in 2017 with CDCA-Leadiant. However, the regulators are claiming that the company had charged Spain’s National Health System 14 times the price of another nearly identical medicine it had marketed in the country in 2010, costing €984 ($1,019) per package in September of 2010 and increasing to €14,618 ($15,146) per package in 2017. The investigation began with a complaint from the Organization of Consumers and Users for the price increase, with the organization noting that the price of the drug was 1,000 times higher than in 2008. Italian and Dutch regulators also initiated proceedings against Leadiant over the price shift. “In order to achieve market exclusivity, Leadiant conducted a series of actions combining lawful behavior—such as obtaining orphan drug designation with the corresponding marketing authorisation—and anti-competitive practices. Among these anti-competitive practices, it maintained an exclusivity clause with the only supplier of the active ingredient authorised to supply CDCA in sufficient quantities and quality. This prevented the emergence of alternative medicines, both industrial and in the form of magistral formulations,” the CNMC’s release stated. On top of the fine, Leadiant must comply with a series of obligations to eliminate the “excessive control” of the active ingredient in Spain and to market the drug at a price negotiated with the Ministry of Health. Endpoints News reached out to Leadiant for comment on the matter but did not receive a response by press time. Another Shkreli-like drug price controversy? Leadiant finds itself in the spotlight after 500-fold price hike — FT Regulators have had eyes on Leadiant for a while as the company gained control of the drug, won European approval and increased the price from €300 a year to more than €150,000, according to the Financial Times in 2018.