Atidarsagene autotemcel

Orchard Therapeutics Receives Swissmedic Approval for Libmeldy in Early-onset MLD

Retrieved on: 
Monday, December 11, 2023

BOSTON and LONDON, Dec. 11, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the Swiss Agency for Therapeutic Products (Swissmedic) has approved Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, for the treatment of early-onset metachromatic leukodystrophy (MLD).

Key Points: 
  • BOSTON and LONDON, Dec. 11, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the Swiss Agency for Therapeutic Products (Swissmedic) has approved Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, for the treatment of early-onset metachromatic leukodystrophy (MLD).
  • Libmeldy aims to correct the underlying genetic cause of MLD by inserting a working copy of the ARSA gene into the genome of a patients’ own HSCs.
  • With more than a cumulative 250 patient-years of follow-up, Libmeldy was generally well-tolerated, with no treatment-related serious adverse events or deaths.
  • “Today’s approval of Libmeldy in Switzerland opens up tremendous new possibilities for eligible children with MLD who previously had no approved treatment options beyond supportive care,” said Leslie Meltzer, Ph.D., chief medical officer of Orchard Therapeutics.

Orchard Therapeutics Reports Third Quarter 2023 Financial Results and Highlights Recent Business Accomplishments

Retrieved on: 
Monday, November 13, 2023

Biologics License Application for OTL-200 in MLD accepted by U.S. FDA under Priority Review; PDUFA date set for March 18, 2024

Key Points: 
  • Biologics License Application for OTL-200 in MLD accepted by U.S. FDA under Priority Review; PDUFA date set for March 18, 2024
    BOSTON and LONDON, Nov. 13, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced several business accomplishments along with its financial results for the quarter ended September 30, 2023.
  • Following the completion of the acquisition, Orchard Therapeutics will become a wholly-owned subsidiary of Kyowa Kirin.
  • The company had approximately 227.3 million ordinary shares, equivalent to 22.7 million American Depositary Shares, outstanding as of September 30, 2023.
  • As a result of the anticipated acquisition by Kyowa Kirin, Orchard Therapeutics has removed its financial guidance around Libmeldy revenue and anticipated cash runway.

Kyowa Kirin to Acquire Orchard Therapeutics

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Thursday, October 5, 2023

TOKYO and LONDON and BOSTON, Oct. 05, 2023 (GLOBE NEWSWIRE) -- Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151), a Japan-based global specialty pharmaceutical company (J-GSP) creating innovative medical solutions utilizing the latest biotechnology, and Orchard Therapeutics plc (Orchard Therapeutics, Nasdaq: ORTX), a global gene therapy leader, today announced the companies have entered into a definitive agreement under which Kyowa Kirin will acquire Orchard Therapeutics for $16.00 per American Depositary Share (ADS) in cash (approximately $387.4 million, or ¥57.3 billion), under which Orchard shareholders will hold an additional contingent value right (CVR) of $1.00 per ADS. An additional $1.00 CVR will be paid for a total of $17.00 per ADS, or approximately $477.6 million (¥70.7 billion) if the conditions are met.

Key Points: 
  • Kyowa Kirin has established a 2030 Vision to consistently create and deliver medicines with life-changing value that ultimately makes people smile, as a J-GSP.
  • “We are excited to announce that we have signed the Transaction Agreement to acquire Orchard Therapeutics, one of the leading providers of HSC gene therapy,” said Takeyoshi Yamashita, Ph.D., Director of the Board, chief medical officer, senior managing executive officer of Kyowa Kirin.
  • Under the terms of the agreement, Kyowa Kirin will initiate a scheme of arrangement to acquire all Orchard Therapeutics’ ADSs at a price of $16.00 per ADS in cash (or aggregated value of approximately $387.4 million, or approximately ¥57.3 billion) at closing, which represents a premium of 144% to Orchard Therapeutics’ volume-weighted average price per ADS over the previous 30 days ended October 4.
  • Following the completion of the acquisition, Orchard Therapeutics will become a wholly-owned subsidiary of Kyowa Kirin.

Orchard Therapeutics Announces Second Closing of Strategic Financing, Resulting in $34 Million of Additional Capital

Retrieved on: 
Monday, June 26, 2023

BOSTON and LONDON, June 26, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that it has completed the second closing of its previously announced securities purchase agreement, resulting in $34 million of new capital before placement agent and transaction fees.

Key Points: 
  • In March 2023, the company completed the first closing of an innovative financing arrangement , led by RA Capital Management with participation from other leading healthcare investors that resulted in $34 million of new capital before placement agent and transaction fees.
  • The second closing comprises the sale of units at a purchase price of $8.00 per unit for aggregate proceeds of $34 million, including the issuance of 4.25 million American Depository Shares (ADSs) as well as warrants.
  • “The initiation of our BLA submission marks a significant de-risking event for Orchard and our OTL-200 program ahead of a potential U.S. approval in MLD,” said Frank Thomas, president and chief operating officer of Orchard Therapeutics.
  • Any offering of the securities under the resale registration statement will only be by means of a prospectus.

Orchard Therapeutics Announces Positive Clinical and Preclinical Data in Programs Targeting Neurometabolic and CNS Disorders at ASGCT

Retrieved on: 
Friday, May 19, 2023

BOSTON and LONDON, May 19, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced multiple clinical and preclinical updates from its portfolio of investigational hematopoietic stem cell (HSC) gene therapies in neurometabolic and neurodegenerative disorders. The data are being featured in several oral presentations at the ongoing 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Los Angeles.

Key Points: 
  • The data are being featured in several oral presentations at the ongoing 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Los Angeles.
  • At baseline, most patients presented with severe joint range of motion impairment, severe acetabular (hip) dysplasia and varying degrees of dorso-lumbar kyphosis.
  • Treatment with OTL-203 was generally well tolerated and demonstrated extensive metabolic correction over four years after treatment.
  • Data from in vivo studies indicate effective GRN protein delivery to the CNS of knockout mice transplanted with gene-modified HSCs.

Orchard Therapeutics Reports First Quarter 2023 Financial Results and Announces Initiation of Rolling Submission for Biologics License Application of OTL-200 for Metachromatic Leukodystrophy to U.S. FDA

Retrieved on: 
Monday, May 15, 2023

BOSTON and LONDON, May 15, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced various business accomplishments along with its financial results for the quarter ended March 31, 2023.

Key Points: 
  • OTL-200 is approved as Libmeldy® (atidarsagene autotemcel) by the European Commission (EC) and Medicines and Healthcare products Regulatory Agency (MHRA).
  • The company has secured reimbursement agreements for Libmeldy in three new markets so far in 2023.
  • Orchard Therapeutics has outlined the following key milestones expected for 2023:
    Libmeldy: Establish qualified treatment centers in Sweden, Spain and Saudi Arabia.
  • The company had approximately 184.3 million ordinary shares, equivalent to 18.4 million American Depositary Shares, outstanding as of March 31, 2023.

Orchard Therapeutics Provides Business Update and Reports 2022 Financial Results

Retrieved on: 
Monday, March 6, 2023

ET

Key Points: 
  • ET
    BOSTON and LONDON, March 06, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced business updates along with its financial results for the quarter and year ended December 31, 2022.
  • Based on the outcome of the Type B meeting, Orchard Therapeutics subsequently requested a pre-BLA meeting which has been scheduled for the second quarter of 2023.
  • Management believes this non-GAAP information is useful for investors, taken in conjunction with Orchard’s GAAP financial statements, because it provides greater transparency regarding Orchard’s operating performance.
  • Management uses these measures, among others, to assess and analyze operational results and trends and to make financial and operational decisions.

Orchard Therapeutics Announces Agreement Enabling Reimbursed Access to Libmeldy for All Eligible MLD Patients in Sweden

Retrieved on: 
Monday, February 27, 2023

Libmeldy received the strongest possible recommendation for use by the NT Council following the completion of a health economic evaluation by FINOSE, a health technology assessment consortium between Finland, Norway and Sweden.

Key Points: 
  • Libmeldy received the strongest possible recommendation for use by the NT Council following the completion of a health economic evaluation by FINOSE, a health technology assessment consortium between Finland, Norway and Sweden.
  • “We are encouraged that the reimbursement agreements secured in multiple European countries to date continue to recognize the value of Libmeldy commensurate with its clinical impact.
  • The agreement with NT Council is a positive step toward enabling access for MLD patients in other Nordic countries.
  • We look forward to continuing to work with reimbursement authorities in the Nordic region and throughout Europe to make Libmeldy available to patients who may benefit from treatment.”