Medicines and Healthcare products Regulatory Agency

Adverum Biotechnologies Reports First Quarter 2023 Financial Results

Retrieved on: 
Thursday, May 11, 2023
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REDWOOD CITY, Calif., May 11, 2023 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases, today reported financial results for the first quarter ended March 31, 2023 and provided an update on its ongoing Phase 2 LUNA trial evaluating ixoberogene soroparvovec (Ixo-vec, formerly referred to as ADVM-022) for the treatment of wet age-related macular degeneration (wet AMD).

Key Points: 
  • We are on track to finish enrolling the study in the second half of 2023,” said Star Seyedkazemi, PharmD, chief development officer of Adverum Biotechnologies.
  • “We plan to provide interim datasets for a percentage of participants at 14 weeks, including aflibercept protein levels, in the third quarter of 2023, as well as preliminary efficacy and safety data in the fourth quarter of 2023.
  • Stock-based compensation expense included in research and development expenses was $1.4 million for the first quarter of 2023.
  • Stock-based compensation expense included in general and administrative expenses was $3.2 million for the first quarter of 2023.

Catalyst Pharmaceuticals Reports Record First Quarter 2023 Results, Achieving 98% Total Revenue Growth Year-over-Year

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Wednesday, May 10, 2023
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Achieved Q1 2023 FIRDAPSE® net product revenue of $57.5 Million, representing a substantial 34% YoY increase.

Key Points: 
  • Achieved Q1 2023 FIRDAPSE® net product revenue of $57.5 Million, representing a substantial 34% YoY increase.
  • Research and Development Expenses: Research and development expenses in the first quarter of 2023 were $3.6 Million, compared to $3.4 Million in the first quarter of 2022.
  • Amortization of Intangible Assets: Amortization of intangible assets was $6.5 Million in the first quarter of 2023, compared to $0.00 in the first quarter of 2022.
  • 2023 Financial Guidance: The Company forecasts full-year 2023 total revenues, including FYCOMPA®, to be between $375 Million and $385 Million, representing a 75% - 80% increase in total revenues compared to 2022.

Arcturus Therapeutics Announces First Quarter 2023 Financial Update and Pipeline Progress

Retrieved on: 
Tuesday, May 9, 2023
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We believe this meaningful milestone is indicative of the broader platform opportunity for our mRNA medicine technologies to result in novel vaccines and therapeutics over the coming years,” said Joseph Payne, President & CEO of Arcturus Therapeutics.

Key Points: 
  • We believe this meaningful milestone is indicative of the broader platform opportunity for our mRNA medicine technologies to result in novel vaccines and therapeutics over the coming years,” said Joseph Payne, President & CEO of Arcturus Therapeutics.
  • Arcturus is amending the protocol to allow the dosing of patients with CF and expects to initiate the enrollment in Q3 2023.
  • Additionally, we expect to collect $90.0 million in the second quarter of 2023, associated with the CSL Seqirus milestones which are in accounts receivable on March 31, 2023.
  • The cash runway remains extended through the beginning of 2026 based on the current pipeline and programs.

Lisata Therapeutics Reports First Quarter 2023 Financial Results and Provides Business Update

Retrieved on: 
Tuesday, May 9, 2023
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BASKING RIDGE, N. J., May 09, 2023 (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc. (Nasdaq: LSTA) (“Lisata” or the “Company”), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, provides a business update and reports financial results for the three months ended March 31, 2023.

Key Points: 
  • Conference call scheduled for today at 4:30 p.m. Eastern time
    BASKING RIDGE, N. J., May 09, 2023 (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc. (Nasdaq: LSTA) (“Lisata” or the “Company”), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, provides a business update and reports financial results for the three months ended March 31, 2023.
  • LSTA1 also has the potential to modify the tumor microenvironment, with the objective of making tumors more susceptible to immunotherapies.
  • The combination of LSTA1 with corresponding standards-of-care in other solid tumor indications is planned for clinical study commencing before the end of the second quarter of 2023.
  • Lisata will hold a live conference call today, May 9, 2023, at 4:30 p.m. Eastern time to discuss financial results, provide a business update and answer questions.

EQRx Resets to Focus on Clinically Differentiated Medicines, Leveraging $1.3 Billion Cash Position; Reports First Quarter 2023 Financial Results

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Monday, May 8, 2023
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ET

Key Points: 
  • ET
    CAMBRIDGE, Mass., May 08, 2023 (GLOBE NEWSWIRE) -- EQRx, Inc. (Nasdaq: EQRX), today announced plans to reset its business and reported financial results for the first quarter ended March 31, 2023.
  • “Going forward, EQRx will leverage its significant scale of capital and team of experienced ‘drug hunters’ towards developing clinically differentiated, high-value medicines,” said Melanie Nallicheri, president and chief executive officer of EQRx.
  • Cash Position: Cash, cash equivalents and short-term investments totaled $1.3 billion as of March 31, 2023.
  • EQRx estimates its year-end cash, cash equivalents and short-term investments position will be approximately $1.1 billion.

Aurinia Pharmaceuticals Announces NICE Recommendation of LUPKYNIS® (Voclosporin) For Adults with Active Lupus Nephritis

Retrieved on: 
Wednesday, May 3, 2023
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The NICE recommendation applies to England and Wales and follows the recent Medicines and Healthcare Products Regulatory Agency (MHRA) authorization of voclosporin licensed in Great Britain for the treatment of active LN in adult patients.

Key Points: 
  • The NICE recommendation applies to England and Wales and follows the recent Medicines and Healthcare Products Regulatory Agency (MHRA) authorization of voclosporin licensed in Great Britain for the treatment of active LN in adult patients.
  • “We are extremely pleased with the NICE recommendation of LUPKYNIS for patients with lupus nephritis.
  • This recommendation follows the earlier approval from the MHRA and further supports access to an important treatment option for those patients.
  • The safety profile of voclosporin and MMF and low-dose corticosteroids was generally comparable to MMF and low-dose corticosteroids alone.

Thorne HealthTech Wins 2023 MedTech Breakthrough Award for Best Overall Medical Device Product for Thorne's OneDraw® Blood Collection Device

Retrieved on: 
Wednesday, May 3, 2023
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NEW YORK, May 3, 2023 /PRNewswire/ -- Thorne HealthTech, Inc. ("Thorne") (NASDAQ: THRN), a leader in delivering innovative solutions for a personalized approach to health and wellness, announced today that its OneDraw® Blood Collection Device has been selected as the Best Overall Medical Device Product in the eighth annual MedTech Breakthrough Awards program.

Key Points: 
  • NEW YORK, May 3, 2023 /PRNewswire/ -- Thorne HealthTech, Inc. ("Thorne") (NASDAQ: THRN), a leader in delivering innovative solutions for a personalized approach to health and wellness, announced today that its OneDraw® Blood Collection Device has been selected as the Best Overall Medical Device Product in the eighth annual MedTech Breakthrough Awards program.
  • MedTech Breakthrough is an independent market intelligence organization that recognizes the "breakthrough" companies, individuals, platforms, and products in today's health, fitness, and medical technology industries.
  • Thorne's OneDraw Blood Collection Device is a single-use system that revolutionizes the collection, stabilization, and transport of whole blood samples, allowing for remote sample collection anytime and anywhere in a convenient and comfortable way.
  • "Thorne's OneDraw device is a true breakthrough in the medical device industry, and we are thrilled to recognize them as the Best Overall Medical Device Product in the 2023 MedTech Breakthrough Awards program," said James Johnson, Managing Director, MedTech Breakthrough.

Horizon Therapeutics plc Announces Initiation of Phase 3 Clinical Trial in Japan Evaluating TEPEZZA® (teprotumumab-trbw) in Adults with Chronic/Low Clinical Activity Score (CAS) Thyroid Eye Disease (TED)

Retrieved on: 
Monday, May 1, 2023
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Horizon Therapeutics plc (Nasdaq: HZNP) today announced that the first patient has been enrolled in a Phase 3 clinical trial in Japan evaluating TEPEZZA for the treatment of adults with chronic Thyroid Eye Disease (TED) and low clinical activity score (CAS), which is a measure of disease activity.

Key Points: 
  • Horizon Therapeutics plc (Nasdaq: HZNP) today announced that the first patient has been enrolled in a Phase 3 clinical trial in Japan evaluating TEPEZZA for the treatment of adults with chronic Thyroid Eye Disease (TED) and low clinical activity score (CAS), which is a measure of disease activity.
  • The trial includes patients with an initial diagnosis of TED between two to 10 years prior to the study and low levels of disease activity.
  • Findings from this study will build on a separate Phase 3 clinical trial (OPTIC-J) that is currently underway in Japan and is evaluating TEPEZZA for the treatment of TED patients with disease duration of nine months or less and higher levels of disease activity.
  • More information about the trial, including eligibility criteria, is available on the Japan Registry of Clinical Trials website (trial ID number jRCT2031220730 ).

Rockwell Medical's Partner Drogsan Pharmaceuticals Submits Marketing Authorization Application in Turkey for Triferic AVNU

Retrieved on: 
Thursday, April 27, 2023
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Triferic AVNU (ferric pyrophosphate citrate injection) is an iron replacement product indicated to maintain hemoglobin in adult patients undergoing hemodialysis.

Key Points: 
  • Triferic AVNU (ferric pyrophosphate citrate injection) is an iron replacement product indicated to maintain hemoglobin in adult patients undergoing hemodialysis.
  • In June 2021, Rockwell entered into license and supply agreements with Drogsan, one of the leading pharmaceutical companies in Turkey, for the rights to commercialize Triferic (dialysate) and Triferic AVNU in Turkey.
  • Under the terms of the agreements, Drogsan will be the exclusive commercialization partner for Triferic (dialysate) and Triferic AVNU in Turkey.
  • "Taking into consideration that Drogsan was granted an accelerated review for Triferic AVNU with the Turkish regulatory authority, we anticipate approval for Triferic AVNU in 2024."

Vertex Announces U.S. FDA Approval for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 2 Through 5 With Certain Mutations

Retrieved on: 
Wednesday, April 26, 2023
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“Since its initial approval, TRIKAFTA has had a significant impact on the CF community, transforming the lives of thousands of people living with cystic fibrosis,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex.

Key Points: 
  • “Since its initial approval, TRIKAFTA has had a significant impact on the CF community, transforming the lives of thousands of people living with cystic fibrosis,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex.
  • The data from this study were recently published in the American Journal of Respiratory and Critical Care Medicine .
  • Additionally, Vertex has submitted applications for the use of TRIKAFTA®/KAFTRIO® in children ages 2 through 5 years of age to other global regulatory authorities, including the European Medicines Agency (EMA) and the Medicines and Healthcare Products Regulatory Agency (MHRA).
  • For more information on TRIKAFTA®, patient assistance programs or to find additional eligibility details, visit TRIKAFTA.com , VertexGPS.com or vertextreatments.com .