EHA

Ryvu Therapeutics Reports 2023 Half-Year Financial Results and Provides Corporate Update

Retrieved on: 
Wednesday, September 13, 2023
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Updated clinical and preclinical data on RVU120 were presented at the European Hematology Associated (EHA) Congress in June 2023.

Key Points: 
  • Updated clinical and preclinical data on RVU120 were presented at the European Hematology Associated (EHA) Congress in June 2023.
  • $14M in non-dilutive grant funding was secured from the Medical Research Agency (ABM) – the largest grant obtained to date by Ryvu.
  • KRAKOW, Poland, Sept. 13, 2023 (GLOBE NEWSWIRE) -- Ryvu Therapeutics (WSE: RVU), a clinical-stage drug discovery and development company focusing on novel small molecule therapies that address emerging targets in oncology, reported today financial results for the first half of 2023 and provided a corporate update.
  • “The last months of 2023 were a productive period for Ryvu as we made significant developments across our clinical programs, collaboration activity, and strengthening our balance sheet”, said Pawel Przewiezlikowski, CEO of Ryvu Therapeutics.

X4 Pharmaceuticals Announces Submission of New Drug Application (NDA) to U.S. FDA for Mavorixafor in WHIM Syndrome

Retrieved on: 
Tuesday, September 5, 2023
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“The submission of our first NDA is a significant milestone in X4's journey to transform the care of those living with rare immunodeficiencies,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals.

Key Points: 
  • “The submission of our first NDA is a significant milestone in X4's journey to transform the care of those living with rare immunodeficiencies,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals.
  • “We’re excited that this submission moves us one step closer to introducing what could be the first approved product in the U.S. for those with WHIM syndrome.
  • X4 has requested priority review for the application which, if granted, would provide a target FDA review period of six months from the application acceptance for filing date.
  • The NDA submission is supported by the results of the global, pivotal, 4WHIM Phase 3 clinical trial of once-daily, oral mavorixafor in individuals with WHIM syndrome.

U.S. FDA Approves Bristol Myers Squibb’s Reblozyl® (luspatercept-aamt) as First-Line Treatment of Anemia in Adults with Lower-Risk Myelodysplastic Syndromes (MDS) Who May Require Transfusions

Retrieved on: 
Tuesday, August 29, 2023
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These results underscore Reblozyl’s ability to address chronic anemia earlier in the treatment journey in a broader range of patients.

Key Points: 
  • These results underscore Reblozyl’s ability to address chronic anemia earlier in the treatment journey in a broader range of patients.
  • HI-E increase of at least 8 weeks was achieved by 74.1% (n=109) of Reblozyl patients vs. 51.3% (n=79) of epoetin alfa patients (p
  • RBC-TI of at least 12 weeks was achieved by 66.7% (n=98) of Reblozyl patients vs. 46.1% (n=71) of epoetin alfa patients (p=0.0003).
  • Patients treated with Reblozyl demonstrated durable responses with nearly 2.5 years of median RBC-TI ≥12 weeks (126.6 weeks, week 1 to end of treatment).

Gracell Biotechnologies Reports Second Quarter 2023 Unaudited Financial Results and Provides Corporate Update

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Monday, August 14, 2023
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ET today

Key Points: 
  • ET today
    SAN DIEGO and SUZHOU, China and SHANGHAI, China, Aug. 14, 2023 (GLOBE NEWSWIRE) -- Gracell Biotechnologies Inc. (NASDAQ: GRCL) (“Gracell” or the “Company”), a global clinical-stage biopharmaceutical company dedicated to developing innovative and highly efficacious cell therapies for the treatment of cancer and autoimmune diseases, today reported second quarter unaudited financial results for the period ended June 30, 2023, and provided corporate updates.
  • “We are delighted with the significant milestones achieved in the past few months across our reprioritized pipeline.
  • The Phase 1b part of the RRMM US IND trial has commenced as patient screening is underway.
  • Financial Results for Second Quarter Ended June 30, 2023
    As of June 30, 2023, the Company had RMB1,188.0 million (US$163.8 million) in cash and cash equivalents and short-term investments.

Legend Biotech Reports Second Quarter 2023 Results and Recent Highlights

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Tuesday, August 15, 2023
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Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global biotechnology company developing, manufacturing and commercializing novel therapies to treat life-threatening diseases, today reported its unaudited financial results for the three and six months ended June 30, 2023.

Key Points: 
  • Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global biotechnology company developing, manufacturing and commercializing novel therapies to treat life-threatening diseases, today reported its unaudited financial results for the three and six months ended June 30, 2023.
  • "We remain committed to exploring the full potential of CARVYKTI® and are pleased with the continued growth of our development program, including two regulatory submissions made during the second quarter," said Ying Huang, Chief Executive Officer of Legend Biotech.
  • License revenue for the six months ended June 30, 2023 was $15.1 million, compared to $50 million for the six months ended June 30, 2022.
  • Collaboration revenue for the three and six months ended June 30, 2023 was $58.2 million and $94.4 million, respectively, compared to $11.9 million for the three and six months ended June 30, 2022.

2seventy bio Reports Second Quarter Financial Results and Recent Operational Progress

Retrieved on: 
Monday, August 14, 2023
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(Nasdaq: TSVT), a leading immuno-oncology cell therapy company, today reported financial results and recent highlights for the second quarter ended June 30, 2023.

Key Points: 
  • (Nasdaq: TSVT), a leading immuno-oncology cell therapy company, today reported financial results and recent highlights for the second quarter ended June 30, 2023.
  • The Company ended the second quarter of 2023 with cash, cash equivalents and marketable securities of $307 million.
  • 2seventy bio believes that this cash position, combined with growing Abecma cashflow and disciplined expense management, provides financial runway into 2026.
  • ET to discuss 2Q 2023 financial results and recent business highlights.

Servier Announces FDA Filing Acceptance and Priority Review for TIBSOVO® (ivosidenib tablets) in the Treatment of IDH1-mutated Relapsed or Refractory (R/R) Myelodysplastic Syndromes (MDS)

Retrieved on: 
Tuesday, August 15, 2023
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BOSTON, Aug. 15, 2023 /PRNewswire/ -- Servier, a leader in oncology committed to bringing the promise of tomorrow to the patients we serve, today announced the U.S. Food and Drug Administration (FDA) has accepted a supplemental New Drug Application (sNDA) and granted Priority Review for TIBSOVO® (ivosidenib tablets) in the treatment of patients with isocitrate dehydrogenase 1 (IDH1)-mutated relapsed or refractory (R/R) myelodysplastic syndromes (MDS). If approved, TIBSOVO would be a first-in-class targeted therapy option for MDS patients within this molecularly defined subset.

Key Points: 
  • If approved, TIBSOVO would be a first-in-class targeted therapy option for MDS patients within this molecularly defined subset.
  • "This filing acceptance and Priority Review for TIBSOVO in patients with relapsed or refractory myelodysplastic syndromes underscores our continued work to advance therapeutic progress across IDH mutated cancers, and if approved in this setting, will bring the first and only targeted therapy to patients living with a significant unmet need."
  • The updated efficacy results were presented at the 2023 European Hematology Association (EHA) Congress.
  • "Today's filing acceptance provides further support for the potential efficacy and acceptable safety profile of TIBSOVO in relapsed or refractory MDS and reinforces the importance of mutational testing in this patient population."

Disc Medicine Reports Second Quarter 2023 Financial Results and Provides Business Update

Retrieved on: 
Friday, August 11, 2023
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Disc plans to initiate a Phase 1 study in healthy volunteers during the second half of 2023.

Key Points: 
  • Disc plans to initiate a Phase 1 study in healthy volunteers during the second half of 2023.
  • Second Quarter 2023 Financial Results:
    Cash Position: Cash and cash equivalents were $377.6 million as of June 30, 2023, which are expected to fund our operational plans well into 2026.
  • Research and Development Expenses: R&D expenses were $12.1 million for the quarter ended June 30, 2023, as compared to $7.7 million for the quarter ended June 30, 2022.
  • Net Loss: Net loss was $15.9 million for the quarter ended June 30, 2023, as compared to $9.6 million for the second quarter of 2022.

Nkarta Reports Second Quarter 2023 Financial Results and Corporate Highlights

Retrieved on: 
Thursday, August 10, 2023
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As previously announced, Nkarta plans to present updated results from its ongoing clinical trial of NKX019 in the second half of 2023.

Key Points: 
  • As previously announced, Nkarta plans to present updated results from its ongoing clinical trial of NKX019 in the second half of 2023.
  • In July 2023, Nkarta announced the appointment of Alyssa Levin, CPA, CA, as Chief Financial and Business Officer.
  • Non-cash stock-based compensation expense included in R&D expense was $2.1 million for the second quarter of 2023.
  • Net loss was $33.3 million, or $0.68 per basic and diluted share, for the second quarter of 2023.

X4 Pharmaceuticals Reports Second-Quarter 2023 Financial Results, Provides Corporate Updates, and Reports Emerging Data from Chronic Neutropenia Clinical Program

Retrieved on: 
Thursday, August 10, 2023
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Additional data from this ongoing Phase 2 trial are expected to be shared in the fourth quarter of 2023.

Key Points: 
  • Additional data from this ongoing Phase 2 trial are expected to be shared in the fourth quarter of 2023.
  • X4 is planning to initiate a Phase 3 clinical trial evaluating mavorixafor in certain chronic neutropenic disorders in the first half of 2024.
  • X4 will host a conference call and webcast today at 8:30 am ET to discuss these financial results and business highlights.
  • The live webcast and slide presentation can be accessed on the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com .