Day: January 7, 2024

Strategic roadmap to achieve blockbuster status for multiple products and expand the Company’s engine for future innovationCOPENHAGEN, Denmark, Jan. 07, 2024 (GLOBE NEWSWIRE) — Ascendis Pharma A/S (Nasdaq: ASND) today introduced selected 2024 corporate milestones and Vision 2030, its strategic roadmap through 2030. Ascendis President and CEO Jan Mikkelsen will present the update tomorrow, January 8, at the 42nd Annual J.P. Morgan Healthcare Conference. “With unwavering focus on our values of Patients, Science, and Passion, we remain on track to fulfill Vision 3×3, with the approval already of two Endocrinology Rare Disease products and value leadership in the U.S. growth hormone market for SKYTROFA,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “Today we are introducing Vision 2030, our...

SAN DIEGO, Jan. 07, 2024 (GLOBE NEWSWIRE) — Singular Genomics Systems, Inc. (Nasdaq: OMIC), a company leveraging novel next-generation sequencing (NGS) and multiomics technologies to empower researchers and clinicians, today announced instrument shipments and preliminary unaudited revenue for the fourth quarter of 2023. The Company shipped eight G4 instruments in the fourth quarter of 2023. Preliminary unaudited revenue for the fourth quarter of 2023 is expected to be approximately $1.0 million, representing growth of approximately 31% compared to $765 thousand during the fourth quarter of 2022. “We are proud of the momentum we built throughout 2023, increasing our installed base of instruments and recently launching two high-impact consumable kits: the F3 flow cells and Max Read flow cells,” said Drew Spaventa, Chairman and Chief...

2023 Total Revenue of ~$399.4M, a 21% Increase Year-Over-Year >2,400 People Living with Fabry Disease on Galafold® Following a Year of Increased Demand Expecting 2024 Galafold Revenue Growth of 11-16% at CER Successful Launches of Pombiliti™ + Opfolda™ Underway in the U.S., U.K., and Germany PRINCETON, N.J., Jan. 07, 2024 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, today provided its preliminary and unaudited 2023 revenue, corporate updates, and full-year 2024 outlook. In 2023, Amicus met or exceeded its strategic priorities, highlighted by:Sustaining double-digit Galafold revenue growth Securing FDA, EMA, and MHRA approvals for Pombiliti + Opfolda Initiating successful global...

Tetra-specific T-cell engager MP0533 for patients with r/r AML and AML/MDS on track to deliver expanded clinical phase 1/2a data in H1 2024 Progress of Radio-DARPin Therapy (RDT) platform: Improved tumor to kidney uptake ratio enables expansion of RDT pipeline; first data on DLL3 as lead candidate; to be co-developed as 212Pb-based RDT in new partnership with Orano Med Introduction of first program from the Switch-DARPin platform: the cKIT x CD16a x CD47 Switch-DARPin allows local conditional immune cell activation for targeted killing of hematopoietic stem cells (HSC) as next-generation conditioning for hematopoietic stem cell transplantation (HSCT) in AML and beyond.ZURICH-SCHLIEREN, Switzerland and CONCORD, Mass., Jan. 07, 2024 (GLOBE NEWSWIRE) — Ad hoc announcement pursuant to Art. 53 LR Molecular Partners AG (SIX: MOLN;...

Investigators from the National Institutes of Health (NIH) report 2 partial responses (PR) in 3 peripheral T-cell lymphoma (PTCL) patients and 1 PR in 2 double-hit diffuse large b-cell lymphoma (DH-DLBCL) patients in ongoing dose-escalation trial of enitociclib in combination with venetoclax and prednisone Vincerx remains on target to report early clinical data from lead VersAptx™ platform compounds, VIP236 (early 2024) and VIP943 (mid 2024) PALO ALTO, Calif., Jan. 07, 2024 (GLOBE NEWSWIRE) — Vincerx Pharma, Inc. (Nasdaq: VINC) (“Vincerx”), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, today announced promising clinical results from a Phase 1 NIH-sponsored study of enitociclib in combination with venetoclax and prednisone for the treatment...

Capital return to be conducted through synthetic share repurchase // Return of up to $300 million – maximum approved by shareholders in 2023 – designed to enable efficient return of cash to shareholders and enhance EPS // Implementation planned to be completed in late January 2024 Venlo, the Netherlands, Jan. 07, 2024 (GLOBE NEWSWIRE) — QIAGEN N.V. (NYSE: QGEN; Frankfurt Prime Standard: QIA) today announced a plan to return up to approximately $300 million (maximum EUR 273 million) to shareholders through a synthetic share repurchase that combines a direct capital repayment with a reverse stock split. QIAGEN has decided to implement the maximum $300 million value of the mandate given at the Annual General Meeting in June 2023, where shareholders gave virtually unanimous approval for the related resolutions. This approach is designed...

CARLSBAD, Calif., Jan. 07, 2024 (GLOBE NEWSWIRE) — Exagen Inc. (Nasdaq: XGN), a leading provider of autoimmune testing solutions, today announces select preliminary unaudited financial results for the full year ended December 31, 2023, and reaffirms its full-year 2023 financial guidance. Select Preliminary Unaudited Financial ResultsRecord AVISE® CTD testing volume of approximately 137,000 units delivered during the year ended December 31, 2023. Cash balance of $36.5 million as of December 31, 2023. Accounts receivables balance of approximately $7 million as of December 31, 2023.Reaffirms Financial GuidanceThe company reaffirms its full-year 2023 total revenue guidance of at least $50 million and full-year 2023 non-GAAP adjusted EBITDA guidance of approximately negative $20 million.“I’m very proud of the strides the Exagen...

Preliminary 2023 Total Revenue of $430 million to $435 million, Crysvita® Revenue of $325 million to $330 million and Dojolvi® revenue of $70 million to $71 million 2024 expected Total Revenue guidance between $500 million to $530 million, Crysvita revenue of $375 million to $400 million, and Dojolvi revenue of $75 million to $80 million Year-end 2023 cash balance of approximately $776 million and 2024 guidance for net cash used in operations expected to be less than $400 million NOVATO, Calif., Jan. 07, 2024 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today reported preliminary unaudited 2023 revenue results, cash and investments at year end 2023, and provided 2024...

Tim Kelly, chief technology officer, Caribou BiosciencesTim Kelly to lead Caribou’s technical operations strategy and execution— Tim Kelly to lead Caribou’s technical operations strategy and execution — — CB-010 ANTLER Phase 1 trial initial dose expansion data and RP2D to be disclosed Q2 2024; Caribou plans to initiate pivotal Phase 3 trial in 2L LBCL by YE 2024 — — CB-011 CaMMouflage Phase 1 trial enrolling patients at dose level 3; initial dose escalation data in r/r MM expected by YE 2024 — — CB-012 AMpLify Phase 1 trial first sites active; trial initiation in r/r AML expected H1 2024 — — Caribou to present at 42nd Annual J.P. Morgan Healthcare Conference on Thursday, January 11, at 11:15 am PST — BERKELEY, Calif., Jan. 07, 2024 (GLOBE NEWSWIRE) — Caribou...

Following a strong end to 2023 with double-digit organic growth in the fourth quarter and solid profitability, the preliminary and unaudited results for 2023 exceed the full-year guidance communicated on 8 November 2023. Key Q4 2023 highlights (preliminary and unaudited):Q4 2023 organic growth accelerates to +12% boosted by LFL (Like-for-Like) growth of +9%. Continued brand momentum drove strong performance across the key trading events of Black Friday and Christmas. Broad-based LFL growth across markets: key markets in Europe remained solid at 5% growth, US accelerated to +10% growth and Rest of Pandora continued strong double-digit growth at +16%.  Strong LFL growth across collections with Moments and Pandora ME delivering +4% and +6% growth, respectively. Timeless accelerated further with +31% growth whilst Pandora Lab-Grown...