New Drug Application

Verastem Oncology Reports First Quarter 2024 Financial Results and Highlights Recent Business Updates

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木曜日, 5月 9, 2024
Research, FDA, Other Health, Biotechnology, General Health, Pharmaceutical, Health, Science, Oncology, Other Science, SGO, Society, Table, Cancer, Part, Fast track (FDA), Investment, Safety, NDA, Food, Abstract, New Drug Application, Gynecologic Oncology (journal), IND, ASCO, RAMP, Accelerated approval (FDA), Patient, Discovery, Research and development, FAK, Bank statement, BMS, Annual general meeting, Therapy, NSCLC, GAAP, Ovarian cancer, Verastem Oncology, Pharmaceutical industry

Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with cancer, today announced business updates and reported financial results for the first quarter ended March 31, 2024.

Key Points: 
  • Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with cancer, today announced business updates and reported financial results for the first quarter ended March 31, 2024.
  • Verastem Oncology ended the first quarter of 2024 with cash, cash equivalents and investments of $110.1 million.
  • Research & development expenses for the 2024 Quarter were $17.7 million, compared to $12.0 million for the 2023 Quarter.
  • Selling, general & administrative expenses for the 2024 Quarter were $10.4 million, compared to $7.3 million for the 2023 Quarter.

Royalty Pharma Reports First Quarter 2024 Results

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木曜日, 5月 9, 2024
FDA, Ulcerative colitis, KarXT, Food, MorphoSys, European Commission, Marketing, New Drug Application, Cystic fibrosis, PDSS, PANSS, Tourette syndrome, Lupus, Docetaxel, Roche, Schizophrenia, Novartis, ADP, Pfizer, Incidence, Bristol Myers Squibb, Patient, Neurology, Acquisition, Sacituzumab govitecan, Olanzapine, Civil service commission, Multiple sclerosis, Positive, Vertex, Sanofi, Teva Pharmaceuticals, Astellas Pharma, European Medicines Agency, Lung cancer, Pharmaceutical industry

During 2024, Royalty Pharma announced new transactions of up to $619 million.

Key Points: 
  • During 2024, Royalty Pharma announced new transactions of up to $619 million.
  • In January 2024, Royalty Pharma acquired a royalty interest in ecopipam for an upfront payment of $49 million and up to $44 million in milestone payments contingent on the achievement of certain regulatory milestones.
  • In May 2024, Royalty Pharma announced a transaction to acquire royalties and milestones on frexalimab owned by ImmuNext for approximately $525 million in cash including estimated transaction costs.
  • Royalty Pharma has provided guidance for full year 2024, excluding transactions and borrowings announced after the date of this release, as follows:

Applied Therapeutics Reports First Quarter 2024 Financial Results

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木曜日, 5月 9, 2024
Sale, FDA, PRV, American College, Diabetic cardiomyopathy, Investment, Priority review, ACC, NDA, Food, Research, Marketing, New Drug Application, MAA, CHMP, Doctor of Philosophy, Disease, Journal, ChromeOS, PDUFA, Patient, Treatment, EMA, JACC, Therapy, HF, European Medicines Agency, Ageing, Pharmaceutical industry

As a result, the Company now expects a decision by the EMA in early first quarter of 2025.

Key Points: 
  • As a result, the Company now expects a decision by the EMA in early first quarter of 2025.
  • Mr. Hooks brings over 30 years of biopharmaceutical experience in leadership, rare disease marketing and sales roles to Applied Therapeutics.
  • Presented Full Results from Phase 3 ARISE-HF Study of AT-001 (caficrestat) in Diabetic Cardiomyopathy at the 2024 American College of Cardiology Annual Scientific Session.
  • Following the presentation, the full study results were published in the Journal of the American College of Cardiology (JACC).

LENZ Therapeutics Reports First Quarter 2024 Financial Results and Provides Business Updates

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水曜日, 5月 8, 2024
FDA, New Drug Application, Presbyopia, Phase 3, Therapy, Patient, Pharmaceutical industry

ET

Key Points: 
  • ET
    SAN DIEGO, May 08, 2024 (GLOBE NEWSWIRE) -- LENZ Therapeutics, Inc. (Nasdaq: LENZ or “LENZ” or the “Company”), a late clinical-stage biopharmaceutical company focused on developing the first aceclidine-based eye drop that has been shown to improve near vision in people with presbyopia, today reported financial results and operational highlights for the first quarter ended March 31, 2024.
  • In February 2024, LENZ launched its unbranded “Eye Am…” campaign to educate and excite eye care professionals about future presbyopia solutions.
  • In March 2024, LENZ Therapeutics announced completion of its merger with Graphite Bio, Inc. and the concurrent $53.5 million private placement, commencing trading on the Nasdaq Global Select Market under the ticker symbol “LENZ” on March 21, 2024.
  • The live webcast from today’s conference call can be accessed here and on the LENZ Therapeutics website at www.LENZ-tx.com in the Investors & Media section.

PolyPid Provides Corporate Update and Reports First Quarter 2024 Financial Results

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水曜日, 5月 8, 2024
SHIELD, Patient, Cancer, Research, New Drug Application, Marketing, Interim analysis, PIPE, Conference, Completeness, G&A

PETACH TIKVA, Israel, May 08, 2024 (GLOBE NEWSWIRE) -- PolyPid Ltd. (Nasdaq: PYPD) (“PolyPid” or the “Company”), a late-stage biopharma company aiming to improve surgical outcomes, today provided a corporate update and reported financial results for the three months ended March 31, 2024.

Key Points: 
  • Unblinded Interim Analysis to be Conducted Once Approximately 400 Patients Complete Their 30-Day Follow-up; Top-line Results Expected in Second Half of 2024
    PETACH TIKVA, Israel, May 08, 2024 (GLOBE NEWSWIRE) -- PolyPid Ltd. (Nasdaq: PYPD) (“PolyPid” or the “Company”), a late-stage biopharma company aiming to improve surgical outcomes, today provided a corporate update and reported financial results for the three months ended March 31, 2024.
  • Top-line results are anticipated in the second half of 2024.
  • The Company’s cash runway now extends into the fourth quarter of 2024 and beyond the expected timing of the planned unblinded interim analysis.
  • PolyPid expects that its pro forma cash balance will be sufficient to fund operations into the fourth quarter of 2024.

Rhythm Pharmaceuticals Reports First Quarter 2024 Financial Results and Business Update

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火曜日, 5月 7, 2024
EMANATE, POMC, FDA, Sale, BV, Shanda, Investment, PES, Body mass index, New Drug Application, BMI, LEPR, LG Chem, LG, Research, Weight, BBS, Bardet–Biedl syndrome, Webcast, MC4R, Prescription, Obesity, Completeness, Patient, Hyperpigmentation, Acquisition, EMA, Polyphagia, Marketing, SIGNAL, Annual general meeting, G&A, The Lancet, Pharmaceutical industry

-- Management to host conference call today at 8:00 a.m. ET --

Key Points: 
  • ET --
    BOSTON, May 07, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, today reported financial results and provided a business update for the first quarter ended March 31, 2024.
  • This pivotal trial is progressing as planned, and we anticipate dosing the first patients with hypothalamic obesity in Japan in the second quarter of 2024.
  • Financial Guidance: For the year ending December 31, 2024, Rhythm anticipates approximately $250 million to $270 million in Non-GAAP Operating Expenses.
  • ET today to review its first quarter 2024 financial results and recent business activities.

Travere Therapeutics Reports First Quarter 2024 Financial Results

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月曜日, 5月 6, 2024
FDA, Total, Risk, Ligand Pharmaceuticals, European Commission, Research, UPCR, Webcast, HCU, IND, EGFR, PDUFA, SIMD, Civil service commission, Diagnosis, PMDA, GAAP, CMA, Congress, Table, PROTECT, Priority review, Food, Abstract, FSGS, Income tax, CSL Vifor, Quarter, Medication, Therapy, SAN, Plasma, Urine, International, Safety, Proteinuria, ANNA, Immunoglobulin A, Homocysteine, Investigational New Drug, Society, Shanda, New Drug Application, Genetic, European, Disease, Patient, Quality of life (healthcare), IPR, Bank statement, Growth, Pharmaceutical industry

Net product sales for the first quarter of 2024 were $40.0 million, compared to $24.2 million for the same period in 2023.

Key Points: 
  • Net product sales for the first quarter of 2024 were $40.0 million, compared to $24.2 million for the same period in 2023.
  • Travere Therapeutics will host a conference call and webcast today, Monday, May 6, 2024, at 4:30 p.m.
  • ET to discuss company updates as well as first quarter 2024 financial results.
  • The Company believes that these non-GAAP financial measures are helpful in understanding its past financial performance and potential future results.

Rhythm Pharmaceuticals Presents Data from Phase 3 Pediatrics Trial at Pediatric Endocrine Society Annual Meeting

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月曜日, 5月 6, 2024
POMC, FDA, Shanda, PES, Body mass index, Food, New Drug Application, BMI, LEPR, Weight loss, BBS, Bardet–Biedl syndrome, MC4R, Obesity, Patient, Rhythm, Annual general meeting, Proopiomelanocortin, Pharmaceutical industry

BOSTON, May 06, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, today announced that the Company delivered three presentations – one oral and two posters – at The Pediatric Endocrine Society’s (PES) Annual Meeting held May 2-5, 2024 in Chicago, IL.

Key Points: 
  • BOSTON, May 06, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, today announced that the Company delivered three presentations – one oral and two posters – at The Pediatric Endocrine Society’s (PES) Annual Meeting held May 2-5, 2024 in Chicago, IL.
  • In the oral presentation, Rhythm showcased data that was previously announced on December 6, 2023, from its 52-week, Phase 3 pediatrics trial in patients between the ages of 2 and younger than 6 years (N=12) with Bardet-Biedl syndrome (BBS) or obesity due to proopiomelanocortin (POMC) or leptin receptor (LEPR) deficiency.
  • Treatment with setmelanotide achieved the primary endpoint with a 3.04 mean reduction in BMI-Z score (a measure of body mass index deviations from what is considered normal) and 18.4 percent mean reduction in body mass index (BMI).
  • Rhythm also presented two additional poster presentations at PES 2024:

Marinus Pharmaceuticals Provides Business Update and Reports First Quarter 2024 Financial Results

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水曜日, 5月 8, 2024
Biotechnology, Pharmaceutical, Health, Epilepsy, FDA, Lennox–Gastaut syndrome, Administration, Investment, NDA, Food, Research, Human services, New Drug Application, CDD, Security (finance), API, Development, Seizure, Data monitoring committee, GABAA, Therapy, Ganaxolone, TSC, Data, Patient, Tuberous sclerosis, Medication, File, GAAP, Pharmaceutical industry

Marinus Pharmaceuticals , Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today reported business highlights and financial results for the first quarter ended March 31, 2024.

Key Points: 
  • Marinus Pharmaceuticals , Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today reported business highlights and financial results for the first quarter ended March 31, 2024.
  • “Our unwavering commitment is to develop innovative treatment options for individuals with seizure disorders,” said Scott Braunstein, M.D., Chairman and Chief Executive Officer of Marinus.
  • Increased full year 2024 projected U.S. ZTALMY net product revenues to between $33 and $35 million from a range of $32 and $34 million.
  • Marinus will focus its resources on the continued commercialization and development of ZTALMY, including the TrustTSC trial.

Innoviva Reports First Quarter 2024 Financial Results; Highlights Recent Company Progress

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水曜日, 5月 8, 2024
Biotechnology, Infectious Diseases, Health, Pharmaceutical, Clinical Trials, Nursing care plan, Investment, IST, Ceftriaxone, New Drug Application, ESCMID, Armata, Acinetobacter, Zoliflodacin, Conference, CNS, Azithromycin, Exercise, Sulbactam, GSK, Patient, Bank, Infection, Gonorrhea, Share repurchase, Growth, Pharmaceutical industry

“Our first quarter financial results continue to demonstrate the successful transformation for Innoviva.

Key Points: 
  • “Our first quarter financial results continue to demonstrate the successful transformation for Innoviva.
  • We are pleased with our first quarter performance, led by the strong launch of our novel therapeutic XACDURO®.
  • Share repurchases: During the first quarter 2024, Innoviva repurchased 0.6 million shares of its outstanding common stock for $9.7 million.
  • In the first quarter 2024, Innoviva invested an additional $35.0 million in one of our assets, Armata, to help advance its lead therapeutic phage candidates.