Microdeletion syndrome

Karyopharm to Participate at Upcoming Investor Conferences

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Tuesday, November 8, 2022
Endometrial cancer, NASDAQ, Microdeletion syndrome, Industry, KPTI, Company, Multiple myeloma, SINE, United Kingdom, View, Ruxolitinib, Science, XPO1, Multimedia, Pharmaceutical industry, Online shopping

Karyopharm Therapeutics Inc. (NASDAQ: KPTI) is a commercial-stage pharmaceutical company pioneering novel cancer therapies.

Key Points: 
  • Karyopharm Therapeutics Inc. (NASDAQ: KPTI) is a commercial-stage pharmaceutical company pioneering novel cancer therapies.
  • Karyopharm has a focused pipeline targeting multiple high unmet need cancer indications, including in multiple myeloma, endometrial cancer, myelodysplastic syndromes and myelofibrosis.
  • For more information about our people, science and pipeline, please visit www.karyopharm.com , and follow us on Twitter at @Karyopharm and LinkedIn .
  • XPOVIO and NEXPOVIO are registered trademarks of Karyopharm Therapeutics Inc.
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Rallybio Reports Third Quarter 2022 Financial Results

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Monday, November 7, 2022
Professional Services, Health, Finance, General Health, Clinical Trials, Pharmaceutical, Biotechnology, Net, G&A, Company, Beta thalassemia, Research, Science, Administration, Corporation, Biotechnology, Microdeletion syndrome, Pregnancy, Woman, Population, HPP, General Administration of Sport of China, Half-life, Iron overload, Nasdaq, University, Research and development, Hematology, ENPP1, U.S. Securities and Exchange Commission, Degenerative disease, Forward-looking statement, Risk, Hypophosphatasia, R, SEC, Erythropoiesis, Patient, Program, Pharmaceutical industry, IND, C5

Recent Business Highlights and Upcoming Milestones:

Key Points: 
  • Recent Business Highlights and Upcoming Milestones:
    In September 2022, Rallybio announced preliminary platelet elimination results from its ongoing Phase 1b proof-of-concept study for RLYB212.
  • Rallybio also announced in September 2022 that dosing under the amended protocol has commenced, which increases the dose of RLYB212.
  • Rallybio currently expects to initiate the multiple ascending dose Phase 1 study of RLYB116 in the first quarter of 2023.
  • Third Quarter 2022 Financial Results:
    Research & Development (R&D) Expenses: R&D expenses were $12.1 million for the third quarter of 2022, compared to $5.0 million for the same period in 2021.

Bionano Genomics Reports Third Quarter 2022 Financial Results and Highlights Recent Business Progress

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Thursday, November 3, 2022
Chronic lymphocytic leukemia, DNA, Society, Potency (pharmacology), IPSC, Cerebellar ataxia, BMA, Regenerative medicine, MD Anderson Cancer Center, Schizophrenia, CRISPR, Company, Infertility, Workflow, Study, Growth, SAN, GAAP, OGM, GLOBE, Total, Ataxia, Genomics, LDT, ASHG, Chromothripsis, Patient, Cancer, CLL, Doctor of Philosophy, MDS, Long, SVS, Research, Hospital, Human genetics, Lineagen, Sale, International Cancer Genome Consortium, Pregnancy loss, NIPT, CTH, Adoption, Long-string instrument, UHMW, American Society of Human Genetics, Chromosome, ATM, RPL, Industry, Microdeletion syndrome, Publication, Certification, Tumors of the hematopoietic and lymphoid tissues, Acute myeloid leukemia, Nasdaq, Peripheral neuropathy, Karyotype, TP53, AML, RFC1, Renewable energy, Video game, Pharmaceutical industry, Medical imaging

SAN DIEGO, Nov. 03, 2022 (GLOBE NEWSWIRE) -- Bionano Genomics, Inc. (Nasdaq: BNGO), today reported financial results for the third quarter ended September 30, 2022.

Key Points: 
  • milestones
    Strong balance sheet with $180.2 million in cash, cash equivalents, and available-for-sale securities at the end of Q3 2022
    SAN DIEGO, Nov. 03, 2022 (GLOBE NEWSWIRE) -- Bionano Genomics, Inc. (Nasdaq: BNGO), today reported financial results for the third quarter ended September30, 2022.
  • We believe our Q3 2022 results, which include the highest quarterly revenues ever for Bionano, reflect continued gains in market development and commercial validation for OGM.
  • Total revenue for Q3 2022 was $7.2 million, up 55% from Q3 2021, and an 8% increase from Q2 2022.
  • Chris Stewart, chief financial officer of Bionano added, Q3 2022 was another outstanding quarter for Bionano.

Corcept Therapeutics Announces Third Quarter Financial Results and Provides Corporate Update

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Thursday, November 3, 2022
Progression-free survival, Exercise, Weight gain, Fatigue, Neck, Investment, Ageing, CORT, Food, Doctor of Pharmacy, Cushing's syndrome, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, GLOBE, Irritability, Muscle, Investor, Intellectual property, NASH, Safety, Neuroinflammation, COVID-19, PIN, Marketing, Intention, Income, Ovary, NDA, Life expectancy, Physician, GRACE, ALS, Prostate, Clinical trial, Depression, Liver disease, NASDAQ, Microdeletion syndrome, Acquisition, Woman, Anxiety, Diabetes, Goal, Wilms' tumor, Insurance, Enrollment, SEC, Patient, MD, Pharmaceutical industry, Corcept Therapeutics

Net income was $34.6 million in the third quarter of 2022, compared to $30.5 million in the third quarter of 2021.

Key Points: 
  • Net income was $34.6 million in the third quarter of 2022, compared to $30.5 million in the third quarter of 2021.
  • Diagnosing and treating patients with a complex disease such as Cushings syndrome requires frequent in-person contact with physicians.
  • Our revenue in the third quarter was affected by fewer than expected in-person interactions as many physician practices have not returned to pre-pandemic patterns of activity.
  • Korlym is an excellent treatment for patients with Cushings syndrome and there are many eligible patients who have yet to receive it.

MacroGenics Provides Corporate Update and Third Quarter 2022 Financial Results

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Thursday, November 3, 2022
Macrogen, Conjugation, Company, ADC, Exercise, Telephone, Teplizumab, Therapy, Food, Cancer, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Microdeletion syndrome, GLOBE, Prescription Drug User Fee Act, Anti-CD3 monoclonal antibody, FDA, B7, MCRPC, D, Webcast, Conference, NSCLC, BLA, Acute myeloid leukemia, Half-life, CD123, Large-cell lung carcinoma, Cytokine release syndrome, Research, CEO, Conference call, Clinical trial, Gilead Sciences, PDUFA, ID, NASDAQ, Investor relations, Mab, Melanoma, Patient, Program, Pharmaceutical industry, Vaccine, T1D, Incyte

ET

Key Points: 
  • ET
    ROCKVILLE, Md., Nov. 03, 2022 (GLOBE NEWSWIRE) -- MacroGenics, Inc. (NASDAQ: MGNX), a biopharmaceutical company focused on developing and commercializing innovative antibody-based therapeutics for the treatment of cancer, today provided an update on its recent corporate progress and reported financial results for the quarter ended September30, 2022.
  • Revenue for the quarter ended September30, 2022 included MARGENZA net sales of $4.4 million, compared to $3.6million for the quarter ended September30, 2021.
  • R&D Expenses: Research and development expenses were $48.2 million for the quarter ended September30, 2022, compared to $49.8 million for the quarter ended September30, 2021.
  • SG&A Expenses: Selling, general and administrative expenses were $15.4 million for the quarter ended September30, 2022, compared to $17.2 million for the quarter ended September30, 2021.

Magenta Therapeutics to Present Data at the 2022 American Society of Hematology (ASH) Annual Meeting

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Thursday, November 3, 2022
Stem cell, Society, Conjugation, Magenta, U.S. Securities and Exchange Commission, Clinical trial, Program, Transplant, Disclosure, Stem cell factor, Food, Sickle cell disease, Pharmacodynamics, GLOBE, ASH, Toxic epidermal necrolysis, Phenotype, Safety, PD, GVHD, Security (finance), Degenerative disease, Control, Patient, Blood, Genetic distance, MDS, Leadership, ADC, B cell, Bone marrow, Biotechnology, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, NHP, Private Securities Litigation Reform Act, Folate targeting, COVID-19, Abstract (summary), Goal, Survival, Primate, CD117, CD137, Microdeletion syndrome, Cell, Uncertainty, Therapy, Growth hacking, Toxicology, Engineering, Lymphatic system, AML, PK, Pharmaceutical industry, Vaccine, Pharmacokinetics

CAMBRIDGE, Mass., Nov. 03, 2022 (GLOBE NEWSWIRE) -- Magenta Therapeutics (Nasdaq: MGTA), a clinical-stage biotechnology company developing novel medicines designed to bring the curative power of stem cell transplant to more patients, today announced that it will make three presentations relating to its ongoing clinical trials at the 2022 American Society of Hematology (ASH) Annual Meeting, to be held in New Orleans from December 10-13, 2022 and virtually. In addition, an academic collaborator will present data from a preclinical program from Magenta’s research platform.

Key Points: 
  • In addition, an academic collaborator will present data from a preclinical program from Magentas research platform.
  • CD117, also known asc-Kit receptor, is highly expressed on hematopoietic stem cells, progenitor cells, and leukemic cells.
  • As described separately, in addition to the results described in the published abstract, Magenta will present updated available clinical data.
  • Magenta explicitly disclaims any obligation to update any forward-looking statements, except to the extent required by law.

Magenta Therapeutics Reports Third Quarter Financial Results and Recent Program Highlights

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Thursday, November 3, 2022
Infection, Stem cell, Society, Overalls, Research, CD45, Magenta, U.S. Securities and Exchange Commission, Clinical trial, Good laboratory practice, Partnership, Bluebird bio, Administration, Program, Disclosure, Mortality, Investigational New Drug, Food, Sickle cell disease, GCSF, GLOBE, IND, ASH, Transplant, Security (finance), Degenerative disease, Patient, Pharmacokinetics, Blood, MDS, DLT, Leadership, ADC, B cell, Bone marrow, Biotechnology, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Private Securities Litigation Reform Act, COVID-19, Granulocyte colony-stimulating factor, American Society of Hematology, Goal, Primate, Time, Cohort, Microdeletion syndrome, Cell, Safety, Disease, Uncertainty, Therapy, Collection, Toxicology, Lymphatic system, Development, AML, Vaccine, Pharmaceutical industry

CAMBRIDGE, Mass., Nov. 03, 2022 (GLOBE NEWSWIRE) -- Magenta Therapeutics (Nasdaq: MGTA), a clinical-stage biotechnology company developing novel medicines designed to bring the curative power of stem cell transplant to more patients, today reported financial results for the third quarter ending September 30, 2022, and recent program highlights.

Key Points: 
  • We are building momentum in the MGTA-117 clinical trial with new clinical results and are making progress across our pipeline, including our second targeted conditioning program CD45-ADC, said Jason Gardner, President and Chief Executive Officer of Magenta Therapeutics, Inc.
  • The program is currently enrolling patients with relapsed/refractory acute myeloid leukemia (AML), and myelodysplastic syndromes (MDS), in a Phase 1/2 dose escalation clinical trial.
  • In addition, Magenta has enrolled a sufficient number of patients to complete Cohort 3, provided that the patients complete their respective dose-limiting toxicity (DLT) observation periods.
  • Magenta has initiated requests for formal engagement with multiple regulatory authorities for the purpose of transitioning the clinical program into transplant-eligible AML and MDS patients.

Keros Therapeutics Reports Recent Business Highlights and Third Quarter 2022 Financial Results

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Thursday, November 3, 2022
Company, Protein, Cytopenia, Private Securities Litigation Reform Act, Degenerative disease, Research, Blood, Growth, Investment, Maintenance, Society, Tissue, Microdeletion syndrome, GLOBE, Hand, Intellectual property, Thrombocytopenia, COVID-19, Therapy, Ruxolitinib, Hypertrophy, U.S. Securities and Exchange Commission, Hypertensive emergency, KROS, ATM, Anemia, Security (finance), SEC, Achievement, Patient, Pharmaceutical industry

Keros continued to build upon the positive momentum of our pipeline programs in the third quarter of 2022.

Key Points: 
  • Keros continued to build upon the positive momentum of our pipeline programs in the third quarter of 2022.
  • Keros reported a net loss of $23.5 million in the third quarter of 2022 as compared to a net loss of $20.3 million in the third quarter of 2021.
  • Research and development expenses were $21.0 million for the third quarter of 2022 as compared to $14.8 million for the same period in 2021.
  • Keros cash and cash equivalents as of September30, 2022 was $239.4 million compared to $230.0 million as of December 31, 2021.

ValenzaBio Announces Positive Preliminary Data from Phase 1b/2a Trial of VB119, an Anti-CD19 Monoclonal Antibody for the Treatment of Primary Membranous Nephropathy

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Thursday, November 3, 2022
FDA, Health, Other Health, Clinical Trials, Pharmaceutical, Biotechnology, Glomerulonephritis, Therapy, Time, Serum, Society, PMN, Safety, Abstract, Poster, Autoantibody, CD19, Urine, ASN, B cell, Microdeletion syndrome, A2, American Society of Nephrology, Disease, Mab, Autoimmunity, Proteinuria, Patient, Pharmacokinetics, Vaccine, Pharmaceutical industry

These data represent the first clinical proof-of-concept for an anti-CD19 antibody in this disease and demonstrate potential differentiation from other therapies used in this indication.

Key Points: 
  • These data represent the first clinical proof-of-concept for an anti-CD19 antibody in this disease and demonstrate potential differentiation from other therapies used in this indication.
  • PMN, also known as idiopathic membranous nephropathy, is an autoimmune glomerular disease and is one of the leading causes of non-diabetic nephrotic syndrome.
  • Available treatment regimens fail to achieve complete remission in most patients with primary membranous nephropathy.
  • The company is advancing a pipeline of differentiated monoclonal antibodies with best-in-class properties, targeting clinically validated mechanisms of action, to provide improved treatment options for patients.

Geron Corporation Reports Third Quarter 2022 Financial Results and Upcoming Expected Milestones

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Thursday, November 3, 2022
Health, Other Science, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Patient, MDS, LR, MF, Company, Lymphatic system, Private Securities Litigation Reform Act, Xi, Exercise, Research, Prejudice, Corporation, Imetelstat, XV, GAAP, RBC, Microdeletion syndrome, Apoptosis, FDA, Intellectual property, Safety, 100,000, Webcast, Pharmacokinetics, U.S. Securities and Exchange Commission, IMPRESS, Geron Corporation, Nasdaq, COVID-19, Agreement, Income, TI, Review, ESA, Clinical trial, Fast Track, New Drug Application, Risk, Jaki, Disease, Security (finance), Insurance, JAK, Depreciation, Fault, Nobel Prize, Pharmaceutical industry, Life insurance, Video game, EU

Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company developing a first-in-class telomerase inhibitor, imetelstat, to treat hematologic malignancies, today reported financial results for the third quarter of 2022 and upcoming expected milestones.

Key Points: 
  • Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company developing a first-in-class telomerase inhibitor, imetelstat, to treat hematologic malignancies, today reported financial results for the third quarter of 2022 and upcoming expected milestones.
  • ET on Thursday, November 3, 2022 to review recent events and third quarter 2022 financial results.
  • This projected non-GAAP financial measure is in addition to, not a substitute for, or superior to, measures of financial performance projected in accordance with GAAP.
  • Geron encourages investors to carefully consider the Companys results under GAAP, as well as the supplemental non-GAAP financial information, to more fully understand Gerons business.