Rare

X4 Pharmaceuticals Reports Third-Quarter 2023 Financial Results and Provides Corporate Updates

Retrieved on: 
Thursday, November 9, 2023
Priority review, FDA, Ageing, PDUFA, Addition, Rare, Physician, Christophe, SG, MD, Conference, Organization, Doctor of Philosophy, PRV, Primary immunodeficiency, R, Society, Neutropenia, ASH, Wart, WHIM, Disease, ID, WHIM syndrome, Immune system, Research and development, Line, File, Security (finance), New Drug Application, NDA, Safety, Conference call, Patient, Diagnosis, State Administrative Expenses, Pharmaceutical industry, Rare-earth element, Hypogammaglobulinemia, Infection, Myelokathexis

BOSTON, Nov. 09, 2023 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today reported financial results for the third quarter ended September 30, 2023 and highlighted key recent and upcoming expected milestones.

Key Points: 
  • ET
    BOSTON, Nov. 09, 2023 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today reported financial results for the third quarter ended September 30, 2023 and highlighted key recent and upcoming expected milestones.
  • The FDA has notified X4 that it does not currently plan to hold an advisory committee meeting to review the filing.
  • Cash, Cash Equivalents, Restricted Cash, and Short-Term Marketable Securities: X4 had $142.7 million in cash, cash equivalents, restricted cash, and marketable securities as of September 30, 2023.
  • The live webcast will be accessible through the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com .

Processa Pharmaceuticals to Present at the 20th Orphan Drugs and Rare Diseases Global Congress 2023 Americas

Retrieved on: 
Thursday, November 9, 2023
Stuart Street, Dunedin, Rare, Safety, Cancer, PCSA, Research and development, Patient, Development, Pharmaceutical industry, Research, MD

HANOVER, MD, Nov. 09, 2023 (GLOBE NEWSWIRE) -- Processa Pharmaceuticals, Inc. (Nasdaq: PCSA) (“Processa” or the “Company”), a clinical-stage pharmaceutical company focused on developing the next generation of chemotherapeutic drugs to improve the efficacy and safety for patients suffering from cancer, announces that Dr. David Young, President of Research and Development, will present at the 20th Orphan Drugs and Rare Diseases Global Congress 2023 Americas.

Key Points: 
  • HANOVER, MD, Nov. 09, 2023 (GLOBE NEWSWIRE) -- Processa Pharmaceuticals, Inc. (Nasdaq: PCSA) (“Processa” or the “Company”), a clinical-stage pharmaceutical company focused on developing the next generation of chemotherapeutic drugs to improve the efficacy and safety for patients suffering from cancer, announces that Dr. David Young, President of Research and Development, will present at the 20th Orphan Drugs and Rare Diseases Global Congress 2023 Americas.
  • The conference is being held on November 16 – 17, 2023 at the Revere Hotel Boston Common, 200 Stuart Street, Boston, MA 02116.
  • Dr. Young will be available for one-on-one meetings throughout the conference.
  • For more information about the conference, click below:

NASP Applauds the Senate Finance Committee for Taking Additional Steps to Address Anticompetitive PBM Practices

Retrieved on: 
Wednesday, November 8, 2023
National Association, Insurance, DIR, NASP, Haemophilia, Rare, Committee, Legislation, Meat extenders, Acquisition, Accreditation, Rheumatoid arthritis, Health, Maintenance, Administration, United States Senate Finance Subcommittee on Health Care, Medicare Part D, Growth, Organ transplantation, Pharmacy, Cystic fibrosis, Specialty pharmacy, Multiple sclerosis, House, Cancer, Patient, Senate, Hospital, Medicare, Medicaid

Washington, DC, Nov. 08, 2023 (GLOBE NEWSWIRE) -- The National Association of Specialty Pharmacy (NASP) thanks the Senate Finance Committee for its bipartisan efforts to address anticompetitive practices that ultimately harm beneficiary access to the specialty pharmacy of their choice.

Key Points: 
  • Washington, DC, Nov. 08, 2023 (GLOBE NEWSWIRE) -- The National Association of Specialty Pharmacy (NASP) thanks the Senate Finance Committee for its bipartisan efforts to address anticompetitive practices that ultimately harm beneficiary access to the specialty pharmacy of their choice.
  • Today’s legislation includes efforts to strengthen and enforce the long-standing federal Any Willing Pharmacy law, an NASP advocacy priority.
  • For these beneficiaries, their pharmacies meet the patients where they are in their local communities, but typically operate from a centralized location.
  • Specialty pharmacies are accredited by an independent, third party nationally recognized accreditation organization ensuring consistent quality of extensive drug management and clinical patient care services.

MaxCyte Reports Third Quarter 2023 Financial Results and Reiterates Full Year 2023 Revenue Guidance

Retrieved on: 
Wednesday, November 8, 2023
LSE, Investment, Rare, Partnership, AutoLISP, Autoimmune disease, Therapy, SPL, Video game

Total revenue of $8.0 million in the third quarter of 2023, a decrease of 25% compared to the third quarter of 2022.

Key Points: 
  • Total revenue of $8.0 million in the third quarter of 2023, a decrease of 25% compared to the third quarter of 2022.
  • Core business revenue of $6.6 million in the third quarter of 2023, a decrease of 33% compared to the third quarter of 2022.
  • SPL Program-related revenue of $1.4 million in the third quarter of 2023, compared to $0.8 million in the third quarter of 2022.
  • Full year 2023 revenue guidance of approximately $34-$36 million total revenue and approximately $28-$30 million of core revenue, and SPL Program-related revenue of approximately $6 million, all consistent with guidance previously provided on October 4th.

Bionano Reports Third Quarter 2023 Results and Highlights Recent Business Progress

Retrieved on: 
Wednesday, November 8, 2023
Spectrum, German Cancer Research Center, Aneuploidy, CMA, MCA, OEM, Sale, Genomics, DSM-IV codes, OGM, NMPA, WES, SVS, IVD, Publication, Rare, Polyploidy, CRISPR, Medtech, International Cancer Genome Consortium, Doctor of Philosophy, GAAP, Acquisition, Research, Poster, Karyotype, Lobotomy Corporation, Genome, Nature Communications, National Medical Products Administration, SNV calling from NGS data, Hospital, Annual general meeting, CGC, Workflow, Bone marrow, KT, CFO, Cancer, ASD, Total, VIA, IPSC, Chromosomal deletion syndrome, SAN, DNA, Outline, Beauty salon, Cryptocurrency, Video game, Vaccine, Management, Medical device, Pharmaceutical industry

Publications featuring OGM increased 61% from the third quarter of 2022 to the third quarter of 2023, including a total of 47 human-focused publications.

Key Points: 
  • Publications featuring OGM increased 61% from the third quarter of 2022 to the third quarter of 2023, including a total of 47 human-focused publications.
  • GAAP gross margin for the third quarter of 2023 was 30%, compared to 25% from the third quarter of 2022.
  • Third quarter 2023 non-GAAP¹ gross margin was 32%, compared to 25% from the third quarter of 2022.
  • Third quarter 2023 non-GAAP operating expense was $31.8 million, compared to $26.3 million in the third quarter of 2022 and $34.6 million in the second quarter of 2023.

AscellaHealth Ranked Number 231 Fastest-Growing Company in North America on the 2023 Deloitte Technology Fast 500™

Retrieved on: 
Wednesday, November 8, 2023
Biotechnology, Pharmaceutical, Managed Care, Oncology, Health, Professional Services, Clinical Trials, Business, Fintech, Deloitte Technology Fast 500, Education, Rare, Creativity, Multimedia, Perseverance, SP, LLP, Telecommunications, Patient, Fine chemical

AscellaHealth , a global healthcare and specialty pharmacy (SP) solutions company, today announced it ranked 231 on the Deloitte Technology Fast 500 ™, a ranking of the 500 fastest-growing technology, media, telecommunications, life sciences, fintech, and energy tech companies in North America, now in its 29th year.

Key Points: 
  • AscellaHealth , a global healthcare and specialty pharmacy (SP) solutions company, today announced it ranked 231 on the Deloitte Technology Fast 500 ™, a ranking of the 500 fastest-growing technology, media, telecommunications, life sciences, fintech, and energy tech companies in North America, now in its 29th year.
  • “While software and services and life sciences continue to dominate the top 10, we are encouraged to see other categories making their mark.
  • “Over the nearly 30 years we’ve been compiling the Technology Fast 500, we’ve seen new categories emerge, growth rates explode, and certain regional markets shine from the bright talent they attract.
  • We are proud of all the winners for achieving this well-deserved honor.”

BPGbio Wins 2023 BioTech Breakthrough Award for Therapeutics Platform of The Year

Retrieved on: 
Wednesday, November 8, 2023
Technology, Clinical Trials, Health Technology, Software, Biotechnology, Pharmaceutical, Health, Science, Artificial Intelligence, Other Science, Drug discovery, GBM, Harvard Medical School, AstraZeneca, CIA, Netherlands Architecture Institute, Biomarker, Rare, Therapy, Government, Oak Ridge National Laboratory, United States Department of Defense, Supercomputing in India, Carcinoma, Icahn School of Medicine at Mount Sinai, HPC, Organization, SCC, Research, Prostate cancer, Glioblastoma, Liver disease, Multimedia, Medicine, Biology, EB, Neurology, Breast cancer, Pancreatic cancer, Sanofi, AI, ORNL, Mount Sinai, Patient, Epidermolysis bullosa, Sarcopenia, Pharmaceutical industry, Medical device, Neuroscience

View the full release here: https://www.businesswire.com/news/home/20231108370629/en/

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20231108370629/en/
    BPGbio named Therapeutics Platform of the Year by Biotech Breakthrough Awards program.
  • The award recognizes BPGbio’s success leveraging their NAi Interrogative Biology Platform to develop late-stage therapeutics and diagnostics for some of the most challenging diseases.
  • “We are honored that BPGbio has won the Therapeutics Platform of the Year from BioTech Breakthrough, a massive achievement for our employees and research partners.
  • BPGbio, our ‘Therapeutics Platform of the Year’ winner, is proving the value of AI in drug discovery for the future of medicine,” said Bryan Vaughn, Managing Director of BioTech Breakthrough Awards.

Rallybio to Present at Upcoming Investor Conferences in November

Retrieved on: 
Wednesday, November 8, 2023
Biotechnology, General Health, Health, Clinical Trials, EST, Conference, Patient, Rare, Corporation, Pharmaceutical industry

Steve Uden, M.D., Chief Executive Officer will present a corporate overview at 3:00 p.m. EST.

Key Points: 
  • Steve Uden, M.D., Chief Executive Officer will present a corporate overview at 3:00 p.m. EST.
  • 6th Annual Evercore ISI HealthCONx Conference in Miami, FL on Tuesday, November 28, 2023.
  • M.D., Chief Executive Officer will participate in a fireside chat at 7:30 a.m. EST.
  • Live webcasts of both presentations will be accessible through the Events and Presentations section of Rallybio’s website at www.rallybio.com .

The New York Center for Rare Diseases at Montefiore to Partner with GeneDx, PacBio and Google Health to Increase Genomic Testing Options for Bronx Families

Retrieved on: 
Wednesday, November 8, 2023
Albert Einstein College of Medicine, Center of excellence, DSM-IV codes, Genetic testing, Rare, RNA, Hope, Genetics, Research, NORD, Children's Hospital at Montefiore, Environment, Disease, Division, National Organization for Rare Disorders, Abraxis BioScience, FACMG, Partnership, CHAM, Google Health, GeneDx, Google AI, DNA encryption, Patient, Hospital, Pediatrics, DNA, Medical imaging, Vaccine, Genomics, Montefiore, The Bronx

BRONX, N.Y., Nov. 8, 2023 /PRNewswire/ -- The New York Center for Rare Diseases (NYCRD) at Montefiore, recognized as a Center of Excellence by the National Organization for Rare Disorders (NORD), is partnering with GeneDx, PacBio and Google Health to deliver genetic diagnoses for Bronx families living with rare diseases. The goal of the new partnership is to help identify the genetic causes of, and best treatments for rare diseases that have remained undiagnosed, despite using today's most advanced tests.

Key Points: 
  • On average, the search for a rare disease diagnosis takes five to seven years and can be fraught with misdiagnoses.
  • The focus of this research project on the Bronx in New York City adds a level of complexity to rare disease diagnostics.
  • "Our aim is to illuminate rare disease mysteries and bring answers and hope to families navigating the complexities of a rare disease."
  • Together with PacBio and Montefiore, we can help improve equity in genomics resources and help end long diagnostic odysseys for patients."

Lumos Pharma Reports Third Quarter 2023 Financial Results and Clinical Development Updates

Retrieved on: 
Tuesday, November 7, 2023
Research, Transgender hormone therapy, American Association of Clinical Endocrinologists, Event, Child, LUMO, Pediatric endocrinology, Therapy, City of Hope National Medical Center, Rare, IGFBP3, Mother, MD, Quarter, SDS, Endocrine Society, Clinical trial, PEM, KOL, Lumos, University, Serum, University of Chile, Political moderate, European Society of Gynaecological Oncology, Growth hormone, Investment, Consistency, AHV, Silviculture, Pharmaceutical industry, Birth control, Endocrinology

AUSTIN, Texas, Nov. 07, 2023 (GLOBE NEWSWIRE) -- Lumos Pharma, Inc. (NASDAQ:LUMO), a clinical-stage biopharmaceutical company focused on therapeutics for rare diseases, today announced that topline results met primary and secondary endpoints for its Phase 2 Dose-Finding OraGrowtH210 Trial and Phase 2 Pharmacokinetic/Pharmacodynamic (PK/PD) OraGrowtH212 Trial evaluating oral LUM-201 for subjects with moderate pediatric growth hormone deficiency (PGHD) who screened PEM-positive utilizing Lumos Pharma’s predictive enrichment marker (PEM) strategy. Lumos also announced its financial results for the quarter ended September 30, 2023.

Key Points: 
  • Lumos also announced its financial results for the quarter ended September 30, 2023.
  • The Company expects cash use of approximately $9.0 to $10.0 million in the fourth quarter of 2023.
  • Net Loss – The net loss for the quarter ended September 30, 2023 was $8.3 million compared to a net loss of $7.3 million for the same period in 2022.
  • Lumos Pharma ended the third quarter 2023 with 7,914,582 shares outstanding.