American Society of Nephrology

XORTX Highlights Achievements of 2023 and Preparation for Registration Clinical Trial

Retrieved on: 
Tuesday, March 19, 2024
Consultant, Nephrology, Provisional application, XRX, Partnership, Chemistry, Kidney disease, Tablet, Orphan, Bryant University, University, EMA, Polycystic kidney disease, Trial of the century, Oxipurinol, MBA, GMP, Diet, PKD, Treatment, Society, Therapy, Patent, Rensselaer Polytechnic Institute, FDA, Marketing, Diagnosis, American Society of Nephrology, Uric acid, Pharmaceutical industry

Dr. Allen Davidoff, CEO of XORTX, stated, “2023 marked a year of substantial clinical, technological and regulatory progress, establishing the foundation for the Company’s 2024 goals.

Key Points: 
  • Dr. Allen Davidoff, CEO of XORTX, stated, “2023 marked a year of substantial clinical, technological and regulatory progress, establishing the foundation for the Company’s 2024 goals.
  • Each of these milestones permit the next step in the Company’s clinical development plan, being a “registration” clinical trial – XRX-OXY-201 in pursuit of accelerated approval and support of the Company’s lead program XRx-008 program for ADPKD.
  • Chemistry and Manufacturing, Clinical and Pre-Clinical Highlights – Produced drug substance for oxypurinol production and produced GMP drug substance; confirmed XORLO™ formulation, produced enhanced bioavailability and produced clinical supply of tablets for clinical trials.
  • August 29, 2023 – XORTX submitted an Orphan Drug Designation application to the EMA for the treatment of ADPKD.

Galera Reports Third Quarter 2023 Financial Results and Recent Corporate Updates

Retrieved on: 
Tuesday, November 14, 2023
NDA, Cisplatin, Disclosure, Galera, Chronic kidney disease, Annual general meeting, Superoxide dismutase, ASN, ASTRO, Food, Type, SBRT, Head, Incidence, Investment, New Drug Application, Clinical trial, American Society of Nephrology, Insurance, Patient, Workforce, Research, Radiation therapy, Death, FDA, CKD, Neoplasm, LAPC, Therapy, Manganese, Enterocyte, GRECO, Cancer, CRL, Symantec Endpoint Protection, Combination, Superoxide dismutase mimetics, Layoff, Society, HNC, Poster, Pharmaceutical industry

MALVERN, Pa., Nov. 14, 2023 (GLOBE NEWSWIRE) -- Galera Therapeutics, Inc. (Nasdaq: GRTX), a clinical-stage biopharmaceutical company focused on developing a pipeline of novel, proprietary therapeutics that have the potential to transform radiotherapy in cancer, today announced financial results for the third quarter ended September 30, 2023 and provided recent corporate updates.

Key Points: 
  • The Company is exploring potential strategic alternatives, as it is not feasible to conduct an additional trial with the Company’s current resources.
  • Research and development expenses were $6.1 million in the third quarter of 2023, compared to $8.1 million for the same period in 2022.
  • General and administrative expenses were $5.0 million in the third quarter of 2023, compared to $4.9 million for the same period in 2022.
  • As of September 30, 2023, Galera had cash, cash equivalents and short-term investments of $28.4 million.

Eloxx Pharmaceuticals Reports Third Quarter 2023 Financial and Operating Results and Provides Business Update

Retrieved on: 
Monday, November 13, 2023
Sale, FSD, RDEB, ASN, Kidney, GBM, American Society of Nephrology, Patient, CFF, FDA, Glomerular basement membrane, G&A, Health, R, JEB, ATM, Society, TEM, OTC, RNA, Proteinuria, Biopsy, Pharmaceutical industry, Phosphorus, Microscope, Cryptocurrency, Alport syndrome

WATERTOWN, Mass., Nov. 13, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (OTC: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today reported its financial results for the three months ended September 30, 2023, and provided a business update.

Key Points: 
  • WATERTOWN, Mass., Nov. 13, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (OTC: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today reported its financial results for the three months ended September 30, 2023, and provided a business update.
  • Data from the Phase 2 study of ELX-02 for Alport Syndrome was included in two presentations at the American Society of Nephrology (ASN) Kidney Week 2023.
  • R&D expenses were $1.3 million for the three months ended September 30, 2023, which included $0.1 million in stock-based compensation.
  • Additionally, we sold shares of common stock through our ATM program during the quarter for gross proceeds of $1.8 million.

Annexon Reports Significant Progress with its Priority Programs and Third Quarter 2023 Financial Results

Retrieved on: 
Monday, November 13, 2023
Molecule, Ophthalmology, Visual acuity, Investment, HD, EMA, Research, Kidney, Autoimmune disease, Driver, C3, Conference, R, Facial muscles, Inflammation, Treatment, Meta-analysis, American Academy, American Society of Nephrology, G&A, Guillain, Society, Letter, Paratriathlon, European Medicines Agency, American Academy of Ophthalmology, Professional, C4, AAO, System, Nephrology, AMD, Immunoglobulin therapy, PACA, Plasma, Disease, Complement component 1q, Medicine, Complement system, FDA, Geographic atrophy, GBS, ARCHER, C5, HIV disease progression rates, Risk management, Pharmaceutical industry, Vaccine, Cryptocurrency, Patient, LN, ALS

EMA orphan drug designation requires that a novel rare disease therapeutic demonstrates the potential for significant benefit over available therapies.

Key Points: 
  • EMA orphan drug designation requires that a novel rare disease therapeutic demonstrates the potential for significant benefit over available therapies.
  • Importantly, Annexon also announced that it has achieved target enrollment of 225 patients in the randomized, double-blind, placebo-controlled Phase 3 trial of ANX005 in patients with GBS.
  • Research and development (R&D) expenses: R&D expenses were $27.9 million for the quarter ended September 30, 2023, reflecting the advancement of the company’s priority programs, including GBS, GA and ANX1502, compared to $27.9 million for the quarter ended September 30, 2022.
  • General and administrative (G&A) expenses: G&A expenses were $6.9 million for the quarter ended September 30, 2023, compared to $8.2 million for the quarter ended September 30, 2022.

CMS Includes the Patient Activation Measure in its Merit-Based Incentive Payment System Starting in 2024

Retrieved on: 
Friday, December 1, 2023
Health, Practice Management, Health Technology, Other Health, Health Insurance, Managed Care, General Health, Patient-centered outcomes, ANA, National Kidney Foundation, NKF, ASN, Organization, National Alliance, White, AHIP, Elective surgery, Kidney disease, ACC, American Society of Nephrology, American Academy, American Diabetes Association, Patient, American Occupational Therapy Association, PAM, University, American Nurses Association, American Academy of Neurology, CMS, Patient Activation Measure, AAOS, Doctor of Public Health, NAMI, Doctor of Philosophy, Medicare, Society, AAN, Mental disorder, MVP Arena, AOTA, Human, Nursing, MIPS, Phreesia

The Patient Activation Measure (PAM) measures a patient’s knowledge, skills and confidence to self-manage their health.

Key Points: 
  • The Patient Activation Measure (PAM) measures a patient’s knowledge, skills and confidence to self-manage their health.
  • Activated patients feel comfortable advocating for themselves and asking for support from their care team to manage their own health.
  • “It’s incredibly gratifying to see CMS include the Patient Activation Measure Performance Measure in its program as a high priority outcome measure.
  • “As a practice that uses the Patient Activation Measure and participates in MIPS, we're incredibly excited to see that PAM will be a MIPS measure going forward.

Alpine Immune Sciences Reports Third Quarter 2023 Financial Results

Retrieved on: 
Tuesday, November 14, 2023
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, Immunoglobulin G, ASN, PMN, UPCR, Investment, Clinical trial, American Society of Nephrology, APRIL, Glomerular filtration rate, Administration, Patient, TACI, Inflammation, Immunoglobulin A, Neonatal Fc receptor, Lupus, Infection, Research program, BAFF, SLE, ALPN, Immunoglobulin M, MD, Development, EGFR, BCMA, Society, NIH Intramural Research Program, Autoimmunity, Proteinuria, AANEM, Pharmaceutical industry, Sewage treatment, Vaccine, Paint, IgA nephropathy, AbbVie, Alpine

Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for autoimmune and inflammatory diseases, today reported financial results and company highlights for the third quarter ended September 30, 2023.

Key Points: 
  • Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for autoimmune and inflammatory diseases, today reported financial results and company highlights for the third quarter ended September 30, 2023.
  • “Following our data presentation, we executed a follow-on equity offering of $150 million to bolster our balance sheet.
  • Cash Position and Runway: As of September 30, 2023, Alpine’s cash and investments totaled $227.2 million compared to $273.4 million as of December 31, 2022.
  • The respective increases of $0.8 million and $2.3 million were primarily attributable to increases in personnel and legal costs.

Calliditas' partner Everest Medicines announces China NMPA's approval of Nefecon® for the treatment of primary IgA nephropathy

Retrieved on: 
Friday, November 24, 2023
National Medical Products Administration, NDA, HKEX, Sustainability, ASN, Prevalence, CALT, Risk, NMPA, Disease, American Society of Nephrology, Patient, CET, Mount Everest, New Drug Application, EGFR, Microhematuria, Society, HIV disease progression rates, Proteinuria, Pharmaceutical industry, IgA nephropathy

China has the highest prevalence of primary glomerular diseases in the world with an estimated five million IgAN patients.

Key Points: 
  • China has the highest prevalence of primary glomerular diseases in the world with an estimated five million IgAN patients.
  • There is a very significant unmet medical need for novel therapies among IgAN patients in China and other Asian countries.
  • Everest also announced that, in addition to Nefecon's approval in mainland China and Macau, it expects a decision on New Drug Application (NDA) approval in Singapore and Hong Kong in the near term.
  • This approval triggers a milestone payment of five million USD to Calliditas, which will be included as revenue in the fourth quarter.

Calliditas' partner Everest Medicines announces China NMPA's approval of Nefecon® for the treatment of primary IgA nephropathy

Retrieved on: 
Friday, November 24, 2023
National Medical Products Administration, NDA, HKEX, Sustainability, ASN, Prevalence, CALT, Risk, NMPA, Disease, American Society of Nephrology, Patient, CET, Mount Everest, New Drug Application, EGFR, Microhematuria, Society, HIV disease progression rates, Proteinuria, Pharmaceutical industry, IgA nephropathy

China has the highest prevalence of primary glomerular diseases in the world with an estimated five million IgAN patients.

Key Points: 
  • China has the highest prevalence of primary glomerular diseases in the world with an estimated five million IgAN patients.
  • There is a very significant unmet medical need for novel therapies among IgAN patients in China and other Asian countries.
  • Everest also announced that, in addition to Nefecon's approval in mainland China and Macau, it expects a decision on New Drug Application (NDA) approval in Singapore and Hong Kong in the near term.
  • This approval triggers a milestone payment of five million USD to Calliditas, which will be included as revenue in the fourth quarter.

Vera Therapeutics Provides Business Update and Reports Third Quarter Financial Results

Retrieved on: 
Thursday, November 9, 2023
APRIL, Hematuria, EGFR, Quarter, Society, American Society of Nephrology, BAFF, Disease, Risk, Serum, Kidney disease, Kidney, Vera, Therapy, Patient, Death, Immunoglobulin A, Proteinuria, Pharmaceutical industry, Cryptocurrency, IgA nephropathy

“Vera presented additional positive data from the Phase 2b ORIGIN trial at Kidney Week 2023, which support the potential of atacicept as a disease-modifying treatment for patients with IgAN.

Key Points: 
  • “Vera presented additional positive data from the Phase 2b ORIGIN trial at Kidney Week 2023, which support the potential of atacicept as a disease-modifying treatment for patients with IgAN.
  • We showed that patients on atacicept achieved durable and significant Gd-IgA1 reduction over 36 weeks regardless of baseline quartile.
  • High serum levels of galactose-deficient IgA1 (Gd-IgA1) are associated with greater risk of end-stage renal disease or death.
  • Vera reported $159.9 million in cash, cash equivalents, and marketable securities as of September 30, 2023.

Equillium Reports Third Quarter 2023 Financial Results and Provides Corporate and Clinical Updates

Retrieved on: 
Wednesday, November 8, 2023
Oncology, Health, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, American College, Lupus nephritis, Harvard Medical School, Education, Gastrointestinal tract, Itolizumab, MPH, Therapy, MPA, Dermatology, G&A, Organic acid, MAD, SAD, R, Society, American Society of Nephrology, ASN, Partnership, Abstract, American College of Rheumatology, UPCR, Investment, Patient, ACR, Alopecia areata, Pharmaceutical industry, MD, Nephrology

Equillium , Inc. (Nasdaq: EQ), a clinical-stage biotechnology company leveraging a deep understanding of immunobiology to develop novel therapeutics to treat severe autoimmune and inflammatory disorders, today announced financial results for the third quarter 2023 and provided corporate and clinical development updates.

Key Points: 
  • Equillium , Inc. (Nasdaq: EQ), a clinical-stage biotechnology company leveraging a deep understanding of immunobiology to develop novel therapeutics to treat severe autoimmune and inflammatory disorders, today announced financial results for the third quarter 2023 and provided corporate and clinical development updates.
  • “The third quarter was focused on clinical execution and expanding education on our multi-cytokine platform and clinical programs ahead of initial data expected later this year,” said Bruce Steel, chief executive officer at Equillium.
  • Cash, cash equivalents and short-term investments totaled $46.3 million as of September 30, 2023, compared to $48.4 million as of June 30, 2023.
  • Net cash used in operating activities in the third quarter of 2023 was $1.9 million.