PH1

Peak Bio, Inc. Awarded Patent with Broad Coverage for Thailanstatin-ADC platform technology (PH-1) a Payload with Novel Immunomodulatory Effects that Target RNA Splicing

Retrieved on: 
Thursday, January 4, 2024
Doctor of Pharmacy, Conference, Family, SF3B1, PCT, Spacecraft, Patent, PH-1, MBA, Cancer, OTCQB, Interim, European Patent Convention, Patient, Therapy, ADC, PH1, Pharmaceutical industry

Pleasanton, CA, Jan. 04, 2024 (GLOBE NEWSWIRE) -- Peak Bio, Inc. ("Peak Bio" or the "Company") (OTCQB) PK: PKBO), announced that it was awarded US Patent No.

Key Points: 
  • Pleasanton, CA, Jan. 04, 2024 (GLOBE NEWSWIRE) -- Peak Bio, Inc. ("Peak Bio" or the "Company") (OTCQB) PK: PKBO), announced that it was awarded US Patent No.
  • The new patent describes properties of potent heteroaryl-substituted Thailanstatin payloads, proprietary non-cleavable and cleavable linkers, and antibody-drug conjugate (ADC) technology derived from it.
  • The patent granted by the USPTO covers claims for the Company’s potent Thailanstatin payload platform specific to the spliceosome protein, SF3B1.
  • This patent application is a divisional patent of the Company’s R&D toxin portfolio on Thailanstatin, termed PH-1, building on the portfolio of previously issued patents (Patent No.

FDA approves Rivfloza™ for children ≥9 years old and adults living with primary hyperoxaluria type 1 (PH1), a rare genetic condition

Retrieved on: 
Monday, October 2, 2023
Senior, Novo Nordisk, Nordisk, PH1, Metabolic disorder, Research, Patient, PH, AUC, Liver, RNA interference, Rare, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, DSM-IV codes, Pharmaceutical industry

We look forward to making our first RNAi treatment available to people living with PH1 and the healthcare professionals partnering on their care."

Key Points: 
  • We look forward to making our first RNAi treatment available to people living with PH1 and the healthcare professionals partnering on their care."
  • "Using the GalXC™ RNAi platform, RivflozaTM targets the liver-specific lactate dehydrogenase enzyme, which is the final step of oxalate production in PH1."
  • RivflozaTM, the first RNAi therapeutic by Novo Nordisk, was developed using the proprietary GalXCTM RNAi technology platform.
  • It is not known if RivflozaTM is safe and effective in children younger than 9 years of age.

Peak Bio, Inc. Announces Filing of Provisional Patent Applications for PH-1, a Novel and Specific Immunomodulatory ADC payload platform targeting RNA splicing

Retrieved on: 
Tuesday, September 19, 2023
Patent, AACR, Doctor of Pharmacy, Therapy, United States Patent and Trademark Office, CMC, SF3B1, PH1, Cancer, MBA, Biological patents in the United States, Patient, PH-1, OTC, ADC, Spacecraft, Pharmaceutical industry, Fine chemical

Peak Bio, Inc. today announced that it has filed provisional patent applications with the United States Patent and Trademark Office (USPTO) covering claims for the Company’s potent Thailanstatin payload platform specific to the spliceosome protein, SF3B1.

Key Points: 
  • Peak Bio, Inc. today announced that it has filed provisional patent applications with the United States Patent and Trademark Office (USPTO) covering claims for the Company’s potent Thailanstatin payload platform specific to the spliceosome protein, SF3B1.
  • These patent applications are the second set of applications related to the Company’s R&D toxin portfolio on Thailanstatin, termed PH-1.
  • Previously, the USPTO had issued a patent (Patent No.
  • US 10,815,246 B2) covering potent Thailanstatin drug payloads, non-cleavable and cleavable linkers, and antibody-drug conjugate (ADC) technology derived from it.

Peak Bio, Inc. Presents Preclinical Data Highlighting Potential of Differentiated Trop2 PH1 (Novel Payload) ADC in Poster at 2023 AACR Annual Meeting

Retrieved on: 
Wednesday, August 23, 2023
Heart, AACR, Doctor of Pharmacy, Therapy, Preclinical imaging, Peak, PH1, MBA, Antibody, MDR, Immune system, Cancer, OTC, FIC, ADC, DAR, Poster, Spacecraft, POC, TGI, IND, Stomach, TACSTD2, Pharmaceutical industry, Fine chemical

In addition, Peak Bio’s lead payload appears to be a poor substrate for multi-drug resistance (MDR) transporters which is a growing concern in the ADC field.

Key Points: 
  • In addition, Peak Bio’s lead payload appears to be a poor substrate for multi-drug resistance (MDR) transporters which is a growing concern in the ADC field.
  • After some encouraging proof-of-concept (POC) data for Peak Bio’s proprietary ADC approach was presented at AACR 2021, the Company was motivated to explore a pipeline of potentially differentiated ADCs as investigational therapeutics in oncology.
  • Trop2 ADCs, conjugated with PH1 toxin at a drug-to-antibody (DAR) ratio of 4, demonstrated single digit nanomolar potency in eight different solid tumor indications.
  • A proprietary Trop2 PH1 ADC (antibody-linker-toxin), had equal or better on-target potency than first-in-class (FIC) Trop2 ADC.

Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2022 Financial Results and Highlights Recent Period Activity

Retrieved on: 
Thursday, February 23, 2023
Science, Other Science, Biotechnology, Research, Pharmaceutical, Health, Genetics, Clinical Trials, TTR, Randomness, Brazilian Health Regulatory Agency, PDUFA, Patisiran, LDL, Polyneuropathy, ATTR, Hypercholesterolemia, Agency, European Medicines Agency, Amyloidosis, FDA, Patient, Bachelor of Medicine, Bachelor of Surgery, CNS, RTE, Growth, Plasma, Therapy, ANVISA, EMA, Type, Cardiomyopathy, Medication, KARDIA, Prescription Drug User Fee Act, Novartis, PH1, RNA interference, Pharmaceutical industry, Video game, Pigment, Alnylam Pharmaceuticals

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the fourth quarter and full year ended December 31, 2022 and reviewed recent business highlights.

Key Points: 
  • Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the fourth quarter and full year ended December 31, 2022 and reviewed recent business highlights.
  • Ranked #2 in Science Magazine’s 2022 Top Employer survey, marking the fourth year Alnylam was featured as one of the top three companies.
  • Alnylam intends to report preliminary topline results from the Phase 1 study of ALN-APP in patients with early onset Alzheimer’s Disease.
  • Financial Results for the Quarter and Year Ended December 31, 2022

Precision BioSciences Recaps 2022 Accomplishments and Outlines 2023 Corporate Priorities and Planned Portfolio Milestones

Retrieved on: 
Monday, January 9, 2023
Science, Other Science, Biotechnology, Research, Pharmaceutical, Oncology, Health, Genetics, IND, Cardiovascular disease, Non-Hodgkin lymphoma, PHH, Lilly, University, Kidney disease, Tissue, Primate, OTC, HBV, CMC, Duchenne muscular dystrophy, Trial of the century, Vivo, Novartis, LNP, Safety, Degenerative disease, FDA, Data, Gene editing, Hepatitis B virus, Completeness, Partnership, Stem cell, DMD, CAR, Hepatocyte, Patient, Mouse, Chromosomal translocation, NHL, Precision, CTA, PH1, FH, Sickle cell disease, Infant, Pharmaceutical industry, Risk management, Vaccine, Antibiotics

Precision BioSciences (Nasdaq: DTIL), a clinical stage gene editing company developing ARCUS®-based ex vivo allogeneic CAR T and in vivo gene editing therapies, today reported on its significant accomplishments in 2022 and announced its corporate priorities and anticipated clinical development and research milestones for 2023.

Key Points: 
  • Precision BioSciences (Nasdaq: DTIL), a clinical stage gene editing company developing ARCUS®-based ex vivo allogeneic CAR T and in vivo gene editing therapies, today reported on its significant accomplishments in 2022 and announced its corporate priorities and anticipated clinical development and research milestones for 2023.
  • “In 2022, we advanced our corporate priorities strengthening the company and made significant progress with our clinical programs and research pipeline.
  • Progress azer-cel to decision point for Phase 2 trial in non-Hodgkin lymphoma (NHL) subjects who have relapsed following autologous CAR T treatment.
  • Present CAR T clinical update in the first quarter of 2023, based on patient accrual and follow-up.

Chinook Therapeutics Provides Business Update and Reports Third Quarter 2022 Financial Results

Retrieved on: 
Thursday, November 10, 2022
LDHA, Lilly, ETA, Method, Serum, CKD, Research, Therapy, Immunoglobulin A, Kidney, EGFR, Chronic kidney disease, SC, Blood pressure, Industry, Blood, 1836 U.S. Patent Office fire, Forward-looking statement, Primary hyperoxaluria, United States Patent and Trademark Office, PH1, Patent, RAS, BNP, European Commission, RNA, SEC, PH2, Cohort, Alport syndrome, Patient, Urine, Tax, COVID-19, Immunoglobulin G, Immunoglobulin M, Nasdaq, Achievement, Clinical trial, Poster, Focal segmental glomerulosclerosis, Trial of the century, GLOBE, FSGS, New Drug Application, ACE, MAD, SAD, SGLT2, Part, Proteinuria, Royal commission, Biomarker, Disease, CVR, Nature, Pharmaceutical industry, Vaccine, Television, UPCR, Chinook, Merck, Evotec, IgA nephropathy, Alport, Atrasentan

SEATTLE, Nov. 10, 2022 (GLOBE NEWSWIRE) -- Chinook Therapeutics, Inc. (Nasdaq: KDNY), a biopharmaceutical company focused on the discovery, development and commercialization of precision medicines for kidney diseases, today provided a business update and reported financial results for the quarter and nine months ended September 30, 2022.

Key Points: 
  • SEATTLE, Nov. 10, 2022 (GLOBE NEWSWIRE) -- Chinook Therapeutics, Inc. (Nasdaq: KDNY), a biopharmaceutical company focused on the discovery, development and commercialization of precision medicines for kidney diseases, today provided a business update and reported financial results for the quarter and nine months ended September 30, 2022.
  • During the third quarter of 2022, we continued advancing our pipeline of clinical and preclinical programs for rare, severe chronic kidney diseases.
  • Quarter and Nine Months Ended September 30, 2022 Financial Results
    Cash Position Cash, cash equivalents and marketable securities totaled $397.7 million at September 30, 2022, compared to $355.1 million at December 31, 2021.
  • Chinook assumes no obligation to update forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

BridgeBio Pharma Announces Positive Phase 1 Data and Phase 2/3 Trial Design for BBP-711, a Potentially Best-In-Class GO Inhibitor for Primary Hyperoxaluria Type 1 (PH1) and Recurrent Kidney Stone Formers

Retrieved on: 
Monday, June 27, 2022
Annual report, Science, Patient, Lists of diseases, LinkedIn, Nephrocalcinosis, Securities Act of 1933, Securities Exchange Act of 1934, Adult, BSA, Liver transplantation, UOX, GLOBE, Hyperoxaluria, COVID-19, Company, U.S. Securities and Exchange Commission, Disease, Plasma, PD, Part, Kidney, Vital signs, PH1, Cancer, ULN, Clinical trial, Drug Quality and Security Act, Half-life, ECG, Nasdaq, Environment, Calcification, SEC, ESPN, Risk, European Society for Primary Immunodeficiencies, Calcium oxalate, Time, Primary hyperoxaluria, Twitter, Body surface area, Therapy, Pharmaceutical industry

PALO ALTO, Calif., June 27, 2022 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced positive Phase 1 data for BBP-711 in healthy volunteers, supporting the development of the investigational therapy for patients with primary hyperoxaluria type 1 (PH1) and recurrent kidney stone formers. The data were shared in a feature oral presentation at European Society for Pediatric Nephrology (ESPN) 2022, taking place in Ljubljana, Slovenia.

Key Points: 
  • BBP-711 is an orally-administered small molecule inhibitor of glycolate oxidase (GO) that is being developed to treat conditions of excess oxalate.
  • Overproduction of oxalate in hyperoxaluria, including PH1 and recurrent kidney stone formers with elevated oxalate, can lead to kidney stone formation, nephrocalcinosis and renal impairment.
  • These data are encouraging for patients with PH1 as BBP-711 could become a novel oral therapy to prevent hyperoxaluria and its long-term consequences.
  • Moreover, BridgeBio operates in a very competitive and rapidly changing environment in which new risks emerge from time to time.

Precision BioSciences Announces Preclinical Data Showcasing Premier In Vivo Gene Editing Capabilities at American Society of Gene & Cell Therapy Annual Meeting

Retrieved on: 
Monday, May 16, 2022
Science, Biotechnology, Research, Pharmaceutical, Health, Infectious Diseases, Genetics, Clinical Trials, RNA transfection, HBV, Lactic acidosis, Washington Convention Center, Liver, Mitochondrial encephalomyopathy, CccDNA, Private Securities Litigation Reform Act, Animal, Mitochondrion, CAR, SEC, LNP, HAO1, Annual report, Serum, Investor, COVID-19, WT, Research, Mouse, MELAS, Growth, Achievement, DNA, Genome, Protein engineering, PH1, Infection, Insurance, PCSK9, Abstract (summary), Gene editing, Hepatitis B, Adult, Ownership, Organic acid, CTA, List, Walter E. Washington Convention Center, Hepatitis B virus, Therapy, Cell, NHP, A-DNA, PHH, Nasdaq, Ornithine transcarbamylase deficiency, Patient, Poster, OTC, Society, Privacy, HAO, Gene, AAV, Intellectual property, Forward-looking statement, Compliance, Walter, Re Recher's Will Trusts, Safety, Technology, SEC filing, Infant, Hepatocyte, Hepatitis, Mutation, Vaccine, D.C, HBsAg, IND

Data from this preclinical study demonstrate Precisions gene editing approach designed to eliminate hepatitis B virus (HBV).

Key Points: 
  • Data from this preclinical study demonstrate Precisions gene editing approach designed to eliminate hepatitis B virus (HBV).
  • These data suggest that LNP-delivered ARCUS mRNA is a promising approach and potential functional cure for chronic hepatitis B.
  • Precision will continue developing its PBGENE-HBV product candidate using LNP delivery and expects to submit an IND/CTA in 2024.
  • Preclinical data presented in this poster demonstrate Precisions gene editing approach to shift mitochondrial DNA (mtDNA) heteroplasmy for the m.3243A>G mtDNA mutation.

Chinook Therapeutics Announces Initiation of Phase 1 Healthy Volunteer Trial of CHK-336, a First-in-Class LDHA Inhibitor to Treat Hyperoxalurias

Retrieved on: 
Tuesday, April 12, 2022
Atrasentan, Calcium, Kidney, Clinical trial, Pharmacokinetics, Kidney failure, COVID-19, Adult, Time, NASDAQ, Chronic kidney disease, Human, RNA, Primary hyperoxaluria, GLOBE, Calcium oxalate, Program, Patient, Preclinical development, PH2, Dialysis, LDHA, IgA nephropathy, SEC, Conditional sentence, Forward-looking statement, Safety, Immunoglobulin A, PH1, Disorder, Industry, Hematuria, Urinary tract infection, Liver, Therapy, Pharmaceutical industry, Chinook

CHK-336 exemplifies Chinooks commitment to discovering and developing novel precision medicines for rare, severe chronic kidney diseases with defined genetic and molecular drivers.

Key Points: 
  • CHK-336 exemplifies Chinooks commitment to discovering and developing novel precision medicines for rare, severe chronic kidney diseases with defined genetic and molecular drivers.
  • The phase 1, single-center, randomized, placebo-controlled double-blind single and multiple ascending dose clinical trial is designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of CHK-336 in up to 104 healthy volunteers.
  • BION-1301, an anti-APRIL monoclonal antibody is being evaluated in a phase 1/2 trial for IgA nephropathy.
  • CHK-336, an oral small molecule LDHA inhibitor for the treatment of hyperoxalurias, is being evaluated in a phase 1 healthy volunteer trial.