PANTHERx Rare Pharmacy’s Post

Targeted pharmacological inhibition of cell cycle regulators has long been considered a promising anti-cancer strategy. Recent advances in our understanding of the molecular basis of cell cycle disruption and the mechanisms of resistance have reignited interest in blocking specific components of the cell cycle machinery. Check out this article to learn about the latest developments in the field. #cancerresearch #pharmacology #cellcycleinhibition https://lnkd.in/ecBEd-52

Setback for anti-B7-H3 ADC drug against prostate cancer. 50% treatment related adverse events leading to high drop out rates. Fierce - " MacroGenics reported five deaths. Investigators deemed two cardiovascular deaths, one in each arm, to be unrelated to the study drug. Another three deaths, all at the higher dose, are under investigation. Two patients died of pneumonitis, an inflammatory lung condition. " -- Despite recent euphoria over ADC, these therapies have always carried toxicity hazards, and have been the primary barrier to widespread adoption of this treatment modality in the past. Even with reassuring commentary by company during investor earnings call, Macrogenic stock is down 70%, from $14.67 to $4.37 per share. -- Disclaimer- Views expressed here are of the Author only

FCRL5-Directed CAR T-Cell Therapy: A Novel Therapeutic Approach for Multiple Myeloma: CAR T-cells specifically targeting FCRL5, when integrated with IL-15, may improve cytotoxicity, thereby enhancing the therapeutic potency of these cells. #finance #pharmacy #lifesciences

The facioscapulohumeral muscular dystrophy (#FSHD) community received very disappointing news this morning with the release of the negative Phase 3 REACH trial topline results from Fulcrum Therapeutics. While the trial did not detect a benefit from losmapimod, it is particularly relevant to recognize the lack of decline in the placebo group. Thank you to Fulcrum Therapeutics for their commitment to release the full dataset from this trial. In diseases without an existing treatment, like FSHD, clinical trial programs are evaluating the efficacy of the novel treatment but also the sensitivity of the outcome measures in detecting change over the study interval. Failure in either treatment or measures can result in a negative trial. Data from this trial will be essential to understand why the placebo group did not decline so that future trial designs and outcomes can be optimized. Thank you to Fulcrum Therapeutics for their commitment to the FSHD community, for developing losmapimod through to the completion of this Phase 3 clinical trial and for committing to releasing the trial data for the benefit of the community. https://lnkd.in/gTH2jaCP

TiNivo-2 Trial Does Not Meet Progression-Free Survival For Patients With Renal Cell Carcinoma: There were positive efficacy and safety results for tivozanib monotherapy consistent with previous analyses. #finance #pharmacy #lifesciences

Be Biopharma, Inc. has received FDA IND clearance for BE-101, a groundbreaking treatment for hemophilia B. The Phase 1/2 clinical trial, BeCoMe-9, will assess BE-101’s safety and efficacy in adults with hemophilia B, starting in late 2024. BE-101, an autologous BCM, aims to insert the human FIX gene into patients’ B cells, enabling sustained FIX activity with reduced dosing frequency. This innovative treatment could significantly improve bleeding control and joint damage in individuals with hemophilia B. Joanne Smith-Farrell, CEO of Be Bio, highlighted that the IND clearance is a major milestone in their mission to provide advanced care for hemophilia B patients. BE-101 offers the potential to transform patient care by providing lasting FIX protection and reducing infusion burdens. Hemophilia B can result in severe consequences like chronic pain and joint damage. Current treatments require frequent infusions, but BE-101 offers a more durable solution with its unique design and potential for sustained FIX levels with redosability. If proven safe and effective in adults, BE-101 could revolutionize hemophilia B treatment for children, enhancing their quality of life and potentially mitigating long-term effects. Learn more about this game-changing therapy: https://lnkd.in/dYHMnxGe Karen Campbell

Congratulations to our friends at Hansa Biopharma (Hansa) on completing the recruitment and randomization phases of their US ConfldeStrial. ConfldeS is a Phase 3 open-label, randomized, controlled trial evaluating the efficacy of imlifidase in kidney transplantation. The data from this trial plays a pivotal role in supporting the Biologic License Application (BLA) submission to the FDA. We recognize Hansa’s dedication to addressing the unmet needs of highly sensitized kidney transplant patients. Read more about their milestone achievement in the article linked below! #MovingMedicineForward #AdvancingHealthcareSolutions #CRO #ThinkGlobalActLocal #ClinicalResearch #ProudFriends #Hansa

Approval of Erdafitinib - inhibitor of FGFR receptor FDA approves erdafitinib for locally advanced or metastatic urothelial carcinoma | FDA On January 19, 2024, the Food and Drug Administration approved erdafitinib (Balversa, Janssen Biotech) for adult patients with locally advanced or metastatic urothelial carcinoma (mUC) with susceptible FGFR3 genetic alterations, as determined by an FDA-approved companion diagnostic test, whose disease has progressed on or after at least one line of prior systemic therapy. Erdafitinib is not recommended for the treatment of patients who are eligible for and have not received prior PD-1 or PD-L1 inhibitor therapy. This approval amends the indication previously granted under accelerated approval for patients with mUC with susceptible FGFR3 or FGFR2 alterations after prior platinum-containing chemotherapy. https://lnkd.in/dnvPPwZ7

According to DelveInsight’s analysis, the market size of thyroid eye disease in the 7MM was around USD 2.5 billion in 2023. The US accounted for ~40% of the total prevalent cases of thyroid eye disease in the 7MM. Prominent companies like Viridian Therapeutics, Inc., Roche, Immunovant, Amgen, Sling Therapeutics, ACELYRIN, INC., Tourmaline Bio, and Lassen Therapeutics are actively developing innovative drugs for thyroid eye disease. Key therapies in development include VRDN-001, ENSPRYNG (satralizumab), Batoclimab, Teprotumumab, Linsitinib, Lonigutamab, TOUR006 - 20, LASN01, and others. These novel therapies are anticipated to enter the market during the forecast period and are expected to transform the treatment landscape. For more detailed insights: https://lnkd.in/gHpVGgQy #ThyroidEyeDisease #HealthcareInnovation #PharmaNews #MedicalResearch #DelveInsight #MarketAnalysis #ViridianTherapeutics #Roche #Immunovant #Amgen #SlingTherapeutics #ACELYRIN #TourmalineBio #LassenTherapeutics

#DrugHunter #DrugDiscovery #Alzheimer #SmallMolecules #PROTAC #CRO #CMO #FFS #FTE In February, Actelion revealed the discovery story of nivasorexant, the first selective orexin-1 receptor antagonist, which completed a Ph. II clinical trial in binge-eating disorder. Additional molecules to make the top ten include a brain-penetrant S2R modulator currently in clinical development for Alzheimer’s disease and an orally bioavailable BTK degrader that has advanced into first-in-human clinical trials for relapsed/refractory B-cell malignancies. NodThera released exciting new details on their two clinical NLRP3 inflammasome inhibitors NT-0796 and NT-0249, which exhibit similar efficacy to the GLP-1 mimetic semaglutide in reversing pre-established obesity. Also featured this month is cilofexor, an intestinally biased FXR agonist discovered by Phenex Pharmaceuticals, and a potent and selective preclinical CDK2 inhibitor from Incyte.