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Innovative Drug R&D newsletter:Pfizer’s TALZENNA apporved by FDA

1. Pfizer’s TALZENNA® in Combination with XTANDI® Receives U.S. FDA Approval

Pfizer announced that the U.S. FDA has approved TALZENNA (talazoparib) in combination with XTANDI (enzalutamide) for the treatment of adult patients with homologous recombination repair (HRR) gene-mutated metastatic castration-resistant prostate cancer (mCRPC). This approval is based on the statistically significant and clinically meaningful rPFS data from the Phase 3 TALAPRO-2 trial, which demonstrated a 55% reduction in the risk of disease progression or death in patients with mCRPC with prospectively identified HRR gene mutations (ATM, ATR, BRCA1, BRCA2, CDK12, CHEK2, FANCA, MLH1, MRE11A, NBN, PALB2, or RAD51C) treated with TALZENNA plus XTANDI vs placebo plus XTANDI (HR 0.45; 95% CI, 0.33–0.61; p<0.0001).

2. U.S. FDA Approves First Anti-Inflammatory Drug for Cardiovascular Disease

AGEPHA Pharma announced that the U.S. FDA has approved LODOCO (colchicine, 0.5 mg tablet) as the first anti-inflammatory atheroprotective cardiovascular treatment demonstrated to reduce the risk of myocardial infarction (MI), stroke, coronary revascularization, and cardiovascular death in adult patients with established atherosclerotic disease or with multiple risk factors for cardiovascular disease. LODOCO reduces cardiac event risk in adult patients with established atherosclerotic cardiovascular disease (ASCVD) by an additional 31% on top of standard of care.

3. Geron Announces Submission of New Drug Application to FDA for First-in-Class Telomerase Inhibitor Imetelstat

Geron Corporation announced the submission to theFDA of a New Drug Application (NDA) for imetelstat for the treatment of transfusion-dependent anemia in adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) who have failed to respond or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESAs). The NDA submission is based on results from IMerge Phase 3, in which the primary endpoint of 8-week transfusion independence (TI) was significantly higher with imetelstat vs. placebo (P<0.001), with median TI duration approaching one year for imetelstat 8-week TI responders. Mean hemoglobin levels in imetelstat-treated patients increased significantly (P<0.001) over time compared to placebo patients. Further, statistically significant and clinically meaningful efficacy results were achieved across key MDS subgroups: ring sideroblast (RS) status, baseline transfusion burden and IPSS risk category. Safety results were consistent with prior imetelstat clinical experience.

4 Precigen Receives Breakthrough Therapy Designation for PRGN-2012 AdenoVerse™ Immunotherapy for the Treatment of Recurrent Respiratory Papillomatosis

Precigen announced that the US FDA has granted Breakthrough Therapy Designation for PRGN-2012 AdenoVerse immunotherapy for the treatment of recurrent respiratory papillomatosis (RRP). The BTD based on the Phase 1 study (NCT04724980) in patients who had an average of 5.8 RRP surgeries (range 3 – 10) in the year prior to PRGN-2012 treatment. PRGN-2012 treatment resulted in 50% of patients (6/12) in Complete Response, requiring no post-treatment surgeries with a minimum follow up of 12 months. PRGN-2012 treatment resulted in a reduction of surgeries in 83% (10/12) patients in the 12 months following treatment.

5 Aardvark Therapeutics Announces FDA Orphan Drug Designation Granted to ARD-101, a Novel Drug Candidate for Prader-Willi Syndrome

Aardvark Therapeutics reported receipt of an Orphan Drug Designation from the FDA for ARD-101, an oral small molecule bitter taste receptor (TAS2R) agonist. FDA decision is based on early results of an ongoing Phase 2 trial of oral ARD-101 in young adults with PWS.

6 Merck Provides Update on Phase 3 KEYNOTE-585 Trial in Locally Advanced Resectable Gastric and Gastroesophageal Junction (GEJ) Adenocarcinomae

MSD announced topline results from the Phase 3 KEYNOTE-585 trial, investigating KEYTRUDA in combination with chemotherapy as neoadjuvant treatment, followed by adjuvant treatment with KEYTRUDA plus chemotherapy, then KEYTRUDA monotherapy in patients with locally advanced resectable gastric and gastroesophageal junction (GEJ) adenocarcinoma. At a pre-specified interim analysis conducted by an independent Data Monitoring Committee, the study met one of its primary endpoints of pCR rate and demonstrated a statistically significant improvement in pCR rates compared with chemotherapy alone. For the primary endpoint of EFS, there was an improvement in the KEYTRUDA arm; however, results did not meet statistical significance per the pre-specified statistical analysis plan. The endpoint of OS was not formally tested since superiority was not reached for EFS.

7 Escient Pharmaceuticals Announces Positive Results from Phase 1 Study of EP262, a First-in-Class Oral MRGPRX2 Antagonist for Mast Cell Mediated Disorders

Escient Pharmaceuticals announced results from a Phase 1 first-in-human study of EP262, a potent, highly selective small molecule antagonist of MRGPRX2. In the Phase 1 study, which enrolled 64 healthy volunteers, EP262 was safe and well-tolerated at all doses tested, at exposures several fold above doses required for efficacy in preclinical disease models. TEAEs for EP262 were all mild, with an incidence that was lower than placebo (33.3% vs. 62.5%) and did not increase with dose. Clinical proof-of-concept studies in chronic inducible urticaria, chronic spontaneous urticaria and atopic dermatitis to start in second half of 2023.



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