Pharma Frontiers: Daily Digest of Global Pharmaceutical News – Sep 19

1.Novartis CDK4/6 Inhibitor New Indication Approved by FDA for Breast Cancer Adjuvant Therapy 

On September 18, Novartis announced that the U.S. FDA has approved Kisqali (ribociclib), a CDK4/6 inhibitor, in combination with an aromatase inhibitor (AI) as adjuvant therapy for patients with high recurrence risk hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) stage II and III early breast cancer (EBC), including those without lymph node metastasis (N0). This approval was based on the positive results of the pivotal phase 3 clinical trial NATALEE, which demonstrated a clinically meaningful and significant reduction of 25.1% in the risk of recurrence (HR=0.749; 95% CI: 0.628, 0.892; P=0.0006) for patients receiving Kisqali combined with endocrine therapy (ET) as adjuvant treatment compared to those receiving only ET, including patients with high-risk N0 status. Improvements in invasive disease-free survival (iDFS) were consistently observed across all patient subgroups.

2.FDA Approves New Indication for Pembrolizumab in Malignant Pleural Mesothelioma 

On September 17, the FDA's website announced that Merck's pembrolizumab (brand name: Keytruda, also known as K-drug) has been approved for a new indication for use in combination with pemetrexed and platinum-based chemotherapy as a first-line treatment for unresectable advanced or metastatic malignant pleural mesothelioma (MPM). This approval was primarily based on the positive outcomes of the phase III KEYNOTE-483 study. The results showed that the median overall survival (OS) was significantly extended in the K-drug group compared to the control group (17.3 vs. 16.1 months, HR=0.79, p=0.0162), with a median progression-free survival (PFS) also significantly lengthened (7.1 vs. 7.1 months, HR=0.80, p=0.0194). Additionally, the confirmed objective response rate (cORR) was higher (52% vs. 29%) and the median duration of response (DOR) was longer (6.9 vs. 6.8 months) in the K-drug group. Presently, only three innovative therapies, including nivolumab (BMS/Ono Pharmaceutical), TTFields (Novocure/Zai Lab), and K-drug, have been approved globally for the treatment of MPM.

3.Daiichi Sankyo/Merck's HER3 ADC Clinical Trial Meets Endpoint, Preparing for Market Application 

On September 18th, Daiichi Sankyo and Merck jointly announced positive results from the Phase 3 clinical trial HERTHENA-Lung02 of their ADC collaboration product, patritumab deruxtecan (HER3-DXd). This study evaluated HER3-DXd in patients with locally advanced or metastatic NSCLC harboring EGFR mutations who had previously received EGFR-TKI therapy. Analysis indicated that the trial met its primary endpoint, demonstrating a statistically significant improvement in PFS compared to platinum plus Pemetrexed induction chemotherapy followed by maintenance therapy with Pemetrexed. OS data were not yet mature at the time of analysis, and the trial will continue to assess the secondary endpoint OS. The observed safety profile was consistent with that seen in previous clinical trials, with no new safety signals identified. Both companies stated that they would engage in further discussions with regulatory authorities worldwide concerning the results of this trial.

4.Boehringer Ingelheim's PDE4B Inhibitor Clinical Trial Successful, Preparing for Market Application

On September 17th, Boehringer Ingelheim announced that its investigational small molecule therapy nerandomilast achieved the primary endpoint in the Phase 3 FIBRONEER-IPF clinical trial, significantly improving lung function in patients with idiopathic pulmonary fibrosis (IPF). Based on these results, Boehringer Ingelheim plans to submit a New Drug Application (NDA) to the FDA and global regulatory bodies for the use of nerandomilast in treating IPF. Nerandomilast, an oral phosphodiesterase-4B (PDE4B) inhibitor, was granted Breakthrough Therapy Designation (BTD) by the FDA in February 2022. This clinical trial marks the first Phase 3 trial in the IPF field to meet its primary endpoint in a decade. Detailed efficacy and safety trial data are expected to be published in the first half of 2025.

5.Infigratinib Granted FDA Breakthrough Therapy Designation

On September 17th, BridgeBio Pharma announced that its investigational oral drug, infigratinib, has been granted Breakthrough Therapy designation by the U.S. FDA. This medication works by inhibiting the activity of FGFR3 and is intended to treat children suffering from achondroplasia. The press release noted that this is the first Breakthrough Therapy designation given by the FDA for a treatment intended for achondroplasia. The designation was awarded based on preliminary clinical evidence from the Phase 2 clinical trial, PROPEL 2. In the trial’s fifth cohort, at a dosage of 0.25 mg/kg/day, oral administration of infigratinib led to a significant and sustained increase in patients' annualized height velocity (AHV). At 12 months, the average increase in AHV relative to baseline was +2.51 cm/year, and at 18 months, +2.50 cm/year, with the increases being statistically significant (p=0.0015).

6.Nura Bio Completes $68 Million Series A Expansion Financing for Broad-Spectrum Neurological Diseases

On September 17th, Nura Bio announced the completion of a $68 million Series A expansion financing, bringing the total for the Series A round to $140 million. This funding round was led by founding investor The Column Group, with participation from existing investors Samsara Bio Capital and Euclidean Capital, as well as new investor Sanofi Ventures. The company's leading investigative therapy, NB-4746, is a brain-penetrant SARM1 inhibitor that has shown potential to prevent axonal degeneration early on and holds vast potential in the treatment of various neurological diseases. NB-4746 has successfully completed a Phase 1 clinical trial in healthy volunteers. Nura Bio plans to initiate a Phase 1b/2 clinical trial in patient populations by 2025.

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!