- **Revolution Medicines** announced that its **RAS(ON) multi-selective inhibitor, daraxonrasib**, has been granted **Orphan Drug Designation** by the U.S. Food and Drug Administration (FDA) for the treatment of **pancreatic cancer**.  **[1678660_72c87e65902e46b3ab623c71d1ba67a9_0]** 
- This designation highlights pancreatic cancer as a **devastating disease with limited therapeutic options** and a **large unmet medical need**, with RAS driver mutations present in nearly all cases.  **[1678660_72c87e65902e46b3ab623c71d1ba67a9_0]** 
- Daraxonrasib is currently being studied in a **global Phase 3 clinical trial (RASolute 302)** for patients with second-line metastatic pancreatic ductal adenocarcinoma (PDAC), and the company plans to initiate two additional Phase 3 trials for first-line and adjuvant treatment in pancreatic cancer.  **[1678660_72c87e65902e46b3ab623c71d1ba67a9_0]** 
- The Orphan Drug Designation provides significant incentives, including **tax credits for clinical trial costs**, **exemption from certain FDA fees**, and up to **seven years of market exclusivity** following approval.  **[1678660_72c87e65902e46b3ab623c71d1ba67a9_0]**

Revolution Medicines' Daraxonrasib Receives FDA Orphan Drug Designation for Pancreatic Cancer

- Perspective Therapeutics presented updated interim results from its Phase 1/2a clinical trial of **[212Pb]VMT-α-NET** at the ESMO Congress 2025, with a data cut-off date of **September 12, 2025**.  **[1678660_096a6a235263465ab13955577b97d786_0]** 
- The drug candidate demonstrated continued **good tolerability** across **55 patients**, with no dose limiting toxicities or Grade 4 or 5 treatment emergent adverse events reported.  **[1678660_096a6a235263465ab13955577b97d786_0]** 
- Efficacy data showed an Objective Response Rate of **44% (7 of 16 patients)** in Cohort 2 (patients with SSTR2 expression in all tumors), with **14 of these 16 patients** remaining free from progression and on study at a median follow-up of **41 weeks**.  **[1678660_096a6a235263465ab13955577b97d786_0]** **[1678660_096a6a235263465ab13955577b97d786_1]** 
- The company expects initial efficacy data for an additional **23 patients in Cohort 2 and 8 patients in Cohort 3** to be presented during **2026**.  **[1678660_096a6a235263465ab13955577b97d786_0]** **[1678660_096a6a235263465ab13955577b97d786_1]** 
- As of **September 30, 2025**, Perspective Therapeutics had approximately **$174.1 million** in cash, cash equivalents, and short-term investments, which is expected to fund operations into **late 2026**.  **[1678660_096a6a235263465ab13955577b97d786_2]** **[1678660_096a6a235263465ab13955577b97d786_3]**

Perspective Therapeutics Presents Updated Interim Clinical Trial Data for [212Pb]VMT-α-NET

- **Prilenia and Ferrer** announced the presentation of new 2-year data for pridopidine at the 2025 HSG HD Clinical Research Congress, demonstrating **significant and clinically meaningful slowing of progression** in Huntington's Disease (HD) patients not taking antidopaminergic medicines (ADMs).  **[1678660_9e6f31f89b3f41719ab040b88e07abf3_0]** 
- The data showed slowing of progression by **65%** for TFC and **59%** for cUHDRS, **88%** for cognition (SWR), and **78%** for motor features (Q-Motor) for pridopidine-treated patients off ADMs over 2 years.  **[1678660_9e6f31f89b3f41719ab040b88e07abf3_2]** 
- A global confirmatory clinical trial for pridopidine in early-stage HD patients is planned to commence in the **first half of 2026**.  **[1678660_9e6f31f89b3f41719ab040b88e07abf3_0]** **[1678660_9e6f31f89b3f41719ab040b88e07abf3_1]** 
- Pridopidine has received **Orphan Drug Designation** in HD and ALS in the US and EU, and **FDA Fast Track Designation** for the treatment of HD.  **[1678660_9e6f31f89b3f41719ab040b88e07abf3_3]**

Prelude Therapeutics (PRLD)