New Drug Designations - January 2025

Shots:  

PharmaShots’ Designation Report provides a concise overview of the latest drug designations granted by major regulatory authorities, including the FDA, EMA, MHLW, and NMPA

The January 2025 report covers designations granted to 53 drugs and 9 medical devices, spanning 18 small molecules, 13 biologics, 13 cell and gene therapies & 9 medical devices among others

Significant trends this month show, Azafaros’ Nizubaglustat secured Orphan Drug Designation (ODD) from both US FDA & EMA to treat GM1 gangliosidosis

Nizubaglustat – Small Molecule

The FDA and EMA have granted Orphan Drug Designation (ODD) to nizubaglustat for treating GM1 gangliosidosis

Topline data from the P-II trial in GM2 and NPC patients demonstrated favorable safety, with preliminary clinical improvements or stabilization observed in most patients

Clinical Trial Authorization (CTA) approvals have been granted for two global P-III trials evaluating nizubaglustat for GM1/GM2 gangliosidoses & Niemann-Pick Type C (NPC) across multiple European countries. Trial initiation is expected in Q2 2025

NMD670 – Small Molecule

FDA Grants ODD to NMD670 for Charcot-Marie-Tooth Disease (CMT). NMD670 (PO, BID) is being evaluated in the P-IIa SYNAPSE-CMT trial (n=80) for efficacy, safety, and tolerability in adults with CMT1 or CMT2 over 21 days across the US and EU

The trial will assess changes in 6-Minute Walk Test (6MWT), 10-Meter Walk/Run Test (10MW/R), and Timed-Up-and-Go Test (TUG)

NMD Pharma is also conducting three global P-II trials of NMD670 for Spinal Muscular Atrophy (SMA) Type 3, Generalized Myasthenia Gravis (gMG), Charcot-Marie-Tooth Disease (CMT)

Data from these trials is expected between H2 2025 and H1 2026.NMD670, a small molecule CIC-1 inhibitor, enhances muscle responsiveness, neuromuscular transmission, and restores function

Amezalpat (TPST-1120) - Small Molecule

FDA Grants ODD to Amezalpat (TPST-1120) for Hepatocellular Carcinoma (HCC). The designation was based on a P-Ib/II trial evaluating amezalpat + Tecentriq + Avastin vs. Tecentriq + Avastin as 1L therapy for inoperable or metastatic HCC

The trial demonstrated Median Overall Survival (mOS) improvement by 6 months, Objective Response Rate (ORR): 30% vs 13%

Amezalpat also showed a survival benefit in sub-populations, including PD-L1-negative and β-catenin-mutated disease, supporting its MoA of direct tumor targeting and immune system enhancement

ELC-100 – Gene Therapy

FDA Grants ODD to ELC-100 for Pancreatic Neuroendocrine Tumors. Currently being assessed in a P-I/II trial to determine its maximum tolerated dose and safety

Last patient enrollment was completed in Fall 2024, with initial results expected in mid-2025

ELC-100, an oncolytic virus-based therapy, selectively infects and destroys neuroendocrine cancer cells

ALG-801 - Protein

FDA Grants ODD to ALG-801 for Pulmonary Arterial Hypertension (PAH)

ALG-801 demonstrated superior efficacy and safety in preclinical studies and P-Ia/Ib trials involving healthy volunteers. The drug is now advancing into a P-II trial

ALG-801, an ActRIIA/IIB hybrid ligand trap, selectively sequesters Smad2/3 pathway-activating ligands, key drivers of PAH and related diseases

CNP-104

The US FDA has granted ODD to CNP-104 for the treatment of primary biliary cholangitis (PBC) following the release of P-IIa topline data

The P-IIa trial evaluated CNP-104 in PBC patients, demonstrating a reduction in disease progression, defined by a decrease in liver stiffness measured by FibroScan at Day 120. The topline data was presented at The Liver Meeting 2024

CNP-104 induces tolerance to pathogenic activated PDC-E2 T-cells, which are responsible for bile duct inflammation, aiming to improve clinical outcomes in PBC

R289 – Small Molecule

The US FDA has granted ODD to R289 for the treatment of myelodysplastic syndromes (MDS), following the grant of Fast Track Designation (FTD) in December 2024

A P-Ib study is currently evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of R289 in patients with relapsed/refractory (r/r) lower-risk MDS

R289 (a prodrug of R835) is an IRAK1/4 dual inhibitor that blocks inflammatory cytokine production in response to Toll-like receptor (TLR) and interleukin-1 receptor (IL-1R) signaling. Dysregulation of these pathways contributes to inflammatory conditions

Usnoflast (ZYIL1) - Small Molecule

The US FDA has granted ODD to usnoflast for the treatment of amyotrophic lateral sclerosis (ALS)

Usnoflast was evaluated in a P-IIa trial against placebo (PBO) in 24 ALS patients, with data expected to be presented at future scientific meetings and published in journals. Additionally, the US FDA has approved a P-IIb trial for Usnoflast

Usnoflast (ZYIL1) is a novel NLRP3 inhibitor being investigated across multiple preclinical models of neuroinflammation, Parkinson’s disease, inflammatory bowel disease (IBD), and multiple sclerosis (MS)

ZL-1310 - Biologic

The US FDA has granted ODD to Zai Lab’s ZL-1310 for treating small cell lung cancer (SCLC)

The designation is based on promising P-Ia/Ib trial data of ZL-1310 as monotx. & with atezolizumab in treatment-experienced ES-SCLC patients, presented at the ENA Symposium 2024

ZL-1310 is a novel ADC targeting DLL3 expressed in neuroendocrine tumors & a validated SCLC target. It combines a humanized anti-DLL3 antibody with a camptothecin derivative payload using the TMALIN platform to address limitations of first-generation ADCs, such as off-target toxicity

MB-105 – Gene Therapy

The US FDA has granted ODD to MB-105 for the treatment of relapsed/refractory (r/r) CD5+ T-cell lymphoma

The ongoing P-I trial evaluating MB-105 in patients with r/r T-cell lymphoma (TCL) and T-cell acute lymphoblastic leukemia (T-ALL) has shown promising safety and efficacy signals, with a 44% overall response rate (ORR) in TCL patients

March Biosciences has raised $28.4 million in Series A financing to advance MB-105 into P-II development and strengthen manufacturing capabilities for future commercialization

Etomoxir- Small Molecule

The US FDA has granted ODD to etomoxir for the treatment of malignant gliomas

Etomoxir is a CPT1 pathway inhibitor that slows tumor growth, supported by preclinical efficacy and pharmacology-toxicology data

Additionally, Numiera Therapeutics received 2:1 matching funds from the State of Colorado’s Office of Economic Development and International Trade

GIVI-MPCs – Cell Therapy

The US FDA has granted ODD to GIVI-MPCs for the treatment of Becker muscular dystrophy (BMD), enabling the creation of new muscle with full-length dystrophin

Additionally, IPS HEART has proposed a P-I/II trial design to the FDA following the completion of the second pre-IND meeting for GIVI-MPCs

GIVI-MPCs is a stem cell therapy designed to regenerate skeletal muscle with full-length human dystrophin in patients with muscular dystrophy

rAAV2-U1a-hELP1 – Gene Therapy

The US FDA has granted ODD and Rare Pediatric Disease Designation (RPDD) to rAAV2-U1a-hELP1 for the treatment of optic neuropathy in familial dysautonomia (FD)

In a familial dysautonomia (FD) mouse model, intravitreal administration of rAAV2-U1a-hELP1 demonstrated a reduction in retinal ganglion cell (RGC) death

BPN-36964 – Small Molecule

The US FDA has granted ODD and RPDD to BPN-36964 for the systemic treatment of familial dysautonomia (FD)

BPN-36964 efficiently crosses the blood-brain barrier (BBB) and corrects ELP1 mRNA splicing, leading to increased ELP1 levels in the body. This mechanism helps prevent progressive neuronal degeneration, as demonstrated in an FD mouse model

ST-01156

The US FDA has granted RPDD and ODD to ST-01156, a molecular glue targeting solid tumor indications

The Investigational New Drug (IND) filing for ST-01156 is expected in H1 2025, with SEED Therapeutics potentially eligible for an FDA Priority Review Voucher upon approval, following the RPDD grant

Elraglusib – Small Molecule

The EMA has granted ODD to elraglusib for the treatment of pancreatic ductal adenocarcinoma (PDAC)

Elraglusib is currently being evaluated in a P-II trial (Actuate-1801 Part 3B), assessing its combination with gemcitabine/nab-paclitaxel (GnP) versus GnP alone as a 1L therapy for metastatic pancreatic cancer

Interim results from the trial indicate an increase in the one-year survival rate and median overall survival (mOS), with topline data expected in H1 2025

Sebetralstat – Small Molecule

Japan’s MHLW has granted ODD and received the New Drug Application (NDA) for sebetralstat to treat hereditary angioedema (HAE) attacks in patients aged ≥12 years

The NDA submission is supported by data from the ongoing open-label extension (OLE) KONFIDENT-S trial and the P-III KONFIDENT trial. The P-III study evaluated sebetralstat (300 mg/600 mg) vs. placebo (PBO) in 136 patients with HAE types 1 & 2, who experienced ≥2 attacks in 90 days, including those on prophylaxis. Participants received ~2 doses per attack for up to 3 attacks over the trial duration

Additionally, the company is transitioning ongoing trial participants to a novel oral disintegrating tablet (ODT) formulation, aiming to support a supplemental NDA (sNDA) filing in 2026

LOAd703 – Gene Therapy

The US FDA has granted Fast Track Designation (FTD) to LOAd703 for the treatment of pancreatic cancer

LOAd703 is being evaluated in combination with gemcitabine + nab-paclitaxel + anti-PD-L1 antibody for pancreatic cancer with P-II trial underway

LOAd703 engineers the tumor microenvironment (TME) to express immune activators & thereby triggers inflammation for improving patient response to traditional checkpoint blockade immunotherapy

[18F]florbetaben – Radiopharmaceutical

The US FDA has granted FTD to [18F]florbetaben for diagnosis of cardiac amyloid light-chain (AL) & amyloid transthyretin-related (ATTR) amyloidosis

[18F]florbetaben is being evaluated in P-III (CArdiag) trial to assess its efficacy in the diagnosis of cardiac amyloidosis

[18F]florbetaben is a radioactive diagnostic agent for PET imaging to estimate β-amyloid neuritic plaque density in adults with cognitive impairment being evaluated for Alzheimer’s or other causes

Cu-64 SAR-BisPSMA – Radiopharmaceutical

FDA has granted FTD to Cu-64 SAR-bisPSMA for PET scans of PSMA+ lesions in pts with BCR post-receiving SoC, based on a P-I/II (COBRA) trial assessing it (same & next day) in patients who had an SoC scan at study entry

Study showed that it localized disease in ~80% patients & detected double the lesions as small as 2mm the next day. Submission also featured its bivalent bisPSMA structure & longer t½ (12.7 hrs vs <2 hrs for F-18/Ga-68) enabling earlier detection

Also, it will enter Co-PSMA IIT assessing its performance vs Ga-68 PSMA-11 & P-III (AMPLIFY) trial (n=220) with rising or detectable PSA post-receiving SoC. P-III data will back the application for FDA approval

VGA039 - Biologic

The US FDA has granted FTD to VGA039 for the treatment of von Willebrand disease (VWD)

VGA039 (mAb) inhibits Protein S to restore balance blood clotting for the treatment of various bleeding disorders

Abenacianine - Fluorescent Imaging Agent

The US FDA has granted FTD to adjunct abenacianine for intraoperative visualization of primary lung cancer, pulmonary lesions, cancerous lymph nodes, & positive surgical margins in patients undergoing surgery for known or suspected lung cancer

The P-II trial of abenacianine showed that 43% (17/40) patients experienced ≥1 clinically significant event (CSE), incl. the detection of hard-to-find tumors, identification of positive margins, & discovery of previously undetected tumors that standard visual & palpation methods might have missed. Data published in The ACS

Enrollment in P-II (VISUALIZE) trial assessing efficacy and safety of abenacianine in patients undergoing surgery for lung cancer is completed with enrollment for P-III (VISUALIZE-2) to initiate in 2025

Posdinemab – Biologic

The US FDA has granted FTD to posdinemab for the treatment of patients with Alzheimer’s disease, following the grant of FTD to JNJ-2056 (being evaluated in P-IIb ReTain Trial) in Jul 2024

Posdinemab is being investigated in P-IIb (AuTonomy) trial assessing it vs PBO in early AD patients; enrollment ongoing

Posdinemab (anti-tau mAb) binds to the pathological phosphorylated tau upon its release from neurons, neutralizing it before it can spread to other neurons

Invikafusp Alfa (STAR0602) - Biologic

The US FDA has granted FTD to invikafusp alfa (STAR0602) for the treatment of advanced colorectal cancer with TMB-H

Designation was based on dose escalation part of P-I/II (STARt-001) trial of STAR0602 in cancer patients who are heavily treated or are unresponsive to SoC. Data was presented at SITC 2024 & ESMO Immuno-Oncology Congress 2024

STAR0602 has entered dose expansion part of P-I/II trial in the end of 2024 with additional efficacy data expected by the end of 2025

STAR0602 targets a common Vβ T cell subset in all cancers, by combining non-clonal TCR activation with T cell co-stimulator in a single molecule to increase levels of clonally enriched effector memory Vβ T cells, amplifying tumor immune responses & promoting tumor clearance

SPG601 – Small Molecule

The US FDA has granted FTD to SPG601 for the treatment of patients with Fragile X Syndrome

SPG601 was assessed in a P-II cross over trial evaluating it vs PBO in adult men with topline data expected in Q2’25

SPG601 is a BK channel activator that binds to and increases activated BK channels to restore synaptic function

LYT-200 - Biologic

The US FDA has granted FTD to LYT-200 for the treatment of acute myeloid leukemia

LYT-200 is being investigated in P-I/II trial assessing LYT-200 ± venetoclax & hypomethylating agents in hematological malignancies (incl. AML and high-risk myelodysplastic syndrome), where it showed early activity as a monotx. as well as in combination

Additionally, it is being evaluated in P-I/II trial as a monotx. & in combination with tislelizumab, where it showed disease control & depicted early signs of anti-tumor activity

Azenosertib – Small Molecule

The US FDA has granted FTD to azenosertib for treating patients with Pt-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer (PROC) who are positive for Cyclin E1 protein levels via immunohistochemistry

Multiple trial results were reported in Jan 2025: topline data of Part Ib (n=102) from P-II (DENALI/ZN-c3-005) trial in Pt-resistant ovarian cancer, final P-Ib (ZN-c3-001) data in solid tumors (incl. 69 PROC patients)

Additionally, data from P-I/II (MAMMOTH/ZN-c3-006; n=61) data assessing azenosertib ± PARP inhibitor & initial data from P-I (ZN-c3-016) assessing azenosertib + BEACON regimen (encorafenib + cetuximab) in BRAF mCRC in partnership with GSK & Pfizer, respectively was also reported

SGT-212 - Gene Therapy

The US FDA has granted FTD to SGT-212 for treating Friedreich’s ataxia (FA) and previously approved its IND on Jan 07, 2025

The P-Ib study will assess safety & tolerability of contemporaneous systemic IV and bilateral IDN administration of SGT-212 in non-ambulatory and ambulatory FA patients for 5yrs., with dosing anticipated during H2’25

SGT-212 is a recombinant AAV-based gene therapy that transfers full-length human frataxin via intradentate nucleus (IDN) in the cerebellar dentate nuclei & IV infusion in the cardiomyocytes. The IDN delivery will be confirmed in real-time using gadolinium MRI contrast agent

NPI-001 – Small Molecule

The US FDA has granted FTD to NPI-001 (N-acetylcysteine amide) tablets for the treatment of retinitis pigmentosa (RP)

NPI-001 boosts glutathione levels to prevent damage from reactive oxygen molecules to retinal cells

DYNE-101 - Antisense Oligonucleotide

The US FDA has granted FTD to DYNE-101 for treating myotonic dystrophy type 1 (DM1). US Accelerated Approval is anticipated to submit in H1’26

DYNE-101 is being tested in the global P-I/II ACHIEVE trial for DM1

DYNE-101 is an antisense oligonucleotide (ASO) linked to an antibody fragment (Fab) that targets the transferrin receptor 1 (TfR1) on muscle cells

Umbilical Cord Outer Lining Stem Cells (UCSLs) - Cell Therapy

The US FDA has granted FTD to UCSLs programs for the treatment of Polymyositis (PM) & Dermatomyositis (DM) patients following the grant of ODD in Dec 2024

The therapy demonstrated positive results in P-I study with 78% clinical improvements & 35% steroid use reduction in 6mos.; favorable safety & efficacy. The initiation of P-II/III (IIMPACT) trial is expected in Q1’25

BBO-8520 – Small Molecule

The US FDA has granted FTD to BBO-8520 for Metastatic NSCLC (KRAS^G12C mutations)

Showed significant tumor inhibition in preclinical models' post-resistance to sotorasib.It is being assessed in a P-I (ONKORAS-101) trial in KRAS^G12C NSCLC patients who previously received KRAS^G12C inhibitors or no targeted therapy

It inhibits both "ON & OFF" states of KRAS^G12C, targeting resistance mechanisms such as KRAS^G12C amplification & RTK activation. Developed via the NCI RAS Initiative, Lawrence Livermore National Laboratory, & BBOT collaboration

Emiltatug Ledadotin (XMT-1660) - Biologic

The US FDA has granted FTD to Emiltatug Ledadotin (XMT-1660) for Advanced/Metastatic Breast Cancer. Targets patients with HER2-low/HER2-negative breast cancer, including TNBC

Intended for those who received anti-TOP1 ADC or are ineligible for/have previously received endocrine therapy

Ersodetug – Biologic

The US FDA has granted Breakthrough therapy designation (BTD) to ersodetug for the treatment of hypoglycemia caused by tumor Hyperinsulinism (HI)

The designation was based on the P-IIb (RIZE) trial of ersodetug, which demonstrated an improvement in hypoglycemia by ≥75% without clinically significant hyperglycemia

Additionally, FDA lifted partial clinical holds on ersodetug allowing inclusion of US sites in ongoing global P-III (sunRIZE) trials in patients (≥3mos.) with congenital HI; topline data expected in H2’25. Also, P-III trial of ersodetug for the treatment of hypoglycemia due to tumor HI will start in H1’25 with topline data expected in H2’26

Ersodetug is a fully human mAb that opposes the effects of over-activated insulin receptors by binding to its unique allosteric site

Tividenofusp Alfa - Protein

The US FDA has granted BTD to tividenofusp alfa (DNL310) for the treatment of patients with Hunter syndrome (MPS II); BLA submission expected in early 2025 under accelerated approval

The P-I/II trial of DNL310 showed benefits in evidence-based surrogate endpoints with early signs of improved clinical outcomes

DNL310 combines iduronate 2-sulfatase (IDS) with Denali’s proprietary Enzyme TransportVehicle (ETV), designed for active brain transport & broad body distribution to target the behavioral, cognitive, & physical symptoms of MPS II

Letetresgene Autoleucel – Cell Therapy

The US FDA has granted BTD to lete-cel to treat inoperable or metastatic myxoid or round cell liposarcoma (MRCLS) patients who had previously received anthracycline-based CT, are positive for HLA-A02:01, HLA-A02:05, or HLA-A*02:06, with tumors expressing NY-ESO-1 antigen

Designation was based on P-II (IGNYTE-ESO) trial assessing lete-cel in patients (N=64) with synovial sarcoma (n=34) or MRCLS (n=30) that showed 42% patients (41% for synovial sarcoma & 43% for MRCLS) had RECISTv1.1 responses (6 CRs & 21 PRs) with mDOR of 12.2mos. (synovial sarcoma: 18.3mos. & MRCLS: 12.2mos.). Data was presented at CTOS 2024

GSK5764227 - Biologic

The US FDA has granted BTD to GSK5764227 (GSK’227) for treating r/r osteosarcoma in patients with disease progression on at least 2 prior lines of therapy

Designation was supported by P-II (ARTEMIS-002) trial (carried out by Hansoh Pharma) assessing the safety & efficacy of GSK’227 to treat patients (n=60; 42 had osteosarcoma) with r/r osteosarcoma plus other unresectable bone & soft tissue sarcomas. Results were highlighted at ASCO 2024

GSK’227 (HS-20093) is a B7-H3-targeted ADC developed by Hansoh Pharma for various solid tumors. In 2023, GSK acquired global rights (excl. China’s mainland, Hong Kong, Macau & Taiwan) for its development & launched a global P-I trial in Aug 2024

Brelovitug - Biologic

The US FDA has granted BTD to brelovitug (BJT-778) for the treatment of chronic hepatitis delta (CHD)

BJT-778 (IgG1 mAB) targets the surface antigen (anti-HBsAg) of the hepatitis B virus to neutralize & clear HDV as well as HBV virus from the body

9MW2821 – Biologic

China’s NMPA has granted BTD to 9MW2821 for the treatment of 1L unresectable, locally advanced or metastatic urothelial carcinoma (la/mUC)

9MW2821 is being evaluated in an ongoing P-III trial in combination with toripalimab, where 40 patients showed ORR of 87.5% with cORR of 80% & DCR of 92.5%. mPFS & DOR were not reached

Carteyva (Relmacabtagene Autoleucel) – Cell Therapy

China’s NMPA has granted BTD to Carteyva for the treatment of 2L r/r large B-cell lymphoma (LBCL) in adults

Designation was supported by P-II (JWCAR029-216) assessing Carteyva’s safety & efficacy in ASCT-ineligible Chinese patients with r/r LBCL who are unresponsive to 1L therapy

IBI343 – Biologic

The NMPA has granted BTD to IBI343 alone for treating CLDN18.2+ advanced pancreatic ductal adenocarcinoma (PDAC) progressed post 1L of systematic therapy

Designation was based on the ongoing P-I trial of IBI343 (6mg/kg, Q3W) in CLDN18.2+ advanced PDAC patients (n=43) treated with at least 1L of therapy (5% received 2L+ of therapy) across China, Australia & the US. It depicted cORR of 23.3%, PFS in 26 pts & mPFS of 5.3mos. at the data cutoff

IBI343 (CLDN18.2 ADC) is also in P-III study for GC. It previously received the NMPA’s BTD for CLDN18.2+ GC progressed post 2L of therapy & PDAC progressed post 1L of therapy as well as the FDA’s FTD for r/r advanced unresectable/metastatic PDAC

CUTX-101 (copper histidinate) - Small molecule

The US FDA has accepted NDA under priority review (PR) for CUTX-101 (copper histidinate) to treat Menkes disease (PDUFA: Jun 30, 2025)

Submission was based on a trial of CUTX-101 vs untreated historical control that depicted significantly improved OS & an 80% reduction in the death risk, with mOS of 177.1 vs 16.1mos.

Nipocalimab - Biologic

The US FDA has accepted BLA & granted priority review to nipocalimab for treating patients with antibody positive (anti-AChR, anti-MuSK, anti-LRP4) gMG, based on P-III (Vivacity-MG3) trial. MAA in this indication has been filed with the EMA on Sep 11, 2024

The P-III study assessed safety & efficacy of nipocalimab + SoC (30mg/kg, IV loading dose followed by 15mg/kg, Q2W) vs PBO for treating adults (n=199; 153 were antibody +ve) with antibody positive or negative gMG for 24wks.

Trial depicted a significant reduction in MG-ADL scores (≥2-point improvement) vs with a sustained disease control after 24wks. in Ab +ve adults vs PBO

Nipocalimab is an investigational monoclonal antibody that blocks FcRn with high affinity, aiming to reduce circulating IgG levels while potentially preserving other immune functions.

Datopotamab Deruxtecan - Biologic

The US FDA has accepted BLA & granted priority review to datopotamab deruxtecan (Dato-DXd) for treatment-experienced patients with LA/M EGFR-mutated NSCLC (PDUFA: Q3’25) which also received BTD from US FDA

BLA & designation were based on P-II (TROPION-Lung05) & P-III (TROPION-Lung01) trials along with supporting data from P-I (TROPION-PanTumor01) study. The combined findings from P-II & P-III depicted confirmed ORR of 47% & mDoR of 7mos., with consistent safety

Dato-DXd is also being assessed in 7 P-III studies, incl. TROPION-Lung14 & TROPION-Lung15 with/without Tagrisso to treat advanced or metastatic EGFRm non-squamous NSCLC

RP1 (Vusolimogene Oderparepvec) - Gene Therapy

The US FDA has accepted BLA & granted priority review to RP1 (vusolimogene oderparepvec) + nivolumab for advanced melanoma, with no advisory committee meeting planned & no potential review issues identified yet (PDUFA: Jul 22, 2025)

Submission was based on primary data from P-III (IGNYTE) trial assessing RP1 + nivolumab vs physician's choice treatment in ~400 patients with anti-PD-1 & CTLA-4 failed melanoma or are ineligible for anti-CTLA-4 treatment

RP1 is a genetically engineered herpes simplex virus armed with a fusogenic protein (GALV-GP R-) & GM-CSF to enhance tumor killing, tumor immunogenicity, & activate systemic anti-tumor response

SL1009 – Small Molecule

The US FDA has accepted NDA & granted priority review to SL1009 (sodium dichloroacetate oral solution) which will be used with a proprietary genetic test to treat pyruvate dehydrogenase complex deficiency (PDCD) (PDUFA: May 27, 2025)

Submission was based on P-III (SL1009-01) trial & a survival (SL1009-02) study showing evidence of mechanistic characterization with nonclinical & clinical evidence of safety & efficacy in PDCD patients

Welireg – Small Molecule

The US FDA has accepted sBLA & granted priority review to Welireg for the treatment of patients (≥12yrs.) with advanced, unresectable, or metastatic pheochromocytoma & paraganglioma (PDUFA: May 26, 2025)

Submission was based on P-II (LITESPARK-015) trial assessing Welireg monotx. (120mg) that showed significant ORR & DOR data in 322 patients; data to be presented

Sunvozertinib – Small Molecule

The US FDA has accepted NDA & granted priority review to sunvozertinib to treat LA/M NSCLC associated with EGFR exon 20 insertion mutations (exon20ins) patients whose disease has progressed on/after Pt-based CT

Submission was based on the WU-KONG1 part B trial assessing the safety & efficacy of sunvozertinib in the above population from Asia, EU, North & South America which showed significant clinical benefits. Results were highlighted at ASCO 2024

Sunvozertinib is an irreversible EGFR inhibitor that targets broad range of EGFRm with wild-type EGFR selectivity

Orpathys (Savolitinib) - Small Molecule

The NMPA has accepted NDA & granted priority review to Orpathys (savolitinib) + Tagrisso (osimertinib) for treating locally advanced or metastatic EGFR-mutated NSCLC with MET amplification in patients progressed on 1L EGFR inhibitor therapy. HUTCHMED will receive milestone from AstraZeneca

Submission was based on P-III (SACHI) study assessing the safety & efficacy of Orpathys + Tagrisso vs Pt-based doublet-CT (pemetrexed plus cisplatin or carboplatin) in this population

Study achieved its 1EP of PFS in a planned interim analysis, as confirmed by the Independent Data Monitoring Committee (IDMC). Enrollment is now complete, and results will be highlighted at future conferences

VG901 – Gene Therapy

The US FDA has granted Rare Pediatric Disease Designation (RPDD) to VG901 for the treatment of retinitis pigmentosa (RP) caused by CNGA1 gene mutations

Additionally, DSMB approved dose escalation in ongoing P-Ib trial of VG901 to evaluate its safety, tolerability & preliminary efficacy

VG901 is a gene therapy that utilizes ViGeneron’s vgAAV capsid to deliver functional CNGA1 gene directly to retinal photoreceptor cells through intravitreal injection.

BPN-36964 – Small Molecule

The US FDA has granted ODD & RPDD to BPN-36964 for systemic treatment of familial dysautonomia

BPN-36964 can efficiently the BBB & correct ELP1 mRNA splicing, increasing its levels in the body to prevent progressive neuronal degeneration, as observed in an FD mouse model

rAAV2-U1a-hELP1 – Gene Therapy

The US FDA has granted ODD & RPDD to rAAV2-U1a-hELP1 for the treatment of optic neuropathy in familial dysautonomia

rAAV2-U1a-hELP1 (intravitreal) showed reduced retinal ganglion cells death when injected in the eyes of an FD mouse model

ST-01156

The US FDA has granted RPDD & ODD to ST-01156 molecular glue to target solid tumor indications

The IND filing for ST-01156 is expected in H1'25, with SEED Therapeutics potentially receiving an FDA priority review voucher upon approval, following the grant of RPDD

AAV8-RK-RetGC – Gene Therapy

The US FDA has granted RPDD to AAV8-RK-RetGC program for the treatment Leber congenital amaurosis caused by GUCY2D mutations (LCA1)

Oesophageal Cancer Detection Tool

The US FDA has granted Breakthrough Device Designation (BDD) to oesophageal cancer detection tool
The tool integrates into clinical workflows to improve early lesion detection & patient outcomes, while allowing endoscopists full control over decision-making

Norm System

The US FDA has granted BDD to Norm system for heart failure management & has been accepted into the FDA’s total product lifecycle advisory program (TAP)

Additionally, FIRE1 has secured $120M funding to complete pivotal clinical trial of the Norm system

The system allows patients to follow physician directed self-management at home, reducing burden on healthcare staff & improving overall health of patients

AK+ Guard

The US FDA has granted BDD to AI-powered AK+ Guard for hyperkalemia detection following its acceptance in FDA’s Total Product Lifecycle Advisory Program (TAP)

AK+ Guard utilizes Lead I ECG data to detect mod. to sev. hyperkalemia, allowing remote monitoring & early intervention for high-risk patients, incl. those with end-stage renal disease, chronic kidney disease (CKD), & other risk factors

Atrial Shunt Technology

The US FDA has granted BDD to novel atrial shunt tech for heart failure with reduced ejection fraction (HFrEF)

Additionally, FDA has granted IDE approval for the pivotal (ALLAY-HFrEF) trial, set to begin in early 2025, to evaluate safety & efficacy in ~350 heart failure patients (LVEF ≤ 40%) symptomatic despite GDMT, while the ALLAY-HF trial in HFpEF is ongoing

pTau 217 Blood Test

The US FDA has granted BDD to pTau 217 blood test for diagnosis of Alzheimer's disease

The test utilizes SPEAR tech which enables ultra-sensitive protein biomarker detection (1 fg/mL) from 1µL of diluted sample using a wash-free, 2-factor authentication system for high specificity & accuracy

DCISionRT Test

The US FDA has granted BDD to DCISionRT test to provide individualized risk assessment & benefits of radiation therapy (RT) in women (30-85yrs.) diagnosed with ductal carcinoma in situ (DCIS)

DCISionRT integrates tumor biology with clinicopathologic factors to provide personalized results by analyzing 7 protein biomarkers & 4 clinical factors to generate a Decision Score, helping physicians optimize RT decisions as well as avoid over or under-treatment

The test predicts radiation therapy benefits after breast-conserving surgery, while also assessing 10yr. recurrence risk, & identifies residual risk post-BCS & radiation

PathChat DX

The US FDA has granted BDD to PathChat DX to transform diagnostic workflows & improve patient outcomes by combining generative AI & multimodal analysis

PathChat assist pathologists in diagnosing complex cases by combining pathology foundation models which are trained on vast histology datasets, with a custom multimodal large language model (MLLM) to analyze high-resolution pathology images & clinical data, improving accuracy and efficiency

Access p‑Tau217/β-Amyloid 1-42 Plasma Ratio

The US FDA has granted BDD to Access p Tau217/β-Amyloid 1-42 plasma ratio for facilitating diagnosis of patients with amyloid pathology associated with Alzheimer's disease

The blood test measures the p-Tau217 to β-Amyloid 1-42 ratio, offering a non-invasive, accessible method for early Alzheimer’s detection, potentially before symptoms appear.

This allows early diagnosis, ensures timely intervention, aids patient stratification for trials, & accelerates Alzheimer’s research & treatment

Defender Implant

The US FDA has granted BDD to Defender implant for meniscus replacement & has been accepted into the FDA’s total product lifecycle advisory program (TAP)

The pilot clinical trial of Defender is expected to start in 2026 with potential US FDA approval by 2029

Laruparetigene Zovaparvovec – Gene Therapy

The US FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to laru-zova (laruparetigene zovaparvovec) for the treatment of X-linked retinitis pigmentosa (XLRP)

Designation was supported by preliminary data from P-II (DAWN & SKYLINE) trials assessing laru-zova in XLRP patients with RPGR gene mutations that showed improved low luminance visual acuity

Additionally, Beacon Therapeutics reported data from various trials incl. 3mos. data from P-II (DAWN) trial, 24mos data from P-II (SKYLINE) trial & ongoing P-II/III (VISTA) trial

Laru-zova expresses the full-length RPGR protein, effectively targeting the full complement of photoreceptor damage in XLRP, incl. both rod & cone degeneration

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