New Drug Designations - December 2024

Shots:

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

The December 2024 report covers designations granted to 43 drugs and 3 devices, encompassing 13 small molecules, 10 biologics, 7 cell and gene therapies & 3 medical devices among others

Significant trends this month show, Nobias Therapeutics’ NB-001 secured the US FDA’s ODD and RPDD to treat DiGeorge syndrome

Ersodetug – Biologic

Sponsor	Rezolute
Indication	Hypoglycemia
Phase	P-III
MOA	Insulin Receptor Antagonists
RoA	Intravenous
Approval Authority	FDA
Date	Dec 03, 2024

The US FDA has granted ODD to ersodetug for the treatment of hypoglycemia caused by tumor HI

The company is expecting to initiate a P-III trial assessing ersodetug in tumor HI patients in 2025 with real-world data exhibited through an expanded access program

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

NB-001 – Antivirals

Sponsor	Nobias Therapeutics
Indication	DiGeorge syndrome (22q11.2 Deletion Syndrome)
Phase	P-II
MOA	mGluR Modulator
RoA	Oral
Approval Authority	FDA
Date	Dec 05, 2024

The US FDA has granted ODD & RPDD to NB-001 for the treatment of patients with neuropsychiatric symptoms related to 22q11.2 deletion syndrome (DiGeorge syndrome/22q11DS)

NB-001 is currently under investigation in a P-II trial assessing NB-001 vs PBO in 22q11DS patients with top-line results presented at CNS Annual Meeting, 2023

NB-001 (mGluR modulator) disrupts neuronal signaling that relieves neuropsychiatric symptoms

Crofelemer – Small molecule

Sponsor	Jaguar Health
Indication	Diarrhea
Phase	P-II
MOA	Chloride channel antagonists
RoA	Oral
Approval Authority	FDA
Date	Dec 17, 2024

The US FDA has granted ODD to crofelemer for treating diarrhea in cholera patients

Crofelemer is under investigation in 3 IIT PoC & 2 P-II studies for SBS-IF ± MVID in the US, EU &/or Middle East/North Africa regions. The first dosing for the same is expected in Dec’24 & Q1’25, whereas IIT PoC results are expected in Q2’25

Additionally, the company intends to pursue ODD & a Tropical Disease Priority Review Voucher for NP-300 to treat diarrhea in cholera patients

ST316 – Peptides

Sponsor	Sapience Therapeutics
Indication	Familial Adenomatous Polyposis
Phase	P-II
MOA	β-catenin Antagonist
RoA	Intravenous
Approval Authority	FDA
Date	Dec 19,2024

The US FDA has granted ODD to ST316 for the treatment of patients with familial adenomatous polyposis (FAP)

ST316 is currently in the P-II study of Phase 1-2 (ST316-101) trial assessing ST316 with relevant SoC & multiple lines of treatment in CRC patients, while the P-I was the dose escalation study in patients having advanced solid tumors with Wnt/β-catenin signaling pathway abnormalities incl. CRC

ST316 (β-catenin & BCL9 antagonist) selectively inhibits Wnt/β-catenin signaling pathway responsible for FAP & >80% of CRCs in patients

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

Sponsor	Restem
Indication	Polymyositis & Dermatomyositis
Phase	P-I
MOA	Unknown
RoA	Intravenous
Approval Authority	FDA
Date	Dec 03, 2024

The US FDA has granted ODD to UCSLs programs for the treatment of Polymyositis (PM) & Dermatomyositis (DM) patients

The therapy demonstrated positive results in P-I study with significant clinical improvements; favorable safety & efficacy plus probable decrease in the need of steroidal therapies. The initiation of P-II/III trials is expected in Q1’2025

BRC-002 – Cannabinoid

Sponsor	Biopharmaceutical Research Company
Indication	Complex Regional Pain Syndrome (CRPS)
Phase	P-I
MOA	Undefined
RoA	Oral
Approval Authority	FDA
Date	Dec 03, 2024

The US FDA has granted ODD to BRC-002 for the treatment of complex regional pain syndrome (CRPS) that is currently under investigation in the IIT P-I study

The company plans to initiate patient enrollment for the P-II study of BRC-002 by the YE’25 & seeks FDA input on the development plan

Azeliragon – Small Molecule

Sponsor	Cantex Pharmaceuticals
Indication	Brain Metastasis
Phase	P-I
MOA	RAGE antagonist
RoA	Oral
Approval Authority	FDA
Date	Dec 09, 2024

The US FDA has granted ODD to azeliragon for the treatment of patients with brain metastasis from breast cancer

Azeliragon (oral, QD) inhibits RAGE interactions with its ligands (incl. HMGB1 & S100 proteins) & is under investigation for brain metastasis, glioblastoma, breast cancer, pancreatic cancer, & hospitalized pneumonia patients

AVG-002 – Gene Therapy

Sponsor	AlveoGene
Indication	Neonatal SP-B Deficiency
Phase	Preclinical
MOA	Gene transference
RoA	Inhalation
Approval Authority	FDA
Date	Dec 03, 2024

The US FDA has granted ODD to AVG-002 for neonatal SP-B deficiency, following the grant of RPDD in Nov’24. AVG-002 is anticipated to enter clinical development & filing is expected by 2028

The Preclinical data showed increased survivability in SP-B deficient murine model with a single dose of AVG-002 plus recovery of lung histology & function in disease-induced tissue after AVG-002 treatment

AVG-002, an inhalable gene therapy, is developed using AlveoGene’s InGenuiTy platform that utilizes a pseudo-typed lentiviral vector to efficiently deliver the gene directly to the alveolar region of the lung

Manufactured Red Blood Cells – Cell Therapy

Sponsor	Safi Biotherapeutics
Indication	Sickle Cell Disease
Phase	Preclinical
MOA	Unknown
RoA	Unknown
Approval Authority	FDA
Date	Dec 18, 2024

The US FDA has granted ODD & RPDD to manufactured RBCs (mRBCs) for use in chronic transfusion to treat sickle cell patients

The company is advancing its validation of mRBCs cGMP manufacturing for clinical studies & anticipates initiating the trials by 2027, post-completion of IND-enabling activities

VGT-1849A – antisense oligonucleotide

Sponsor	Vanda Pharmaceuticals
Indication	Polycythemia Vera
Phase	Preclinical
MOA	JAK2 inhibitor
RoA	Unknown
Approval Authority	FDA
Date	Dec 20, 2024

The US FDA has granted ODD to VGT-1849A for the treatment of patients with polycythemia vera (PV)

VGT-1849A is a selective ASO-based JAK2 inhibitor with the potential to decrease JAK2V617F-driven pathogenic signaling, thereby reducing malignant proliferation & survivability of hematopoietic cells

Sparsentan – Small Molecule

Sponsor	Renalys & Travere
Indication	IgA nephropathy
Phase	P-III
MOA	Angiotensin II & Endothelin A receptor antagonists
RoA	Oral
Approval Authority	MHLW
Date	Dec 02, 2024

The Japanese MHLW has granted ODD to sparsentan, being investigated under P-III study in Japan for treating IgA nephropathy. It was FDA approved under brand name Filspari for slowing kidney function among adults in Sep 2024

Renalys secured its development & commercialization rights from Travere Therapeutics across Japan, South Korea, Taiwan, Brunei, Cambodia, Indonesia, Laos, Malaysia, Myanmar, the Philippines, Singapore, Thailand & Vietnam as per an agreement signed in Jan 2024

Filspari (QD, oral) is a non-immunosuppressive medication for IgAN that blocks endothelin-1 and angiotensin II pathways to reduce glomerular injury

PT217 – Biologic

Sponsor	PTC Therapeutics
Indication	Neuroendocrine Prostate Cancer
Phase	P-II
MOA	CD47 & DLL3 Inhibitors
RoA	Parenteral
Approval Authority	FDA
Date	Dec 4, 2024

The US FDA has granted FTD to PT217 for treating metastatic de novo or treatment-emergent neuroendocrine prostate cancer (NEPC)

PT217 is being investigated under P-I/II (SKYBRIDGE) trial for its safety, tolerability, pharmacokinetics and preliminary efficacy in patients with advanced or refractory cancers with DLL3 expression and another P-I trial in China (CTR20242720)

PT217 is a first-in-class bispecific antibody targeting DLL3 & CD47 and being developed for SCLC & neuroendocrine carcinoma (NEC), incl. NEPC. Phanes has also partnered with Roche to study it in combination with atezolizumab

AdAPT-001 – Immunotherapy

Sponsor	EpicentRx
Indication	Soft Tissue Sarcoma
Phase	P-II
MOA	TGFβ inhibitors
RoA	Intratumoral
Approval Authority	FDA
Date	Dec 5, 2024

The US FDA has granted FTD to AdAPT-001 + nivolumab/atezolizumab for the treatment of r/r advanced or metastatic soft tissue sarcoma (STS) post disease progression after at least 1L of therapy

The Designation was supported by data from P-I/II trials assessing AdAPT-001 as monotherapy or in combination with PD-1/PD-L1 inhibitors showed PFS of ~8.5mos. in patients with STS & other tumors

AdAPT-001 is an oncolytic adenovirus-delivered TGF-βR inhibitor that neutralizes TGFβ locally to decrease T cell function & sensitize STS tumors to checkpoint inhibitors

Fluzone & Flublok + Novavax – Vaccine

Sponsor	Sanofi
Indication	Influenza and COVID-19
Phase	P-I/II
MOA	Immunostimulants
RoA	Intramuscular
Approval Authority	FDA
Date	December 11, 2024

The US FDA has granted FTDs to two combinations, Fluzone (influenza protein-based trivalent vaccine) + Novavax (COVID-19 vaccine) & Flublok + Novavax, for preventing influenza and COVID-19 infections in subjects (50+ yrs.)

Fluzone High-Dose and Flublok outperformed standard-dose flu vaccines in preventing influenza and reducing flu-related hospitalizations in older adults. The Novavax COVID-19 vaccine has shown better tolerability as a booster and high efficacy as primary vaccination in P-III studies

Sanofi has launched 2 P-I/II studies to assess the safety and immune response of combination vaccines to prevent influenza A, influenza B & COVID-19

NCT06695117: Combines Fluzone High-Dose (TIV-HD) with the Novavax COVID-19 vaccine for individuals aged 50+ yrs.

NCT06695130: Combines Flublok (RIV3) with the Novavax COVID-19 vaccine for the same age group

IMM-1-104 – Small Molecule

Sponsor	Immuneering
Indication	Advanced Melanoma
Phase	P-IIa
MOA	MAPK Inhibitor
RoA	Oral
Approval Authority	FDA
Date	Dec 12, 2024

The US FDA has granted FTD to IMM-1-104 for the treatment of unresectable or metastatic NRAS-mutant melanoma patients progressed or intolerable to PD-1/PD-L1 checkpoint inhibitors

IMM-1-104 (QD) inhibits MAPK pathway to stimulate RAS activity that impacts cancer cells selectively & is in P-IIa trial for the treatment of advanced solid tumors incl. melanoma

LPCN 1148 – Small Molecule

Sponsor	Lipocine
Indication	Sarcopenia
Phase	P-II
MOA	Androgen receptor agonists
RoA	Oral
Approval Authority	FDA
Date	Dec 17, 2024

The US FDA has granted FTD to LPCN 1148 for the treatment of sarcopenia in decompensated cirrhosis patients

LPCN 1148 was subjected to a recent PoC P-II study in decompensated cirrhosis patients, showing improved sarcopenia & related clinical outcomes

LPCN 1148 (oral) contains testosterone dodecanoate (androgen receptor agonist) & utilizes multimodal approach to manage cirrhosis & related comorbidities

NX-5948 – Small Molecule

Sponsor	Nurix Therapeutics
Indication	R/R Waldenstrom’s Macroglobulinemia
Phase	P-II
MOA	Bruton’s Tyrosine Kinase Degrader
RoA	Oral
Approval Authority	FDA
Date	Dec 19, 2024

The US FDA has granted FTD to NX-5948 for the treatment of r/r Waldenstrom’s macroglobulinemia (WM) in patients who received at least 2L of treatment, incl. a BTK inhibitor

The designation is followed by P-I data evaluating the safety & efficacy of NX-5948 & the company is enrolling WM patients in a P-Ib (expansion cohort) trial with results expected in 2025

NX-5948 is a novel oral small molecule that degrades BTK proteins through cereblon E3 ligase complex without affecting other cereblon neo-substrates

BGC101 – Cell Therapy

Sponsor	BioGenCell
Indication	Critical Limb Threatening Ischemia
Phase	P-II
MOA	Cell replacements
RoA	Intramuscular
Approval Authority	FDA
Date	Dec 19, 2024

The US FDA has granted FTD to BGC101 for the treatment of Critical Limb Threatening Ischemia (CLTI) patients to prevent amputations, disease progression & relieve pain

BGC101 is under investigation in the P-II trial, which completed patient recruitment & is being conducted in the US, EU & Israel

BGC101 utilizes BioGenCell’s TRACT platform to stimulate tissue regeneration in the damaged limb by developing personalized cell therapies through the immune & stem cells obtained from the patient

SC291 – Cell Therapy

Sponsor	Sana Biotechnology
Indication	B-cell mediated autoimmune disease
Phase	P-I
MOA	Immunologic cytotoxicity
RoA	Intravenous
Approval Authority	FDA
Date	Dec 2, 2024

The US FDA has granted FTD to SC291 for the treatment of r/r SLE, incl. extrarenal lupus & lupus nephritis

SC291 is being evaluated in the GLEAM trial in B-cell mediated autoimmune disease patients (incl. lupus nephritis, extrarenal lupus, plus ANCA-associated vasculitis with ongoing enrolment & initial results expected in 2025

SC291, an allogeneic CAR T cell therapy, is developed using Sana’s hypoimmune platform that targets CD19 protein expressed on the surface of B cells

R289 – Small Molecules

Sponsor	Rigel Pharmaceuticals
Indication	Lower-risk Myelodysplastic Syndrome
Phase	P-I
MOA	IRAK1/4 dual inhibitor
RoA	Oral
Approval Authority	FDA
Date	Dec 2, 2024

The US FDA has granted FTD to R289 in patients with previously-treated transfusion dependent lower-risk myelodysplastic syndrome (LR-MDS)

The P-Ib study is currently assessing the safety, tolerability, pharmacokinetics & preliminary efficacy of R289 among patients with r/r lower-risk MDS, with recruitment underway

R289 (prodrug of R835) is an IRAK1/4 dual inhibitor that works by blocking inflammatory cytokine production in response to TLR and IL-1R signaling. Dysregulation of these pathways contributes to inflammatory conditions

LP-184 – Small Molecule

Sponsor	Lantern Pharma
Indication	Triple Negative Breast Cancer
Phase	P-I
MOA	DNA synthesis inhibitors
RoA	Oral
Approval Authority	FDA
Date	Dec 3, 2024

The US FDA has granted FTD to LP-184 for the treatment of Triple Negative Breast Cancer (TNBC) in patients, following the grant of FTD to treat Glioblastoma in Oct 2024

The preclinical data showed tumor regression (107-141%) in both PARPi (PARP inhibitor) resistant & PARPi sensitive tumors across 10 TNBC PDX models. Recent preclinical data was presented at Immuno-Oncology Summit 2024

LP-184 is being assessed in a P-Ia trial to evaluate its safety and tolerability in a broad range of solid tumor patients, incl. TNBC

LP-184 leverages Lantern’s AI platform, RADR & is activated into its cytotoxic form by the enzymatic action of Prostaglandin Reductase 1 (PTGR1), to produce anti-tumor effects

CRB-701 – Biologic

Sponsor	Corbus Pharma
Indication	R/R Metastatic Cervical Cancer
Phase	P-I
MOA	Anti Nectin 4
RoA	Intravenous
Approval Authority	FDA
Date	Dec 9, 2024

The US FDA has granted FTD to CRB-701 for the treatment of patients with r/r metastatic cervical cancer

CRB-701 is being assessed in a 3-part, P-I trial evaluating its safety, PK & efficacy to treat advanced solid tumors linked to elevated Nectin-4. Enrollment of the dose escalation part has been completed which will be conducted in the US & Europe, initial data is expected to be reported in Q1’25

CRB-701 (SYS6002) is an ADC with MMAE as the cytotoxic payload & site-specific, cleavable linker to target Nectin-4 expression on the cancer cells

LBT-SA7 – Vaccine

Sponsor	LimmaTech Biologics
Indication	Skin And Soft Tissue Infections
Phase	P-I
MOA	Immunostimulants
RoA	Parenteral
Approval Authority	FDA
Date	Dec 19, 2024

The US FDA has granted FTD to LBT-SA7 to avert skin and soft tissue infections caused due Staphylococcus aureus

LBT-SA7 (multivalent toxoid vaccine) will be assessed in a P-I trial in the US, to demonstrate its safety & immunogenicity in 130 subjects (18 – 50yrs.), with initial results expected in H2’25

ATX101- Peptides

Sponsor	Allay Therapeutics
Indication	Post-surgical Pain Following Total Knee Replacement Surgery
Phase	P-III (Planned)
MOA	Protein interaction domain and motif inhibitors
RoA	Subcutaneous, Intravenous
Approval Authority	FDA
Date	Dec 04, 2024

The US FDA has granted BTD to ATX101 for treating adults with post-surgical pain following total knee replacement surgery

Designation was based on dose-ranging P-II exploratory study of ATX101 vs bupivacaine (SoC) in 112 subjects, showing sustained pain relief for up to 2wks., decreased opioid use & associated side effects plus improved functional activities & satisfaction for up to 60 days. A registrational P-IIb trial in 200 individuals is planned in 2025 across the US

ATX101 is an investigational formulation of bupivacaine (intracellular sodium ion channel blocker) and a biopolymer indicated for relieving pain post total knee arthroplasty (TKA)

Datopotamab Deruxtecan (Dato-DXd) – Biologic

Sponsor	Daiichi Sankyo
Indication	EGFR-Mutated Non-Small Cell Lung Cancer
Phase	P-III
MOA	Anti-TROP2 ADC
RoA	Intravenous
Approval Authority	FDA
Date	Dec 09, 2024

The US FDA has granted BTD to Dato-DXd for treating locally advanced or metastatic EGFR-mutated NSCLC in adults progressed on an EGFR tyrosine kinase inhibitor (TKI) and Pt-based CT

Designation was based on P-II (TROPION-Lung05) & P-III (TROPION-Lung01) studies, with the results highlighted at ESMO 2024

Daiichi Sankyo and AstraZeneca have filed a new BLA seeking accelerated approval for treating locally advanced or metastatic EGFR-mutated NSCLC in adults who received prior systemic therapies, incl. an EGFR-directed therapy

Tolebrutinib – Small Molecule

Sponsor	Sanofi
Indication	Non-relapsing Secondary Progressive Multiple Sclerosis
Phase	P-III
MOA	BTK inhibitor
RoA	Oral
Approval Authority	FDA
Date	Dec 13, 2024

The US FDA has granted BTD to tolebrutinib for treating adults with non-relapsing secondary progressive multiple sclerosis (nrSPMS)

Designation was based on P-III (HERCULES) study assessing the safety & efficacy of tolebrutinib vs PBO to treat nrSPMS that showed a 31% delay in 6mos. confirmed disability progression onset, with 10% vs 5% having confirmed disability improvement

Liver enzyme elevations (>3xULN) occurred in 4.1% vs 1.6%, with 0.5% showing ALT >20xULN within 90 days. Most cases resolved without intervention, and increased monitoring has mitigated serious liver issues

Regulatory filings of tolebrutinib are being finalized in the US & prepared in the EU. In addition, the P-III (PERSEUS) study in primary progressive MS is underway, with results expected in H2’25

Jemperli – Biologic

Sponsor	GSK
Indication	dMMR/MSI-H Rectal Cancer
Phase	P-II
MOA	PD-1-blocking antibody
RoA	Intravenous
Approval Authority	FDA
Date	Dec 16, 2024

The US FDA has granted BTD to Jemperli for treating locally advanced dMMR)/MSI-H rectal cancer

Designation was based on P-II study (conducted with Memorial Sloan Kettering Cancer Center) in dMMR rectal cancer patients (n=42), showing a 100% clinical CR in all, without any evidence of tumors on MRI, endoscopy, PET scan, or digital rectal exam

Furthermore, sustained cCR was observed at a median follow-up of 26.3mos. in first 24 of them and is underway to assess the patients; safety profile aligned with known data without grade 3+ AEs. GSK’s registrational P-II (AZUR-1) trial is ongoing to validate this data

Trodelvy – Biologic

Sponsor	Gilead
Indication	Extensive-Stage Small Cell Lung Cancer
Phase	P-II
MOA	DNA topoisomerase I inhibitors
RoA	Intravenous
Approval Authority	FDA
Date	Dec 17, 2024

The US FDA has granted BTD to Trodelvy for treating adult with ES-SCLC whose disease has progressed on Pt-based CT

The designation was based on P-II (TROPiCS-03) study ES-SCLC cohort, showing promising antitumor activity in both Pt-resistant (PR) & sensitive (PS) disease as a 2L treatment for ES-SCLC. Gilead plans to start a P-III trial

P-III trials will assess Trodelvy alone and in combinations across various cancers, in collaboration with academic, industry & global partners

Sacituzumab Tirumotecan – Biologic

Sponsor	Merck
Indication	EGFR Mutated Non-Squamous NSCLC
Phase	P-I/II
MOA	DNA topoisomerase I inhibitors
RoA	Intravenous
Approval Authority	FDA
Date	Dec 03, 2024

The US FDA has granted BTD to sac-TMT (Kelun-Biotech holds the Greater China rights) for treating advanced or metastatic non-squamous EGFR-mutated (exon 19 deletion/exon 21 L858R) NSCLC in patients progressed post TKI & Pt-based CT

Designation was supported by P-II expansion part of P-I/II study and two P-II trials of sac-TMT in EGFR-mutated NSCLC patients treated with at least two prior therapies, highlighted at ASCO 2023

Merck is developing sac-TMT alone & combined with Keytruda under 10 global P-III trials for solid tumors, incl. TroFuse-004 (sac-TMT vs CT) & TroFuse-009 (sac-TMT vs doublet CT) in previously treated EGFR-mutated NSCLC

Zorevunersen – Antisense oligonucleotides

Sponsor	Stoke Therapeutics
Indication	Dravet Syndrome
Phase	P-I/II
MOA	SCN1A gene targeting therapy
RoA	Intrathecal
Approval Authority	FDA
Date	Dec 04, 2024

The US FDA has granted BTD to zorevunersen for treating Dravet syndrome with a confirmed mutation, not associated with gain-of-function, in the SCN1A gene

The P-I/IIa and open label studies have depicted significant & sustained seizure reduction as well as improved cognition & behavior. The drug was well tolerated

The Company is in ongoing talks with regulators about a P-III study of zorevunersen and will provide an update by YE’24

SER-155

Sponsor	Seres Therapeutics
Indication	Bloodstream Infections
Phase	P-Ib
MOA	Bacteria replacements
RoA	Oral
Approval Authority	FDA
Date	Dec 09, 2024

The US FDA has granted BTD to SER-155 for reducing bloodstream infections (BSIs) in adults, receiving allogeneic hematopoietic stem cell transplant (allo-HSCT) to treat hematological malignancies

Designation was based on P-Ib study of SER-155 vs PBO, showing a 77% reduction in bacterial BSIs (10% vs 42.9%), shorter period of antibiotic use (9.2 vs 21.1 days) and reduced febrile neutropenia & gastrointestinal pathogen domination. Seres is looking for a collaboration to support its development

SER-155 (oral) is a biotherapeutic aimed at preventing bacterial bloodstream infections and AMR-related outcomes in allo-HSCT patients by decolonizing GI pathogens and improving immune tolerance

Tobevibart – Biologic and Elebsiran – RNA Therapy

Sponsor	Vir Biotechnology
Indication	Chronic Hepatitis Delta
Phase	P-II
MOA	Virus internalization inhibitors
RoA	Subcutaneous
Approval Authority	FDA & EMA
Date	Dec 12, 2024

The US FDA & the EMA have granted BTD & PRIME Designation, respectively, to tobevibart & elebsiran for the treatment of chronic hepatitis delta (CHD) patients

The designations were based on a P-II (SOLSTICE) trial evaluating tobevibart ± elebsiran in CHD patients with results presented at AASLD The Liver Meeting. The P-III (ECLIPSE) trial assessing tobevibart & elebsiran in CHD is to initiate in H1’25

Orpathys and Tagrisso – Small Molecules

Sponsor	HUTCHMED
Indication	EGFR-Mutated NSCLC
Phase	P-III
MOA	Orpathys: MET tyrosine kinase inhibitor Tagrisso: irreversible EGFR TKI
RoA	Oral
Approval Authority	NMPA
Date	Dec 12, 2024

The China’s NMPA has granted BTD to Orpathys (MET tyrosine kinase inhibitor) and Tagrisso (irreversible EGFR TKI) combination for treating locally advanced or metastatic EGFR+ NSCLC with MET amplification after progressing on EGFR inhibitor therapy

The combination is being assessed under P-III (SACHI) study for its safety & efficacy in comparison with Pt-based doublet-CT (pemetrexed + cisplatin/carboplatin) to treat locally advanced or metastatic EGFR+ NSCLC

The 1EP includes PFS by investigator evaluation while other EPs are PFS by IRC evaluation, OS, ORR, DoR, DCR, time to response (TTR) & safety

Imfinzi – Biologic

Sponsor	AstraZeneca
Indication	Muscle-Invasive Bladder Cancer
Phase	P-III
MOA	PD-L1 inhibitor
RoA	Intravenous
Approval Authority	FDA
Date	Dec 06, 2024

The US FDA has accepted & granted priority review to sBLA of Imfinzi for treating muscle-invasive bladder cancer (MIBC), with decision anticipated in Q2’25. Further submissions are under review across the EU, Japan & other regions

sBLA was based on P-III (NIAGARA) study assessing perioperative Imfinzi + neoadj. CT followed by Imfinzi adj. vs CT alone treatment in patients (n=1,063) with MIBC, with data presented at ESMO 2024 & published in the NEJM

Interim analysis showed reduced disease progression, recurrence, or death risk by 32% (mEFS: not reached vs 46.1mos.), 67.8% vs 59.8% were event free at 2yrs. Imfinzi also reduced death risk by 25% (mOS not reached), 82.2% vs 75.2% were alive at 2yrs.

Avutometinib + Defactinib

Sponsor	Verastem Oncology
Indication	Recurrent KRAS Mutant Low-Grade Serous Ovarian Cancer
Phase	P-III
MOA	Avutometinib: RAF/MEK Clamp Defactinib: Selective FAK Inhibitor
RoA	Oral
Approval Authority	FDA
Date	Dec 30, 2024

The US FDA has accepted & granted priority review (PDUFA: Jun 30, 2025) to NDA of avutometinib + defactinib for treatment-experienced KRAS-mutated recurrent LGSOC adults, under accelerated approval pathway. Launch is expected in mid-2025

NDA was based on P-I (FRAME) trial & P-II (RAMP 201) trial of avutometinib (3.2mg, BIW) ± defactinib (200mg, BID) in LGSOC patients, identifying the combination as go-forward treatment in Part A; Parts B & C will assess the regimen’s safety & efficacy & Part D will explore low-dose regimen

P-II showed significant ORRs & good tolerability. The P-III (RAMP 301) study will confirm results for this indication & is recruiting LGSOC patients regardless of KRAS mutations for indication expansion

Taletrectinib – Small Molecule

Sponsor	Nuvation Bio
Indication	ROS1+ve NSCLC
Phase	P-II
MOA	ROS1 TKI
RoA	Oral
Approval Authority	FDA
Date	Dec 23, 2024

The US FDA has accepted NDA of taletrectinib (next-generation ROS1 TKI) and granted priority for treating advanced ROS1+ NSCLC, with the decision anticipated on Jun 23, 2025

Submission was based on pooled data from ongoing P-II [TRUST-I (China) & TRUST-II (global)] studies assessing the safety & efficacy of taletrectinib monotx. for treating advanced NSCLC, with the data highlighted at ESMO 2024

Pooled analysis showed cORR of 89% & intracranial cORR of 77% with mDoR of 44mos. & mPFS of 46mos. in TKI-naïve patients (n=160). In TKI-pretreated patients (n=113), cORR was 56% & 62% for G2032R mutations, with intracranial cORR of 66%, mDoR of 17mos. & mPFS of 10mos.

101-PGC-005 – Antivirals

Sponsor	PIF Partners
Indication	Systemic Juvenile Idiopathic Arthritis (sJIA) Flares
Phase	P-III
MOA	Glucocorticoid receptor agonists
RoA	Unknown
Approval Authority	FDA
Date	Dec 03, 2024

The US FDA has granted RPDD to 101-PGC-005 (‘005) for treating systemic juvenile idiopathic arthritis (sJIA) flares

101-PGC-005 (Type IA prodrug of dexamethasone targeting CD206+ macrophages) is currently being investigated under P-III study to treat ARDS induced by COVID-19 across 9 sites in India

NB-001

Sponsor	Nobias Therapeutics
Indication	DiGeorge syndrome (22q11.2 Deletion Syndrome)
Phase	P-II
MOA	mGluR Modulator
RoA	Oral
Approval Authority	FDA
Date	Dec 05, 2024

The US FDA has granted ODD & RPDD to NB-001 for the treatment of patients with neuropsychiatric symptoms related to 22q11.2 deletion syndrome (DiGeorge syndrome/22q11DS)

NB-001 is currently under investigation in a P-II trial assessing NB-001 vs PBO in 22q11DS patients with top-line results presented at CNS Annual Meeting, 2023

NB-001 (mGluR modulator) disrupts neuronal signaling that relieves neuropsychiatric symptoms

THIO – Small Molecule

Sponsor	MAIA Biotechnology
Indication	Pediatric-Type Diffuse High-grade Gliomas
Phase	P-II
MOA	Telomere targeting agent
RoA	Unknown
Approval Authority	FDA
Date	Dec 16, 2024

The US FDA has granted RPDD to THIO for treating pediatric type diffuse high-grade gliomas (PDHGG)

Alongside RPDD, the drug has also been designated with ODD for hepatocellular carcinoma (HCC), small cell lung cancer (SCLC) and glioblastoma

THIO (6-thio-dG or 6-thio-2’-deoxyguanosine) is a telomere-targeting candidate that is being investigated for NSCLC

HORA-PDE6b – Gene Therapy

Sponsor	EyeDNA Therapeutics (Coave’s Subsidiary)
Indication	Retinal Dystrophy
Phase	P-I/II
MOA	Gene transference
RoA	Subretinal
Approval Authority	FDA
Date	Dec 17, 2024

The US FDA has granted RPDD to HORA-PDE6b for treating inherited retinal dystrophy (IRD) caused by PDE6b gene mutations

HORA-PDE6b is being studied under P-I/II trial for its safety & efficacy, with the 24mos. follow-up data highlighted at ARVO 2024

The company is seeking for accelerated approval across the US & the EU for patients with this indication

HORA-PDE6b, an AAV5-based gene therapy, delivers a functional PDE6b gene to the subretinal space, restoring protein synthesis in photoreceptors and potentially halting retinal degeneration in PDE6b-deficient patients

Relutrigine – Small Molecule

Sponsor	Praxis Precision Medicines
Indication	Dravet Syndrome
Phase	P-II
MOA	Sodium channel antagonists
RoA	Oral
Approval Authority	FDA
Date	Dec 18, 2024

The US FDA has granted RPDD to relutrigine for treating Dravet syndrome

Relutrigine is being assessed under P-II (EMBOLD) study for its safety & efficacy in reducing seizures. Findings from SCN2A and SCN8A patients in cohort 1 were highlighted at AES 2024

Study depicted a 46% reduction in PBO-adjusted monthly motor seizure during double-blind, 30% were seizure free, 77% reduction in median seizure rate during long-term extension & notable improvements in alertness, communication & seizure severity

The company has begun recruitment for second, registrational cohort for SCN2A and SCN8A patients, with topline data anticipated in H1’26

Manufactured Red Blood Cells

Sponsor	Safi Biotherapeutics
Indication	Sickle Cell Disease
Phase	Preclinical
MOA	Unkown
RoA	Unkown
Approval Authority	FDA
Date	Dec 18, 2024

The US FDA has granted ODD & RPDD to manufactured RBCs (mRBCs) for use in chronic transfusion to treat sickle cell patients

The company is advancing its validation of mRBCs cGMP manufacturing for clinical studies & anticipates initiating them by 2027, post-completion of IND-enabling activities

Magnetic EEG Resonance Therapy (MeRT) System

Sponsor	Wave Neuroscience
Indication	Post-Traumatic Stress Disorder
Phase	N/A
MOA	N/A
RoA	N/A
Approval Authority	FDA
Date	Dec 03, 2024

The US FDA has granted BDD to Magnetic EEG Resonance Therapy (MeRT) system as an adjunctive treatment of post-traumatic stress disorder (PTSD)

The MeRT system uses a proprietary algorithm to personalize Transcranial Magnetic Stimulation (TMS) treatment based on an individual’s brain wave data

FieldForce Ablation System

Sponsor	Field Medical
Indication	Ventricular Tachycardia
Phase	N/A
MOA	N/A
RoA	N/A
Approval Authority	FDA
Date	Dec 05, 2024

The US FDA has granted BDD to FieldForce Ablation System for sustained monomorphic scar-related ventricular tachycardia (VT), which makes it eligible for Total Product Life Cycle Advisory Program (TAP) Pilot

The FieldForce Ablation System consists of a single-point contact force PFA catheter with proprietary FieldBending tech, enabling precise targeted lesions and large-volume transmural lesions in the ventricle

MeMed Severity Test

Sponsor	MeMed
Indication	Suspected Sepsis
Phase	N/A
MOA	N/A
RoA	N/A
Approval Authority	FDA
Date	Dec 10, 2024

The US FDA has granted BDD to MeMed Severity test to manage suspected acute infections and suspected sepsis

The test assesses various proteins in the blood sample using advanced host-response technology and identifies the risk of suspected acute infection deteriorating to severe outcomes within 72hrs. or death within 14 days through ML

Tobevibart and Elebsiran

Sponsor	Vir Biotechnology
Indication	Chronic Hepatitis Delta
Phase	P-II
MOA	Virus internalization inhibitors
RoA	Subcutaneous
Approval Authority	EMA
Date	Dec 12, 2024

The US FDA & the EMA have granted BTD & PRIME Designation, respectively, to tobevibart & elebsiran for the treatment of chronic hepatitis delta (CHD) patients

The designations were based on a P-II (SOLSTICE) trial evaluating tobevibart ± elebsiran in CHD patients with results highlighted at AASLD The Liver Meeting. The P-III (ECLIPSE) trial assessing tobevibart & elebsiran in CHD is to initiate in H1’25

GSK’227 – Biologic

Sponsor	GSK
Indication	Relapsed Extensive-stage Small-cell Lung Cancer
Phase	P-I
MOA	B7-H3-targeting ADC
RoA	Intravenous
Approval Authority	EMA
Date	Dec 16, 2024

The EMA has granted PRIME designation to GSK5764227 (GSK’227 or HS-20093), B7-H3-targeted ADC, for treating relapsed extensive-stage SCLC based on preliminary data from P-I (ARTEMIS-001) study (carried out by Hansoh Pharma)

The P-I trial is assessing safety, tolerability & anti-tumor activity of GSK’227 in locally advanced or metastatic solid tumors (incl. ES-SCLC) patients (n=>200), with data highlighted at WCLC 2024. A global P-I trial has begun to support its registrational filing

GSK secured GSK’227’s exclusive worldwide rights (excl. mainland mainland China, Hong Kong, Macau & Taiwan) from Hansoh as per an agreement b/w them

AAV2-hAQP1 – Gene Therapy

Sponsor	MeiraGTx Holdings
Indication	Grade 2/3 Radiation-Induced Xerostomia
Phase	P-II
MOA	Gene transference
RoA	Undisclosed
Approval Authority	FDA
Date	Dec 09, 2024

The US FDA has granted RMAT designation to AAV2-hAQP1 for treating Grade 2/3 radiation-induced xerostomia (RIX)

Designation was based on P-I (AQUAx) study showing significantly improved patient-reported outcomes & saliva production without treatment-related SAEs or DLTs. Results were highlighted at AAOM 2024