Drug & Device Pipeline News

January 2, 2024

This week’s Pipeline features a phase 1 trial approval for autism and epilepsy in Italy, a phase 3 trial start for prostate cancer and an FDA approval of a high-risk neuroblastoma drug.

Company	Drug/Device	Medical Condition	Status
Trials Authorized
Avstera Therapeutics	AVS100	Locally advanced or metastatic solid tumors	IND for a phase 1 trial approved by the FDA
ImPact Biotech	Padeliporfin VTP	Unresectable pancreatic adenocarcinoma	IND for a phase 1 trial approved by the FDA
Senti Biosciences	SENTI-202	Relapsed/ refractory hematologic malignancies including acute myeloid leukemia	IND for a phase 1 trial approved by the FDA
CV6 Therapeutics	CV6-168 plus infusional 5-FU	Colorectal cancer and other cancers	Approval for a phase 1a trial granted by the UK’s regulatory authority
IAMA Therapeutics	IAMA-6	Autism and epilepsy	Approval for a phase 1 trial granted by Italy’s regulatory authority
Asieris Pharmaceuticals	APL-2301	Acinetobacter baumannii infections	Approval for a phase 1 trial granted by Australia’s regulatory authority
Uvax Bio	UVAX-1107 and UVAX-1197 vaccines	HIV prevention	Approval for a phase 1 trial granted by Australia’s regulatory authority
Creative Medical Technology	AlloStem cell therapy	Chronic lower back pain	IND for a phase 1/2 trial approved by the FDA
Senhwa Biosciences	Silmitasertib	Community-acquired pneumonia caused by viral infection	Approval for a phase 2 trial granted by Taiwan’s regulatory authority
NRx Pharmaceuticals	NRX-101	Complicated urinary tract infections	IND for a phase 3 trial approved by the FDA
Trials Initiated
Agomab Therapeutics	AGMB-447	Idiopathic pulmonary fibrosis	Initiation of a phase 1 trial
Almirall	ALM223	Autoimmune disease	Initiation of a phase 1 trial
BioLineRx	Motixafortide	CD34+ hematopoietic stem cell mobilization for gene therapies in sickle cell disease	Initiation of a phase 1 trial
Cullinan Oncology	CLN-617	Advanced solid tumors	Initiation of a phase 1 trial
HighField Biopharmaceuticals	HFK1	HER2 low and HER2+ solid tumors	Initiation of a phase 1 trial
Immusoft	ISP-001	Mucopolysaccharidosis type I	Initiation of a phase 1 trial
Kiromic	Deltacel	Stage 4 metastatic non-small cell lung cancer	Initiation of a phase 1 trial
Maze Therapeutics	MZE829	APOL1 kidney disease	Initiation of a phase 1 trial
PepGen	PGN-EDODM1	Myotonic dystrophy type 1	Initiation of a phase 1 trial
SciTech Development	ST-001 nanoFenretinide	T-cell non-Hodgkin's lymphoma	Initiation of a phase 1 trial
Tyra Biosciences	TYRA-200	Advanced solid tumors with activating FGFR2 gene alterations	Initiation of a phase 1 trial
Ethris	ETH47	Viral asthma exacerbations	Initiation of a phase 1 trial in the UK
MBrace Therapeutics	MBRC-101	Advanced metastatic solid tumors refractory to standard treatment	Initiation of a phase 1/1b trial
1ST Biotherapeutics	FB849	Advanced solid tumors	Initiation of a phase 1/2 trial
BioCardia	BCDA-03 allogeneic mesenchymal stem cells	Ischemic heart failure of reduced ejection fraction	Initiation of a phase 1/2 trial
Galapagos	GLPG5301 CAR-T therapy	Relapsed/refractory multiple myeloma	Initiation of a phase 1/2 trial
Carina Biotech	CNA3103	Metastatic colorectal cancer	Initiation of a phase 1/2a trial
Aulos Bioscience	AU-007	Unresectable locally advanced or metastatic solid tumor cancers of the skin and kidney	Initiation of phase 2 portion of a phase 1/2 trial
IO Biotech	IO102-IO103 plus pembrolizumab	Neoadjuvant/adjuvant solid tumors	Initiation of a phase 2 trial
WAVE Life Sciences	WVE-N531	Duchenne muscular dystrophy in boys amenable to exon 53-skipping	Initiation of a phase 2 trial
BioCity Biopharma	BC3402 plus durvalumab	Advanced hepatocellular carcinoma	Initiation of a phase 1b/2 trial in China
Tiziana Life Sciences	Intranasal foralumab	Nonactive secondary-progressive multiple sclerosis	Initiation of a phase 2a trial
Carrick Therapeutics	Samuraciclib plus fulvestrant	HR+ HER2- advanced breast cancer in patients previously treated with a CDK4/6 inhibitor	Initiation of a phase 2b trial
Akero Therapeutics	Efruxifermin	Precirrhotic non-alcoholic steatohepatitis/metabolic dysfunction-associated steatohepatitis (NASH/MASH)	Initiation of a phase 3 trial
Akero Therapeutics	Efruxifermin	Noninvasively diagnosed NASH/MASH or nonalcoholic fatty liver disease/metabolic dysfunction-associated steatotic liver disease	Initiation of a phase 3 trial
Amylyx Pharmaceuticals	AMX0035	Progressive supranuclear palsy	Initiation of a phase 3 trial
Apnimed	AD109	Obstructive sleep apnea	Initiation of a phase 3 trial
AstraZeneca Daiichi Sankyo	Datopotamab deruxtecan plus durvalumab	Stage II-III triple negative breast cancer or HR low, HER2 low or negative breast cancer	Initiation of a phase 3 trial
AstraZeneca Daiichi Sankyo	Datopotamab deruxtecan plus durvalumab	Locally recurrent inoperable or metastatic triple negative breast cancer with PD-L1-expressing tumors	Initiation of a phase 3 trial
Clarity Pharmaceuticals	64Cu-SAR-bisPSMA (diagnostic agent)	Prostate cancer	Initiation of a phase 3 trial
Dermata Therapeutics	DMT310	Moderate-to-severe acne	Initiation of a phase 3 trial
Lynk Pharmaceuticals	LNK01001	Rheumatoid arthritis with inadequate response or intolerant to bDMARDs	Initiation of a phase 3 trial
Oculis	OCS-01 eye drops	Inflammation and pain following cataract surgery in patients with diabetic macular edema	Initiation of a phase 3 trial
Rezolute	RZ358	Congenital hyperinsulinism	Initiation of a phase 3 trial
Travere Therapeutics	Pegtibatinase	Classical homocystinuria	Initiation of a phase 3 trial
Nicox	NCX 470	Ocular hypertension	Initiation of a phase 3b trial
Approvals
Chiesi Global Rare Diseases	Filsuvez (birch triterpenes) topical gel	Epidermolysis bullosa	Approved by the FDA
GC Biopharma	Alyglo (immune globulin intravenous, human-stwk) 10 percent liquid	Primary humoral immunodeficiency in patients 17 and older	Approved by the FDA
Ionis AstraZeneca	Wainua (eplontersen)	Polyneuropathy of hereditary transthyretin-mediated amyloidosis	Approved by the FDA
Merck	Welireg (belzutifan)	Advanced renal cell carcinoma following a PD-1 or PD-L1 inhibitor and a VEGF-TKI	Approved by the FDA
US WorldMeds	Iwilfin (eflornithine)	High-risk neuroblastoma	Approved by the FDA
Astellas Pfizer	Padcev (enfortumab vedotin-ejfv) plus pembrolizumab	Locally advanced or metastatic urothelial cancer	Approved by the FDA for expanded indication
Arcutis Biotherapeutics	Zoryve (roflumilast) topical foam	Seborrheic dermatitis in patients 9 and older	Approved by the FDA for new indication and formulation
Glaukos	iDose TR (travoprost intracameral implant)	Open-angle glaucoma or ocular hypertension	Approved by the FDA