Shots:

• PharmaShots' designation report provides a concise overview of several drugs and their designations by the US FDA, EC, and China’s NMPA. This month’s report includes 13 small molecules, 7 devices, 3 biologics ADC, 3 gene therapy, 1 antineoplastics, 1 protein, 1 peptide, 1 recombinant enzyme & 1 vaccine
• NRx Pharmaceuticals’ NRX-101 small molecule, focused on the treatment of complicated urinary tract infections (cUTI) & Pyelonephritis is the drug to receive both QIDP & FTD from the US FDA
• PharmaShots has compiled a list of a total of 26 drugs and 5 devices awarded with designations by multiple regulatory bodies in Jan 2024

NTX-001

• Neuraptive Therapeutics’ NTX-001 received ODD from the US FDA for peripheral nerve injury
• NTX-001 is the only surgical novel technology treatment that works by enhancing functional performance by preventing Wallerian degeneration in patients with peripheral nerve injury after PEG-fusing severed axons

IRL201104

• The ODD was granted by the US FDA based on the results from the P-IIa (RVLO 121-04) study evaluating the safety, efficacy, & tolerability of IRL201104 (at 3 doses for 2wks.) in patients with active EoE
• The results demonstrated a significant sustained improvement after last dose in patient reported DSQ for 4wks, reduction in eosinophils, CD4+, & CD8+ cells, and an increase in T & B cells vs PBO
• Revolo is planning to initiate the P-IIb study in 2024 where higher doses of IRL201104 is expected to be evaluated in patients with active EoE

DWN12088

• The EMA has granted ODD to Daewoong Pharmaceutical's Bersiporocin (DWN12088) for idiopathic pulmonary fibrosis (IPF). Previously DWN12088 has also received FTD from the US FDA
• The P-I study of Bersiporocin on healthy subjects (n=162) has been completed by demonstrating its safety & PK in Korea & Australia. In USA & Korea, the multinational P-II study is in progress

BX004

• BiomX’s BX004 receives ODD from the US FDA to treat Chronic Pulmonary Infection caused by Pseudomonas Aeruginosa associated with Cystic Fibrosis. BX004 also received FTD from the US FDA for the same indication in Aug 2023
• The P-Ib/IIa study consist of 2 parts in which part 1 of the study evaluates the BX004’s (in SAD & MAD designs) safety, PKs & microbiologic/clinical activity in CF patients (n=9) & part 2 evaluates the safety & efficacy in CF patients (n=34) in randomized (2:1) vs PBO 
• BX004 is an anti-infective molecule which degrades epithelial surface and is developed using BiomX’s proprietary BOLT platform

SGT-003

• Solid Biosciences’ SGT-003 received ODD from the US FDA for Duchenne muscular dystrophy. SGT-003 also received FTD in Dec 2023
• Solid Bioscience is in process to obtain IRB approval to conduct P-I/II study, with expected recruitment in mid-to-late Q1 2024
• SGT-003 developed using proprietary, rationally designed capsid (AAV-SLB101), which contains R16-R17 nNOS binding domain. The mechanism of action and functionality is proven by preclinical study

PRGN-2012

• Precigen’s PRGN-2012 receives ODD from the EMA for the treatment of Recurrent Respiratory Papillomatosis (RRP) & for this treatment Precigen also receive accelerated approval & BTD from the US FDA 
• PRGN-2012 presently being assessed in single arm, P-I/II study in the US. P-I study showed safety profile & easy administration benefit & after 2 yrs. of continuous treatment, 50% of patients remain surgery-free
• The presentation of the P-II study data & BLA submission under an accelerated approval with the US FDA are expected to be done in Q2’24 & H2’24 respectively 
• PRGN-2012 is based on the company’s AdenoVerse platform, & developed by using gorilla adenovector technology that optimized antigen design
• Precigen preparing for the commercial launch in the US and expected to launch in 2025

DOC1021

• Diakonos Oncology’s DOC1021 received ODD from the US FDA for the treatment of malignant glioma incl. diagnosed/refractory glioblastoma multiforme (GBM). It also received ODD for malignant glioma (incl. Newly diagnosed/ refractory GBM) along with FTD
• The single-arm P-I study for glioblastoma has completed the enrollment and evaluates the safety & feasibility of DOC1021 in glioblastoma patients (n=16) across 4 dosing cohorts
• So far DOC1021 is safe and well tolerated and 13/16 patient are alive and progression free. Looking for sites for P-II trial
• DIakonos working with Cellipont bioservices for manufacturing optimization
• Diakonos Oncology also has 2 other vaccines for pancreatic cancer & angiosarcoma in clinical development

Pravibismane

• Microbion's Pravibismane received a second ODD from the US FDA to treat non-tuberculous mycobacterial (NTM) Infections
• The in vivo & in vitro studies illustrate the unique and highly differentiated & potent activity of Pravibismane vsto presently available treatments for NTM infections
• The company plans to start P-III & P-I studies for evaluating the topical Pravibismane on chronic wounds & for inhaled Pravibismane to treat NTM lung infections respectively

ABSK021

• Abbisko Therapeutics’ Pimicotinib (ABSK021) received ODD from the EMA for the treatment of an inoperable tenosynovial giant cell tumor (TGCT). Pimicotinib also received PRIME designation from the EMA in Jun 2023
• In the P-Ib study, after 1 yr. follow-up period Pimicotinib (50 mg, QD) showed an ORR of 87.5 % presented at CTOS 2023. The P-I study of Pimicotinib has been completed in the US & the US FDA granted FTD for unresectable TGCT
• Abbisko is also assessing Pimicotinib for other indications & received approval from China’s NMPA for conducting a P-II study for chronic graft-versus-host disease (cGVHD)
• Abbisko signed a license agreement with Merck & received a non-refundable down payment of $70M in Dec 2023 to commercialize the Pimicotinib for all indications in the Chinese mainland, Hong Kong, Macau & Taiwan. For worldwide development & commercialization, If Merck opts-in, Abbisko will receive a $605.5M milestone amount & on annual sales, double-digit royalty

JUV-161

• Juvena Therapeutics’ JUV-161 received ODD from the US FDA to treat Myotonic Dystrophy Type 1 (DM1)
• In animal models, JUV-161 illustrates the potential to restore muscle fiber formation, counter muscle atrophy, boost metabolism, and increase muscle strength & endurance. Human trials expected to initiate in 2024
• JUV-161 is an engineered human IGF-2 protein developed using Juvena’s AI- based drug discovery platform. JUV-161 is an SC formulation which showed muscle fiber formation ability.

GC1130A

• GC Biopharma’s GC1130A received ODD from the EMA for Sanfilippo Syndrome type A. Previously GC1130A has also received ODD & RPDD from the US FDA in Jan 2023
• GC Biopharma & Novel Pharma collaborated in 2020 for the development of GC1130A, an intracerebroventricular (ICV) Enzyme Replacement Therapy (ERT) candidate
• GC1130A has been formulated using GC biopharma’s proprietary recombinant protein manufacturing technology which makes it compatible with cerebrospinal fluid at high protein concentrations

PTX-252

• Pleco Therapeutics’ lead compound PTX-252 received ODD from the US FDA based on PTX-252's ability to address Acute Myeloid Leukaemia (AML). Recently completed preclinical phase and soon expected to enter clinical stage
• Pleco Therapeutics & Belgian company Hyloris Pharmaceuticals SA have collaborated for the development of PTX-252

SLS009

• SELLAS Life Sciences’ SLS009 received FTD from the US FDA for the treatment of r/r Acute Myeloid Leukemia
• The P-IIa study evaluates SLS009’s safety, tolerability, and efficacy combined with venetoclax/azacitidine at two dose levels (45mg and 60mg) having 5-10 participants in each arm. The 1EPs of the study are CRc, DOR additionally OS, EFS, PK & PD
• In the 45 mg study arm 8/9 (89%) patients remain alive with follow-up of 2-7 mos. & 6 continue the treatment. In 7/8 (87.5%) patients a reduction in bone marrow (≥50%) was observed. AML Patients who failed previously with aza/ven therapy receive SLS009 as monotx. in the P-I study & demonstrates durable CR with no MRD
• The additional data of 45mg & 60mg cohort will be reported in Q1’24 & Q2’24 respectively

KYV-101

• Kyverna Therapeutics’ KYV-101 received FTD from the US FDA for the treatment of Multiple Sclerosis
• The KYV-101 is presently being assessed in 2 studies in patients with lupus nephritis in which ≥½ of patients do not reach CR & have more chances of kidney failure
• KYV-101, CAR T-cell therapy targets CD19, a protein indicated on the surface of B cells that is recognized by the patient’s own modified T cells that remove B cells from the patient’s body
• The NIH designed the CAR in KYV-101 to improve tolerability, presently also being evaluated in the P-I study in oncology patients (n=20) & results were presented in Nature Medicine
• Kyverna is also planning to evaluate KYV-101 in patients with systemic sclerosis, myasthenia gravis, and multiple sclerosis

Rina-S, PRO1184

• ProfoundBio’s Rinatabart Sesutecan (Rina-S; PRO1184) received FTD from the US FDA for Advanced Ovarian Cancer
• The P-I/II (PRO1184-001) study of Rinatabart Sesutecan evaluates the safety, tolerability, PK & antitumor activity in patients with selected locally advanced and/or metastatic solid tumors incl. ovarian cancer & endometrial cancer. Results from Part A (Dose Escalation) were reported in Nov 2023 and Part B (Dose Expansion) is enrolling across the US and China
• Rina-S, PRO1184 is a novel, proprietary hydrophilic exatecan-based linker-drug consists of folate receptor-alpha (FRα) conjugated to sesutecan that targets ADC for the treatment of ovarian, endometrial cancer & other FRα-expressing cancers

Avutometinib + Sotorasib

• Verastem Oncology’s Avutometinib + Sotorasib received FTD from the US FDA for the treatment of KRAS G12C-Mutant Non-Small Cell Lung Cancer (NSCLC)
• The P-I/II (RAMP 203) study assessing the safety & efficacy of avutometinib + sotorasib (4.0 mg, 21/28 days, BIW + 960 mg, 28/28 days, QD; PO, RP2D) in patients with KRAS G12C-mutant NSCLC who previously have been treated &/or not treated with KRAS G12C inhibitor
• The results demonstrated that avutometinib + sotorasib showed similar PK results as avutometinib alone. No drug-drug interactions were observed between avutometinib and sotorasib. Based on DLT assessment, Avutometinib 4.0 mg PO BIW 21/28 days + sotorasib 960 mg PO QD 28/28 days was selected as RP2D
• The RAMP 203 study is actively enrolling patients for the expansion phase & results are expected to be updated in H1’24

SGX945- Dusquetide

• Soligenix’s SGX945 (Dusquetide) received FTD from the US FDA for the treatment of oral lesions associated with Behçet's Disease.
• The P-I study consists of 2 phases, SAD & MAD (n=54, n=30) which evaluates the safety, tolerability, and PK at single & multiple doses of SGX945 (Dusquetide) [(0.15, 1, 2, 3, 5 and 8 mg/kg), (0.5, 1.5, 3, 4.5 and 6.5 mg/kg daily), IV] in healthy subjects vs PBO
• The P-II & P-III studies evaluate the efficacy & +ve results of SGX942 (Dusquetide) in oral mucositis subjects (n=350) who had received chemoradiation therapy for head and neck cancer
• The US FDA will review the sections of NDA before the complete submission because, on a rolling basis, Soligenix will be eligible to submit NDA for SGX945

NX-5948

• Nurix Therapeutics’ NX-5948 received FTD from the US FDA to treat r/r chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL and SLL)
• FTD was based on the P-I study safety & efficacy data. The P-Ia/Ib study evaluates NX-5948 (50-200mg) in patients with CLL (n=6/7) & demonstrates 3 PRs that were ongoing as of Oct 17, 2023.Presented in ASH meeting in Dec 2023.
• The ongoing P-Ia study evaluates NX-5948 safety & tolerability in patients with r/r B cell malignancies who previously received 2 lines of therapy & P-Ib evaluates the efficacy of NX-5948 at the same doses selected in P-Ia up to 7 arms in r/r B cell malignancies incl. CLL, SLL, DLBCL, MCL, WM, MZL, FL & PCNSL
• The P-Ia/Ib study is enrolling patients in the US, UK & the Netherlands & Data on higher doses & longer treatment is anticipated in 2024
• NX-5948 is a small molecule, orally bioavailable, investigational drug that is very effective against tumor cell lines that are resistant to BTK inhibitor therapies

PF614-MPAR

• Ensysce Biosciences’ PF614-MPAR opioid received BTD from the US FDA for Prescription overdose
• PF614-MPAR capsule is designed next generation opioid for drug abuse & overdose, each capsule consists of built-in protection which will provide pain relief to the patient if the excess amount of API has been consumed

TIVDAK (tisotumab vedotin-tftv)

• The sBLA application of TIVDAK (tisotumab vedotin-tftv) has been accepted by the US FDA for priority review to treat recurrent/metastatic Cervical cancer & PDUFA date assigned is May 9, 2024
• The acceptance of sBLA was based on the P-III (innovaTV 301) study which evaluates the safety & efficacy profile of TIVDAK vs CT (topotecan, vinorelbine, gemcitabine, irinotecan, or pemetrexed) as monotx in patients (n=502) with recurrent/metastatic Cervical Cancer. The 1EP was OS & 2EPs were PFS, ORR, time to response, duration of response
• The study demonstrates the OS of 30%, PFS of 30%, ORR of 17.8% vs 5.2%, the complete response of 2.4% & DCR of 75.9% in the TIVDAK arm vs 58.2% in the CT arm

HMPL-523

• HUTCHMED’s Sovleplenib (HMPL-523) NDA has been accepted in China & received priority review from the NMPA for the treatment of primary immune thrombocytopenia (ITP). Sovleplenib also received BTD from the NMPA for the same indication in Jan 2022
• The NDA is based on the P-III (ESLIM-01) study that evaluates Sovleplenib in patients (n=188) with primary ITP who had previously received at least 1L of standard therapy
• Sovleplenib met the 1EP & 2EPs which showed significant increase in durable response vs PBO and safety & response rate respectively. The results were published in The Lancet Haematology 
• Sovleplenib is also being assessed in P-I study for indolent non-Hodgkin lymphoma & P-II & P-III studies for warm antibody autoimmune hemolytic anemia (AHA)

ENHERTU (fam-trastuzumab deruxtecan-nxki)

• AstraZeneca & Daiichi Sankyo’s ENHERTU, sBLA has been accepted & granted priority review from the US FDA to treat metastatic HER2+ solid tumors & with expected PDUFA in Q2’24
• The sBLA is based on the results from the ongoing P-II (DESTINY-PanTumor02) study for ENHERTU that illustrates a DoR that induces survival benefits in patients who previously get treatment across HER2-expressing metastatic solid tumors incl. other tumors. DESTINY-Lung01 & DESTINY-CRC02 study data also supports this submission
• The results demonstrate the PFS of 6.9 mos., OS of 13.4 mos., ORR of 37.1% & DoR of 11.3 mos. In HER2+ solid tumors whereas ORR of 61.3%, PFS of 11.9 mos., OS of 21.1 mos., & DoR of 22.1 mos. demonstrate in patients with the highest response to HER2+ solid tumors
• The sBLA will be reviewed under RTOR. Results from DESTINY-PanTumor02 were presented at the 2023 ESMO and published in the Journal of Clinical Oncology.

4D-710

• 4DMT’s aerosolized 4D-710 received RPDD from the US FDA for the treatment of Cystic Fibrosis Lung Disease
• 4D-710 is currently being evaluated in the P-I/II (AEROW) study in patients with CF who can’t tolerate or are not eligible for presently approved CFTR modulators
• In the 1st & 2nd arms, (1E15 vg and 2E15 vg, respectively) patients (n=7) illustrate promising & reproducible CFTR expression, significantly above normal levels. Moreover, patients (n=3) in 1st arm also illustrate durable improvement or stabilization of both quality of life and pulmonary function in 12 mos.
• Low dose of 4D-710 is being evaluated in the dose exploration 3rd arm (5E14 vg)
• The company is expected to present interim results of the AEROW study in mid-2024
• 4D-710 consists of codon-optimized CFTR∆R transgene and a developed, targeted next-generation vector, A101

DF-003

• Drug Farm’s DF-003 received RPDD from the US FDA for the treatment of ROSAH Syndrome
• The ongoing P-I part-1 study evaluates safety, tolerability & PKs of DF-003 (3 x 1mg) in SAD in healthy subjects (n=64) vs PBO while part 2 will evaluates DF-003 (3 x 1mg) in MAD in subjects (n=32) based on part 1 data

CT-155

• Boehringer Ingelheim and Click Therapeutics’ CT-155 received BDD from the US FDA for -ve symptoms associated with Schizophrenia
• CT-155 is a novel software that was developed as an investigational PDT (prescription digital therapeutic), accessible in mobile devices & has the potential to treat -ve symptoms of schizophrenia over current SoC
• CT-155, if approved could be used as an adjunctive therapy along with SoC pharma products
• CT-155 is presently being assessed in P-II registration study CONVOKE since May 2023 and have shown positive and supportive results from previous studies

CanScan

• Geneseeq’s CanScan receives BDD from the US FDA for a multi-cancer early-detection solution. CanScan assay kit also received CE approval in Jan 2023
• The ongoing P-I (Jinling Cohort) study evaluates CanScan, and DECIPHER (Detecting Early Cancer by Inspecting ctDNA Features) on 13 tumors (n=15,000). The 1EPs are sensitivity, specificity, +ve/-ve predictive value & 2EPs are accuracy, how many no. of tumors it can detect. The study is expected to be completed in 2024
• CanScan is developed on the company’s highly sensitive MERCURY multi-omics technology & uses WGS by circulating ctDNA for providing 99% precision & prediction of tissue of origin (TOO) tumors for diagnosis

RENOVITE, BMP-2

• Renovos Biologics’ (investee company of Biocomposites) RENOVITE BMP-2 receives BDD from the US FDA for interbody spinal fusion
• RENOVITE BMP-2 is in development on proprietary synthetic nano clay gel that is safe & does not leach BMP-2 with the gel. It is an injectable, easy-to-use gel that contains a growth factor, BMP-2 that stimulates the growth of bone-forming cells
• RENOVITE BMP2 aims to replace bone graft material and has a property to biodegrade after new bone formation

OsStic

• Biomimetic Innovations’ OsStic received BDD from the US FDA as an Injectable Structural Bio-Adhesive Bone Void Filler
• OsStic is a novel technology that enhances structural stability, bio adhesive for reduction while standard fixation does not give enough support for functional mobilization

ShortCut

• Pi-Cardia’s ShortCut receives BDD from the US FDA for TAVR procedures associated with coronary obstruction
• The ShortCut is developed by Pi-Cardia as a dedicated leaflet modification device that is a part of the leaflet modification product portfolio which includes TMVR & Leaflex

REBUILD

• AbSolutions’ REBUILD received BDD from the US FDA as a Bioabsorbable (“REBUILD”) abdominal wall closure device
• REBUILD device inserts by suture-less procedure on a wide area of tissue to maintain midline fascial structures during the after-surgery healing period for reducing the complication of long-term foreign body risk

NRX-101

• NRx Pharmaceuticals’ NRX-101 received QIDP & FTD from the US FDA for the treatment of complicated urinary tract infections (cUTI) & Pyelonephritis
• The company is eligible for the rolling submission of NDA after receiving FTD & actively seeking partners for commercialization of NRX-101 in urology, infectious disease and/or women's health
• NRX-101 has D-cycloserine (DCS) as an active antibiotic ingredient mostly used for tuberculosis developed in 1950 which was later flunked due to CNS adverse effects. Based on NRx analysis, NRx is assessing low dose of DCS which can provide therapeutic advantage without raising CNS events

Related Post: New Drug Designations - December 2023