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 (Device)
Sponsor
Neuraptive Therapeutics
Indication
Peripheral Nerve Injury
Phase
P-II
MOA
Surgical Product (Undisclosed details)
Approval Authority
FDA
Date
Jan 15, 2024
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
Source: https://www.businesswire.com/news/home/20240115132196/en/Neuraptive-Therapeutics-Inc.-Receives-Orphan-Drug-Designation-for-its-Lead-Development-Asset
DWN12088 – (Small Molecule)
Sponsor
Daewoong Pharmaceutical’s
Indication
Idiopathic Pulmonary Fibrosis (IPF)
Phase
P-II
MOA
Prolyl-tRNA Synthetase inhibitor
Approval Authority
EMA
Date
Jan 29, 2024
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
Source: https://www.prnewswire.com/news-releases/daewoong-pharmaceuticals-bersiporocin-receives-orphan-drug-designation-in-europe-to-treat-idiopathic-pulmonary-fibrosis-302046478.html
IRL201104 – (Peptides)
Sponsor
Revolo Biotherapeutics
Indication
Eosinophilic Esophagitis (EoE)
Phase
P-IIa
MOA
Immune regulatory protein
Approval Authority
FDA
Date
Jan 30, 2024
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
Source: https://revolobio.com/2024/01/30/revolo-biotherapeutics-receives-orphan-drug-designation-from-the-u-s-fda-for-its-first-in-class-peptide-as-a-potential-treatment-for-eosinophilic-esophagitis/#:~:text=GAITHERSBURG%2C%20MD%20and%20CAMBRIDGE%2C%20UK,U.S.%20Food%20and%20Drug%20Administration%20
DOC1021 – (Vaccine)
Sponsor
Diakonos Oncology
Indication
Glioblastoma Multiforme (GBM)
Phase
P-I
MOA
Dendritic Cell Vaccine
Approval Authority
FDA
Date
Jan 02, 2024
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
Source: https://www.businesswire.com/news/home/20240102095438/en/Diakonos-Oncology-Completes-Phase-1-Glioblastoma-Trial-Recruitment-Receives-FDA-Orphan-Drug-Designation
ABSK021 – (Small Molecule)
Sponsor
Abbisko Therapeutic
Indication
Tenosynovial Giant Cell Tumor
Phase
P-Ib
MOA
CSF-1R inhibitor
Approval Authority
EMA
Date
Jan 09, 2024
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
Source: Abbisko Therapeutics – 上海和誉生物医药科技有限公司-Abbisko Therapeutic announces that EMA has granted orphan drug designation for its CSF-1R inhibitor Pimicotinib （ABSK021）
Pravibismane (Small Molecule)
Sponsor
Microbion
Indication
Non-tuberculous Mycobacterial (NTM) Infections
Phase
P-I
MOA
Antibiotic
Approval Authority
FDA
Date
Jan 30, 2024
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
Source: https://microbioncorp.com/_news/microbion-s-pravibismane-granted-second-fda-orphan-drug-designation-for-the-treatment-of-non-tuberculous-mycobacterial-ntm-infections
BX004 – Small Molecule
Sponsor
BiomX
Indication
Chronic Pulmonary Infection
Phase
P-Ib/IIa
MOA
Phage therapy
Approval Authority
FDA
Date
Jan 04, 2024
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
Source: https://ir.biomx.com/news-events/press-releases/detail/101/biomx-receives-orphan-drug-designation-from-the-u-s-food
SGT-003 – (Gene Therapy)
Sponsor
Solid Biosciences
Indication
Duchenne Muscular Dystrophy (DMD)
Phase
P-I/II
MOA
AAV-SLB101 Capsid
Approval Authority
FDA
Date
Jan 16, 2024
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
Source: https://www.solidbio.com/about/media/press-releases/solid-biosciences-granted-fda-orphan-drug-designation-for-duchenne-muscular-dystrophy-gene-therapy-candidate-sgt-003
PRGN-2012 – (Antineoplastics)
Sponsor
Precigen
Indication
Recurrent Respiratory Papillomatosis (RRP)
Phase
P-I/II
MOA
Gorilla AAV Genece therapy
Approval Authority
EU
Date
Jan 16, 2024
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
Source: https://investors.precigen.com/news-releases/news-release-details/precigen-receives-orphan-drug-designation-prgn-2012-treatment
GC1130A – (Recombinant Enzyme)
Sponsor
GC Biopharma
Indication
Sanfilippo Syndrome (type A)
Phase
Preclinical
MOA
Enzyme Replacement Therapy
Approval Authority
EMA
Date
Jan 22, 2024
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
Source: https://www.prnewswire.com/news-releases/european-medicines-agency-ema-grants-orphan-drug-designation-odd-to-gc-biopharmas-treatment-for-sanfilippo-syndrome-type-a-302041406.html
JUV-161 – Protein
Sponsor
Juvena Therapeutics
Indication
Myotonic Dystrophy Type 1 (DM1)
Phase
Preclinical
MOA
human IGF-2 protein
Approval Authority
FDA
Date
Jan 23, 2024
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.
Source: https://www.businesswire.com/news/home/20240123210304/en/Juvena-Therapeutics-Receives-FDA-Orphan-Drug-Designation-for-JUV-161-for-the-Treatment-of-Myotonic-Dystrophy-Type-1
PTX-252 – (Small Molecule)
Sponsor
Pleco Therapeutics
Indication
Acute Myeloid Leukaemia (AML)
Phase
N/A
MOA
Anti- Neoplastics
Approval Authority
FDA
Date
Jan 16, 2024
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
Source: https://plecotherapeutics.com/news/39/86/Pleco-Therapeutics-receives-FDA-Orphan-Drug-Designation.html
SLS009 – (Small Molecule)
Sponsor
SELLAS Life Sciences
Indication
r/r Acute Myeloid Leukemia (AML)
Phase
P-IIa
MOA
highly selective CDK9 inhibitor
Approval Authority
FDA
Date
Jan 09, 2024
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
Source: https://www.sellaslifesciences.com/investors/news/News-Details/2024/SELLAS-Life-Sciences-Receives-FDA-Fast-Track-Designation-for-SLS009-for-Treatment-of-RelapsedRefractory-Acute-Myeloid-Leukemia-and-Provides-Updated-Data-for-Phase-2a-Study-of-SLS009-in-RelapsedRefractory-Acute-Myeloid-Leukemia-Patients/default.aspx
KYV-101 – (Gene Therapy)
Sponsor
Kyverna Therapeutics
Indication
Multiple Sclerosis
Phase
P-II
MOA
CD19-CART
Approval Authority
FDA
Date
Jan 19, 2024
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
Source: https://kyvernatx.com/press-releases/kyverna-therapeutics-granted-fda-fast-track-designation-for-kyv-101-in-the-treatment-of-patients-with-refractory-progressive-multiple-sclerosis/
SGX945- Dusquetide – (Small Molecule)
Sponsor
Soligenix
Indication
Behçet’s Disease
Phase
P-I
MOA
Innate Defense Regulator
Approval Authority
FDA
Date
Jan 8, 2024
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
Source: https://ir.soligenix.com/2024-01-08-FDA-Grants-Soligenix-Fast-Track-Designation-for-Dusquetide-in-the-Treatment-of-Oral-Lesions-of-Behcets-Disease
NX-5948 – (Small Molecule)
Sponsor
Nurix Therapeutics
Indication
Relapsed or Refractory CLL and SLL
Phase
P-I
MOA
Selective BTK degrader
Approval Authority
FDA
Date
Jan 16, 2024
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
Source: https://ir.nurixtx.com/news-releases/news-release-details/nurix-therapeutics-receives-us-fda-fast-track-designation-nx
Rina-S, PRO1184 (Biologics ADC)
Sponsor
ProfoundBio
Indication
Ovarian Cancer
Phase
P-I/II
MOA
FRα-ADC
Approval Authority
FDA
Date
Jan 05, 2024
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
Source: https://www.prnewswire.com/news-releases/profoundbio-announces-rinatabart-sesutecan-fda-fast-track-designation-for-patients-with-advanced-ovarian-cancer-302026902.html
Avutometinib + Sotorasib (Small Molecules)
Sponsor
Verastem Oncology
Indication
KRAS G12C-Mutant NSCLC
Phase
P-I/II
MOA
RAF/MEKi + KRAS G12Ci
Approval Authority
FDA
Date
Jan 18, 2024
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
Source: https://investor.verastem.com/news-releases/news-release-details/verastem-oncology-granted-fast-track-designation-combination
PF614-MPAR – (Small Molecule)
Sponsor
Ensysce Biosciences
Indication
Multi-Pill Abuse Resistance (MPAR)
Phase
P-I
MOA
Opioid mu receptor agonists
Approval Authority
FDA
Date
Jan 23, 2024
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
Source: https://ir.ensysce.com/news/press-releases/detail/139/ensysce-biosciences-announces-fda-breakthrough-therapy
HMPL-523 (Small Molecule)
Sponsor
Hutchmed Limited
Indication
Primary Immune Thrombocytopenia (ITP)
Phase
P-III
MOA
Syk kinase inhibitors
Approval Authority
NMPA
Date
Jan 11, 2024
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)
Source: https://www.hutch-med.com/nda-acceptance-in-china-for-sovleplenib-for-the-treatment-of-primary-immune-thrombocytopenia-with-priority-review-status/
TIVDAK (tisotumab vedotin-tftv) – (Biologics ADC)
Sponsor
Genmab and Pfizer
Indication
Recurrent or Metastatic Cervical Cancer
Phase
P-III
MOA
Tissue Factor (TF) – MMAE ADC
Approval Authority
FDA
Date
Jan 09, 2024
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
Source: https://ir.genmab.com/news-releases/news-release-details/tivdakr-tisotumab-vedotin-tftv-supplemental-biologics-license
ENHERTU (fam-trastuzumab deruxtecan-nxki) – (Biologic ADC)
Sponsor
AstraZeneca and Daiichi Sankyo
Indication
Metastatic HER2-Positive Solid Tumors
Phase
P-II
MOA
HER2 ADC
Approval Authority
FDA
Date
Jan 29, 2024
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.
Source: https://www.astrazeneca-us.com/media/press-releases/2024/enhertu-fam-trastuzumab-deruxtecan-nxki-granted-priority-review-in-the-us-for-patients-with-metastatic-her2-positive-solid-tumors-01292024.html
DF-003 (Small Molecule)
Sponsor
Drug Farm
Indication
ROSAH Syndrome
Phase
P-I
MOA
alpha-kinase 1 (ALPK1) inhibitor
Approval Authority
FDA
Date
Jan 13, 2024
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
Source: https://drug-farm.com/news
4D-710 – (Gene Therapy)
Sponsor
4DMT
Indication
Cystic Fibrosis Lung Disease
Phase
P-I/II
MOA
Gene transference
Approval Authority
FDA
Date
Jan 23, 2024
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
Source: https://4dmt.gcs-web.com/news-releases/news-release-details/4dmt-receives-rare-pediatric-disease-designation-fda-aerosolized
CT-155 – (Device)
Sponsor
Boehringer Ingelheim and Click Therapeutics
Indication
Schizophrenia
Phase
P-III
MOA
Neurobehavioral Intervention
Approval Authority
FDA
Date
Jan 04, 2024
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
Source: https://www.clicktherapeutics.com/press/click-therapeutics-and-boehringer-ingelheim-announce-fda-breakthrough-device/
CanScan -(Device)
Sponsor
Geneseeq
Indication
Multi-Cancer Early Detection
Phase
P-I
MOA
N/A
Approval Authority
FDA
Date
Jan 03, 2024
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
Source: https://na.geneseeq.com/fda-grants-breakthrough-designation-for-geneseeqs-multi-cancer-early-detection-solution/
RENOVITE, BMP-2 – (Device)
Sponsor
Biocomposites
Indication
Interbody Spinal Infusion
Phase
N/A
MOA
Bone Morphogenic Protein 2
Approval Authority
FDA
Date
Jan 04, 2024
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
Source: https://biocomposites.com/news/renovos-breakthrough-device-designation/
OsStic – (Device)
Sponsor
Biomimetic Innovations
Indication
Bone Void Filler
Phase
N/A
MOA
N/A
Approval Authority
FDA
Date
Jan 19, 2024
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
Source: https://www.pbcbiomed.com/uncategorized/fda-grants-global-medical-device-company-biomimetic-innovations-breakthrough-device-designation-for-osstic-synthetic-injectable-structural-bio-adhesive-bone-void-filler/
ShortCut – (Device)
Sponsor
Pi-Cardia
Indication
Coronary Obstruction
Phase
N/A
MOA
N/A
Approval Authority
FDA
Date
Jan 22, 2024
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
Source: https://www.businesswire.com/news/home/20240122184265/en/
REBUILD – (Device)
Sponsor
AbSolutions Med
Indication
Incisional Hernia
Phase
N/A
MOA
N/A
Approval Authority
FDA
Date
Jan 24, 2024
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
Source: https://www.globenewswire.com/news-release/2024/01/25/2816255/0/en/AbSolutions-Med-Inc-Receives-FDA-Breakthrough-Device-Designation-for-its-REBUILD-Bioabsorbable-Abdominal-Wall-Closure-Device.html
NRX-101 (Small Molecule)
Sponsor
NRx Pharmaceuticals
Indication
complicated urinary tract infections (cUTI) and Pyelonephritis
Phase
Preclinical
MOA
Anti-Biotic
Approval Authority
FDA
Date
Jan 16, 2024
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
Source: https://ir.nrxpharma.com/2024-01-16-NRx-Pharmaceuticals,-Inc-Nasdaq-NRXP-Announces-FDA-Qualified-Infectious-Disease-Product-QIDP-and-Fast-Track-Designation-of-NRX-101-in-Complicated-Urinary-Tract-Infection-and-Pyelonephritis
Related Post: New Drug Designations – December 2023
