New Drug Designations

Shots:

PharmaShots' designation report provides a concise overview of several drugs and their designations by the FDA, NMPA and EMA. This month’s report includes designations allotted to 7 small molecules, 5 biologics, 8 cell & gene therapies, 2 recombinant fusion protein, and 3 devices
This month, ChromaDex’s Nicotinamide Riboside Chloride received US FDA’s ODD & RPDD for Ataxia Telangiectasia and Aurion Biotech’s Neltependocel (AURN001) gained FDA’s BTD & RMAT for corneal endothelial disease
We have compiled a list of a total of 22 drugs and 3 devices awarded with designations by multiple regulatory bodies in June 2024

BE-101

Be Bio will conduct the P-I/II (BeCoMe-9) trial of BE-101 for treating severe or moderately severe Hemophilia B during H2’24
BE-101 is a first-in-class B Cell Medicine (BCM) developed to deliver the human FIX gene into primary human B cells for active FIX expression

Tafenoquine

Tafenoquine is being investigated under the P-II study for its safety and efficacy vs PBO for the treatment of acute babesiosis hospitalized patients (n=24) who are at less risk of relapsing; patients will also receive SoC antimicrobial regimen
Furthermore, the company has collaborated with Tufts Medical Center to carry out the clinical evaluation, the enrollment for which will be initiated on June 13, 2024, with additional recruitment sites at prominent university hospitals across the Northeast US

AGMB-447

AGMB-447, a small molecule lung-restricted ALK5 inhibitor, is being investigated under P-I trial for the treatment of idiopathic pulmonary fibrosis (IPF)

SL-172154

SL-172154 is being assessed under the P-I studies for treating Pt-resistant ovarian cancer (NCT04406623, NCT05483933) as well as AML & HR-MDS (NCT05275439)
SL-172154 (SIRPα-Fc-CD40L) is an ARC fusion protein induces anti-tumor response by activating CD40 costimulatory receptor through simultaneous inhibition of CD47/SIRPα checkpoint interaction

RCT1100

The company is assessing RCT1100 under P-I trial for the safety and tolerability of its single ascending dose to treat PCD caused due to pathogenic mutations in the DNAI1 gene. Recruitment is ongoing for part B of the trial
RCT1100, developed using SORT lipid nanoparticle (LNP) delivery platform, demonstrated effective translation of DNAI1 mRNA into DNAI1 protein, restoring ciliary function across PCD models
RCT1100 is an inhaled mRNA therapy that works by transferring DNAI1 mRNA into the airway cells, which gets translated into DNAI1 protein to regain ciliary function

Nicotinamide Riboside Chloride

Along with ODD, the US FDA has also granted RPDD to nicotinamide riboside chloride for treating Ataxia Telangiectasia
Its IND submission with the US FDA is planned to carry out clinical study under the guidance of Dr. Vilhelm (Will) Bohr, Prof., University of Copenhagen and Scientific Advisor to ChromaDex

UCART22

The ODD was supported by the positive opinion from the EMA Committee for Orphan Medicinal Products
UCART22 (allogeneic CD22-CAR T) is being investigated under the P-I/II (BALLI-01) dose-escalation & expansion trial for its safety, expansion, persistence and clinical activity for treating r/r ALL
The last data showed a 67% response rate with UCART22-P2 (manufactured in-house) at dose level 2, vs 50% with UCART22-P1 at dose level 3 (manufactured externally). Further updates are anticipated by YE’24

EryDex System

The US FDA has granted FTD to EryDex System as a treatment of Ataxia-Telangiectasia (A-T)
The P-III trial of EryDex (#IEDAT-02-2015) shows safety & efficacy results in A-T. Another, P-III NEAT (#IEDAT-04-2022) is an ongoing study globally evaluating EryDex in patients to treat A-T, in n=~86 (age= 6-9 yrs.) & n=~20(age≥10yrs.)
This pivotal P-III study has been undertaken by a Special Protocol Assessment (SPA) agreement with the FDA and initiated to conduct the study in the US, UK, and the EU

BNT324/DB-1311

BioNTech and DualityBio ’s BNT324/DB-1311 (ADC) has received the US FDA’s FTD to treat advanced or metastatic CRPC
FTD was based on the P-I/II ongoing study preliminary data shows safety & efficacy in adults with advanced or metastatic solid tumors
Additionally, the FDA has also granted FTD to BNT323/DB-1303 P-I/II study for advanced solid tumors & P-III global study for metastatic breast cancer and BTD for endometrial cancer in 2023. In January 2024, BNT325/DB-1305 (TROP2) received FDA’s FTD to treat adults with platinum-resistant ovarian epithelial cancer

TUB-040

The US FDA has granted FTD to the company’s TUB-040, based on P5 technology, to treat Pt-resistant ovarian cancer. The preclinical models, incl. ovarian cancer, depicted its effective & durable responses
TUB-040 monotx. is being assessed in the P-I/IIa (NAPISTAR 1-01) trial for its safety with P-I finding the MTD or the identified dose for optimization as well as P-IIa designed for dose optimization & preliminary efficacy across the US, UK, Spain, Belgium & Germany
TUB-040 is an ADC that comprises an IgG1 antibody linked with Topoisomerase I inhibitor Exatecan via a cleavable linker system. It targets Napi2b which is highly expressed in ovarian cancer and lung adenocarcinoma

Lunresertib + Camonsertib

The US FDA has granted FTD to the combination of lunresertib & camonsertib for treating CCNE1 amplified and FBXW7 or PPP2R1A-mutated Pt-resistant ovarian cancer
Lunresertib with camonsertib is being developed under P-I (MYTHIC Module 2) study at RP2D to treat ovarian and endometrial cancers with CCNE1 amplification or FBXW7/PPP2R1A mutations. Data from 20-30 patients each are anticipated in Q4’24
The combination was also designated with FTD in Q3’23 for CCNE1 amplified, or FBXW7 or PPP2R1A mutated endometrial cancer

GC1130A (NP3011)

GC Biopharma and Novel Pharma’s GC1130A (NP3011) has received the US FDA’s FTD for the treatment of MPSIIIA (Sanfilippo syndrome Type A)
GC1130A will be assessed under the P-I study for its safety & tolerability among MPSIIIA (Sanfilippo syndrome Type A) patients in Korea, Japan & the US

IBI343

The US FDA has granted FTD to IBI343 (recombinant human anti-Claudin 18.2 monoclonal ADC) as a treatment of r/r advanced unresectable or metastatic pancreatic ductal adenocarcinoma (PDAC)
IBI343 is being assessed in the P-I study for its safety, tolerability, DLTs to determine MTD and/or RP2D & preliminary efficacy for treating locally advanced unresectable or metastatic solid tumors across China and Australia
The results, as of Jan 2024 highlighted at ASCO’24, demonstrated PR achieved by 7/25 patients (PDAC: 5, BTC: 2) with ORR of 28% & DCR of 80%. In the 6mg/kg dose group, ORR was 40% among 10 evaluable PDAC patients with CLDN18.2 1/2/3+≥60%

CT-0525

CT-0525 (ex vivo, gene-modified autologous CAR-Monocyte) is being assessed under the P-I study for its safety, tolerability, and manufacturing feasibility to treat locally advanced, unresectable or metastatic HER2 expressing solid tumors
The trial will recruit patients with HER2 expressing solid tumors pregressed on SoC and will comprise 2 dose escalation arms

ABD-147

The US FDA has granted ODD to ABD-147 for treating ES-SCLC patients, progressed after Pt-based CT
The company anticipates P-I FIH study assessing ABD-147 for treating ES-SCLC progressed after Pt-based CT during H2’24
ABD-147 is a radiopharmaceutical biologic therapy that transfers 225Ac to DLL3 expressing solid tumors

Neltependocel (AURN001)

The US FDA has granted both BTD & RMAT to AURN001 for treating corneal edema secondary to corneal endothelial disease
Aurion has concluded recruitment & dosing for its P-I/II (CLARA) trial across the US & Canada, involving 97 subjects with corneal edema secondary to corneal endothelial dysfunction, to evaluate 3 doses of AURN001 with Y-27632
The drug is approved in Japan

NT-501

The US FDA grants priority review to the BLA of NT-501 for the treatment of MacTel, with the decision anticipated on Dec 17, 2024
NT-501 is an ocular implant intended to slow the disease progression by transferring ciliary neurotrophic factor (CNTF) directly to the retina

Tagrisso

The US FDA has accepted & granted priority review to Tagrisso's sNDA for treating inoperable, stage III EGFRm NSCLC post chemoradiotherapy (CRT) based on P-III (LAURA) study, with the decision expected in Q4’24
The P-III (LAURA) study assesses Tagrisso (80mg, oral, QD) vs PBO to treat inoperable, stage III EGFRm NSCLC patients (n=216) whose disease did not progress post Pt-based CRT. Patients were switched from PBO upon progression
Study depicted 84% reduction in the disease progression or death risk with 39.1mos. vs 5.6mos. mPFS & a PFS benefit in all prespecified subgroups, OS data was immature; though a favorable trend was seen, its evaluation is underway. Results were highlighted at ASCO 2024 & published in the NEJM

Tafasitamab

The NMPA has accepted and granted priority review to the BLA of tafasitamab + lenalidomide to treat r/r DLBCL adults, ineligible for ASCT
Tafasitamab + lenalidomide has received accelerated & conditional approval by the US FDA & EMA, respectively, for the same

Ribitol (BBP-418)

BridgeBio has crossed its interim analysis recruitment target with topline interim data of P-III (FORTIFY) trial anticipated in 2025
BBP-418 is being assessed under P-III (FORTIFY) for its safety & efficacy to treat limb-girdle muscular dystrophy type 2I/R9, with 12mos. interim analysis for evaluating glycosylated αDG as a surrogate endpoint for accelerated approval. Its recruitment is ongoing across the US, UK, EU & Australia
The 1EP, assessed at 36mos., includes the North Star Assessment (NSAD) for limb-girdle type muscular dystrophies
On FDA’s approval of the drug, BridgeBIo may be eligible to receive priority review voucher
Interim results have been published in Journal of Muscle Research & Cell Motility

SLS009

The US FDA has granted RPDD to the company’s SLS009 for the treatment of ALL among pediatric population
On NDA approval, SELLAS will be eligible to receive Priority Review Voucher
Sellas has already received PRVs in past and sold them for an average of >$100M

Nicotinamide Riboside Chloride

Along with RPDD the US FDA has also granted ODD to nicotinamide riboside chloride for treating Ataxia Telangiectasia
Its IND submission with the US FDA is planned to carry out clinical study under the guidance of Dr. Vilhelm (Will) Bohr, Prof., University of Copenhagen and Scientific Advisor to ChromaDex

VIO Skin Platform

The US FDA has granted BDD to VIO Skin Platform (VIO) for assessing lesions suspicious of basal cell carcinoma (BCC) and squamous cell carcinoma (SCC)
The device incorporates VIO technology and VIO.ai NMSC (CADx/CADt software) for lession classification, allowing physicians to make clinical decisions

DynamX Sirolimus-Eluting Coronary Bioadaptor System

The US FDA has granted BDD to DynamX Coronary Bioadaptor System for improving coronary luminal diameter, restoring hemodynamic modulation & decreasing plaque progression symptomatic ischemic heart disease patients
The data from BIOADAPTOR RCT study of DynamX bioadaptor vs SoC showed TLF rate reduction of 65% (1.9% vs 5.5%) & 78% (1.9% vs 8.7%) reduction in the critical LAD artery vessels

AMT-130

The designation was supported by interim data from P-I/II trial of AMT-130 and analysis comparing 24mos. results with non-concurrent natural history cohort
The US P-I/II study assesses the safety, tolerability & efficacy of AMT-130 to treat Huntington’s disease patients (n=26) with 10 patients receiving low-dose & 16 receiving high-dose
The European P-Ib/II trial of AMT-130 is to assess the safety, PoC and optimal dose for P-III confirmatory trial among 13 patients with early Huntington’s disease across 2 dose cohorts (6 in low-dose & 7 in high-dose) for accelerated registration
Patient dosing is underway across the 3rd cohort (up to 12 patients) to assess both doses of AMT-130 with perioperative immunosuppression. Recruitment is anticipated to conclude in the H2’24

NRTX-1001

The US FDA has granted RMAT designation to NRTX-1001 for drug-resistant mesial temporal lobe epilepsy (MTLE)
The P-I/II study assesses the safety & efficacy of NRTX-1001's single administration to treat drug-resistant unilateral MTLE, with 1st stage involving 16 individuals (8 to be treated at a starting dose and 8 at a higher dose)
NRTX-1001 is a regenerative cell therapy from human pluripotent stem cells. It uses fully differentiated GABA releasing interneurons for long-term GABAergic inhibition, repairing hyper-excitable neural networks

Neltependocel (AURN001)

The US FDA has granted both BTD & RMAT to AURN001 for treating corneal edema secondary to corneal endothelial disease
Aurion has concluded recruitment & dosing for its P-I/II (CLARA) trial across the US & Canada, involving 97 subjects with corneal edema secondary to corneal endothelial dysfunction, to evaluate 3 doses of AURN001 with Y-27632
The drug is approved in Japan

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