K-Bio receives FDA Orphan Drug Designations with novel drugs

Targets the U.S. market…10 FDA designations in the first half of the year

Clinical trials are being conducted in various diseases, such as Fabry disease, sarcoma, and lymphomas

In the first half of the year, Korean biopharmaceutical companies succeeded in obtaining numerous Orphan Drug Designations.

 

Few patients have rare diseases but developing innovative new drugs can create added value and an exclusive position in the market.

 

In the United States, if a drug is designated as an orphan drug, it is provided seven years of the market exclusivity period.

 

Additionally, the company is provided with support for development and reduced taxes.

 

After completing a phase 2 trial, the drug can be conditionally sold.

 

As a result, biopharmaceutical companies in Korea actively seek FDA orphan drug designation.

 

According to industry sources on July 8th, the U.S.

 

Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to novel drug candidates from Boryung, Rzynomics, NeoImmunetech, SPARK Biopharma, SN BioScience, Ingenium Therapeutics, Dr.

 

Noah Biotech, Hanmi Pharm and GC Biopharma, and GI Innovation.

 

FDA ODD program grants companies that have developed treatments for fewer than 100,000 patients with rare and incurable diseases incentives such as expedited review, reduced taxes, and market exclusivity.

 

K-Bio pharmaceuticals with FDA-approved Orphan Drug Designations in the first half of the year.

 

(From the top, by month) Novel drug candidates from Boryung (BR-101801), Rzynomics (RZ-001), NeoImmunetech (NT-I7), SPARK Biopharma (SBP-401), SN BioScience (SNB-101), Oscotec (cevidoplenib), Ingenium Therapeutics (Gengleucel), Dr.

 

Noah Biotech (NDC-011), Hanmi Pharm and GC Biopharma (LA-GLA), and GI Innovation (GI-102).

Boryung’s BR-101801, a novel drug candidate to treat blood cancer, was the first to receive FDA ODD this year.

 

Boryung is investigating the potential of BR-101801 in various blood cancers, including peripheral T-cell lymphoma and mycosis fungoides.

 

In January, it received the approval for the treatment of angioimmunoblastic T-cell lymphoma.

 

BR101801 is the first-in-class drug candidate to inhibit phosphoinositide 3-kinase (PI3K)γ/ δ and DNA-dependent protein kinase (DNA-PK).

 

It can effectively induce cell death through triple target inhibition and suppress a cancer protein c-Myc.

 

In recently disclosed phase 1b study results, BR101801 was demonstrated as a potential candidate to treat various lymphomas.

 

The clinical studies evaluated the efficacy and safety of BR101801 in patients with peripheral T-cell lymphoma (11 patients), T-cell lymphoma (11 patients), diffuse large B-cell lymphoma (2 patients), and marginal zone lymphoma (1 patient).

 

Over a follow-up of 12.9 months (median value), the objective response rate (ORR) in 19 evaluable patients with peripheral T-cell lymphoma and T-cell lymphoma was 31.6%.

 

4 patients showed complete response (CR), and 2 showed partial response (PR).

 

The progression-free survival (PFS) was confirmed to be 7.5 months, but overall survival (OS) and duration of response (DOR) did not reach median values.

 

For the safety profile, the most common adverse reactions occurred were rashes, an increase in AST/ALT, and coughs.

 

Treatment-associated death did not occur.

 

Boryung plans to apply for a Phase 2 Investigational New Drug (IND) application next year and continue to investigate the efficacy of BR101801.

 

NeoImmuneTech’s NT-I7 received an ODD in the treatment of pancreatic cancer.

 

NT-I7 is a novel drug candidate that targets interleukin (IL)-7, which regulates T-cell development and function.

 

It has been investigated for various indications.

 

Besides the current ODD for pancreatic cancer, NT-I7 received ODDs in the treatment of CD4 lymphocytopenia (2019), multifocal leukoencephalopathy (2020), and glioblastoma (2023).

 

The current ODD in the treatment of pancreatic cancer was based on the Phase 2a results investigating Keytruda combination therapy.

 

The study evaluated the effectiveness and safety of NT-I7 in combination with Keytruda in 50 patients with metastatic colorectal cancer and 48 pancreatic patients who had previously undergone treatments.

 

The clinical results indicated that 3 out of 48 patients with pancreatic cancer have shown partial response (PR).

 

The median overall survival time (OS) was 11.1 months.

 

Additionally, NeoImmuneTech plans to investigate the potential of NT-I7 in combination with cancer vaccines.

 

A Fabry disease treatment, ‘LA-GLA,’ which is under joint development by Hanmi Pharm and GC Biopharma, successfully received an ODD in the United States.

 

LA-GLA is formulated for once-per-month subcutaneous administration.

 

Fabry disease is a type of lysosomal storage disorder (LSD) resulting from a deficiency in a particular enzyme due to a genetic cause, leading to metabolic alterations.

 

Last month, Hanmi Pharm disclosed results demonstrating that LA-GLA inhibits cell death in podocytes, which is important for kidney function in Fabry disease patients.

 

Also, LA-GLA has significantly improved the peripheral sensory functions and histopathological features of nerve cells.

 

Last month, GI Innovation’s GI-102, a candidate immunotherapy for cancer, received FDA ODD.

 

The company is developing GI-102, which acts on CD80 and interleukin (IL)-2.

 

IL-2 is involved in immune cell proliferation and activation, and CD80 blocks CTLA-4, a receptor preventing immune cells from attacking cancer cells.

 

According to the clinical results disclosed at the American Society of Clinical Oncology (ASCO 2024) annual meeting, held in Chicago, U.S., response rates of GI-102 have been confirmed in various diseases, such as melanoma and non-small cell lung cancer (NSCLC).

 

An ORR of 17.4% was observed in 23 patients (7 skin melanoma patients, 4 NSCLC patients, and 3 ovarian cancer patients).

 

The reported overall ORR was 42.9%, and the disease control rate (DCR) was 85.7%, including three cases of partial response (cPR) confirmed in patients with metastatic skin melanoma who had previously experienced ICB.

 

GI Innovation aims to obtain conditional approval for its GI-102 and is assessing the potential for technology transport.

 

They are also examining the potential of using GI-102 in combination with NK cell therapy, as well as GI-102 monotherapy.