First-in-human Phase 1a/1b clinical trial will evaluate orally available,
RGT-61159 designed to selectively target MYB RNA and inhibit
oncogenic MYB protein production
Preclinical data supporting the clinical trial
were recently presented at the American Society of Clinical
Oncology Annual Meeting (ASCO 2024)
WOBURN,
Mass., July 10, 2024 /PRNewswire/ -- Rgenta
Therapeutics, a biotechnology company pioneering the development of
a new class of oral small molecules targeting RNA and RNA
regulation for oncology and neurological disorders, announced the
clearance of its Investigational New Drug application (IND) by the
U.S. Food and Drug Administration (FDA) for RGT-61159, which is
being developed for the potential treatment of adenoid cystic
carcinoma (ACC), colorectal cancer (CRC) and other solid tumors as
well as acute myeloid leukemia (AML).
"Clearance of our first IND application is a significant
milestone in Rgenta's mission to develop oral, small molecule
RNA-targeting medicines to treat previously incurable diseases,"
said Simon Xi, Ph.D., co-founder and chief executive officer of
Rgenta. "We look forward to initiating clinical studies of
RGT-61159 with a first-in-human Phase1a/1b clinical study in adults with ACC and CRC to
potentially provide a new therapeutic option for patients with
these difficult-to-treat cancers."
"Development of small molecule drugs targeting oncogenic drivers
such as MYB, has proven challenging in the past," said Travis Wager, Ph.D., co-founder, president and
chief scientific officer. "RGT-61159 demonstrates potent inhibition
of oncogenic MYB protein production and significant inhibition of
tumor growth at tolerated doses in preclinical models of ACC and
other cancers, and we are excited to move this novel therapeutic
into clinical evaluation."
About RGT-61159
RGT-61159 is an orally available small
molecule designed to specifically modulate splicing of the
transcription factor MYB resulting in the inhibition of oncogenic
MYB protein production, which has the potential to inhibit
proliferation or induce cell death of cancer cells that overexpress
MYB protein. MYB acts as a master regulator of cell proliferation
differentiation processes and its aberrant expression has been
demonstrated in multiple forms of human cancer including adenoid
cystic carcinoma (ACC), acute myeloid leukemias (AML), T-cell acute
lymphoblastic leukemias (T-ALL), colorectal cancer (CRC), small
cell lung cancer (SCLC) and breast cancer. Additional information
on the planned Phase 1a/1b clinical
trial can be accessed at clinicaltrials.gov (NCT06462183).
About Adenoid Cystic Carcinoma (ACC)
It is estimated
that approximately 200,000 people are living with ACC throughout
the world including 11,000 in the US. While it is a rare cancer,
ACC is the second most common cancer type arising in the salivary
gland and is an aggressive malignancy with a tendency to infiltrate
surrounding nerves and metastasize to distant sites. Overactivation
of the MYB oncogene has been described as a hallmark of ACC and is
noted in over 90% of ACC. Treatment for ACC is extremely
challenging and may include surgery and/or radiation, which often
fails to control local tumor recurrence and distant metastases.
There are no effective targeted therapies available for patients
with recurrent and/or metastatic disease. There is thus an unmet
medical need for new therapeutic targets and treatment strategies
for patients with this fatal cancer.
About Colorectal Cancer (CRC)
CRC is the third most
prevalent cancer and the second leading cause of cancer-related
mortality worldwide. According to the World Health Organization, in
2022, more than 1.9 million cases of CRC were diagnosed. Despite
the improved early detection of CRC and the recent success of
targeted therapeutics, approximately 15%-30% of patients present
with metastases and 20%-50% of patients with initially localized
disease will develop metastases. Patients with relapsed or
refractory CRC who have exhausted all the available standard of
care therapy options, have a very poor prognosis. MYB is
significantly overexpressed in 80-85% of CRC and has been
frequently found to be a predictive biomarker of tumor
aggressiveness and poor prognosis. Developing novel therapies to
treat patients with metastatic CRC remains a major unmet medical
need.
About Rgenta Therapeutics
Rgenta Therapeutics is
developing a pipeline of oral, small-molecule RNA-targeting
medicines with an initial focus on oncology and neurological
disorders. Our proprietary platform mines the massive genomics data
to identify targetable RNA processing events and design
small-molecule glues to modulate the interactions among the
spliceosome, regulatory proteins, and RNAs. Our lead programs and
unique approach are unlocking the therapeutic potential of
historically undruggable targets in human diseases. Learn more at
http://www.rgentatx.com.
Contacts
Investors:
Sylvia Wheeler
Wheelhouse Life Science Advisors
Swheeler@wheelhouselsa.com
Elizabeth Wolffe, Ph.D.
Wheelhouse Life Science Advisors
Lwolffe@wheelhouselsa.com
Media
Aljanae Reynolds
Wheelhouse Life Science Advisors
Areynolds@wheelhouselsa.com
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SOURCE Rgenta Therapeutics