FDA Approves Cytalux

On Target Laboratories, Inc., a privately-held biotechnology company developing fluorescent imaging agents to target and illuminate cancer during surgery, today announced that the U.S. Food and Drug Administration (FDA) has approved Cytalux for adult patients with ovarian cancer as an adjunct for intraoperative identification of malignant lesions. Cytalux is the first targeted fluorescent imaging agent that illuminates ovarian cancer intraoperatively, enabling the detection of more cancer for removal. Cytalux, administered by standard IV in as little as one hour before surgery, binds to folate receptors that are overexpressed in most epithelial ovarian cancersi and illuminates intraoperatively under near-infrared light.

"Complete removal of all malignant tissue is the goal of ovarian cancer surgery, however identifying all lesions can be challenging," said Dr. Janos L. Tanyi, MD, PhD, Associate Professor of Obstetrics and Gynecology, University of Pennsylvania Perelman School of Medicine and Investigator on the Phase 2 and 3 studies. "In the Phase 3 study, additional cancer was detected in 27% of patients, showing great promise in the ability of Cytalux to help surgeons identify malignant lesions that may otherwise be missed during surgery."

Ovarian cancer is the number one cause of gynecologic cancer death in the United Statesii. Cytoreductive surgery is a well-established treatment for ovarian cancer, however, a study showed that among patients reported to have undergone optimal cytoreduction, 40% were found to have measurable disease on 30-day postoperative imagingiii. Cytalux serves as an adjunctive tool for surgeons to identify additional malignant ovarian cancer lesions that may have been missed by standard visual inspection and palpation, increasing the detection of more cancer during surgery.

Adverse reactions consisting of nausea, vomiting, abdominal pain, flushing, indigestion, chest discomfort, and itching were reported during the administration of Cytalux (see additional Important Safety Information below).

"This FDA approval is a significant milestone towards achieving On Target's mission to make cancer visible during surgery so it can be removed more completely," said Chris Barys, President and Chief Executive Officer of On Target Laboratories. "We are excited about the potential impact Cytalux can have for patients in their fight against ovarian cancer. Our goal is to make Cytalux a standard of care for ovarian cancer surgery and we look forward to exploring the use of our technology for patients suffering from other cancers."

Cytalux received Priority Review and both Fast Track and Orphan designations from the FDA. Additionally, Cytalux is being investigated in cancer of the lung in a Phase 3 trial under Fast Track designation.

About Fluorescence-Guided Surgery

To date, there have been limited ways for surgeons to confidently assess the location and full extent of cancerous tissue while operating. On Target Laboratories' targeted fluorescent imaging agents are comprised of a near-infrared dye and a targeting molecule, or ligand, that binds to receptors overexpressed on cancer cells. The imaging agents illuminate the cancerous tissue, which may enable surgeons to detect more cancer that otherwise may have been left behind.

On Target's first novel compound, Cytalux, targets folate receptors commonly found on many cancers, such as ovarian cancer. A single dose of the agent is administered via intravenous infusion prior to surgery to help the surgeon identify additional malignant tissue during the operation using a near-infrared imaging system.

About On Target Laboratories, Inc.

On Target Laboratories discovers and develops targeted fluorescent imaging agents to illuminate cancer during surgery so cancerous tissue can be removed more completely. Their fluorescent imaging technology, based on the pioneering work of Philip S. Low, PhD, Purdue University's Presidential Scholar for Drug Discovery and the Ralph C. Corley Distinguished Professor of Chemistry, aims to reduce the uncertainty associated with finding and removing cancerous tissue during surgical procedures, helping surgeons to provide a more precise and complete surgical resection. Cytalux, the Company's first product, received FDA approval for ovarian cancer in November 2021 and is being studied in the Phase 3 ELUCIDATE Trial for lung cancer in the US. For more information visit www.ontargetlabs.com and www.cytalux.com.

About Cytalux Phase 3 (006 Study) Clinical Study Results

FDA approval for Cytalux is based on data from a Phase 3 registration trial which demonstrated that in 27% of patients, Cytalux identified additional lesions which would have been left behind (N=134, 95% CI [0.196, 0.352]). In a subgroup analysis of patients with confirmed ovarian cancer who underwent surgery after chemotherapy (called interval debulking surgery), the rate was 40% of patients (N=58, 95% CI [0.270, 0.534]). This subgroup analysis utilized a smaller analysis set than the primary endpoint and was not adjusted to control for error, so the results may be overstated and should be interpreted cautiously.

Patient-level false positive rate of Cytalux with near-infrared fluorescent light with respect to the detection of ovarian cancer lesions confirmed by central pathology was 20.2% with 95% confidence interval (13.7%, 28.0%).

What is Cytalux?

Cytalux is an FDA-approved optical imaging agent indicated in adult patients with ovarian cancer as an adjunct for intraoperative identification of malignant lesions.

Important Safety Information

Infusion-Related Reactions
Adverse reactions consisting of nausea, vomiting, abdominal pain, flushing, indigestion, chest discomfort, and itching were reported during the administration of Cytalux. Your doctor may treat you with antihistamines and/or anti-nausea medication.

Pregnancy
Cytalux may cause fetal harm when administered to a pregnant woman. There are no available human data to evaluate for a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. Contact your healthcare provider with a known or suspected pregnancy.

Folate Supplement Usage
Folic acid may reduce the detection of cancerous tissue with Cytalux. Patients should stop taking folate, folic acid, or folate-containing supplements 48 hours before administration of Cytalux.

Risk of Misinterpretation
Errors may occur with the use of Cytalux. Sometimes cells may light up even if they are not cancerous or those that are cancerous may not light up. Also, cancerous or non-cancerous cells from other areas may light up, such as areas of the bowel, kidneys, lymph nodes, and inflamed tissue.

Adverse Reactions
The most common side effects of Cytalux reported in clinical trials were nausea, vomiting, abdominal pain, flushing, indigestion, chest discomfort, itching, and allergic reaction during administration or infusion.

Tell your healthcare provider if you have any side effect that bothers you or that does not go away. These are not all the possible side effects of Cytalux. For more information, ask your healthcare provider.

Call your doctor for medical advice about side effects. You may report side effects to On Target Laboratories at 1-844-434-9333 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

i Kalli KR, Oberg AL, Keeney GL, et al. Folate receptor alpha as a tumor target in epithelial ovarian cancer. Gynecologic Oncology. 2008;108(3):619-626.
ii Centers for Disease Control and Prevention. June 8, 2021. Ovarian Cancer Statistics. Accessed August 2021. https://www.cdc.gov/cancer/ovarian/statistics/index.htm
iii Eskander RN, Kauderer J, Tewari KS, Mannel RS, Bristow RE, O'Malley DM, Rubin SC, Glaser GE, Hamilton CA, Fujiwara K, Huh WK, Ueland F, Stephan JM, Burger RA. Correlation between Surgeon's assessment and radiographic evaluation of residual disease in women with advanced stage ovarian cancer reported to have undergone optimal surgical cytoreduction: An NRG Oncology/Gynecologic Oncology Group study. Gynecologic Oncology. 2018 Jun;149(3):525-530. doi: 10.1016/j.ygyno.2018.03.043.

SOURCE On Target Laboratories, Inc.