CIO Bulletin

Targazyme is developing the next generation of cancer immunotherapy medicine, one where cancer patients with solid cancer tumors are treated with the power of their own immune system quickly, affordably and with minimal side-effects’

Each year sees a couple of dozen new drugs licensed for use, but in their wake, tens of thousands of candidate drugs will fall by the wayside. The research anddevelopment journey of those new drugs that make it to market will have taken around 15 years.

Before a drug can reach a patient, it must go through rigorous testing to determine whether it is safe, and effective at treating the condition it was developed for and to ascertain the correct dosage and appropriate administration route. Simply put, biotech drug development is a massive undertaking.

Immunotherapies, which harness the body’s natural defenses, have revolutionized cancer treatment. A survey by market research firm InCrowd revealed that hematologists and oncologists considered chimeric antigen receptor (CAR) T-cell therapies the most exciting new development in cancer treatment.

While exciting, these genetically engineered adoptive cell therapies have mostly worked only for patients with blood cancers, but have had less success in treating treating patients with solid cancer tumors. This is a serious limitation since solid tumors such as colon, breast and lung cancer accounts for approximately 98 percent of total new cancer patients annually. Additionally, the high costs of immune cell preparation results in patients being charged about half a million dollars per treatment.

Targazyme is focused on improving immuno-oncology treatments to address the unmet medical needs of 100 million new cancer patients worldwide each year. They are developing the next generation of immune cell therapies called Tumor Infiltrating Lymphocyte (TIL) therapies with their potential for successfully treating cancer patients with solid tumors.

TILs are immune cells that can move from the blood into a tumor. TILs can recognize and kill cancer cells. In cancer immune therapy, TILs are removed from a patient’s tumor, grown in large numbers in a laboratory, and then given back in order to kill the cancer cells.

Targazyme’s TILs are differentiated from other immune cell therapies thanks to the use of their novel fucosylation technology. Cell-cell interactions are controlled by the molecules on the cell’s surfaces. Cells use modifications of the sugars coating their surfaces to regulate these interactions. Fucosylation is the addition of a type of sugar to the cell surface, enabling it to better interact with other cells. Without fucosylation, fewer than 5 percent of transplanted immune cells reach the solid tumors – which is why immune oncology for solid tumors has not been working. Targazyme’s fucosylated TILs reach their solid tumor targets far more effectively.

Preclinical testing data also shows that Targazyme’s fucosylation platform also increases the effectiveness of the transplanted TILs killing machinery and enables intra-tumor penetration of the TILs. All of these changes improve the tumor-killing abilities of their TILs, enabling patients to receive potentially more efficacious and affordable immune cell therapies to treat their solid cancer tumors.

Targazyme’s fucosylation technology has been validated through work published in respected medical journals such as Clinical Cancer Research and IBJ by collaborators at MD Anderson Cancer Center and Harvard. These studies point to Targazyme- manufactured immune cell therapies as a potential alternative to the highly toxic chemotherapy, radiation, and radical surgery that traditionally constitute standard of care. The data points to a day where, with Targazyme’s immunotherapy medicine, patients with solid cancer tumors can be successfully treated with the power of their own immune system.

Targazyme is now raising capital to drive its novel immunotherapy medicine through clinical trials to reach and cure patients.

In conversation with Lynnet Koh, CEO and Founder of Targazyme

Q. Walk us through the journey of the company right from its beginning to the point where it unwaveringly stands today?

Targazyme was founded withthe mission to successfully develop plop the next generation of cancer immunotherapy for metastatic cancer patients, one that can potentially enable cancer patients with solid tumors to be successfully cured with the power of their own immune system safely, affordably, and quickly.

What drove the creation of this mission to make a life-changing difference for cancer patients and families worldwide with our novel medicine was the pain and helplessness I felt in the space of 5 years when I lost several close family members and friends to lymphoma, colon cancer, breast and lung cancers. I could not understand why despite the trillions of dollars spent on cancer research, people I loved went through so much pain and died when they had cancer.

Seeing the pain worldwide, I made a personal commitment to make a big difference to hopefully change the course of cancer medicine and patient outcomes in a big way worldwide by starting Targazyme with the goal to get our potentially transformative drugs to the finishing line to help cancer patents.

Q. What other major illnesses can your TZ 101 and TZ 102 products address besides cancer?

Targazyme has a cell-enabling patent-protected platform technology with multiple products in its portfolio that have the potential to transform cancer patient outcomes as well as patients with blood disorders, autoimmune diseases such as Lupus, MS, ALS, cardiovascular diseases such as myocardial infarction, diabetic retinopathy, critical limb ischemia, to improve in outcomes in regeneration medicine and anti-aging. While the potential of our products to help patients with a wide range of significant illnesses is tremendous, we have been laser-focused on making a life-changing difference for cancer patients and their families.

Mention some of the prominent achievements of your company.

We started Targazyme with nothing but a product idea and a personal commitment to address compelling unmet medical needs in cancer and to remove the pain for cancer patients and their families worldwide.

We have come a long way since then! Now our novel patent- protected TZ 101 has been proven safe and efficacious in phase 2 clinical trials at MD Anderson Cancer Center for blood cancer patients undergoing stem cell transplantation. This has resulted in a FDA Phase 3 Special Protocol Assessment Award which provides Targazyme a clear pathway towards first approval in the United States. We have a phase 3 peripheral blood registration trial pending.

In addition to TZ 101 for treating patients with blood cancers, blood disorders, and autoimmune diseases undergoing hematopoietic stem transplantation, we now have have our second product TZ 102 Tumor Infiltrating Lymphocyte Therapy which we aim to drive into clinical development in collaboration with Yale Medical Center and MD Anderson Cancer Center. This is exciting because it may be the first successful approach to treating solid tumors with the power of the patient’s own immune system, because we are utilizing a basic mechanism that controls cell-cell interactions, our technology has also been shown in preclinical testing to have the potential to help prevent solid organ transplant rejection, help treat autoimmune diseases such as Type 1 Diabetes, Lupus, MS, help enable better outcomes for patients with cardiovascular diseases such as myocardial infarction, diabetic retinopathy, critical limb ischemia etc. Having said that, we have been and will continue to be laser-focused on the development of our oncology/ hematology assets.

Targazyme has 37+patents and an additional 35 patent pending applications worldwide providing- vital freedom to operate and /the ability to obstruct competitors with our patents. Further, Targazyme has, multiple FDA orphan drug and BLA designations which collectively provide market exclusivity additional years of protection from generic drugs, savings on FDA filing fees, and a 50% tax savings for future acquirers of cumulative Targazyme R&D spending.

Q. What does the future hold for your company and its customers? Are exciting things on the way?

We will be raising/allocating significant resources to clinical grade manufacturing, advancing into Registration Trials with TZ101, and advancing into the clinic with TZ102 Tumor Infiltrating Lymphocyte Therapies for the treatment of cancer patients with solid cancer tumors. We want to seek product approval from FDA and other regulators to begin selling our first product, TZ101, and we want to achieve clinical validation for our second product, TZ102 Tumor Infiltrating Lymphocytes therapies.

We hope to demonstrate in clinical trials that our clinical drug candidates can help enable the potentially curative treatment of stem cell transplantation to become much safer and to work more effectively, thereby improving outcomes for terminally ill cancer patients. With TZ 102 Tumor Infiltrating Lymphocyte therapies, we hope to usher in the next frontier of cancer medicine, one where our treatments can harness the patients’ own immune system to obliterate the cancer without awful side effects of current treatments.

To ensure our platform technology also helps patients with other diseases such as auto-immune diseases, cardiovascular diseases, regenerative medicine, we aim to pursue out-licensing deals with other bio-pharmaceutical companies with commercialization capabilities. This strategy will also help us build a licensing revenue- generating business to hopefully provide our shareholders, multiple shots on goal with shareholder value creation.

Helping provide better drugs to combat cancer and save lives

Lynnet Koh is the Founder and CEO of Targazyme. She has been described as a visionary, a major technology/product innovator, a mission-driven, ethical company builder and a ‘chief product officer’– driven by a commitment to developing best-in-class medicine to make a life-changing difference for cancer patients and their families, to help employees/consultants grow to their very best selves and to build a great company that is built to last.

Lynnet is huge on ensuring decisions impact both short-term and medium- and long-term results and being patient-driven and patient-inspired! She is driven to make this world a much better place and leave a legacy that is built to last!