Cancer affects millions of people annually, and expenditures for cancer care are expected to increase over the years. The American Cancer Society estimates there will be approximately 1.74M cancer cases for 2018, or around 4,700 new cancer diagnoses per day. According to the National Cancer Institute, National expenditures for cancer care in the U.S. alone totaled nearly $125B in 2010 and could reach over $150B in 2020.
Patients have started to adopt small molecule targeted cancer therapies due to their minimal adverse effects, as well as the high incidence rates of cancer. Unlike chemotherapies and traditional cytotoxic medications, which work by destroying cells that are dividing at a rapid rate, small molecule targeted cancer therapy destroys cancer cells with high precision. The main problem with using chemotherapies is the fact that they could harm normal cells during treatment. However, treatments should only be targeting cancer cells. With small molecule targeted cancer therapy, normal cells are able to survive and only suffer minimal damage.
Small molecule targeted cancer therapies work by blocking cancerous cell growth and destroying targeted cancer cells that result in the growth of tumors and carcinogenesis. That said, this treatment is considered to be significantly more effective than traditional methods. Small molecule targeted cancer treatments have an array of applications in various cancers, such as melanoma, prostate cancer, lymphoma, breast cancer, multiple myeloma and all other cancers.
Now, there’s one small company trading on the Nasdaq that is potentially on the brink of Food and Drug Administration (FDA) approval for one of its small molecule drug candidates. With positive Phase II data for one of its treatments, this company could take a piece of the small molecule targeted cancer therapy market and potentially grow its earnings within the foreseeable future.
Onconova Therapeutics (NASDAQ: ONTX)
Dr. Ramesh Kumar co-founded Onconova Therapeutics (NASDAQ: ONTX) in 1998, and is headquartered in Newtown, PA. Currently, Onconova Therapeutics is a Phase III stage biopharmaceutical company primarily focused on developing and discovering novel small molecule drug candidates to treat cancer. The company is developing its pipeline of targeted anti-cancer treatments, which are designed to work against specific cellular pathways critical to cancer cells. While targeting these pathways, ONTX looks to minimize damage to normal cells.
The company has advanced three different novel product candidates to the clinic and has multiple active pre-clinical programs. Now, the advanced clinical trials involves one of the company’s lead compounds, rigosertib, which is aimed at unmet medical needs of myelodysplastic syndromes (MDS) patients.
Onconova Therapeutics (NASDAQ: ONTX) is committed to providing cancer patients with novel solutions that could be more beneficial than traditional treatments. The company is looking to develop products that could improve outcomes for cancer patients by direct addressing the disease, the side-effects and recurrence.
Onconova Therapeutics Lead Compound With Promising Data
ONTX recently presented promising data from its Phase II expansion study of oral rigosertib and azacitidine combination in patients with myelodysplastic syndromes (MDS), often referred to as “bone marrow failure disorder.” MDS are primary bone marrow stem cell disorders, which are on the rise due to the disease’s prevalence in the elderly and a longer life span for the population. Although MDS is primarily found in the elderly population, it could also affect younger patients.
Rigosertib belongs to a novel class of organic molecules:
Source: Taylor & Francis
According to the MDS Foundation, “Failure of the bone marrow to produce mature healthy cells is a gradual process, and therefore MDS is not necessarily a terminal disease. Some patients do succumb to the direct effects of the disease: reduced blood cell and/or reduced platelet counts may be accompanied by the loss of the body’s ability to fight infections and control bleeding. In addition, for roughly 30% of the patients diagnosed with MDS, this type of bone marrow failure syndrome will progress to acute myeloid leukemia (AML).”
Oral rigosertib has been developed as a single agent in combination with azacitidine. Previous studies of the single-agent treatment in Low-Risk and (LR) MDS patients with intermittent oral rigosertib treatment at a dose of 560 mg BID showed a transfusion independence rate of 44%. Now, oral rigosertib in combination with azacitidine (AZA) is being studied in patients with high-risk (HR) MDS.
The Phase II in study showed promising results. The initial Phase II study with oral rigosertib in combination with AZA in HR MDS patients demonstrated an overall response rate of 76%, 62% in patients following hypomethylating agent (HMA) failure and 85% in HMA naive patients.
According to Professor and Chief of the MDS Section at the MD Anderson Cancer Center Guillermo Garcia-Manero stated, “Choices are very limited for higher risk MDS, with two HMAs as the only drugs approved by the Health Authorities for these patients. There is an urgent need to develop novel approaches, including combination therapies that can improve the outcomes in patients who require an HMA. The previously presented studies of the combination regimen of oral rigosertib with azacitidine have demonstrated impressive evidence of efficacy in HMA naïve and HMA refractory patients with higher-risk MDS.”
Dr. Garcia-Manero added, “Since the success of a combination therapy is greatly influenced by the safety and tolerability of the regimen, the new results of improved tolerability are of great importance for the proposed pivotal study of this combination. The ability to ensure longer duration of treatment without interruption or dose reduction due to an acceptable safety profile can ensure optimal benefit for patients. We look forward to participating in the planned Phase 3 study of this novel approach, which combines two agents with distinct mechanisms of action for the potential benefit of frontline MDS patients.”
With the company participating in the planned Phase III study of rigosertib and AZA, if it announces positive data for that study, it could potentially be a positive catalyst for investors to take seriously.
Another study is being conducted on oral rigosertib in combination with AZA. This treatment demonstrated a higher complete remission rate among HMA-naive HR-MDS patients. The responses are more durable and occur more rapidly with the combined treatments.
ONTX is conducting studies for IV Briciclib. Briciclib is an experimental anti-cancer drug, which is administered intravenously once per week as increasing doses in adult patients with advanced cancer and solid tumors.
Recilisib is a radioprotectant, modifying cell cycle distribution patterns in cancer cells, which are subjected to radiation therapy. Now, this candidate is currently in the Phase I stage of clinical trials.
Strong Leadership Could Bring These Treatments to the Market
ONTX has a strong management team, which is key for any biopharmaceutical company looking to market its products.
President and CEO Dr. Ramesh Kumar received his Ph.D. in Molecular Biology and trained at the National Cancer Institute. He’s had positions in Research & Development (R&D) at Princeton University, Bristol-Myers Squibb, DNX (which was later known as Nextran, a subsidiary of Baxter), Kimeragen (later Valigen). Onconova Therapeutics CEO has over 50 publications in gene therapy and recombination, molecular oncology, and transgenic animals. Moreover, Dr. Kumar is an inventor in eight U.S. patents.
Chief Medical Officer and Senior Vice President of Research and Development, Dr. Steven M. Fruchtman, has a plethora of experience in both small and large biopharmaceutical companies. Dr. Fruchtman led multiple successful clinical development programs while serving in senior positions at Novartis, Spectrum Pharmaceuticals, Ortho Biotech Products, Allos Therapeutics and Syndax Pharmaceuticals. That said, with his experience, he could potentially lead Onconova Therapeutics’ candidates to successful clinical trials.
Senior Vice President Product Development, Dr. Manoj Maniar, also led the development and commercialization of multiple pharmaceutical products and medical devices during his career.
Not only does Onconova Therapeutics (NASD: ONTX) have a strong management team, it also developed a partnership with Symbio Pharmaceuticals. ONTX has been collaborating with Symbio Pharmaceuticals for over five years, and they are looking to develop and commercialize rigosertib in Japan and Korea.
Now, SymBio has an exclusive license for Japan and Korea. The company would develop and commercialize rigosertib in these countries. In turn, Onconova would receive an upfront payment and development milestones tied to the progress of rigosertib. Moreover, it would receive sales milestone payments plus royalties on net sales.
The Bottom Line
Overall, Onconova Therapeutics could potentially see a rise in its stock price due to a number of reasons, such as:
- Positive data for its lead compound
- Pivotal Phase III trial INSPIRE on four continents for rigosertib
- Unique platform targeting Ras pathways, which is one of the most sought after targets in the oncology industry
- Rigosertib is protected by issued patents and has been awarded Orphan Designation for MDS in the United States, Europe and Japan.
- Oral rigosertib, in combination with azacitidine, is being developed for a much larger patient population.
- Extensive pipeline with clinical and several pre-clinical stage drug candidates
With the need for innovative cancer therapies that provide minimal damage to normal cells, Onconova Therapeutics (NASD: ONTX) could provide cancer patients with a “new” treatment with favorable outcomes. Since the cancer treatment market is so large, Onconova Therapeutics only needs to execute and take a small piece of the market to reap the rewards.