1stOncology™: Cancer Intelligence Service – Page 12705 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Zydus Cadila Launches Enzalutamide, making Prostate Cancer therapy highly affordable

On May 8, 2020 Zydus Cadila, a global innovation driven healthcare company, reported that it is launching Enzalutamide, a highly effective drug for the treatment of Prostate Cancer, under the brand name ‘Obnyx’ in India (Press release, Zydus Cadila, MAY 8, 2020, View Source [SID1234558211]). In a step that can significantly reduce treatment cost by almost 70%, the drug is priced at Rs 5995 (weekly therapy) reducing the monthly treatment cost to less than Rs. 27000. The current MRP of Enzalutamide drug ranges from Rs. 70000 to Rs 80000 for a monthly therapy and can be a huge financial burden for the elderly patients as they need to continue the therapy for a long period of time. This price reduction will benefit many prostate cancer patients to adhere to the treatment.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

Schedule Your 30 min Free Demo!

One of the important aspects of prostate cancer treatment is reducing the effect of androgens (a male reproductive hormone) on prostate gland. Many patients require Androgen Receptor Targeted Therapies like Enzalutamide which works by blocking the effects of androgen to stop the growth and spread of prostate cancer cells. More importantly, it is a preferred option in patients with significant liver, heart and kidney diseases which is very common in the elderly men. Enzalutamide has an advantage of being taken through oral route. Zydus’ Obnyx scores over other formulations in the market as it is a soft gel capsule filled with liquid, similar to the innovator drug. This has been specially developed through in-house efforts. The other formulations available in the market are hard gelatin capsules.

Prostate cancer is one of the leading cancer in males in India and the risk increases with age. About 1 out of 9 men have risk of developing prostate cancer in lifetime. The incidence is nearly 60% in men over the age of 65 years. Other risk factors such as obesity, family history and improper diet have been identified as the main contributing factors towards an increased incidence of prostate cancer.

FDA Approves Retevmo (selpercatinib) for Treatment of Advanced NSCLC

On May 8, 2020 Bonnie J Addario Lung Cancer Foundation reported that the U.S. Food and Drug Administration approved Retevmo (selpercatinib) capsules to treat three types of tumors – non-small cell lung cancer, medullary thyroid cancer and other types of thyroid cancers – in patients whose tumors have an alteration (mutation or fusion) in a specific gene (RET or "rearranged during transfection") (Press release, Bonnie J Addario Lung Cancer Foundation, MAY 8, 2020, View Source [SID1234557485]). Retevmo is the first therapy approved specifically for cancer patients with the RET gene alterations.

"Innovations in gene-specific therapies continue to advance the practice of medicine at a rapid pace and offer options to patients who previously had few," said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. "The FDA is committed to reviewing treatments like Retevmo that are targeted to specific subsets of patients with cancer."

Specifically, the cancers that Retevmo is approved to treat include:

Non-small cell lung cancer (NSCLC) that has spread in adults,
Advanced medullary thyroid cancer (MTC) or MTC that has spread, in patients 12 and older who require systemic therapy (a treatment option that spreads across the entire body, is not targeted), and
Advanced RET fusion-positive thyroid cancer in those age 12 and older that requires systemic therapy that has stopped responding to radioactive iodine therapy or is not appropriate for radioactive iodine therapy.
Retevmo is a kinase inhibitor, meaning it blocks a type of enzyme (kinase) and helps prevent the cancer cells from growing. Before beginning treatment, the identification of a RET gene alteration must be determined using laboratory testing.

The FDA approved Retevmo on the results of a clinical trial involving patients with each of the three types of tumors. During the clinical trial, patients received 160 mg Retevmo orally twice daily until disease progression or unacceptable toxicity. The major efficacy outcome measures were overall response rate (ORR), which reflects the percentage of patients that had a certain amount of tumor shrinkage, and duration of response (DOR).

Efficacy for NSCLC was evaluated in 105 adult patients with RET fusion-positive NSCLC who were previously treated with platinum chemotherapy. The ORR for the 105 patients was 64%. For 81% of patients who had a response to the treatment, their response lasted at least six months. Efficacy was also evaluated in 39 patients with RET fusion-positive NSCLC who had never undergone treatment. The ORR for these patients was 84%. For 58% of patients who had a response to the treatment, their response lasted at least six months.

Efficacy for MTC in adults and pediatric patients was evaluated in those 12 years of age and older with RET-mutant MTC. The study enrolled 143 patients with advanced or metastatic RET-mutant MTC who had been previously treated with cabozantinib, vandetanib or both (types of chemotherapy), and patients with advanced or metastatic RET-mutant MTC who had not received prior treatment with cabozantinib or vandetanib. The ORR for the 55 previously treated patients was 69%. For 76% of patients who had a response to the treatment, their response lasted at least six months. Efficacy was also evaluated in 88 patients who had not been previously treated with an approved therapy for MTC. The ORR for these patients was 73%. For 61% of patients who had a response to the treatment, their response lasted at least six months.

Efficacy for RET fusion-positive thyroid cancer was evaluated in adults and pediatric patients 12 years of age and older. The study enrolled 19 patients with RET fusion-positive thyroid cancer who were radioactive iodine-refractory (RAI, if an appropriate treatment option) and had received another prior systemic treatment, and eight patients with RET fusion-positive thyroid cancer who were RAI-refractory and had not received any additional therapy. The ORR for the 19 previously treated patients was 79%. For 87% of patients who had a response to the treatment, their response lasted at least six months. Efficacy was also evaluated in eight patients who had not received therapy other than RAI. The ORR for these patients was 100%. For 75% of patients who had a response to the treatment, their response lasted at least six months.

The most common side effects with Retevmo were increased aspartate aminotransferase (AST) and alanine aminotransferase (ALT) enzymes in the liver, increased blood sugar, decreased white blood cell count, decreased albumin in the blood, decreased calcium in the blood, dry mouth, diarrhea, increased creatinine (which can measure kidney function), increased alkaline phosphatase (an enzyme found in the liver and bones), hypertension, fatigue, swelling in the body or limbs, low blood platelet count, increased cholesterol, rash, constipation and decreased sodium in the blood.

Retevmo can cause serious side effects including hepatotoxicity (liver damage or injury), elevated blood pressure, QT prolongation (the heart muscle takes longer than normal to recharge between beats), bleeding and allergic reactions. If a patient experiences hepatotoxicity, Retevmo should be withheld, dose reduced or permanently discontinued. Patients undergoing surgery should tell their doctor as drugs similar to Retevmo have caused problems with wound healing.

Retevmo may cause harm to a developing fetus or a newborn baby. Health care professionals should advise pregnant women of this risk and should advise both females of reproductive potential and males patients with female partners of reproductive potential to use effective contraception during treatment with Retevmo and for one week after the last dose. Additionally, women should not breastfeed while on Retevmo.

Retevmo was approved under the Accelerated Approval pathway, which provides for the approval of drugs that treat serious or life-threatening diseases and generally provide a meaningful advantage over existing treatments. The FDA also granted this application Priority Review and Breakthrough Therapy designation, which expedites the development and review of drugs that are intended to treat a serious condition, when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies. Additionally, Retevmo received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

The FDA granted approval of Retevmo to Loxo Oncology, Inc., a subsidiary of Eli Lilly and Company.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Ayala’s IPO Raises $55M to Test Two Former Bristol Myers Cancer Drugs

On May 8, 2020 Ayala Pharmaceuticals reported that it has two clinical-stage cancer drugs licensed from Bristol Myers Squibb (Press release, Ayala Pharmaceuticals, MAY 8, 2020, View Source [SID1234557442]). Now it has $55 million to take those drugs further than the pharmaceutical giant did.

On Thursday evening, Ayala priced its IPO, which consisted of 3.7 million shares sold for $15 each. That price was the midpoint of the targeted $14 to $16 per share range, but the Rehovot, Israel-based company was able to increase the number of shares in the offering by 10 percent. Ayala is expected to begin trading on the Nasdaq Friday under the stock symbol "AYLA."

Ayala’s lead candidate, AL101, is an injectable small molecule that blocks gamma secretase, an enzyme that activates the Notch signaling pathway. This pathway plays a role in both solid tumors and blood cancers. Bristol (NYSE: BMY) had tested the drug in three Phase 1 studies—all of them failures.

Ayala is trying for a better outcome. In its prospectus, the company says patients in the Bristol studies weren’t selected according to whether their cancers were characterized by Notch activation. Ayala uses DNA sequencing technology to identify patients whose cancers are most likely to respond to AL101. The company is currently testing the drug in an open-label Phase 2 study enrolling patients with adenoid cystic carcinoma, a rare form of cancer affecting secretary glands, such as the salivary glands.

Of the estimated 3,400 patients who have adenoid cystic carcinoma, Ayala says about 1,700 have disease that is recurrent or metastatic. The company calculates that 18 to 20 percent of these patients have cancers with Notch-activating mutations. There are no FDA-approved drugs for this cancer.

As of April 28, Ayala has some early data safety and efficacy data from its Phase 2 test of AL101, according to the prospectus. Additional data will be presented at medical conferences in the second half of this year. Ayala also plans to test AL101 in two other cancers characterized by Notch pathway activation: triple negative breast cancer and acute lymphoblastic leukemia.

Ayala’s second compound, an oral drug called AL102, is also a gamma secretase inhibitor. The company is developing the former Bristol compound as a treatment for desmoid tumors, which occur in connective tissue. There are currently no FDA-approved therapies for desmoid tumors, though SpringWorks Therapeutics (NASDAQ: SWTX) is in the hunt testing a drug licensed from Pfizer (NYSE: PFE). The SpringWorks compound is in Phase 3 testing. Ayala plans to start a Phase 2 trial for its desmoid tumor drug candidate in the second half of this year.

AL102 is also being developed as a potential treatment for multiple myeloma under a partnership with Novartis (NYSE: NVS). The plan is to evaluate the drug in combination with Novartis’s B-cell maturation antigen therapies. The agreement gives the Swiss pharma giant the option to license the Ayala drug. A Phase 1 study has started but patients have not yet been dosed, according to the prospectus. The agreement calls for Novartis to pay up to $4.3 million of Ayala’s research and development expenses.

Ayala plans to use $13 million to $14 million to advance AL101 through the adenoid cystic carcinoma Phase 2 study, the company says in its IPO documents. Another $11 million to $12 million is earmarked for the planned mid-stage study in triple negative breast cancer; $6 million to $7 million for the acute lymphoblastic leukemia test. AL102 will require $7 million to $8 million for the Phase 2 study in desmoid tumors.

Ayala formed in 2017, the same year it licensed AL101 and AL102 from Bristol. The biotech says it has raised $46.3 million since its launch. The company’s largest shareholder is Israel Biotech Fund, which holds a 25.5 percent post-IPO stake, according to the prospectus. Novartis owns 5.2 percent of the biotech; Bristol 4.6 percent.

Akebia Therapeutics to Present at RBC Capital Markets Global Healthcare Virtual Conference

On May 8, 2020 Akebia Therapeutics, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease, reported that its President and Chief Executive Officer, John P. Butler, will participate in the 2020 RBC Capital Markets Global Healthcare Virtual Conference on Wednesday, May 20 at 10:20 a.m. ET (Press release, Akebia, MAY 8, 2020, View Source [SID1234557413]). A live webcast and replay of Akebia’s presentation will be available on the Company’s website at www.akebia.com.

Immunic, Inc. Reports First Quarter 2020 Financial Results and Highlights Recent Activity

On May 8, 2020 Immunic, Inc. (Nasdaq: IMUX), a clinical-stage biopharmaceutical company focused on developing best-in-class, oral therapies for the treatment of chronic inflammatory and autoimmune diseases, reported financial results for the first quarter ended March 31, 2020 and highlighted recent activity (Press release, Immunic, MAY 8, 2020, View Source [SID1234557435]).

"The global coronavirus disease 2019 (COVID-19) pandemic has led to a period of intense, additional focus for Immunic, as we quickly recognized the potential of our lead asset, selective oral DHODH inhibitor, IMU-838, as a possible therapeutic for the treatment of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infections, given its already well-recognized, broad-spectrum antiviral effects," stated Daniel Vitt, Ph.D., Chief Executive Officer and President of Immunic. "After performing preclinical testing, we recently announced that, in cellular assays, IMU-838 successfully demonstrated antiviral activity against SARS-CoV-2 at blood concentrations which are well below those associated with IMU-838 dosing regimens studied in our ongoing and previous clinical trials. Based on these positive results, we are preparing a phase 2 clinical development program for IMU-838 as a potential treatment for this devastating disease."

Dr. Vitt continued, "On the heels of this encouraging preclinical data, we were able to bolster our financial position with a successful $15.0 million financing. As a result, we expect to be well funded through key, near-term milestones including top-line data from both our phase 2 trial of IMU-838 for relapsing-remitting multiple sclerosis (RRMS), expected in the third quarter of this year, as well as results from the investigator-sponsored phase 2 trial for IMU-838 in primary sclerosing cholangitis (PSC), expected in early 2021."

First Quarter 2020 and Subsequent Highlights

April 2020: Completed a $15.0 million registered direct offering led by institutional investor, Altium Capital.
April 2020: Announced that lead asset, IMU-838, has successfully demonstrated preclinical activity against clinical isolates of SARS-CoV-2 associated with COVID-19. As a result, the company is now exploring the feasibility of conducting a prospective, multicenter, randomized, placebo-controlled, double-blind phase 2 clinical trial in patients with moderate COVID-19.
April 2020: Reported several changes to the company’s executive team, including the resignation of Chief Financial Officer, Sanjay S. Patel, CFA, and the promotion of Glenn Whaley to the position of Vice President Finance, Principal Financial and Accounting Officer. Additionally, announced that Duane Nash, MD, JD, MBA, current Chairman of the Board of Directors, has temporarily assumed the role of Executive Chairman.
January 2020: Exercised option to obtain the exclusive worldwide rights to commercialization of a group of compounds, designated by the company as IMU-856, aimed at restoring intestinal barrier function, from Daiichi Sankyo Co., Ltd.
Anticipated Clinical Milestones

Top-line data from the phase 2 EMPhASIS trial of IMU-838 in RRMS is expected to be available in the third quarter of 2020.
Completion of preclinical and manufacturing activities necessary for initiation of phase 1 clinical studies of IMU-856 are expected during the first half of 2020.
The current, single ascending dose part of the ongoing phase 1 trial of IMU-935, being conducted in Australia, is planned to be followed by a multiple ascending dose (MAD) portion in healthy volunteers and a safety evaluation in patients with mild-to-moderate psoriasis as a third part of this phase 1 trial. Based on the temporary pausing of trials in healthy volunteers imposed by Ethics Committees in Australia due to COVID-19, the MAD portion of the trial is expected to start in the second half of 2020.
Top-line data from the phase 2, investigator-sponsored proof-of-concept clinical trial for IMU-838 in PSC, being conducted at the Mayo Clinic, is expected to be available in early 2021.
Top-line data from the phase 2 CALDOSE-1 trial in ulcerative colitis (UC) is expected to be available during the fourth quarter of 2021.
Upcoming Events

Immunic will host a virtual R&D Day on May 19, 2020. Immunic’s management and invited key opinion leaders, specializing in multiple sclerosis and inflammatory bowel disease, will discuss today’s treatment options for, and the unmet medical needs of, chronic inflammatory and autoimmune diseases, as well as clinical progress of the company’s selective oral immunology programs and their potential advantages over the current treatment landscape. Management will also discuss the company’s COVID-19 program.
Financial and Operating Results

Research and Development (R&D) Expenses were $6.4 million for the three months ended March 31, 2020, as compared to $3.4 million for the same period ended March 31, 2019. The $3.1 million increase was primarily attributable to (i) increased development costs for the phase 2 clinical trials of IMU-838 in RRMS and UC, the IMU-935 phase 1 clinical trial, and preclinical activities with IMU-856, (ii) increased employee costs related to the growth in headcount and (iii) other expenses.
General and Administrative (G&A) Expenses were $2.6 million for the three months ended March 31, 2020, as compared to $1.3 million for the same period ended March 31, 2019. The $1.3 million increase is primarily due to becoming a public company and expanding operations into the United States.
Other Income was $0.5 million for the three months ended March 31, 2020, as compared to $0.3 million for the same period ended March 31, 2019. The $0.2 million increase is primarily attributable to research and development tax incentives for clinical trials in Australia as a result of increased spending on clinical trials in Australia.
Net Loss for the three months ended March 31, 2020 was approximately $8.5 million, or $0.79 per basic and diluted share, based on 10,749,460 weighted average common shares outstanding, compared to a net loss of approximately $4.3 million, or $5.09 per basic and diluted share, based on 846,953 weighted average common shares outstanding for the same period ended March 31, 2019.
Cash and Cash Equivalents, as of March 31, 2020, were $18.6 million. With these funds and the money raised in equity issuances in April 2020, including approximately $13.9 million in net proceeds from the registered direct offering closed on April 27, 2020, and an additional $2.3 million in net proceeds from ATM issuances, the company expects to be able to fund operations beyond twelve months from the date of the issuance of this earnings release.