NEW DRUGS ON THE HORIZON FOR 2019

NEW DRUGS ON THE HORIZON FOR 2019

by Daniel Dupuis

There are a number of new medications in development that offer help for patients suffering from a number of different diseases.

Cancer research, much like last year, is prominent among both small biotech firms, in addition to large pharmaceutical companies. The main focus is on how they might maintain or increase efficacy, while hoping to mitigate life-altering side effects and/or safety concerns.

Many other sectors have also seen some major breakthroughs, among them ALS, Multiple Systems Atrophy, complicated urinary tract infections and chronic migraines.

This list includes some major developments in oncology drug development, in addition to a new treatment for plaque psoriasis and a promising formulation for Alzheimer’s disease.

Talazoparib For Breast Cancer

Talazoparib is a new medication from Pfizer that is currently in phase 3 trials that offers some hope for women with advanced stage breast cancer (with the BRCA1 and BRCA2 gene mutation). The BRCA gene mutations are common among aggressive breast and ovarian cancers.

Patients that took part in the study lived longer without their cancer progressing by an average of three months more than women treated with standard chemotherapy. While three months may seem to be an incremental change, it is a substantial period of time relevant to improvements within the cancer sector. There are many drugs that have been approved in the past that have improved life expectancy by periods of time that are measured in days, rather than weeks.

In the current trial, researchers found that the women who were randomly selected to receive talazoparib had a higher response rate to treatment than women who received standard chemotherapy: 63 percent versus 27 percent.

The drug does, however, have side effects. Among women receiving talazoparib, 55 percent had blood disorders, mostly anemia, compared with 38 percent of those receiving standard chemotherapy.

In addition, 32 percent of the women receiving talazoparib had other side effects, which is only slightly less than the rate of 38 percent for those in the trial that were on standard chemotherapy.

New Drug from Galera Therapeutics Reduces Effects of Radiation

Within the oncology sector, there are a number of approaches to mitigating the life-altering side effects and safety risks associated with cancer therapies, which includes the many immuno-therapies and combination drugs that are currently under investigation.

Galera Therapeutics has pursued a novel approach to helping reduce a prominent side effect of radiation therapy. The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to GC4419 for the reduction of the duration, incidence and severity of mouth lesions induced by radiotherapy for head and neck cancer.

Results from Galera’s 223-patient, double blind, randomized, placebo-controlled Phase 2b clinical trial demonstrated GC4419’s ability to dramatically reduce the duration of mouth lesions from 19 days to 1.5 days (92 percent), the incidence of lesions through completion of radiation by 34 percent and the severity by 47 percent. It also indicated that GC4419 increased tumor response to radiation therapy while preventing toxicity in normal tissue. Mouth lesions are one of the most commonly cited side effects among people under radiotherapy.

While mouth lesions are its lead indication, GC4419 is also being studied in combination with SBRT for its anti-tumor effect in a Phase 1/2 trial of patients with locally advanced pancreatic cancer.

Aneustat Is a True Breakthrough Medication for Prostate Cancer

Aneustat is an immunotherapy that is truly groundbreaking. It is taken orally and offers substantial efficacy, yet has a negligible side effect profile and presents no known safety risks. It is a multivalent, multifunctional platform drug. It is identified as a platform drug because it has been subject to studies that pair it with a number of currently utilized medications for prostate cancer. Studies indicate that it can significantly increase efficacy, while reducing side effects, and even more importantly, drug resistance. Combination therapy has become an increasingly popular option and Aneustat has been proven to be effective as both a stand-alone medication and as a component of combination therapy while being paired with numerous cancer drugs.

Aneustat is scheduled to begin a phase 3 study in collaboration with Johns Hopkins that will focus on Aneustat with enzalutimide for post-surgical, radiation recurrent prostate cancer, while another study at the Tisch Cancer Center at Mt. Sinai will examine Aneustat in combination with docataxel for metastatic castration resistant prostate cancer.

New Alzheimer’s Drug Slows Memory Loss in Early Trial Results

The long, discouraging quest for a medication that works to treat Alzheimer’s disease reached a potentially promising milestone. For the first time in a large clinical trial, a drug was able to both reduce the plaques in the brains of patients and slow the progression of dementia.

The drug, currently known as BAN2401, is a result of a cooperative initiative of Eisai, a Japanese company, and Biogen, based in Cambridge, Mass. Eisai is the maker of Aricept, which is one of the few drugs that can help slow early memory decline, but which is effective for only about six to nine months, while Biogen is the maker of another Alzheimer’s treatment, aducanumab, that has shown early promise in a small Phase 1 trial in both reducing amyloid and slowing cognitive decline.

More extensive trials will be needed to know if the new drug is truly effective, but if the results, presented recently at the Alzheimer’s Association International Conference in Chicago, are borne out, the drug may be the first to successfully attack both the brain changes and the symptoms of Alzheimer’s.

Aside from a couple of medications that can slow memory decline for a few months, there is no effective treatment for Alzheimer’s, which affects about 44 million people worldwide, including 5.5 million Americans. It is estimated that those numbers will triple by 2050.

Realistic goals for the current treatment of Alzheimer’s are not focused on cognitive improvement, but instead are merely demonstrating that a drug might slow the progression of the disease. On a battery of cognitive and functional tests measuring memory and skills like planning and reasoning, the performance of the high-dose BAN2401 group declined at a rate that was 30 percent slower than the rate of decline in the placebo group.

Risankizumab Offers New Hope for People with Psoriasis

There is no shortage of drugs used to treat autoimmune conditions. Indeed, two drugs in this class, Humira® and Enbrel®, are currently first and second in overall spending for all medications in the U.S.

Risankizumab is currently being evaluated by the FDA for the treatment of plaque psoriasis, an autoimmune disease. Risankizumab has demonstrated greater efficacy in reducing psoriasis symptoms in clinical trials vs. the currently available medications.

Study participant’s lesions improved by 90%, while also offering complete clearance among some of the subjects. 82% of patients had a 90% clearance rate, while 49% reported 100% clearance.

Looking forward, risankizumab is also being studied for other autoimmune conditions, such as Crohn’s disease and ulcerative colitis.