A Growing Market For Rare Disease Treatments Opens Up New Opportunities

NEW YORK – There are currently thousands of health conditions that affect people’s lives for which few or no effective treatment options currently exist. Each one of these conditions is an unaddressed market need in addition to a humanitarian need since these patients and the rare diseases that affect them get overlooked a lot of times by Big Pharma. The US Food and Drug Administration provides grants, other incentives, and expedited regulatory pathways to pharmaceutical companies to encourage focus on these rare and orphan diseases. In February, the FDA released a study highlighting the progress drug companies have made on this front and reaffirming the urgent need for novel treatments. Meanwhile, companies like Bristol-Myers Squibb Company (NYSE:BMY), Johnson & Johnson (NYSE:JNJ), Tryp Therapeutics (CSE:TRYP) (OTCQB:TRYPF), Amgen, Inc. (NASDAQ:AMGN), and Pfizer Inc. (NYSE:PFE) are dedicating massive resources to addressing these needs.

Tryp Therapeutics Advances Important Treatments

One company that fully recognizes the market opportunity presented by unmet clinical needs is Tryp Therapeutics (CSE:TRYP) (OTC:TRYPF). The company is focused on developing compounds with known activity and/or safety profiles and adapting them for the treatment of rare and orphan diseases. One advantage of this approach is the opportunity to quickly advance its development programs and reduce development costs through the combination of the FDA’s established regulatory pathways with available third-party preclinical and clinical data.

Tryp currently has two active development programs. The first is focused on the psychedelic compound psilocybin for treating neuropsychiatric disorders, particularly for the treatment of chronic pain disorders like fibromyalgia, a serious condition characterized by widespread musculoskeletal pain accompanied by fatigue, sleep, memory, and mood issues. There are currently three FDA approved treatments for fibromyalgia but all come with debilitating side effects that limit long-term use.

Tryp’s second ongoing development program is focused on razoxane for the treatment of soft tissue sarcomas (STS), a rare type of cancer that begins in the tissues that connect to and support other body structures. Razoxane has already had multiple Phase 2 clinical trials completed on it for the treatment of STS, and Tryp believes it will qualify for orphan drug status. The company plans on conducting a bridging study with the FDA this year to replicate the successful Phase 2 clinical data that was done previously.

On March 18, Tryp Therapeutics announced that the company has submitted a provisional patent describing novel methods for the formulation, delivery, and dosing of psychedelics resulting in a potential reduction in the time spent by patients in the dissociative state. Tryp says that its proprietary methods will improve how psychedelics are administered across a broad range of indications, improving the safety profile of treatments, and methods to reduce risk of abuse and addiction.

“We are seizing the opportunity to further differentiate our intellectual property position in psychedelics through core improvements to the formulating, delivery, and dosing of active ingredients,” Tryp Therapeutics President and Chief Science Officer Jim Gilligan, Ph.D. said in the company’s release. “I am continually impressed with the ingenuity and determination of our world-class team of scientists and advisors. Their innovations are empowering Tryp’s work to significantly expand the availability of treatment options for those patients with unmet medical needs by demonstrating the safety and efficacy of our psychedelic drug programs.”

Major Pharmaceutical Players Target Rare and Orphan Diseases

In late 2019, Bristol-Myers Squibb Company (NYSE:BMY) completed its acquisition of Celgene Corporation, the leading company in the rare and orphan drug space by market share. In February 2021, the Motley Fool reported that Bristol-Myers has been seeing a significant boost from Celgene’s portfolio of rare and orphan treatments.

In December 2020, Johnson & Johnson’s (NYSE:JNJ) subsidiary Janssen Pharmaceutical Companies announced the longer-term results from the combined Phase 1b/2 study evaluating the efficacy and safety of ciltacabtagene autoleucel (cilta-cel) for the treatment of patients with relapsed and/or refractory multiple myeloma. A year earlier, Janssen received Breakthrough Therapy Designation, as well as orphan drug designation, from the FDA for cilta-cel, granting the company an expedited development and regulatory review process.

On February 3, Amgen, Inc. (NASDAQ:AMGN) announced a three-year, Golden Ticket sponsorship of the BioLabs LA at The Lundquist Institute (TLI) life sciences co-working space to “accelerate the development of new therapies, medical devices, and diagnostics to advance and improve human health.” The Lundquist Institute is a state-of-the-art research institute with more than 100 principal investigators working on more than 600 research studies, including therapies for numerous, and often fatal orphan diseases.

Pfizer Inc. (NYSE:PFE) announced on February 26 that the European Medicines Agency has validated for review the Marketing Authorization Application for the company’s recombinant human growth hormone somatrogon, which is intended to be administered once-weekly for the treatment of pediatric patients with growth hormone deficiency. Pfizer says that the announcement is part of the company’s commitment to support patients suffering from the rare condition.

Each rare condition for which few or no treatment options are available represents an opportunity for a pharmaceutical development company to serve a vital need. Companies like Tryp Therapeutics have worked out how to develop these important therapies quickly and cost effectively.

Click here to find out more about Tryp Therapeutics.


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