Fulcrum Therapeutics recently announced the interim results from their Phase 1 trial of FTX-6058 in a press release. This selective small-molecule inhibitor of EED is in development for the treatment…
For many patients, certain treatments can be expensive, invasive, or difficult to access. But it doesn't have to be this way. According to aHUS News, researchers are running two separate…
Orphan drug designation is granted in the United States to expedite the development and review of drugs and biologics intended to treat patients with rare conditions (over 200,000 Americans). Once…
For years, researchers have evaluated Italfarmaco's givinostat for patients with Duchenne muscular dystrophy (DMD). In fact, a Phase 2/3 clinical trial showed that long-term givinostat treatment helped slow respiratory decline…
HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma patients haven't seen a drug approval for their disease state in a decade. That is until the FDA approved Keytruda in combination with…
Patients with relapsed or refractory mantle cell lymphoma (R/R MCL) may have a new treatment option, based on positive results from a Phase 1/2 trial of Zanubrutinib published in Blood…
On July 30, 2021, biopharmaceutical company Cyclo Therapeutics shared that it had released new and promising data regarding the safety, efficacy, and tolerability of Trappsol® Cyclo™ ("Trappsol Cyclo") for patients with…
Over the last few months, researchers have been enrolling and dosing patients with hereditary angioedema (HAE) from Europe, Israel, and Canada within the Phase 2 RAPIDe-1 clinical trial. According to…
In the past, ibrutinib has been used to treat patients with a variety of conditions, including chronic lymphocytic leukemia (CLL), mantle cell lymphoma (MCL), and chronic graft-versus-host disease (cGVHD). According…
Despite drug developers' best efforts, the path to approval is not always easy. Ask biotechnology company Ardelyx, whose potential treatment option tenapanor was recently rejected by the FDA. According to…
In the United States alone, an estimated 300,000 people have moderate-to-severe chronic idiopathic urticaria (CIU), or chronic hives which are either recurrent or last for over 6 weeks, which…
Previously, biopharmaceutical company XyloCor Therapeutics ("XyloCor") performed the first portion (dose-escalation) of the Phase 1/2 EXACT clinical trial evaluating its investigational treatment XC001 (encoberminogene rezmadenovec) for patients with refractory…
Near the end of June 2021, pharmaceutical company Crinetics Pharmaceuticals, Inc. ("Crinetics") shared via news release that the first patient had been dosed in the Phase 3 PATHFNDR-1 clinical trial.…
Medical studies are crucial players in the journey to develop new and more efficacious treatment options for patients with rare diseases. On June 10, 2021, Vertex Pharmaceuticals Incorporated ("Vertex") shared…
The FDA has recently granted their approval to a Phase 2 trial of FHL-301, a treatment for those in the earlier stages of Parkinson's disease. Forest Hills Lab will conduct…
According to a news release from late June 2021, biopharmaceutical company Pharming Group N.V. ("Pharming") completed enrollment for a Phase 2/3 clinical trial evaluating leniolisib for patients with activated phosphoinositide…
While myasthenia gravis (MG), an autoimmune neuromuscular disorder, can be treated, there is a smaller subset of patients who are treatment-refractory. Those whose condition is not easily treated are thought…
In the company's Phase 2 proof-of-concept clinical trial, biopharmaceutical company Harbour BioMed ("Harbour") sought to understand the impact of batoclimab (HBM9161) on patients with generalized myasthenia gravis (gMG). The…
Clinical trials can be crucial in determining potential treatment options for a variety of conditions. According to Pharma Biz, biopharmaceutical company Nordic Nanovector ("Nordic") recently shared promising data from the…
Acknowledgment: This story is sponsored by Takeda and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant, vetted and valuable information to the…
Clinical trials are important research tools to help understand the safety, efficacy, and tolerability of potential treatments. According to a news release from July 19, 2021, biotechnology company Prometheus Biosciences,…
According to the Cancer Network, orelabrutinib, a Bruton tyrosine kinase (BTK) inhibitor, was recently granted Breakthrough Therapy designation within the United States. While the treatment is also indicated for patients…
Breakthrough Therapy designation is granted by the FDA as a way to accelerate drug development and review. In particular, the designation is granted to drugs or biologics for serious conditions,…
In a news release from mid-July, 2021, biopharmaceutical company Strongbridge Biopharma plc ("Strongbridge") shared the publication of new post hoc analyses regarding KEVEYIS (dichlorphenamide) for patients with Primary Periodic Paralysis…
Thus far, retifanlimab, a drug designed to treat patients with anal cancer, has received Orphan Drug designation and Priority Review status. However, the approval process has not been entirely easy.…
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