Aridis Enrolling CF Patients to Test AR-501 in Chronic Lung Infections

By:
ALICE MELÃO

Aridis Pharmaceuticals has enrolled the first healthy participant in its Phase 1/2a clinical trial to evaluate the antibacterial potential of its investigational candidate, AR-501 (gallium citrate), against chronic lung infections in patients with cystic fibrosis (CF).

The study (NCT03669614) is expected to enroll approximately 48 healthy adult volunteers and 48 adult CF patients with chronic lung infections across 15 sites in the United States.

Participants will be randomized to receive one of three doses of AR-501, or a placebo, self-administered once a week using a hand-held nebulizer.

The company expects to announce results from Phase 1 during the fourth quarter of 2019, and from Phase 2a in the fourth quarter of 2020.

“We are pleased to initiate this exciting program with the first subject enrolled,” Wolfgang Dummer, MD, PhD, chief medical officer of Aridis, said in a press release. “Through this trial, we anticipate safety, pharmacokinetic, and exploratory efficacy data that will enable us to further explore the potential of AR-501 in the treatment of life-threatening bacterial infections in cystic fibrosis patients.”

AR-501 is an inhalable formulation of gallium being developed to treat pulmonary bacterial infections. It works by starving bacteria of iron, and inhibiting their iron-dependent metabolic processes necessary for the infection to progress, a mechanism very different from that of common antibiotics.

Preclinical studies have demonstrated that AR-501 holds a broad antibacterial activity with unique benefits, compared to current standard-of-care antibiotics, working against antibiotic-resistant strains such as Pseudomonas aeruginosa and B. cepaciaaccording to the company.

Also, data from a Phase 2 clinical trial (NCT02354859) showed that intravenous gallium is safe and can effectively improve the lung function of CF patients.

“The recent safety and efficacy demonstration of intravenous gallium from a Phase 2 clinical study in CF patients gives us optimism of the prospect inhaled delivery of gallium (AR-501), which is a more direct, local route of delivery to the site of infection in the lungs and less systemic exposure,” Dummer said.

The new Phase 1/2a trial is being conducted in collaboration with the Cystic Fibrosis Foundation (CFF), and is led by Noah Lechtzin, MD, director of the Adult Cystic Fibrosis Program and associate professor of medicine at Johns Hopkins University.

The U.S. Food and Drug Administration recently granted Fast Track Designation and Qualified Infectious Disease Product Designation (QIDP) to AR-501. These are expected to support and expedite the therapy’s development and regulatory review.

Original article: https://cysticfibrosisnewstoday.com/2018/12/14/aridis-started-enrolling-in-phase-1-2a-trial-to-test-ar-501-antibacterial-potential/

AbbVie Awards $25K to Two Students with Cystic Fibrosis

Two students living with cystic fibrosis (CF) were selected as AbbVie’s 2018 Thriving Undergraduate and Thriving Graduate Students as a part of the AbbVie CF Scholarship program. Vinnie Acedo-Holmquist and Jackie Johnson were each the recipient of a $25,000 scholarship in their pursuit of higher education.

                    

Vinnie, the 2018 Thriving Undergraduate Student, is currently attending Chandler Gilbert Community College and pursuing a degree in Business Administration. Jackie, the Thriving Graduate Student, is currently studying Physician Assistant Studies at Saint Catherine University. Both have demonstrated strength and resilience in facing CF head-on, and serve as inspirations for other students pursing their dreams of higher education.

Earlier this year, 40 undergraduate and graduate CF students were selected as 2018 AbbVie CF Scholars and received $3,000 scholarships. The students then had the opportunity to earn the title of Thriving Undergraduate or Graduate Student along with an additional $22,000 in scholarship funding by participating in a public voting period. Vinnie and Jackie were named Thriving Students based on a combination of their academic achievements, community involvement/extracurricular activities, essay and creative presentation scores, as well as the number of public votes cast.

To learn more about these students and the scholarship program, visit the AbbVie Scholarship website,www.AbbVieCFScholarship.com.

If you have any questions or would like to schedule an interview with either student to learn more about their experiences and goals, feel free to contact me at (212) 583-2701 or burseyp@ruderfinn.com for additional information. 

About the AbbVie CF Scholarship

The AbbVie CF Scholarship was established 26 years ago in recognition of the financial burdens many families touched by CF face and to acknowledge the achievements of students with CF. Since its inception, the scholarship program has awarded over $3.2 million in scholarships to over 1,000 students. The AbbVie CF Scholarship is part of AbbVie’s ongoing commitment to the CF community, which is comprised of more than 30,000 people in the United States. As of 2016, more than half of the CF population are 18 years or older.

  1. 1. Cystic Fibrosis Foundation. About Cystic Fibrosis. Diagnosed with Cystic Fibrosis. Available at: https://www.cff.org/What-is-CF/Diagnosed-with-Cystic-Fibrosis/. Accessed September 2018.

I’m Drowning – A researcher-patient’s plea for broader inclusion in cystic fibrosis trials

By: Ella Balasa

I’ve always known cystic fibrosis (CF) is a progressive disease; it destroys lung cells, tightens the small airways in the bottom of my chest, and each day takes me closer to the time when it will have ravaged my lungs. I had never really questioned if there was some way this process could be altered. I accepted that it couldn’t.

Recently, however, this has changed. The epicenter of new CF research is the development of medications that will slow, stop, and hopefully even reverse the effects and damage that CF inflicts on the body. The possibility of the cells in my lungs functioning to their full potential — with CF transmembrane conductance regulator protein function restored and working correctly, expelling chloride out of my cells, hydrating the surface of my lungs, and halting the thick sticky mucus that has caused my airways to be enveloped in a suffocating cloak for all these years — is like a feeling of being rescued when you are drowning.

Unfortunately, I am still drowning.

“I’m very sorry, Ms. Balasa, but you will not be able to be a participant in this clinical trial.” This was the response I received during one of my searches for these drug trials. Excited by the possibility of participating, finding one recruiting at my local adult clinic, I reached out to study coordinators and was informed that I met all but one criterion to participate in the studies. This specific criterion has prevented me from prior trial participation involving other investigational medications treating the symptoms of CF, including anti-infectives and anti-inflammatories.

Most CF studies, including phase I, II, and III trials, require a lung function minimum of at least 40% FEV1 (forced expiratory volume in one second). My FEV1 is 25%, so I am excluded from these trials. Many patients face a similar situation. The 40% threshold biases samples toward a young patient population, as this degenerative condition causes steadily decreasing lung function with time. Furthermore, as CF treatment has rapidly progressed and increased patients’ life expectancies, there are now more adults with CF in the U.S. than children, according to the CF Foundation Patient Registry.

As a patient who works in the science field, I started to ask myself: Where does that number come from? Should this one variable be such a deciding factor? Are we getting comprehensive results from these studies if a subset of patients is omitted? Are investigators using eligibility criteria from a prior study without determining whether the exclusions are scientifically justifiable?

To continue reading, please visit MedPage Today.