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First in Human: A vaccine for Down syndrome-related Alzheimer's disease

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First in Human: A vaccine for Down syndrome-related Alzheimer's disease

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This is a recurring column on clinical research in the early stages of development, which is referred to as phase 1. These are treatments being used for the first time in a small number of human patients to determine safety, dosing and general pharmacological activity.

While vaccines have snatched much of the scientific limelight in the last 12 months due to the unprecedented speed of development for a clutch of effective COVID-19 therapies, Alzheimer's disease remains a focus of patient and investor attention ahead of a crucial and long-awaited U.S. regulatory decision on Biogen Inc.'s aducanumab, due next month.

Switzerland's AC Immune SA has neatly combined the two by developing a vaccine to target Down syndrome-related Alzheimer's disease. Recent data showed that the novel anti-amyloid-beta ACI-24 vaccine demonstrated encouraging immunogenicity and safety in phase 1b clinical testing in people with Down syndrome, or DS, and the company plans to advance the vaccine into mid-stage trials to treat and prevent the progression of Alzheimer's disease in this patient group.

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Lausanne, Switzerland-based AC Immune reported in March that the study showed the generation of anti-Abeta antibodies in adults with DS vaccinated with ACI-24, which was safe, well tolerated and resulted in no serious side effects. But the next clinical trial has been delayed to ensure the safety of study participants, due to the high vulnerability of people with DS to severe COVID-19.

"There is broad potential for our optimized Abeta vaccine formulation across Abeta-driven diseases, including DS-related, genetic, and sporadic [Alzheimer's disease]," AC Immune CEO Andrea Pfeifer said in a March 16 statement.

"We look forward to initiating a follow-on clinical trial in DS as soon as the threat to this vulnerable patient population from COVID-19 subsides. In the interim, we are in discussions with the FDA on a potentially accelerated development pathway for the optimized vaccine formulation and expect to file an investigational new drug application for the new formulation in Q4 2021."

New way of treating liver disease

The spread of the sedentary Western lifestyle has fueled a global boom in liver disease. Traditionally caused by alcoholism and hepatitis, rising cases of diabetes and obesity across the world are responsible for a surge in fatty liver disease and liver cirrhosis, also known as end-stage liver disease. Once the liver which cleans the body's blood of toxins fails, it sets off a fatal cascade of organ failure throughout the body.

With about 1 in 4 people in the U.S. likely to be affected by this silent epidemic, many large pharmaceutical companies, including AstraZeneca PLC and Novo Nordisk A/S, are tackling earlier-stage diseases like nonalcoholic steatohepatitis, or NASH. At the later stage of liver disease, Versantis AG, a spinout from ETH Zürich, Switzerland's highly regarded research university, is working on an alternative to a liver transplant and recently reported positive results from a phase 1b clinical trial in decompensated cirrhosis.

"Producing a drug or coming up with a new innovation that will be a magic bullet for these patients has been more difficult than the industry thought in the beginning," Versantis CEO Vincent Foster said in an interview with S&P Global Market Intelligence. "And for us, it's really good that we're not into this hyper-competitive space of early NASH, but really are solving the problem of later-stage liver disease."

Zürich-based Versantis said its VS-01 therapy was found to be safe and well tolerated in a first-in-human study led by Jonel Trebicka, a professor at the Goethe University Hospital Frankfurt. A potentially lifesaving, multi-organ support therapy, VS-01 aims to reverse acute-on-chronic liver failure by enhancing the clearing out of ammonia and other toxins from the body.

The product is injected into the abdominal cavity over seven days and, over a period of three hours a day, captures the excess of toxins, which are then removed in a subsequent process, said Versantis co-founder and COO Meriam Kabbaj.

"The goal of VS-01 is really to be able to efficiently and rapidly capture this excess of toxin that the liver can no longer metabolize or clean out of the blood circulation," Foster said. "We inject the product, and we can remove it out of the patient's body."

Hope for triple negative breast cancer

SNL ImagePEP-Therapy's PEP-010 is being tested on breast and ovarian cancer.
Source: Omar Kafeel


Paris-based PEP-Therapy SAS, a biotechnology company working on targeted oncology treatments, is carrying out a first-in-human trial in patients with recurring solid tumor cancers, notably triple negative breast cancer and ovarian cancer. Founded in 2014 based on research from French cancer center the Institut Curie and Sorbonne University, PEP-Therapy's lead candidate PEP-010 uses novel cell-penetrating and interfering peptides, or CP&IP, technology.

Sponsored by the Institut Curie, the phase 1 study will evaluate intravenous PEP-010 both on its own and in combination with chemotherapy drug paclitaxel in patients with recurrent or metastatic solid tumors.

About 10% to 20% of breast cancers are triple negative meaning they do not respond to hormonal therapies or medicines that target the HER-2 protein receptors.

"PEP-010 aims to fight cancers with poor prognosis for which there are only few or no therapeutic alternatives," said PEP-Therapy CEO and co-founder Antoine Prestat. "We look forward to starting this trial in the coming weeks and to generating results that we hope will confirm the promising results seen in preclinical studies."