Latest News – 12 July 2018
PharmaKrysto announces licence agreement with Rutgers University and Orphan status

a life-changing condition.

There is a group of adults and children who experience severe, frequent pain, kidney damage and a risk of kidney failure , caused by deposits of large amino acid crystals in their kidneys. This medical condition is called Cystinuria.

We are an early-stage research and development company — our mission is to save kidneys and prevent pain in people with Cystinuria.

The Problem.

Cystinuria is caused by faulty genes that cause the kidneys to leak amino acids into the urine. This problem in amino acid retention causes high concentrations of an amino acid called cystine in the urine. This crystallises, as large crystal-stones, in the kidneys. These crystal-stones block the flow of urine, causing pain and infection. Surgery is often the only way of removing them. Damage to the kidneys caused by surgery, and repeated infection can cause progressive and permanent kidney failure. Severely affected patients can have many kidney stone events each year, causing significant disruption to work, family life as well as the education of younger patients.

There are about 45,000 in the US and 100,000 people in the EU with Cystinuria; most people have their first experience of cystine crystal-stones causing pain or other symptoms in their teens
or early 20’s.

There have been no new treatments introduced for over 30 years for people with Cystinuria.
Current treatments to prevent cystine stones are effective in around half of the patients that take them. These older treatments have significant side effects, including skin rashes, liver and
bone marrow disorders.

The experiences of people with cystinuria and discussion on current and new potential treatments have been featured in the Spring 2018 edition of the digital magazine RareRevolution here.

The Solution.
We founded PharmaKrysto to prevent pain and the risk of losing kidneys in people with Cystinuria. We have been working with a group of scientists who have discovered a method of blocking the growth of cystine crystals, through the use of newly invented molecules. These have the potential to become a medication that could be taken as a tablet and prevent the growth of cystine crystal-stones. They work in a completely different way to current therapies and could become a useful treatment without severe side effects. Our goal is to develop these molecules to see if they are safe and effective in preventing crystal-stones forming in people with Cystinuria.
Our Path Forward.

We are an experienced team of drug developers who were inspired to set up PharmaKrysto when we understood what people with cystinuria experience; and how a new and effective therapy could change their lives in a positive way.

We have completed early discussions with medicines regulators, who are supportive of our plans to develop new treatments for children and adults with this condition.  We are now at the stage of preparing for the first studies in humans, through developing the manufacturing process and adding to our knowledge of the safety of our molecules.  These clinical trials will enable us to learn about the safety and effectiveness of a treatment before it can be made more widely available to people with Cystinuria.

Our Team.
Julian Howell

Co-Founder and CEO of the company,
Julian studied medicine and surgery in London before continuing his career in pharmaceutical development. He started his medicine development career at SmithKline Beecham and Roche and more recently as the Chief Medical Officer at smaller biopharma companies. Julian has been involved in multiple new medicines approvals in the US and EU and has led development teams across a range of
therapy areas. He completed an MBA at
Cranfield University.

Carl Sterritt

Co-Founder and Non-Executive Chairman, Carl has20 years’of management and executive level experience in pharmaceutical development and commercialisation in both large and small companies. Previously, Carl held senior management roles at United Therapeutics and Encysive Pharmaceuticals, working on innovative therapies for the treatment of pulmonary arterial hypertension. Carl is currently Chief Executive of Shield Therapeutics, which he also founded.

Dr. Jackie Mitchell

Co-Founder, PreClinical and Regulatory Consultant, Jackie has over 20 years’ experience in regulatory affairsand early stage development. She holds an MA in biochemistry from OxfordUniversity, where she also obtained a doctorate in immunology and molecular biology. Jackie has worked in regulatory affairs for large, medium and small pharmaceutical companies, including Boehringer Ingelheim and Abbott. She has been involved in a broad range of global, EU and national new medicines approvals, including in the US and EU Kaletra and Humira.

Please be aware that we are not able to offer any individual medical advice and we do not have treatments that we can offer on a ‘compassionate use’ basis

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