WHAT IS

What is MDS?

An aggressive, prolonged and increasingly
prevalent blood cancer

AGGRESSIVE & PROLONGED: From nausea, dizziness and shortness of breath to bleeding disorders and frequent infections – MDS is a cluster of haematological malignancies causing a wide range of grueling symptoms. 40% of MDS patients are categorized as high or very high risk and receive a survival prognosis of around 2 years (1)1.Cancer.org. MDS is often referred to as “pre-leukemia” – a third of patients go onto develop acute myeloid leukemia (AML). MDS attacks the elderly:

PREVALENT: MDS is growing increasingly prevalent due to a rising elderly population, combined with slowly improving treatment (3)3. US National Library of Medicine 2017, Myelodysplastic Syndromes and Acute Myeloid Leukemia in the Elderly  over recent decades. In 2019, there were 238,000 cases worldwide, forecast to increase to 313,500 cases by 2028 (4)4. Datamonitor MDS Spotlight Nov 2020.

…BUT WITH A CHALLENGING FIRST-LINE TREATMENT: However, the first-line treatment for MDS – Azacitidine (commercial name Vidaza) – remains uncomfortable, inconvenient and demanding for predominantly elderly patients.

They must make daily visits to their nearest infusion clinic for one week every 28-day treatment cycle, spending a total of around 30 hours in-clinic per cycle. Injections need to be carefully monitored, with an approx. 2-hour recuperation period after each. Azacitidine requires complete blood counts to be carried out before and after each treatment cycle, to monitor response and toxicity. Strong anti-emetics are routinely administered before injections to treat the nausea and vomiting that are common side effects.

Sources
(1)
Cancer.org
(2) https://www.healthline.com/health/mds-prognosis
(3) US National Library of Medicine 2017, Myelodysplastic Syndromes and Acute Myeloid Leukemia in the Elderly
(4) Datamonitor MDS Spotlight Nov 2020

Patient Case

PATIENT CASE

Meet Anna

I’m on my 4th round of Aza now and my platelet count’s back to normal. But it’s been tough. The first 2 rounds particularly were hard. Even with the nausea medication I vomited a lot and couldn’t hold my job down - I literally couldn’t stay standing for long periods. But I hang in there, you’ve got to stay positive.

DIAGNOSIS: Anna was diagnosed with MDS on her 64th birthday – one year before she was due to retire.

PROGNOSIS: Anna is categorized as at intermediate-1 risk in the International Prognostic Scoring System (IPSS). This gives her a survival prognosis of 3.5 – 4 years, with a 33% chance of developing leukemia (acute myeloid leukemia AML) within the next five years.

IMPACT: A committed, active Principal of a 2,000-pupil middle secondary school, Anna had to take early retirement during her first month of treatment despite her intermediate risk classification.

INJECTIONS

From 7 daily to
1 monthly injection

Controlled release, smarter care

NEX-18 would require a single monthly injection – replacing the seven daily doses per treatment cycle currently needed.

Today Azacitidine suffers from an inconvenient dosage regimen, where daily subcutaneous injections are given for seven days in each monthly cycle (i.e. 7 injections per month). In reality, patients are typically treated in a technically off-label 5-2-2 regimen from Monday to Friday, and Monday and Tuesday of the following week, or receive a modified higher dose during five consecutive days, to fit hospital outpatient hours. This  regimen is not only inconvenient for patients, it is also costly for providers compared to the single monthly injection that NEX-18 is planned to be. NEX-18 would fill a real unmet medical need.

REDUCING SIDE EFFECTS

Reducing Azacitidine’s
side effects

Controlled release, increased comfort

Azacitidine’s side effects are, according to patient testimony (1)https://mdspatientsupport.org.uk/latest-news/mds-patient-stories-2/, typically most severe at the beginning of each treatment cycle and during the patient’s first two treatment cycles.

NEX-18 aims to significantly reduce side effects. With NEX-18 the active substance in Azacitidine is released slowly into the bloodstream –avoiding the initial, potentially toxic, ‘burst’ of high drug concentration thought to cause many of Azacetidine’s side effects. NEX-18’s PharmaShell nano-covering completely envelopes Azacitidine, dissolving slowly and releasing a low, sustained dose of the active substance over the month.

In the peer-reviewed paper the European Journal of Pharmacology and Bio pharmacology, PharmaShell reduced the initial burst of a sample drug indomethacin by 28 times the strength of the original formulation, sustaining the drug release over several weeks.(Insert full reference)

AZACITIDINE associated with:

ANAEMIA

causing fatigue, weakness, dizziness, light-headedness, shortness of breath, irregular heartbeat, chest pain, cold hands and feet.

NEUTROPENIA

an abnormally low neutrophil white blood cell count that causes increased risk of infections, fever (temperatures of 38°C or higher), chills, sweating, sores in the throat or mouth, abdominal pain.

THROMBOCYTOPENIA

an abnormally low platelet count, causing easy or excessive bruising (purpura), superficial bleeding into the skin, appearing as rash of pinpoint-sized reddish-purple spots (petechiae), usually on the lower legs, prolonged bleeding from cuts, bleeding from your gums or nose, blood in urine or stools. (2)https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4000014/