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:
Median age at diagnosis 76 yrs
Majority of patients over 65 yrs
Only 6% of patients below 50 yrs (2)https://www.healthline.com/health/mds-prognosis
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.
Incident cases of
Latin America and the Caribbean
…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.
Patients 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.
(3) US National Library of Medicine 2017, Myelodysplastic Syndromes and Acute Myeloid Leukemia in the Elderly
(4) Datamonitor MDS Spotlight Nov 2020
Treatment with NEX-18 will offer 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 often treated in a technically off-label 5-2regimen 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, but it is also costly for providers compared to a single monthly injection of NEX-18.. NEX-18 would consequently fill a real unmet medical need for patients as well as relatives and health care providers.
REDUCING INITIAL BURST
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.
Nausea, dizziness, anxiety, anaemia, diarrhoea, muscle / joint pain, neutropenia, infections, sepsis, disrupted sleep, kidney problems
By reducing the initial ‘burst’ of high drug concentration with NEX-18, there is a potential to also reduce acute side-effects of the treatment. NEX-18’s PharmaShell covering completely envelopes azacitidine, dissolving slowly and releasing a low, sustained dose of the active substance over the cycle.
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)