Feb 22, 2019
First Cohort Dosed In U.S. Phase 1 Study Of Subcutaneously Administered Sevuparin

Modus Therapeutics AB, a company developing innovative treatments for patients with high unmet medical needs and a focus on sickle cell disease (SCD), announces that the first cohort has been successfully dosed in its Phase 1, Single Ascending Dose Study to Explore the Pharmacokinetics and Safety & Tolerability of Subcutaneous Sevuparin Injections in Healthy Volunteers (HV). This study complements a large Phase 2 study which completed enrollment in January 2019 and will report data in mid-2019. “This Phase 1 study launches an important pillar of our strategy to treat vaso-occlusion and thereby reduce pain by providing SCD patients with a novel, non-opioid, therapy that can be dosed at home,” commented John Öhd MD, PhD, CMO of Modus Therapeutics. “While our recent Phase 2 study focused on treating patients with vaso-occlusive crisis (VOC) by intravenous (IV) sevuparin infusion, the Phase 1 study with subcutaneously-administered (SQ) sevuparin will pave the way for a therapy that could be used ‘on demand’ as a patient feels an oncoming painful VOC.”

The VOCs, or “painful crises”, are a hallmark of SCD and are believed to contribute to the patient’s gradual accumulation of complications such as organ failure and stroke.  Research suggests that reduction of the number of VOCs should improve patient outcomes and could increase the lifespan of these patients.

“We see an incredible opportunity for subcutaneous sevuparin as the only therapy in development with the potential to be used at home for the treatment of vaso-occlusion, expanding treatment options for SCD patients to the early hours of a VOC” Dr. Öhd continues.

Sevuparin is an innovative, proprietary polysaccharide drug with anti-adhesive, anti-aggregate and anti-inflammatory effects due to its multimodal mechanism of action. A large Phase 2 study of IV administered sevuparin completed enrollment in January 2019 with data readout expected mid-2019.  This study is evaluating the potential for sevuparin to restore blood flow and prevent further microvascular obstructions in adolescents and adults with acute VOCs resulting from their SCD. The double-blind, placebo-controlled Phase 1 study will include approximately 24 HVs in three ascending single-dose cohorts in a U.S. clinical trial facility.

“It is important to recognize the strong role that patient research had in informing this important element of Modus’ sevuparin development program.  Through this research we have observed that patients are often aware that they are about to develop painful crisis, are keen to have access to an at-home therapy that they can take in the first days of pain to prevent a hospital visit”, said Ellen K. Donnelly, PhD, CEO of Modus Therapeutics.  “This study is also an important milestone for Modus as sevuparin’s clinical development is now taking place in the United States (U.S.) under the IND that was approved by the FDA in November 2018,” added Dr. Donnelly.

SCD is an inherited blood disorder which is characterized by severely painful VOCs that lead to organ damage due to a lack of oxygen delivery to the organs. Progressive organ damage limits the life expectancy of patients with SCD and lifetime medical care costs can exceed $1 million per patient with an estimated $1 billion spent annually on the disease in the U.S. In the U.S alone SCD is believed to affect approximately 100,000 individuals.

There is currently no pharmaceutical product available that targets the vaso-occlusive activity during a VOC in SCD patients. At present, the standard of care relies on strong intravenous pain medications and SCD patients often must be hospitalized to be treated