Results from Phase 1b/2a Trial Show Substantial Reduction of RA Symptoms Based on ACR and DAS28 Disease Scores
- Primary and secondary endpoints of the study successfully met
- MOR103 showed excellent safety data at all doses administered
- First anti-GM-CSF antibody to demonstrate proof-of-concept in patients
- ACR20 score of up to 68% at week 4
- Strong onset of therapeutic effect as early as 2 weeks
- Anti-inflammatory effects supported by MRI analysis
MARTINSRIED / MUNCHEN, Germany I September 20, 2012 I MorphoSys AG (FSE: MOR; Prime Standard Segment, TecDAX) today announced results from the phase 1b/2a clinical trial evaluating its proprietary HuCAL antibody MOR103 in rheumatoid arthritis (RA) patients. The positive data make MOR103 the first anti-GM-CSF antibody to demonstrate clinical efficacy in RA. The results demonstrate the compound’s potential to become an important new drug in an area of unmet medical need. MorphoSys will submit a late-breaking abstract for a forthcoming conference to present the clinical trial results before the end of the year.
"We are very excited by the results of the study," commented Dr. Arndt Schottelius, Chief Development Officer of MorphoSys AG. "The ACR20 score ranks amongst the highest seen for a biological compound in RA after four weeks of treatment. The excellent efficacy and safety data we observed underline MOR103’s potential to become a first-in-class treatment modality for RA patients, with a novel and differentiated mechanism of action. We expect to present the data at the most relevant medical conference for RA later this year. The quality of the data demonstrates the capabilities of our discovery and development organization and mark a significant step in MorphoSys’s strategy to bring innovative drugs to patients."
The best response was achieved in the 1.0 mg/kg dose cohort with an ACR20 score of 68% at week 4, which was significantly higher than in the control arm (p < 0.0001). Of particular importance was the fast onset of action observed: within 2 weeks, up to 40% of patients achieved an ACR20 score. Improvement of DAS28 scores was rapid and significant over the treatment period of the study. MRI scans revealed a reduction of synovitis according to the RAMRIS system at week 4.
MOR103 was safe and well-tolerated at all doses administered. There were no drug-related serious adverse events. No obvious differences in the adverse event rate between the MOR103 and placebo groups were observed.
"These are excellent results," commented the principal investigator of the study, Professor Harald Burkhardt, Professor of Rheumatology and Head of the Division of Rheumatology at Frankfurt University. "Considering the short first- in-patient study, MOR103 demonstrated very promising clinical activity and fast onset of action with favorable safety data."
In the randomized, double-blind, placebo-controlled phase 1b/2a trial in 96 mild to moderate RA patients, MOR103 was administered in four weekly doses of 0.3 mg/kg, 1.0 mg/kg or 1.5 mg/kg. The trial, which was designed to look in particular at the onset of the therapeutic effect, was conducted in 26 centers in Germany, Netherlands, Poland, Bulgaria and Ukraine. The majority of the trial participants were on a stable regimen of disease modifying anti-rheumatic drugs. The primary endpoint of the trial was to determine the safety and tolerability of multiple doses of MOR103 in patients with active RA. Secondary outcome measures were pharmacokinetics, immunogenicity, and the drug’s potential to improve clinical signs and symptoms of RA as measured by DAS28, ACR20/50/70 and EULAR response criteria, MRI imaging for synovitis and bone edema as well as patient reported outcomes.
Based on these compelling data, MorphoSys will now proceed with its plans to seek a commercial partner for further development of the program. In addition to the RA study, MOR103 is currently being evaluated in a phase 1b dose- escalation study in multiple sclerosis. Results of a phase 1 pharmacokinetic study in healthy volunteers to evaluate a subcutaneous formulation of MOR103 will be available shortly.
Overview of Study Results:
+——————–+——-+————–+————–+————-+
|Results at day 28 |Placebo| MOR103 | MOR103 | MOR103 |
|(Majority of | | [0.3 mg/kg] | [1.0 mg/kg] | [1.5 mg/kg] |
|patients were on | | | | |
|stable regimen of | | | | |
|DMARDS) | | | | |
+——————–+——-+————–+————–+————-+
|Number of patients | 27 | 24 | 22 | 23 |
+——————–+——-+————–+————–+————-+
|Proportion of | | | | |
|patients achieving | 7% | 25% | 68% | 30% |
|ACR20 | | | | |
+——————–+——-+————–+————–+————-+
|Proportion of | | | | |
|patients achieving | 4% | 4% | 23% | 9% |
|ACR50 | | | | |
+——————–+——-+————–+————–+————-+
MorphoSys will hold a public conference call and webcast tomorrow, Friday, September 21, 2012 at 03:00 p.m. CEST (09:00 a.m. EST, 02:00 p.m. BST) to present more information on the results of the MOR103 clinical trial.
Dial-in number for the Conference Call (listen-only):
Germany: +49 89 2444 32975
For U.K. residents: +44 20 3003 2666
For U.S. residents: +1 202 204 1514
Please dial in 10 minutes before the beginning of the conference.
In addition, MorphoSys offers participants the opportunity to follow the presentation through a simultaneous slide presentation online at http://www.morphosys.com/conference-calls.
A live webcast, slides, webcast replay and transcript will be made available at http://www.morphosys.com/conference-calls.
About MOR103:
MOR103 is a fully human antibody against GM-CSF (granulocyte macrophage- colony stimulating factor). GM-CSF was originally identified as a growth factor for granulocytes and macrophages but has more recently been identified as an inflammatory mediator in autoimmune disorders such as RA. GM-CSF stimulates stem cells to produce granulocytes and other macrophages and subsequently activates these differentiated immune cells leading to an increased production of pro- inflammatory cytokines, chemokines and proteases and thereby ultimately to articular destruction. The antibody MOR103 blocks GM-CSF and reduces its pro- inflammatory activity.
About clinical trials in RA:
Rheumatoid arthritis, or RA for short, is traditionally considered a chronic, inflammatory autoimmune disorder that causes the immune system to attack the joints and affects in particular a membrane, called synovium, which lines each movable joint. It is a disabling and painful inflammatory condition, which can lead to substantial loss of mobility due to pain and joint destruction. The disease affects approximately 4-6 million people worldwide. To analyze the effect of a compound in a clinical trial in RA, disease scores such as ACR- a measure summarizing improvement in the number of tender and swollen joints, pain scale, patients’ and physicians’ assessment of improvement and certain laboratory markers – and DAS28 scores are used. ACR 20 describes the percentage of study participants who achieved a 20 percent improvement in tender or swollen joint counts as well as 20 percent improvement in three other disease- relevant criteria. For the DAS28 score 28 swollen joints and 28 tender joints are assessed.
About MorphoSys:
MorphoSys developed HuCAL, the most successful antibody library technology in the pharmaceutical industry. By successfully applying this and other patented technologies, MorphoSys has become a leader in the field of therapeutic antibodies, one of the fastest-growing drug classes in human healthcare. The company’s AbD Serotec unit uses HuCAL and other antibody technologies to generate superior monoclonal antibodies for research and diagnostic applications.
Together with its pharmaceutical partners, MorphoSys has built a therapeutic pipeline of more than 70 human antibody drug candidates for the treatment of cancer, rheumatoid arthritis, and Alzheimer’s disease, to name just a few. With its ongoing commitment to new antibody technology and drug development, MorphoSys is focused on making the healthcare products of tomorrow. MorphoSys is listed on the Frankfurt Stock Exchange under the symbol MOR. For regular updates about MorphoSys, visit http://www.morphosys.com
SOURCE: Morphosys
