Synovo wins orphan drug status for immune modulator SYD003
NOVEMBER 13, 2014
Press Release | Synovo GmbH
Tuebingen, Germany: – The European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) have granted orphan drug designation to Synovo’s first-in-class investigational compound SYD003 tumor-targeted immune modulator.
The innovative German-based drug discovery services company today announced that SYD003 will be evaluated for safety and efficacy in patients with pancreatic cancer as well as other cancers.
The candidate is the product of a multi-year international program sponsored in part by European Research funding.
Targeted at pancreatic cancer
Pancreatic cancer is one of the most lethal cancer types. Each year in the U.S. and Europe, roughly 150,000 people are diagnosed with pancreatic cancer, which has a five-year survival rate of less than six per cent with the majority of patients succumbing within six to 12 months after diagnosis.
“The orphan drug designation is an important regulatory achievement towards our goal of developing tumor-targeted immune modulators for pancreatic cancer,” said Dr. Michael Burnet, Managing Director of Synovo.
“We are pleased to continue developing SYD003, a first in class compound with a unique mode of action in cancer,” added Dr. Burnet.
Synovo Pharmacist Enriqueta Vallejo said that the main potential advantages of SYD003 were that it could be combined with many therapies and appeared better tolerated than many conventional cancer treatments.
“SYD003 is a novel compound that causes the suppressed immune cells around a tumor to become active. Under the influence of SYD003, these cells can directly attack the tumor, or activate other immune cells that were suppressed by the tumor during its growth,” said Ms. Vallejo.
The pre-clinical studies evaluating SYD003 in pancreatic cancer models over a number of years demonstrated remarkable activity for a non-cytotoxic compound. Such compounds are more easily tolerated than conventional chemotherapy. Indeed, in earlier safety studies in human subjects, the active ingredient of SYD003 was well tolerated at doses much higher than those planned for pancreatic cancer trials.
Switching the immune response
European Research Fellow Dr. Linda Sandin, a Swedish scientist now working with Synovo in Tübingen, Germany, explained that SYD003 worked by ‘switching’ the key role of immune cells from enemy to friend.
“The immune system is normally very efficient at detecting small tumors and kills most of them soon after they arise as small cell clusters,” Dr. Sandin explained.
“However, most tumors that become malignant evolve to secrete signals that cause the immune response to be suppressed so that immune cells that detect the growing tumor are rendered inactive as soon as they contact it. Not only that, but many aggressive tumors are also very effective in causing the immune cells to co-operate with the tumor helping it to grow even faster,” Dr. Sandin said.
“These seduced cells not only orchestrate a local immune suppression, but also make it possible for tumor cells to migrate and encourage blood vessels to grow into the tumor. This leads to metastatic disease and poor prognosis.”
“SYD003 interrupts the tumor’s signaling to these cells and causing them to be aggressive toward the tumor. This immune attack on the tumor is itself helpful, but it also supports and enhances other types of therapy like chemo and antibody-therapy, to make them more effective, ” said Dr. Sandin.
Synovo is now working with its partners and collaborators to move SYD003 into clinical trials.
Aspects of the discovery and development of SYD003 have been conducted within European projects MACROCEPT, KINACEPT, NANOFOL Seventh Framework Program (FP7) and the TIMCC Marie Curie Action.
Notes for Editors:
About Orphan drug status
To qualify for EMA orphan designation, a medicine must:
- Be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating
- Address a condition whose prevalence in the EU is not more than 5 in 10,000 or unlikely to support commercial marketing
- Offer a diagnosis, prevention or treatment of the condition for which no other authorized or equivalent therapy exists
The European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) examines applications for orphan designation. Following orphan designation, sponsors can benefit from a number of incentives, including protocol assistance and market exclusivity.
Synovo GmbH is a privately held company based in Tübingen, Germany, focusing on pharmaceutical research and development. Synovo provides services to customers in the pharmaceutical industry and conducts research on its own behalf.
Synovo’s in house research is mainly focused on inflammation, infection, oncology, ophthalmology, process chemistry and formulation. Through collaborations with public sector and biotech partners, Synovo has also developed novel candidates for clinical development in airway diseases (COPD and CF orphan designated), ophthalmology (eye acute neuropathies) and CNS (Parkinson’s disease).
Synovo’s main pharmacological activities are based on assays and disease models for the selection of drug candidates with optimal in vivo properties. Synovo works in partnership with public sector laboratories to improve its systems and extend its technologies to protein and nucleic acid drugs.
The company was founded by Dr. Michael Burnet, a former Zeneca project leader with a broad interest in chemical biology and its application in improving small molecule discovery efficiency. Its 30-strong team works in a new facility with an optimised for chemistry and pharmacology studies.
SYD003 modifies the tumor-supportive microenvironment characteristic of pancreatic cancer and tilts it towards a less immunosuppressive mode and a better overall anti-tumor response. It causes the levels of key signaling molecules in the tumour environment to change in support of an immune response against the tumour. This, in turn, increases the number and activation of anti-tumour cells such as T-cells and Natural Killer cells.
SYD003 demonstrated outstanding efficacy in Gold Standard pancreatic cancer models. It significantly prolonged survival and reduced the incidence of metastases, alone and in combination with the current standard of care.
SYD003 is not a cytotoxic drug and is therefore expected to be well tolerated by the patients and to be a positive addition to standard of care.
After demonstrating outstanding efficacy in pancreatic cancer models during pre-clinical development and achieving the orphan drug status by the EMA and the FDA, SYD003 has become a candidate for clinical development.
About pancreatic cancer
Pancreatic cancer is the fourth leading cause of cancer death in the EU and US. It is a high life-threating condition with an extremely low survival rate because it is frequently diagnosed at advanced stages after metastases have already formed. In pancreatic cancer, the tumor builds a biological barrier around itself, managing to modulate the environment and generate a supportive niche of expansion. This makes it particularly resistant to conventional chemotherapy. The most effective current regime provides median progression free survival in the range of 11 months.
Renate Burnet, Business Development Manager, Synovo
Tel: 49 7071 6397812
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