BIO is very excited to be hosting the BIO Investor Forum next week in San Francisco. The BIO One-on-One Partnering System has already scheduled over 640 meetings, which is a 50% increase over last year. Investor and company registration is also trending higher, including the number of presenting companies. The One-on-One Compass has been profiling these companies slated to present at the conference, and recently spoke to the CEO of Regado Biosciences, David Mazzo. Please read below to learn more about what’s ahead for this newly public company.
Tell us about Regado Biosciences.
Regado is a public (NASDAQ: RGDO) biopharmaceutical company focused on the development of novel, first-in-class, actively controllable antithrombotic drug systems for acute (hospital) and sub-acute (hospital and physician’s office) cardiovascular indications. We are pioneering the discovery and development of two-component drug systems consisting of a therapeutic aptamer and its specific active control agent. Our lead program, REG1, just began a global phase 3 trial in the indication of PCI (Percutaneous Coronary Intervention).
REG1 is Regado’s lead drug candidate. Can you describe REG1?
REG1 is an actively controllable anticoagulant system targeting coagulation Factor IXa for use in patients with a wide variety of coronary syndromes undergoing a percutaneous coronary intervention (PCI), a hospital-based procedure used to mechanically open or widen obstructed coronary arteries.
REG1 consists of pegnivacogin, an anticoagulant aptamer, and its complementary active control agent, anivamersen. Pegnivacogin is a highly specific and potent Factor IXa inhibitor. Pegnivacogin is pegylated and, as such, has a very long half-life (>24 hours). Anivamersen, the active control agent, is the Watson-Crick base pair complement of a 15 nucleotide snippet of pegnivacogin. It is a single-stranded oligonucleotide (a biological polymer consisting of a relatively small number of nucleotides chemically bound in a linear sequence that forms a chain-like structure) and, importantly, has no pharmacologic activity other than to bind to and modulate the therapeutic effect of pegnivacogin. Because anivamersen is not pegylated, it has a very short (<5 minutes) half-life by design.
Both pegnivacogin and anivamersen are administered by intravenous bolus injection using weight-based dosing. As a result, the onset of action of both agents is extremely rapid. By adjusting the dose of anivamersen relative to pegnivacogin, the anticoagulant effect of pegnivacogin can be precisely and rapidly controlled or eliminated.
The combination of the choice of Factor IXa as the target, the specificity of pegnivacogin and anivamersen and the dose of each agent administered allows physicians to achieve unprecedented levels of anticoagulation in patients that would be practically unattainable or unsafe to use with existing anticoagulants lacking active, specific control.
Can you discuss details of the REGULATE-PCI clinical trial and where it is in the clinic?
Last month, we announced the enrollment of the first patient in REGULATE-PCI clinical trial, our Phase 3, PROBE design (Prospective, Randomized, Open-label, Blinded-Endpoint) superiority study comparing the effects of Regado’s REG1 to bivalirudin in patients undergoing PCI electively or for the treatment of unstable angina or non-ST elevated myocardial infarction (N-STEMI).
REGULATE-PCI calls for the enrollment of 13,200 patients evenly divided between REG1 and the comparator, bivalirudin. The trial is expected to enroll over two years. Significantly, there are 3 interim analyses prescribed in the study design calling for safety evaluations after 1000 patients enrolled and after 25% enrollment has been achieved and a safety and efficacy analysis after 50% enrollment is attained. All three interim analyses are planned for 2014 (2Q14, 3Q14 and 4Q14, respectively). The primary endpoint of the trial is efficacy compared to bivalrudin based on a composite of death, nonfatal myocardial infarction, nonfatal stroke and urgent target lesion revascularization through day three. The principal secondary endpoint is safety compared to bivalrudin as measured by major bleeding events through day three. The trial is powered to show superiority in efficacy and non-inferiority or superiority in safety against bivalirudin. If successful, REGULATE-PCI will become the cornerstone of Regado’s international new drug applications, planned for filing in early 2016.
Why is there such a significant need for this new technology?
PCI procedures involve a significant risk of ischemic events, including death, stroke, myocardial infarction and the need for repeat revascularization of the artery. Because of this risk, powerful anticoagulant drugs are administered prior to and throughout the PCI procedure. However, anticoagulants create a significant risk of major bleeding events. As a result, interventional cardiologists are forced to make a compromising medical decision because they lack the means to simultaneously reduce the risks of ischemic and major bleeding events. Additionally, PCI procedure costs are a major expenditure for the health care system and overall PCI cost reductions (i.e., pharmacoeconomic benefits) contributed by any new anticoagulant are highly desirable.
REG1 is the first and only anticoagulant to demonstrate the ability to reduce both ischemic and major bleeding events in a clinical trial for PCI (the randomized, partially blinded, 640 subject Phase 2b “RADAR” trial) while providing pharmacoeconomic benefits.
Why should investors closely watch Regado?
REG1 has performed consistently throughout development and is expected to perform similarly in phase 3. Additionally, because of the nature of the design of REGULATE-PCI, including three key near-term value inflection points in the planned interim analyses in 2014, the trial is continuously de-risking. This trial, if successful, will result in a REG1product profile that includes superior efficacy and non-inferior or superior safety as well as significant savings to the hospital and healthcare systems. With this profile, REG1 is expected to become the new standard of care and ultimately, market leader in anticoagulant therapy in PCI.
Regado is presenting Tuesday, 10/8 at 10:30am. We look forward to seeing them at the conference! Follow our hashtag, #BIF13 for updates!
*Company snapshots and interviews are meant to be previews of presentations given at BIO events by way of answering set questions. BIO does not substantiate or validate any claims mentioned in company snapshots or presentations.