Post-Con BIO CEO Company Snapshot: Immunovaccine

A few weeks ago, the BIO CEO & Investor Conference concluded in New York City. With a 30% increase in One-on-One Partnering meetings and sold out company presentations, industry execs gathered to do business, and hear the latest from innovative companies.

As a precursor to the event, the company snapshot campaign highlighted a few of the companies who would be participating onsite. This year, the snapshot feature concludes with an interview from Marc Mansour, Ph.D., Chief Operating Officer of Immunovaccine. Read on to learn more about their work, and why 2014 should be a significant year for the company.

What is your company’s lead product or technology?

Immunovaccine develops cancer immunotherapies and infectious disease vaccines based on the Company’s DepoVax™ technology platform. DepoVax is a patented formulation that provides controlled and prolonged exposure of antigens and adjuvants to the immune system. Immunovaccine has advanced two T cell activation therapies for cancer through Phase I human clinical trials. Lead cancer vaccine therapy, DPX-Survivac, which has delivered some of the strongest immune responses against a cancer target seen to date in Phase I testing, is expected to enter Phase II clinical studies in ovarian cancer and glioblastoma (brain cancer) in 2014.

How does your company go about differentiating itself from the competition?

There are several key details that differentiate Immunovaccine from competitors. First and foremost, we believe our proprietary DepoVax vaccine adjuvanting platform is a powerful technology that provides our vaccine candidates an unrivaled boost in strength and duration of therapeutic impact. Secondly, is the impressive data we have achieved to date with our lead cancer vaccine, DPX-Survivac. The immune response and activity triggered by DPX-Survivac in our Phase I studies is among the strongest reported for a cancer vaccine. Finally, we think our strategic focus on developing cancer vaccines as part of combination therapy sets us apart. We always thrive to conduct clinical studies of our cancer vaccines in combination with potentially synergistic compounds like immune modulators and other immunotherapies when possible.

What are the upcoming milestones and long-term priorities for your company?

2014 promises to be a significant year for Immunovaccine, particularly as it pertains to our work in the area of cancer vaccines. We have been very successful to date in Phase I clinical studies – delivering some of the strongest immune responses reported to date for a cancer vaccine. These results set the stage for a strategic Phase II clinical program for our lead oncology asset, DPX-Survivac. We intend to initiate Phase II trials of DPX-Survivac in at least two tumor types during 2014. In the long-term, we are focused on successfully completing clinical development of our cancer vaccine candidates as quickly as possible in an effort to provide much needed treatment options to patients.

Tell us something about your company that investors might not know.

One thing that we take great pride in at Immunovaccine has been our ability to aggressively advance our clinical development programs in a capital efficient manner, often via strategic relationships and non-dilutive financing vehicles. We have combined a variety of grants from government agencies, as well as collaborations with academic institutions and research organizations, to provide significant capital resources when fundraising was very challenging. We would also point to the fact that with the frenzy of M&A activity in the cancer immunotherapy space, our lead cancer vaccine, DPX-Survivac, represents one of the industry’s few remaining non-partnered, high-value cancer immunotherapy assets. That places Immunovaccine in an enviable position as companies’ appetites for compelling cancer immunotherapy assets remains high.

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Thank you to Immunovaccine and all of our presenters from the 16th Annual BIO CEO & Investor Conference. We know the industry is eagerly looking forward to the year ahead, and we hope to see everyone again to discuss at the 17th annual event!

BIO CEO: Five Prime Therapeutics Company Snapshot

Five Prime Therapeutics is another company that will be presenting at next week’s BIO CEO & Investor Conference. They will be among 20 other presenters at the event who debuted an IPO in the last two years. Read below to learn more about their work, and what to expect from them at the event.

What is your company’s lead product or technology?

Five Prime focuses on discovering and developing novel protein therapeutics for cancer and inflammatory diseases. We leverage our comprehensive library of 5,700 human extracellular proteins and proprietary high-throughput screening technologies to produce new targets for therapeutics for partners and ourselves. Our most advanced candidate is FP-1039 (GSK3052230), a fibroblast growth factor (FGF) ligand trap being developed with GSK for solid tumors. A Phase 1b study in FGFR1-amplified lung cancer and other tumors is underway.

How does your company go about differentiating itself from the competition?

We spent 7 years successfully developing a platform to accelerate protein therapeutic discovery based on:

  • a proprietary library of more than 5,700 human extracellular proteins that we believe is the most comprehensive collection of fully functional extracellular proteins available
  • proprietary technologies for producing and testing thousands of proteins at a time.

We believe our platform can:

  • identify novel medically relevant protein targets and protein therapeutics that have little or no previously known biological function
  • determine the best protein target among alternatives
  • identify new targets more quickly and efficiently than previously possible.

Your company went public last year. What are your company’s goals and priorities for 2014? 

Our goals in 2014 are to continue to progress our pipeline products in clinical development (FP-1039 for cancers in Phase 1b, FPA008 for inflammatory diseases in Phase 1, and FPA144 for gastric cancer, projected to enter Phase 1 by end of year), advance our research with our collaborators at GSK and UCB in the areas of muscle disease, respiratory disease, fibrosis and CNS disorders, establish a new discovery collaboration, and expand and accelerate our internal research efforts in cancer immunotherapy.

Tell us something about your company that investors might not now.

We are leveraging our discovery platform in cancer immunotherapy, focusing on

  • Unknown binding partners for known targets (e.g., TIM3, VISTA, B7-H3/H4)
  • Novel pathways that regulate anti-tumor immunity

Five Prime Therapeutics will be presenting Monday, February 10th at 2:00pm in Basildon. For those not at the conference and are interested in hearing webcasts from companies, select presentations will be available online on our website. We look forward to seeing everyone!

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Company snapshots 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.

BIO CEO Company Snapshot: Tetraphase Pharmaceuticals

The BIO CEO & Investor Conference is now just a week away, and buzz for the event has never been greater. Tomorrow, Inside BIO is hosting its first ever twitter chat to discuss the annual buyside survey, which has been getting great responses from the investor community. Presentation spots are still sold out as well, and we’re continuing to highlight those companies slated to present. One such company is Tetraphase Pharmaceuticals, who went public this year. Read on to hear about their antibiotic drugs, and what they see in store for them in 2014 and beyond.

tetraphase

 

What is your company’s lead product or technology?

Our lead candidate is eravacycline, a novel, fully synthetic tetracycline antibiotic that has demonstrated potent activity against a broad spectrum of bacteria, including many of the multidrug-resistant (MDR) Gram-negative bacteria highlighted as urgent public health threats by the CDC. We are currently studying the safety and efficacy of eravacycline in two Phase 3 clinical trials in the indications of complicated intra-abdominal infections (cIAI) and complicated urinary tract infections (cUTI). We expect data from both trials in the first half of 2015 and to file an NDA for eravacycline in the U.S. by the end of 2015.

How does your company go about differentiating itself from the competition?

As a company, Tetraphase is unique amongst its peers, as we have built a product pipeline of novel antibiotics using our internal proprietary antibiotic discovery engine. Our chemistry technology allows for the modification of the tetracycline scaffold in ways never before possible, overcoming resistance and revitalizing the tetracycline class of antibiotics, long considered one of the safest of classes. We believe Tetraphase represents a compelling investment opportunity in the Gram-negative space, given that we are the only public company where successful market entrance offers significant upside potential in valuation. In addition, eravacycline, which was discovered using our technology platform, is the only Gram-negative candidate in late-stage development that has the potential to be used as a monotherapy with convenient once- or twice-daily dosing, and that is expected to be available in both IV and oral formulations.

Your company went public last year. What are your company’s goals and priorities for 2014?

In addition to our IPO last March, we also completed a follow-on offering in November, raising total gross proceeds of approximately $130 million during 2013. With these proceeds, we are currently executing on our Phase 3 clinical program of eravacycline. In mid-2014, we expect to have data from the lead-in portion of the Phase 3 cUTI clinical trial that will identify an oral dose of eravacycline to take into the pivotal portion of the study. We also own worldwide rights to eravacycline – announcements of single or multiple partnerships for development and commercialization rights to eravacycline outside the U.S. are possible during 2014. We continue to make progress on our pipeline as well, and expect to file an IND on our second clinical candidate TP-271, an antibiotic being developed with funding from NIAID to combat serious biothreat pathogens.

Tell us something about your company that investors might not know. 

Those not familiar with the antibiotic space may not know that Tetraphase is benefiting directly from new regulatory guidelines and legislative action. In response to the rise in MDR infections and increased mortality rates and also to encourage the development of new antibiotics, the FDA no longer requires two pivotal studies per indication, permitting companies to file for regulatory approval with two Phase 3 studies in distinct indications. For eravacycline, we will be seeking approval from one Phase 3 study each in cIAI and cUTI. In addition, Tetraphase is benefitting from the GAIN (Generating Antibiotics Incentives Now) Act of 2012. Through the Act, eravacycline has received QIDP (Qualified Infectious Disease Product) designation for both the cIAI and cUTI indications, making it eligible for Fast-Track status, priority review, and an additional five years of U.S. market exclusivity should it ultimately receive FDA approval.

Investors also may not know that eravacycline actually has very broad-spectrum activity – covering not only Gram-negative bacteria, but also Gram-positive and anaerobic bacteria. Although we are pursuing Gram-negative indications currently, eravacycline has some of the most potent activity amongst currently marketed antibiotics against Gram-positive bacteria, including MRSA and VRE.

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Tetraphase’s President and CEO, Guy MacDonald, will be presenting on the second day of the conference, Tuesday, February 11th at 3:00pm in Basildon. You can see the full list of presenting companies here.

Company snapshots 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.

BIO CEO Company Snapshot: Conatus Pharmaceuticals

The Company Snapshots have returned for our next One-on-One Partnering event, the BIO CEO & Investor Conference, taking place in New York City this February. The companies that will be profiled in these snapshots will all be presenting at the event, and we hope this provides the opportunity to learn more about them, and catch up on what’s new.

Conatus-Pharma-logo

What is your company’s lead product or technology?

Conatus’s lead product is emricasan, a first-in-class oral medicine designed to reduce inflammation and excessive levels of a specific form of cell death known as apoptosis—two underlying causes of liver damage across a broad spectrum of diseases. Emricasan has been evaluated in over 500 human subjects in six Phase 1 studies completed in healthy volunteers and four randomized, placebo-controlled Phase 2 trials completed in a variety of liver diseases.

How does your company go about differentiating itself from the competition?

Emricasan is a pan-caspase protease inhibitor designed to reduce the activity of all ten human caspases-enzymes that mediate inflammation and apoptosis. Seven caspases are specifically involved in the process of apoptosis while three caspases specifically activate pro-inflammatory cytokines and are not directly involved in apoptosis. We believe that by reducing the activity of these enzymes, emricasan has the potential to interrupt the progression of liver disease regardless of the initial cause. We believe there are no other drugs, either approved or in development, using this mechanism which offers both broad spectrum and self-limiting liver disease activity.

Your company went public last year. What are your company’s goals and priorities for 2014?

Conatus expects top-line results in 1H14 from its ongoing Phase 2b trial in patients with acute-on-chronic liver failure (ACLF), and is planning a Phase 2 trial to initiate in 1H14 in patients with nonalcoholic steatohepatitis (NASH), a Phase 2b trial to initiate in 2H14 in patients with chronic liver failure (CLF), and a Phase 2b trial to initiate in 2H14 in patients who developed liver fibrosis post-orthotopic liver transplant due to hepatitis C virus infection (HCV-POLT), who achieved sustained virus response (SVR) to antiviral therapy, but still have underlying liver fibrosis (HCV-POLT-SVR).

Tell us something about your company that investors might not know.

Emricasan was developed initially by the founders of Idun Pharmaceuticals, who sold the company to Pfizer and founded Conatus in 2005. After further development by Pfizer to prepare the drug to advance to Phase 3 trials and commercialization, Conatus reacquired Idun in 2010 and resumed its development independently.

BIO Investor Forum Interview with Regado Biosciences

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.

regado

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.