Jim Greenwood Celebrates JOBS Act Turning Five (Op-Ed in The Hill)

Five years ago today, the Jumpstart Our Business Startups (JOBS) Act became law. The JOBS Act – designed to enhance capital formation for emerging companies – passed in Congress with an overwhelming bipartisan vote.  In an Op-Ed appearing in The Hill, BIO President and CEO Jim Greenwood celebrates this anniversary with hopes to build on its success and continue working with Congress to support such sensible policies.

The JOBS Act struck the perfect balance between incentivizing companies to going public and still keeping important investor protections in place. It increased the flow of capital to innovative businesses without a revenue-generating product, yet. This law gave such biotech start-ups the opportunity to save on unnecessary compliance burdens and invest those funds into research and development – making science the priority and new life-saving treatments a reality. As Greenwood summarizes in BIO’s statement released today:

“The extraordinary impact of the JOBS Act shows the dramatic effect that smart policymaking can have on the search for groundbreaking cures and treatments.”

BIO also released new data today showing how the JOBS Act has left a significant impression in the industry after five short years. Here are some highlights that make today’s anniversary worth celebrating:

  • Compared to only 55 biotech IPOs just five years prior, there are now an unprecedented 212 biotech IPOs.
  • Emerging biotech companies have raised $17 billion through JOBS Act IPOs. As a result, an additional $16 billion was raised through follow-on offerings.
  • The FDA has approved 18 new treatments from JOBS Act companies, and an additional 696 therapies are currently in the pipeline.
  • Oncology companies make up the largest share of JOBS Act IPOs (25%).

Thanks to the JOBS Act, biotech companies currently employ over 27,000 people and have a market value of more than $111 billion. It is clear that America is the undisputed leader of a global industry transforming medicine, farming, and energy production. We hope Congress builds on the success of the JOBS Act and fosters continued growth of emerging public biotech companies.

Read Greenwood’s full Op-Ed here.

View the infographic summarizing the impact of the JOBS Act on the biotech industry here.

#BIO2017: A Peek at Partnering | Protalix Biotherapeutics’ Bill Taylor

Breaking Buzz is BIO’s newest blog series that reaches across the globe to bring you an insider’s preview into the hottest international and partnering trends coming to San Diego for the BIO International Convention. 

Breaking Buzz Sits Down with Partnering King

BIO’s One-on-One Partnering™ opens April 10 and it’s not too early to register, set up a profile and start requesting and accepting meetings before schedules get filled. BIO will host a Webinar on April 5 to explain everything you need to know about the partnering system and offer tips to ensure a successful BIO 2017.

Recently we connected with Industry veteran Bill Taylor, one of BIO’s most prolific partnering delegates, having single-handedly scheduled and attended 55 formal meetings, over just three days, during last year’s International Convention in San Francisco. This impressive number of well-executed meetings wasn’t beginner’s luck. Mr. Taylor first attended BIO when he was with Pharmacia in 2001 and since then has racked up a lot of meetings, many of which resulted in more meetings. We can all agree that with more meetings often come lasting partnerships and potential product commercialization agreements.

Mr. Taylor, now Vice President, Business Development for Protalix Biotherapeutics, sat down with Breaking Buzz to share some of his strategies on how to pull off a great number of effective meetings in a meager amount of time.

BB:  What’s the single most important task when it comes to scheduling partnering meetings?

TAYLOR: It will seem obvious, but the most important thing I’ve learned is to DO YOUR HOMEWORK. I usually start researching companies within three months of the Convention because otherwise it can feel like diving into a mosh pit of 20,000 people. The online instructions are a great place to start. A close second most important task though, is to make sure you populate your profile really well and use well-chosen key words to highlight your company interests and offerings, not just name and location.

Bill Taylor, VP Business Development, Protalix Biotherapeutics

BB:  What is the most important part of a company profile?

TAYLOR: In the Protalix profile I talk about what we’re doing and what our pipeline is, which allows other companies looking for opportunities to send me an invite. You’ve got to recognize that your company profile on the system is a two-way street in that respect. Our profile will read something like this:

Protalix is a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by our proprietary ProCellEx® plant cell-based protein expression system. Our pipeline consists of proprietary, potentially clinically superior versions of recombinant therapeutic proteins that target established pharmaceutical markets with a focus on rare diseases.

BB:  How many people do you think should attend a partnering meeting from each company?

TAYLOR:  Two to three, tops. One or two from the company, and myself. That said, sometimes companies want to bring their science and technical experts to do some drill-down for their initial diligence questions. Knowing this in advance will save time.

BB:  What should a company expect to achieve during partnering meetings?

TAYLOR: Like most things you get what you put into it. So, if I’ve done my homework, I expect to walk away with well-grounded conversations that cover the full width of our pipeline; conversations where I can articulate our value propositions, while conveying the commercial opportunities that our products represent. BIO is unique because it provides a very broad band forum in which to meet up and engage with a large number of companies across a vast range of geographies.

BB:  Should a company lock-down their schedule, or keep it flexible?

TAYLOR: I find that there’s no lack of people pinging me with lots and lots of invites, so I have to be highly disciplined about my scheduling strategy, while remaining open-minded to unforeseen possibilities. The challenge is to get your scheduled meetings locked down early, but it’s equally important to have some safety valve time-slots available to ensure a meeting can actually occur if it comes in on a last-minute blitz.

BB:  This will be your 16th consecutive year attending the BIO International Convention. What keeps you coming back?

TAYLOR: BIO is a lot like Homecoming. There may be nearly 20,000 people but we have a lot in common; we’re a collegial group that live and survive by the networks we’ve created over the years. Even waiting for a coffee — or a beer at the end of the day — you’ll strike up a conversation with someone who may end up as a great resource or they might be someone you’ve met at a past event.  It’s a place for former colleagues to re-connect, meet up with old contacts and make new connections. In that respect it really does feel like Homecoming.

BB: Does that make you the BIO International Convention Homecoming King?

TAYLOR: I guess so. You should see my convention badge collection!  After all these years, I’m still just a science geek!

Athena Staton serves as executive editor for BIO Buzz, the Official Show Daily for the BIO International Convention, and BIO’s Breaking Buzz blog series.

#BIO2017: A Peek at Partnering | Protalix Biotherapeutics’ Bill Taylor

Breaking Buzz is BIO’s newest blog series that reaches across the globe to bring you an insider’s preview into the hottest international and partnering trends coming to San Diego for the BIO International Convention. 

Breaking Buzz Sits Down with Partnering King

BIO’s One-on-One Partnering™ opens April 10 and it’s not too early to register, set up a profile and start requesting and accepting meetings before schedules get filled. BIO will host a Webinar on April 5 to explain everything you need to know about the partnering system and offer tips to ensure a successful BIO 2017.

Recently we connected with Industry veteran Bill Taylor, one of BIO’s most prolific partnering delegates, having single-handedly scheduled and attended 55 formal meetings, over just three days, during last year’s International Convention in San Francisco. This impressive number of well-executed meetings wasn’t beginner’s luck. Mr. Taylor first attended BIO when he was with Pharmacia in 2001 and since then has racked up a lot of meetings, many of which resulted in more meetings. We can all agree that with more meetings often come lasting partnerships and potential product commercialization agreements.

Mr. Taylor, now Vice President, Business Development for Protalix Biotherapeutics, sat down with Breaking Buzz to share some of his strategies on how to pull off a great number of effective meetings in a meager amount of time.

BB:  What’s the single most important task when it comes to scheduling partnering meetings?

TAYLOR: It will seem obvious, but the most important thing I’ve learned is to DO YOUR HOMEWORK. I usually start researching companies within three months of the Convention because otherwise it can feel like diving into a mosh pit of 20,000 people. The online instructions are a great place to start. A close second most important task though, is to make sure you populate your profile really well and use well-chosen key words to highlight your company interests and offerings, not just name and location.

Bill Taylor, VP Business Development, Protalix Biotherapeutics

BB:  What is the most important part of a company profile?

TAYLOR: In the Protalix profile I talk about what we’re doing and what our pipeline is, which allows other companies looking for opportunities to send me an invite. You’ve got to recognize that your company profile on the system is a two-way street in that respect. Our profile will read something like this:

Protalix is a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by our proprietary ProCellEx® plant cell-based protein expression system. Our pipeline consists of proprietary, potentially clinically superior versions of recombinant therapeutic proteins that target established pharmaceutical markets with a focus on rare diseases.

BB:  How many people do you think should attend a partnering meeting from each company?

TAYLOR:  Two to three, tops. One or two from the company, and myself. That said, sometimes companies want to bring their science and technical experts to do some drill-down for their initial diligence questions. Knowing this in advance will save time.

BB:  What should a company expect to achieve during partnering meetings?

TAYLOR: Like most things you get what you put into it. So, if I’ve done my homework, I expect to walk away with well-grounded conversations that cover the full width of our pipeline; conversations where I can articulate our value propositions, while conveying the commercial opportunities that our products represent. BIO is unique because it provides a very broad band forum in which to meet up and engage with a large number of companies across a vast range of geographies.

BB:  Should a company lock-down their schedule, or keep it flexible?

TAYLOR: I find that there’s no lack of people pinging me with lots and lots of invites, so I have to be highly disciplined about my scheduling strategy, while remaining open-minded to unforeseen possibilities. The challenge is to get your scheduled meetings locked down early, but it’s equally important to have some safety valve time-slots available to ensure a meeting can actually occur if it comes in on a last-minute blitz.

BB:  This will be your 16th consecutive year attending the BIO International Convention. What keeps you coming back?

TAYLOR: BIO is a lot like Homecoming. There may be nearly 20,000 people but we have a lot in common; we’re a collegial group that live and survive by the networks we’ve created over the years. Even waiting for a coffee — or a beer at the end of the day — you’ll strike up a conversation with someone who may end up as a great resource or they might be someone you’ve met at a past event.  It’s a place for former colleagues to re-connect, meet up with old contacts and make new connections. In that respect it really does feel like Homecoming.

BB: Does that make you the BIO International Convention Homecoming King?

TAYLOR: I guess so. You should see my convention badge collection!  After all these years, I’m still just a science geek!

BIO Releases Largest Study Ever on Clinical Development Success Rates

On Wednesday, BIO released the largest ever study of clinical development success rates. The study, conducted in partnership with Amplion and Biomedtracker, recorded and analyzed 9,985 clinical and regulatory phase transitions, across 1,103 companies. Using clinical trial data from the past decade, “Clinical Development Success Rates 2006-2015” compares groups of diseases, drug modalities and other attributes to generate the most comprehensive analysis, to date, of biopharmaceutical R&D success.

“This study provides a wealth of information about drug development success rates across a broad range of indications,” said Cartier Esham, PhD, BIO’s Executive Vice President, Emerging Companies. “The results may be used to pinpoint disease areas and phases of development where the industry has been most successful in recent years, as well as areas presenting challenges along the capital intensive-pathway of drug development.”

Key findings from the study include:

  • Clinical trial programs that used selection biomarkers saw an overall likelihood of approval (LOA) from Phase I of 25.9%, compared to 8.4% when no selection biomarkers were used.
  • The overall LOA from Phase I for all developmental candidates was 9.6%, and 11.9% for all indications outside of Oncology.
  • Of the 14 major disease areas studied, Hematology had the highest LOA from Phase I (26.1%) and Oncology had the lowest (5.1%).
  • Oncology drugs were approved the fastest of all 14 disease areas.
  • Rare disease programs had higher success rates at each phase of development vs. the overall dataset.
  • Chronic diseases with high populations had lower LOA from Phase I vs. the overall dataset.

The study relied upon years of clinical program monitoring and data entry by Informa’s Biomedtracker service. BIO has long partnered with Biomedtracker to calculate success rates based on this data. More recently, BIO and Biomedtracker partnered with Amplion, the inventors of BiomarkerBase, to analyze the effects of biomarkers in clinical trial success.

“Combining Amplion’s biomarker database with Biomedtracker’s clinical transition records we were able to, for the first time, quantify the benefit of using selection biomarkers in drug development,” said study author David Thomas, CFA, BIO’s Senior Director of Industry Research. “In combination with the rare disease section of the report, these results appear to be revealing the overall strength in targeting well-defined, homogenous patient populations.”

A full version of the report is available for download here.

Thomas will be discussing the report on June 6 at the 2016 BIO International Convention during his session, “The State of the Innovation Industry.”

Filed under: Business and Investments, Health, Inside BIO Industry Analysis, , , , , , , , , ,

2015 FDA Approvals: Highest Levels in Over a Decade

2015 was a blockbuster year for FDA approvals of novel new medicines, many of them for serious and life-threatening conditions. As Dr. John Jenkins of the FDA’s Center for Drug Evaluation and Research (CDER) notes in a recent blog post, “During this past year, we approved many new drugs to treat various forms of cancer, including four to treat multiple myeloma, and others to treat lung, skin, breast, brain, colorectal, and other cancers. We also approved new drugs to treat heart failure, high cholesterol, cystic fibrosis, and irritable bowel syndrome, as well as the first approved reversal agent for a commonly-used blood thinner. And, for the second consecutive year, we approved more drugs to treat rare diseases than any previous year in our history.”

BIO’s Industry Analysis team has tallied the number of novel medicines approved in 2015 by both FDA’s Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) divisions. The combined number is a spectacular 48 novel approvals.

This number is higher than what has been widely been reported in the media as most outlets only report CDER’s tally of 45 new drugs. However, three novel biologics were approved by CBER in 2015, bringing the total to 48. The three biologic therapies approved by CBER are: 1) Imlygic, an oncolytic virus cancer therapy from Amgen that selectively replicates in and kills tumor cells, 2) Bexsero, a meningococcal disease vaccine from Novartis, and 3) Adynovate, Baxalta’s pegylated FVIII protein for the treatment of Hemophilia A: Below are some additional 2015 findings:

  • 12 (25%) of the 2015 approved novel drugs received Breakthrough Therapy Designation
  • 8 (16%) of the 2015 approved novel drugs received Accelerated Approval
  • 43 (90%) of the 48 novel drugs were first cycle approvals
  • 33 (69%) of the novel drugs were small molecule and 15 (31%) were biologics
  • 11 (23%) of the novel approvals were First-in-Class (new mechanism of action) drugs

Here’s a breakdown by disease area:

That’s the kind of innovation that BIO member companies work hard to bring to patients each and every day. Here’s to 2016!

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.