BIO’s Next Generation Partnering System to Open April 2015

You’ve found your lead investor. Now it’s time to get to the next stage, and the best place to do so is the 2015 BIO International Convention. You have a meeting with Pfizer at 9:00 a.m. followed by a meeting with AstraZeneca/MedImmune at 9:30 a.m. Then a 30 minute break. Then you have four meetings back to back with Sofinnova, Takeda Ventures, Gates Foundation and Merck. After a quick lunch it’s back to partnering: make the pitch and build relationships for when the time is right. Check out the rich set of service providers that will help you on your way.

Getting these meetings was no small feat. But you got them and you’ll make the most of them because BIO’s next generation partnering system, One-on-One Partnering™, and Inova’s collaboration module, One-on-One Partnering™ Plus, will help you.

One-on-One Partnering and One-on-One Partnering Plus will help you minimize the time you need to manage your partnering workflow, information and content, and maximize the time available to do your job. Imagine no data entry after the BIO International Convnetion. All your meetings and information about the companies and individuals you met with can now seamlessly transfer to One-on-One Partnering Plus, a powerful partnering workflow tool. BIO’s new partnering system will maximize partnering collaboration within your company with social bookmarking, voting, comment threads, meeting history, Outlook integration, task management and other features that apply both to partnering at BIO events and your year-round partnering workflow.

It’s time to dramatically increase your partnering ROI. BIO and INOVA, the leader in partnering relationship management solutions, have teamed to help you get there.

See you at BIO 2015.

BIO Investor Forum Company Snapshot: Syntrix Biosystems

1.     What is your company’s lead product or technology?

Omnitram (O-desmethyltramadol) is a novel  oral analgesic developed by Syntrix that alleviates pain via a dual mechanism involving mu-opioid receptor activation and inhibition of the reuptake of norepinephrine.  Omnitram is in clinical development and has just completed a Phase 1b double-blind, placebo-controlled clinical trial (N=40) in experimental pain.

2.     What sets your company apart from competitors?

Omnitram is a proprietary solution for pain relief that is thought to be less addictive than traditional opioids and capable of treating a wider spectrum of pain types than traditional opioids.  Omnitram is active without requiring metabolic activation.  Other available analgesics require metabolic activation and are ineffective in many patients due to genetics or drug-drug interactions.  Omnitram exploits the active metabolite of an FDA approved analgesic and may offer a shortened and less costly regulatory path to approval.

3.     What has been your company’s most exciting recent accomplishment or milestone?

The Omnitram IND opened in March 2014.  Syntrix was able to start and complete enrollment of the Phase 1b trial within only seven months of opening the IND, hitting this milestone well ahead of schedule.

4.     How has the NIH SBIR program helped you in working to achieve your long-term goals? (50-75 words)

Drug development is a long and difficult path that requires patience and a reliable capital source.  For Syntrix, the NIH SBIR program has been that patient and consistent source of capital.  With over $25 million in NIH SBIR capital raised to date, the NIH SBIR program has allowed us to successfully mature our drug pipeline from early discovery stage on through phase 2 clinical testing.

5.     What do you hope to accomplish through your company presentation and participation at the BIO Investor Forum?

Connect with potential strategic pharmaceutical partners and equity investors.

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

Syntrix has two other drug candidates in its clinical pipeline.  SX-682 is a novel oral CXCR1/2 inhibitor being developed for cancer and inflammatory diseases such as COPD.  The IND for SX-682 is scheduled to open in the next year.  LD-aminopterin is a once-a-week oral therapy in phase 2 clinical testing for inflammatory diseases that include psoriasis and rheumatoid arthritis.  Each of the three drug candidates in the Syntrix pipeline are directed to multi-billion dollar therapeutic markets.

www.syntrixbio.com

Click here to learn more about BIO Investor Forum.

Understanding the Biotech Market in Brazil

The biotechnology market in Brazil has seen enormous activity this year in both the areas of industrial biotechnology and healthcare. In a recent webinar that discussed the latest results of the annual Scientific American Worldview report, panelists were especially excited by the partnering opportunities in Brazil. Mike May, Editorial Director, Scientific American Worldview, commented, “Brazil is, as a country, like a master of partnering. They partner in the country between industry and government. They partner outside the country with industry and industry in other places. They have huge networks of partnering. They’re bringing industries in from other countries to help them with things where maybe they don’t have the technology… So you know, it’s like they’re finding creative ways to deal with some of the challenges they have that, say, the United States doesn’t have.”1

Building on this movement, BIO and Biominas have partnered to co-host the first annual BIO Latin America September 9-11, 2014 in Rio de Janeiro, Brazil, the industry’s must-attend conference for innovation and collaboration in Latin America’s rapidly-growing biotechnology sector. Here is a look at the current state of the biotech market in Brazil.

Industrial Biotechnology

Brazil is well known for its research and investment in industrial biotechnology, particularly in regards to cellulosic sugars and agribusiness. In April, several companies including Amyris, BP, Dow Chemical, DuPont, and Novozymes came together to launch of the Brazilian Industrial Biotech Association (ABBI) to promote dialogue with stakeholders, policy makers, and the public about advancing industrial biotechnology in Brazil. The trade group aims to improve current patent laws in light of new biotechnology advancements, support investments in R&D, laboratory infrastructure, and capacity and training for skilled and technical labor.

At the launch event for ABBI, Greg Stephanopoulos, from the Department of Chemical Engineering at MIT, said that industrial biotechnology would help Brazil take a leadership position in the 21st Century global economy2.

Biotech Sales Growth among BRIC Countries

Despite representing under 5% of global biotech sales, BRIC countries (Brazil, Russian, India, and China) have seen a 29.3% compound annual growth rate (CAGR) from 2010-2012.

Figure 1, BRIC Biotechnology Sales Growth for the top 100 Global Pharma Companies, 2010-2012 (source: EvaluatePharma)

More Biotech Companies in Brazil Focus on Human Health than Agribusiness

Biotechnology makes up an especially large portion of the current bioscience enterprises in Brazil. Out of 271 bioscience enterprises, 143 (or 51%) are biotechs. Out of the 143 biotech enterprises, the largest segment by application (33%) is comprised of companies with a human health focus.

While Brazil is valued as a leader in industrial biotechnology and is known for their investment and R&D in cellulosic sugars and biofuels, there is a portion of a the biotech market that is focused on human health. In 2012, there were 89 biotech companies dedicated to the development of new medications (small molecules and biological), diagnostics, vaccines, cell therapy, regenerative medicine and tissue engineering, advanced method for assisted reproduction, genetic and molecular testing, etc.3

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Figure 2. Number of Biotech Enterprises in Brazil (source: pwc/Biominas)

fg3Figure 3: Brazilian Biotech Companies by Application (source: pwc/Biominas)

The Healthcare Market in Brazil

Brazil is currently the largest healthcare market in Latin America, covering almost one-fourth of the population3. It also has the highest per head healthcare spending in Latin America, which has seen steady growth year over year alongside Brazil’s growing population of 195 million4.

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Figure 4, As Brazil’s Population Grows, So Does Its’ Health Spending (source: Sutherland Global Services)

The pharmaceutical market in Brazil grew at a CAGR of nine per cent from 2004-20105,6. According to Business Monitor International (BMI),7 Brazil’s pharmaceutical market is projected to reach $26.2bn in 2014. A report by Deloitte8 found that Brazil’s government has moved to align the drug regulatory environment with international standards, including intellectual property (IP) reforms carried out in recent years.

Deal making between global pharma giants and smaller Brazilian companies are helping to drive the success of the healthcare industry in Brazil, such as Sanofi’s purchase of Medley, Brazil’s largest generic-drug company and third-largest pharmaceutical maker9, and Amgen’s acquisition of privately-held Brazilian generics firm Bergamo, for $215 million10.

This demonstrated growth of the healthcare industry in Brazil and recent deal making activity highlights the importance of accessing the innovation and partnering opportunities in Latin America’s rapidly-growing life science industry. Registration for BIO Latin America is now open. We hope to see you there!

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Partner with Top Asian Biopharma Executives at BIO Asia

On April 8-9, 2014, a diverse group of innovative companies and Japanese dealmakers will come together in a collaborative setting for partnering meetings, company presentations, networking and targeted programming at the 11th annual BIO Asia International Conference.

Japan’s presence as the 2nd largest pharmaceutical industry in the world, bolstered by an evolving Asia-Pacific partnering landscape, means biopharma companies cannot ignore the deal opportunities that are being created in this dynamic market.

Industry leaders have recognized Japan as being ripe with partnership opportunities for biopharma companies this year. Joseph Panetta, president and CEO of Biocom, told Genetic Engineering & Biotechnology News (GEN), “Japan’s moves toward building biopharma appear to give U.S. companies a faster option for expansion into Asia than has been seen after a decade of engagement with China.” Similarly, JP Morgan healthcare specialist Scott Braunstein has touted the direct access that the Japanese pharma landscape has afforded Western companies, saying, “’Many pharma companies have a free run into Japanese markets as it has become so open.”

This kind of participation demonstrates why two days spent at BIO Asia will help your company build the important relationships that are required to succeed in the Asian partnering environment. BIO’s One-on-One Partnering™ system makes this possible by enabling senior executives to arrange private meetings to explore licensing and research collaborations.

BIO Asia also provides a platform for emerging and established innovative Western and Asian biopharma companies to reach new potential partners and tell their story. If you are interested in applying for a company presentation slot, we encourage you to submit your application today. Less than 5 spots remain. See the list of currently confirmed 2014 presenting companies here.

There’s no better time to strengthen your ties with the Japanese biotechnology sector. Additional details about this year’s program, BIO One-on-One Partnering™ and presentation opportunities, as well as registration information, can be found at www.bio.org/bioasia. We look forward to welcoming you to Tokyo!

Find Your Next Asia Partnership in Tokyo at BIO Asia

On April 8-9, 2014, the BIO Asia International Conference will enter its eleventh year. This year, biopharma industry leaders will convene at the Grand Hyatt in Tokyo, Japan to make the cross border deals that will advance collaboration between Asia and the West. This event has become the premier customized partnering event for Western and Asian biopharma executives. According to JP Morgan healthcare specialist Scott Braunstein, Japan has become one of the most critical markets for pharma and biotech firms. Japan currently holds the spot for the second largest pharmaceutical market in the world.

Asian biopharmas at this year’s plenary sessions will include Acucela Inc., TaiGen Biotechnology Co., Ltd., Pfizer (Asia), Baxter (Japan), and Taiwan Liposome Company, Ltd. The speakers and the full descriptions for the 2014 panels can be viewed here. In addition, the following list of Asian biopharma companies will be available for One-on-One Partnering, with more being added daily:

Acucela, Inc.
Allergan Singapore Pte Ltd.
Asahi Kasei Corporation
ASLAN Pharmaceuticals Pte Ltd.
Astellas Pharma, Inc.Baxter
Beijing Fogangren Bio-Pharm Tech, Co., Ltd.
Chugai Pharmaceutical Co., Ltd.
Curadev Pharma Pvt Ltd.
Daiichi Sankyo, Inc.
Dong-A ST
Dr.Reddy’s Laboratories Ltd.
Eddingpharm
Fresenius Kabi Asia Pacific Ltd.
Generon (Shanghai) Corporation
GlaxoSmithKline
Global Health Innovative Technology Fund (GHIT Fund)
Golden Biotechnology Corporation
Hisamitsu Pharma
Hua Medicine
ICS Convention Design, Inc.
Ildong Pharmaceutical Co., Ltd.
JCR Pharmaceuticals Co., Ltd.
Jeil Pharmaceutical
JCR Pharma
JT Pharma
Kissei Pharmaceutical Co., Ltd.
Lilly China Research and Development Co., Ltd.
Lundbeck Research China
Mitsubishi Tanabe Pharma Corporation
Mitsui Chemicals Inc.
MSD K.K.
Nippon Kayaku Co., Ltd.
Ono Pharmaceutical Co., Ltd.
Pluristem Therapeutics, Inc.
ReqMed Company, Ltd.
Sosei Co. Ltd.
TaiGen Biotechnology Co., Ltd.
Taiho Pharmaceutical Co., Ltd.
Taiwan Liposome Company Co., Ltd. (TLC)
Takeda Pharmaceutical Co., Ltd.
Tanaka International, LLC
Tech Manage Corp.
Teva Pharmaceutical Industries Ltd.
Tokyo Stock Exchange, Inc.

There is growing interest in U.S. partnerships by Japanese biopharmas, as evident in several recent deals including Ajinomoto’s $175 million acquisition of San Diego-based Althea Technologies last year. One reason why Japan has become popular among Western biotech and pharma companies is the direct access available in Japan to end markets. Leading biotech companies such as Celgene can sell their products straight into the Japanese market and enjoy overwhelming success.

Registration for the BIO Asia International Conference is open, and BIO’s One-on-One PartneringTM System has launched. Don’t miss this chance to capitalize on the growing opportunities for collaboration available in Asia- register today!

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.