Dr. Yuan-Tseh Lee Joins Fellow Scientists on Biotechnology Company Advisory Board

SANTA BARBARA, Calif.--(BW HealthWire)--May 7, 2001--EpiGenX Pharmaceuticals Inc., announced today that Dr. Yuan-Tseh Lee has joined a distinguished advisory board which has been assembled to guide the Company in the development of epigenetic-based diagnostics and therapies for cancer and infectious disease. The Scientific Advisory Board, composed of leading scientists in the biotechnology and academic community, will be instrumental in accelerating the development and commercialization of the company's technologies.

Dr. Yuan-Tseh Lee joins an elite group including Michael T. Bowers, Ph.D., Professor of Chemistry and Thomas C. Bruice, Ph.D., Professor of Chemistry and Biochemistry from University of California, Santa Barbara; Joseph F. Costello, Ph.D., on the faculty of University of California, San Francisco, Department of Neurological Surgery; Melvin Louis DePamphilis, Ph.D., Section Chief for the Eukaryotic DNA Replication and Gene Expression unit of the Molecular Growth Laboratory at the National Institute of Child Health and Human Development; as well as Jeffrey L. Ebersole, Ph.D., Professor of Oral Health Research and Director, Center for Oral Health Research, from University of Kentucky, College of Dentistry and Arthur D. Riggs, Ph.D., Associate Director of the Beckman Research Institute of the City of Hope National Medical Center.

EpiGenX's Scientific Advisory Board now features expertise covering cancer biology, infectious disease, bioorganic chemistry and mass spectrometry technologies. Epigenetic strategies entail developing the advanced understanding of gene regulation, and its manipulation in order to fight disease, particularly cancer and infections.

"We are extremely honored to have Dr. Lee join our advisory board," commented Larry Bymaster, President and Chief Executive Officer, EpiGenX. "He has unparalleled expertise in molecular beam technology and we are pleased that he has agreed to provide counsel to our team of scientists."

"Dr. Lee is universally recognized as a world leader in design and use of customized state-of-the-art instrumentation for chemical analysis," noted Alec Wodke, Director of Epigenomics, EpiGenX. "We look forward to his guidance in defining the technology directions for our High Throughput Epigenetic applications." 

 EpiGenX Scientific Advisory Board is now composed of:

• Michael T. Bowers, Ph.D., a Professor of Chemistry at UCSB, and one of the world's leading scientists in the field of mass spectrometry has served for over 10 years as, Editor, International Journal of Mass Spectrometry and Ion Processes and serves as Associate Editor Journal of the American Chemical Society. Dr. Bowers his Ph.D. in 1966 from the University of Illinois. His Awards include: Nobel Laureate Signature Award of the American Chemical Society (1988); American Chemical Society Award for Outstanding Achievement in Mass Spectrometry (1996); Guggenheim Fellowship (1994); and UCSB Faculty Research Lecturer (1994). He is a Fellow of the American Physical Society and of the American Association for the Advancement of Science.

• Thomas C. Bruice, Ph.D., a Professor of Chemistry and Biochemistry at UCSB, was listed among the world's 50 most cited chemists for the period from 1984 to 1991. He has been elected a Member of the National Academy of Sciences (1974), a Fellow of the American Academy of Arts and Sciences and a Fellow of the AAAS (1989) as well as the Royal Society of Chemistry. His awards include: NIH Career Development (1979); Lifetime Investigator and MERIT Awards; Guggenheim Fellow (1979); UCSB Faculty Research Award (1970); American Chemical Society Arthur C. Cope Scholar Award (1987), Richard C. Tolman Medal (1979), Repligan Medal in Biochemistry (1987), Alfred Bader Medal in Bioorganic and Bioinorganic Chemistry (1988), and the James Flack Norris Award (1996) in physical organic chemistry.

• Joseph F. Costello, Ph.D., serves on the faculty at University of California, San Francisco, Department of Neurological Surgery, and is a leading expert in the field of methylation, including identifying non-random and tumor-specific methylation patterns in human malignancies. His awards include the James S. McDonnell Foundation 21st Century Scientist Award. Dr Costello is a member of the Brain Tumor Research Center at UCSF and has had his work featured in editorials published in Nature Genetics, Science, The Scientist and Wired Magazine. He serves as an ad hoc reviewer for Nature Genetics, Proceedings of the National Academy of Science, Cancer Research, and Genes Chromosomes & Cancer.

• Melvin Louis DePamphilis, Ph.D., is the Section Chief for the Eukaryotic DNA Replication and Gene Expression unit of the Molecular Growth Laboratory at the National Institute of Child Health and Human Development, Bethesda, MD. He previously served as a Laboratory Head and Full Member of the Roche Institute of Molecular Biology, an Adjunct Professor in the Department of Biological Sciences at Columbia University, and a Professor in the Department of Biological Chemistry at Harvard Medical School. Dr. DePamphilis is one of the country's leading experts in the initiation of DNA replication in mammalian chromosomes; gene  expression at the beginning of mammalian development. He currently serves on the editorial board of the following journals: Molecular Reproduction and Development, Molecular Biology Reports, Gene Therapy & Molecular Biology, Cell Structure and Function and Molecular and Cellular Biology.

• Jeffrey L. Ebersole, Ph.D., is Professor of Oral Health Research and Director, Center for Oral Health Research, at University of Kentucky, College of Dentistry. Earlier in his career, he was Professor of Periodontics and Microbiology at The University of Texas Health Center and, in the mid-1980's, Associate Clinical Professor of Oral Biology and Pathophysiology at the Harvard School of Dental Medicine. Dr. Ebersole's research area is B cell biology and antibodies in secretory immunity and periodontal immunobiology. He has had NIH funding for over 25 years. His primary research emphasis is in the development, specificity and functional abilities of antibodies in the oral cavity.

• Yuan-Tseh Lee, Ph.D., a Nobel laureate in chemistry in 1986, is  the current President of Academia Sinica, the highest research institute in Taiwan. In 1986, Lee shared the Nobel Prize in Chemistry with Dudley R. Herschbach and John C. Polanyi for helping to apply the technology and theory of physics to chemistry. In his research, Lee extended Herschbach's "crossed molecular beam technique" to analyze larger and more complex molecules. Dr. Lee received his doctorate from the University of California at Berkeley in 1965. His world-leading laboratory now contains seven very sophisticated molecular beam apparati, which were specially designed to pursue problems, associated with reaction dynamics, photochemical processes, and molecular spectroscopy. His awards include: Alfred P. Sloan Fellow (1969 - 1971); Fellow, American Academy of Arts and Science (1975); Fellow, American Physical Society, (1976); John Simon Guggenheim Fellow (1976 - 1977); Member, National Academy of Sciences (1979); Member, Academia Sinica, Taiwan, China (1980); Ernest O. Lawrence Award, U.S. Department of Energy (1981); Peter Debye Award of Physical Chemistry, American Chemical Society (1986) and the National Medal of Science in 1986.

• Arthur D. Riggs, Ph.D., a noted molecular biologist, serves as the Chair, Division of Biology, and Associate Director of the Beckman Research Institute of the City of Hope National Medical Center,  which has been designated a Comprehensive Cancer Center by NCI.  Dr. Riggs' was one of the scientific founders of Genentech. He is a pioneer in developing an understanding of how methylation acts  as an epigenetic control. Currently he is studying X chromosome inactivation and the biological roles of DNA methylation. Dr. Riggs' scientific achievements span three decades at City of Hope.

EpiGenX has accepted invitations to make presentations at two industry conferences this month. The company will be presenting at the Innovative Drug Development Conference in New York on May 8th and the C21 Biotech Convergence Conference in Monterey Bay, California on May 23rd.

EpiGenX Pharmaceuticals is pioneering the development of epigenetic-based diagnostics and therapies tailored to the needs of individual patients. Epigenetic strategies entail developing the advanced understanding of gene regulation, and its manipulation in order to fight disease, particularly cancer and infections. The company's technology platforms all involve DNA methylation. EpiGenX has three commercialization pathways. Its drug discovery tools, EpiHiTS(TM) Technology include a suite of high-throughput assays for the rapid discovery of new epigenetic-based drugs. The company is developing novel and safe therapeutics to fight cancer and infectious diseases through epigenetic mechanisms. EpiGenX is also developing a suite of high-throughput diagnostic and epigenomics technologies to generate information on the activity and inactivity of genes in relation to various diseases. EpiGenX Pharmaceuticals, Inc., is located in Santa Barbara, CA. For more information, call 805-964-4486 or log on to http://www.epigenx.com

Award Abstract : 

DESCRIPTION (provided by applicant): We propose to develop a new class of anticancer therapeutics based on inhibiting DNA cytosine methyltransferase (DNMT). Such compounds will form the basis for subsequent drug development, with potential applications to cancer therapy. DNA methylation plays an essential role in regulating gene expression; inhibiting the enzyme is a validated anticancer strategy. Sporadic forms of cancer have an epigenetic component, which are amenable to reversible intervention; thus are inherently less cytotoxic. We have developed an enzyme-based high throughput screen and identified small molecule DNMT inhibitors in an initial 8,000 compound screen. We have purchased a 50,000 compound library, and from this historical library will be submitted to a secondary cell-based screen to detect inhibition of methylation of a promoter containing a green fluorescence protein (GFP) reporter. Lead compounds will be tested in cell-based assays to determine their ability to alter the methylation pattern and expression of specific genes, in which hypermethylation has been suggested to play a role in tumorigenesis. The best candidates will be submitted to a panel of cytotoxicity studies and used as a design platform for the synthesis of new compounds. We expect to develop small molecule inhibitors with acceptable efficacy and toxicity profiles for testing in animal models of human cancers. PROPOSED COMMERCIAL APPLICATION: NOT AVAILABLE

BOTHELL, Wash., Feb. 12 -- Nastech Pharmaceutical Company Inc. (Nasdaq: NSTK - News) announced today that its Board of Directors has approved a plan to further reduce operating expenses and align the company's workforce with its strategic, business and clinical development requirements. 

"The workforce reduction of approximately 50 employees will enable us to drive our key clinical development and RNAi programs forward in a more efficient manner for the benefit of our shareholders," stated Steven C. Quay, M.D., Ph.D., Chairman and CEO of Nastech. "The savings by this action are estimated to be not less than $11 million during the 2008 fiscal year." 

• About Nastech :   Nastech is a clinical stage biopharmaceutical company focusing on the development and commercialization of innovative therapeutic products based on its proprietary molecular biology-based drug delivery technologies and, through its wholly-owned subsidiary, MDRNA, Inc., based on its proprietary ribonucleic acid interference technology. Nastech and its collaboration partners are developing products for multiple therapeutic areas including osteoporosis, obesity, diabetes, autism, respiratory diseases and inflammatory conditions. Additional information about Nastech is available at http://www.nastech.com.   

BOTHELL, WA--(Marketwire - February 14, 2012) - Marina Biotech, Inc. (OTCQX: MRNA), a leading nucleic acid-based drug discovery and development company, today announced the closure of its Cambridge site and the consolidation of all research and development efforts at its headquarters in Bothell, WA. In addition, the Company announced dosing of the first patient in Cohort 2 in the Dose Escalation Phase of the START-FAP (Safety and Tolerability of An RNAi Therapeutic in Familial Adenomatous Polyposis) clinical trial with CEQ508.

"After considerable review, we have decided to close our Cambridge site and transfer those research and development efforts to our headquarters in Bothell, WA," said J. Michael French, President & CEO of Marina Biotech. "This move will not affect the START-FAP trial as noted by our announcement today of Cohort 2 dosing. Further, we expect to continue to develop the tkRNAi platform in our labs in Bothell. The closing of the Cambridge site is consistent with our continued efforts to reduce our cash utilization. The individuals affected by this decision were all part of the former Cequent team who then, and certainly over the past several years, were instrumental to the development of CEQ508 and the successful execution of the START-FAP trial. I want to thank them for their efforts and wish them all well in their future endeavors."

About two years ago Mirna Therapeutics Inc., which is developing a new type of cancer therapy, sought to solve one of its toughest technical challenges through a partnership with Marina Biotech Inc. Liking what it sees so far, Mirna has now gained the right to use Marina's drug-delivery capabilities more extensively.

Mirna develops oncology drugs that mimic the effects of certain microRNA molecules found in cells. Its lead drug, MRX34, mimics miR-34, a tumor-suppressing molecule that's lost or expressed at reduced levels in most solid tumors and blood cancers.

ProNAi Therapeutics Inc. has collected $59.5 million after clinical studies showed that its lead drug could help non-Hodgkin's lymphoma patients who are running out of options.

ProNAi, based in Plymouth, Mich., develops nucleic acid therapies for cancer and other diseases. Its lead product, PNT2258, targets the BCL-2 gene to inhibit cancer cell proliferation and promote tumor cell death. Other companies working in this area include Genentech Inc., whose pipeline includes a small-molecule BCL-2 protein inhibitor for chronic lymphocytic leukemia and non-Hodgkin's lymphoma.

It wouldn’t be a stretch to call 2018 the year RNA-based treatments, such as those applying RNA interference (RNAi) or small interfering RNA (siRNA), finally reached the proverbial tipping point predicted ever since Andrew Z. Fire and Craig C. Mello won the 2006 Nobel Prize in Physiology or Medicine for discovering RNAi.

Earlier this month, Moderna Therapeutics carried out the largest-ever initial public offering (IPO) for a biotech. And in August, Alnylam won the first-ever FDA approval for an RNAi drug in the U.S. Both companies appear on GEN’s first-ever list of top biopharmas focused on leveraging RNA-based treatments.

Yet at least one company that didn’t make the list nevertheless left its mark in RNA-based drug development this past year. In June, Translate Bio (which changed its name last year from RaNa Therapeutics) completed its own smaller IPO, raising approximately $113.4 million in net proceeds. Two other companies changed their names this year: RXi Pharmaceuticals became Phio Pharmaceuticals on November 19, while Marina Biotech became Adhera Therapeutics on October 9.

According to Grand View Research, the global antisense and RNAi therapeutics market size is expected to expand at a compound annual growth rate (CAGR) of 8.6% between 2018 and 2025, when it is projected to reach $1.81 billion.

Below is a list of the top 10 biopharmas focused on developing RNA-based drugs—five public companies, and five private companies. The public companies are ranked by their 2017 revenues, whether from sales of products or services, or from collaborations and R&D activity. On that basis, Translate Bio didn’t generate enough revenue to be among the top-five public companies.

Private companies are ranked by the total capital they have raised, as disclosed by the companies themselves, either in press statements or in responses to GEN queries. Each company is listed with a short explanation of their recent activity.

Public Companies

5. Arrowhead Pharmaceuticals

Revenue: $31.408 million (Fiscal Year ending September 30, 2017); $16.142 million (FY 2018)

Arrowhead is half of 2018’s biggest collaboration deal, an eye-popping more-than-$3.7-billion partnership with Johnson & Johnson’s Janssen Pharmaceuticals to develop an RNAi treatment candidate for hepatitis B virus (HBV), plus up to three other RNAi therapies against targets to be selected by Janssen. The deal gives Janssen a global exclusive license to Arrowhead’s ARO-HBV program, designed to develop a third-generation subcutaneously administered RNAi therapeutic candidate as a potentially curative therapy for patients with chronic HBV. On November 9, Arrowhead announced positive Phase I/II results for ARO-HBV, saying it “effectively reduced all measurable viral products, including HBsAg [hepatitis B surface antigen].” Arrowhead also trumpeted positive Phase I results for ARO-AAT, a second-generation RNAi treatment for a rare genetic liver disease associated with alpha-1 antitrypsin (AAT) deficiency.

4.Akcea Therapeutics
Revenue: $55.209 million (2017); $54.670 million (Q1–Q3 2018)

Akcea’s revenues the first three quarters of this year were almost as much as the company generated all of last year, thanks to $12 million in licensing revenue from its collaboration with PTC Therapeutics, plus a more-than-doubling of R&D revenue. All that R&D revenue came from Akcea’s collaboration with Novartis, launched last year, with Akcea amortizing $75 million shelled out by the pharma giant, which also purchased Ionis stock at a premium of $33 million. Akcea is an affiliate of Ionis Pharmaceuticals. In July, the two companies won European Commission approval of their Tegsedi™ (inotersen), an RNA-targeted treatment for polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR) in adults. The FDA followed suit three months later.

3. Alnylam Pharmaceuticals

Revenue: $89.912 million (2017); $53.875 million (Q1–Q3 2018)

Alnylam won the first-ever FDA approval for an RNA interference (RNAi) drug in the U.S. on August 10, when the agency authorized Onpattro™ (patisiran) for polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adults. On August 30, the European Commission followed suit and approved Onpattro, which is designed to interfere with RNA production of an abnormal form of the protein transthyretin (TTR). Onpattro has already begun generating revenue, $0.5 million in Q3 to be precise—a fraction of the company’s nine-month revenue, nearly all of it from collaborations. On December 6, Alnylam announced its R&D plans for 2019; they include continuing the global rollout of Onpattro and launching a Phase III trial for ATTR amyloidosis candidate vutrisiran.

2. Moderna

Revenue: $205.825 million (2017); $113.921 million (Q1–Q3 2018)

Moderna made history on December 7 when it traded its first shares on the NASDAQ Global Select Market, in the largest-ever biotech IPO. While Moderna shares finished the day at $18.60, down $4.40 from the IPO price, the company still raised $604.3 million from the sale of 26.3 million shares—capital it says will fund mRNA technology platform development as well as further develop its 21-candidate clinical pipeline and its drug discovery and manufacturing capabilities. Moderna’s mRNA treatments differ by coding region, which according to the company gives its investigational mRNA medicines “a very software-like quality,” and can combine different mRNA sequences encoding for different proteins in a single mRNA medicine.

1. Ionis Pharmaceuticals

Revenue: $507.666 million (2017); $407.559 million (Q1–Q3 2018)

More than half of Ionis’ revenue (55%) came from R&D, the rest from commercial activity—including a near-tripling of royalties (to $168 million) from spinal muscular atrophy (SMA) treatment Spinraza® (nusinersen), co-developed with Biogen. On December 6, Biogen exercised its option to develop BIIB067 for a subtype of ALS based on positive Phase I data. As a result, Ionis received $35 million upfront and could see up to $55 million in milestones plus royalties from Biogen. Ionis suffered a setback in August when it, and its Akcea Therapeutics affiliate, received an FDA complete response letter for their NDA for Waylivra™ (volanesorsen), a candidate for familial chylomicronemia syndrome. Waylivra remains on track for potential approval in Europe, and Akcea has reported continuing talks with the FDA on a regulatory path for the treatment.

Private Companies

5. Gotham Therapeutics 

Total capital raised: $54 million

Gotham is perhaps the newest company to make this list, based on the $54 million Series A financing with which it debuted on October 10. While founding investor Versant Ventures, Forbion and S.R. One co-led the round. Gotham attracted two big-name investors of note—the biotech giant Celgene and Alexandria Venture Investments, the strategic venture capital arm of Alexandria Real Estate Equities. Versant created and seed-funded Gotham based upon the research discoveries of co-founder Samie Jaffrey, M.D., Ph.D., a pioneer in epitranscriptomics whose work has shed light on the role of post-transcriptional mRNA modifications in health and disease. The seed funding enabled Gotham to build a platform designed to assess the impact of RNA-modifying proteins on disease biology and developed small molecules against priority targets. Gotham says the Series A financing will allow it to establish clinical proof of concept and invest in a pipeline of preclinical candidates with potential to treat diseases intractable to classical approaches.

4. Quark Pharmaceuticals

Total capital raised: $61.4 million

Quark Pharmaceuticals hasn’t announced a financing round since 2010, when it completed a $10 million private financing by existing investors, namely funds of SBI Holdings Group in Japan. Yet, Quark has kept busy developing novel siRNA therapeutics for unmet medical needs. At the American Society of Nephrology (ASN) 2018 Kidney Week Meeting, held October 23–28 in San Diego, Quark presented positive data from a one-year mortality follow-up of a Phase II trial (NCT02610283) assessing its teprasiran (previously QPI-1002), an siRNA targeting the p53 gene, in subjects at high risk for acute kidney injury following cardiac surgery. Also in Quark’s pipeline is QPI-1007, a siRNA candidate targeting the gene Caspase 2 and being developed to treat non-arteritic ischemic optic neuropathy (NAION). Crunchbase tallies $61.4 million in total capital raised by Quark, which at deadline hadn’t responded to GEN queries seeking to verify that figure.

3. Gradalis

Total capital raised: $89 million

Gradalis has been on an expansion track this year, closing on a $55 million Series C financing and upgrading its current Good Manufacturing Practice (cGMP) manufacturing facility in Carrollton, TX, to support the launch of a Phase III registration trial of its immunotherapy platform Vigil™ plus chemotherapy drugs Irinotecan and Temozolomide in Ewing’s sarcoma (NCT03495921). Ewing’s sarcoma is one of several advanced cancer indications for which Vigil is being developed; the platform is also in combination studies with checkpoint inhibitors targeting advanced gynecological and other women’s cancers. Gradalis raised $10 million in a Series B round in 2013, and $10 million in a Series A round six years earlier.

2. CureVac

Total capital raised: About €450 million (about $511 million)

CureVac delivered promising news in November at the 2018 Society of Immunotherapy of Cancer (SITC) Annual Meeting: One patient treated with its RNA-based solid tumor candidate CV8102 saw complete regression of injected and non-injected lesions while four patients achieved stable disease in a Phase I trial (NCT03291002). A TLR-/RIG-I agonist, CV8102 is one of four clinical-phase candidates for CureVac, which has collaborations with the Bill & Melinda Gates Foundation, Boehringer Ingelheim, CRISPR Therapeutics, Arcturus Therapeutics, Acuitas, and Eli Lilly—with which CureVac is partnering to develop up to five cancer vaccines under a potentially $1.795 billion-plus collaboration. In May, CureVac said it will expand its financial and scientific presence in Boston while retaining its global HQ and manufacturing in Tübingen, Germany.

1. BioNTech

Total capital raised: More than $1 billion

BioNTech raised a hefty $270 million Series A financing in December 2017 led by the Redmile Group, and intended to advance its clinical immunotherapy pipeline, led by its mRNA-based Individualized Vaccines Against Cancer (IVAC®) Mutanome and FixVAC® cancer vaccines. The Series A came about 10 years after the company attracted $180 million in seed funding in December 2008. BioNTech turned heads again in August by scooping up $120 million upfront, equity and near-term research payments from Pfizer under a collaboration to develop mRNA-based vaccines for prevention of influenza. The collaboration could also generate $305 million for BioNTech in milestone payments from Pfizer, which joined several other biopharma giants in partnering with BioNTech, including Genentech, a member of the Roche Group, Eli Lilly, Sanofi, Genmab, and Bayer Animal Health.