La Jolla, California, September 12, 2019 (PRNEWSWIRE) – Avidity Biosciences, a privately-held biotechnology company pioneering Antibody Oligonucleotide Conjugates (AOCs™), today announced that Arthur A. Levin, Ph.D., Chief Scientific Officer, is scheduled to present at the 2019 Myotonic Annual Conference on Saturday, September 14 at 4:00 p.m. ET. The conference will be held September 13-14, 2019 in Philadelphia.

“Although oligonucleotides have shown promise for the treatment of muscle diseases, delivery has proven to be a challenge to the development of effective therapeutics,” said Dr. Levin. “Avidity has developed a powerful approach to overcome the oligonucleotide delivery challenge, and our AOCs are uniquely suited to treat myotonic dystrophy. We welcome the chance to share an update on our progress and our plans with the the myotonic dystrophy community at this year’s conference.”

About Avidity Biosciences, Inc.

Avidity Biosciences, Inc. is a privately-held biotech company pioneering Antibody-Oligonucleotide Conjugates (AOC™). AOCs combine the tissue selectivity of monoclonal antibodies and the precision of oligonucleotide-based therapeutics to overcome barriers to the delivery of oligonucleotides and target genetic drivers of disease. Using its AOC™ technology, Avidity has demonstrated significant and sustained reductions of  disease-related mRNAs expression in murine and primate models with ED50 of < 1mg/kg siRNA and and EC50 in the nanomolar range.  The company is focused on advancing an internal pipeline of therapeutic candidates to treat diseases including myotonic dystrophy, Duchenne muscular dystrophy, Facioscapulohumeral muscular dystrophy (FSHD) and other disorders. In addition, Avidity and Eli Lilly announced a strategic partnership in April 2019 to utilize Avidity’s AOC™ technology to pursue therapeutic targets initially focused on immunology and other indications. Avidity has raised $30 million in venture financing from a top-tier group of investors, including Takeda Ventures, Alethea Capital, Alexandria Real Estate Equities, Brace Pharma, EcoR1 Capital, F-Prime Capital, Moore Venture Partners, and Boxer Capital of Tavistock Group. For more information, please visit www.aviditybio.com.

Contacts:
Company and Media:
Leslie Ann Kerins
(858) 401-7900
leslie@aviditybio.com

La Jolla, California, August 22, 2019 (PR NEWSWIRE) – Avidity Biosciences, a biotechnology company pioneering Antibody-Oligonucleotide Conjugates (AOCs™) focused in rare muscle disorders and other serious diseases, today announced the appointment of Noreen Henig, M.D. and Edward Kaye, M.D., to the company’s board of directors.

“I am delighted to welcome Noreen and Ed to the Avidity board,” said Troy Wilson, Ph.D., Executive Chairman of the Board of Directors. “They bring deep experience in the development of oligonucleotide-based drug candidates and have held leadership roles in leading private and public companies. Their insight and perspective will be invaluable as we continue to advance our pipeline of therapeutic programs, optimize our AOC platform and prepare to initiate our first clinical study.”

Noreen Henig, M.D. brings over 20 years of clinical and development leadership experience at biopharmaceutical companies, in addition to an extensive background in academic research and medicine. Dr. Henig has served as Chief Medical Officer of Breath Therapeutics since July 2018. Breath Therapeutics was acquired by Zambon SpA in July 2019 for up to 500 Million Euros and is now a Zambon Group Company.  Previously, she served as Chief Development Officer of ProQR Therapeutics from 2013-2017, where she led preclinical and clinical development. ProQR had a successful cross-over round and NASDAQ IPO as a preclinical company in 2014.  Earlier in her drug development career, she spent six years at Gilead Sciences, most recently as the  Senior Director, Medical Affairs for Respiratory and PAH. Throughout her career, Dr. Henig held several senior academic positions at California Pacific Medical Center and Stanford University School of Medicine. She earned her M.D. with Distinction in Immunology from Albert Einstein College of Medicine, completed training in Internal Medicine at the University of California San Francisco and Pulmonary and Critical Care Medicine at the University of Washington.

“Avidity’s platform and assets have almost limitless therapeutic potential,” said Dr. Henig. “I am impressed with the Avidity team and technology, and I look forward to guiding their strategic plan to quickly bring their molecules to clinical development. Avidity’s therapeutic programs in rare genetic diseases and immunology are important advancements for patients in need.”

Edward Kaye, M.D., has served as CEO and a director of Stoke Therapeutics since October 2017. Dr. Kaye previously served as CEO and a director of Sarepta Therapeutics, where he led the successful push to win FDA approval of Exondys 51, a pioneering drug for Duchenne muscular dystrophy. Before taking on the CEO role at Sarepta, he served as the company’s Chief Medical Officer for six years, leading the development of Exondys 51. Earlier in his career, Dr. Kaye spent 10 years at Genzyme Corporation, most recently as Group Vice President of Clinical Development, where he supervised clinical research in programs focused on lysosomal storage disease and genetic neurological disorders. Dr. Kaye currently serves as a member of the boards of directors at Cytokinetics, Inc. and the Massachusetts Biotechnology Council. Prior to entering the biotech industry, Dr. Kaye trained in pediatrics, pediatric neurology and biochemical genetics. He was on the research staff at Massachusetts General Hospital and Tufts University Medical Center and was the chief of biochemical genetics at the Children’s Hospital of Philadelphia. He remains on the pediatric neurology staff at Boston Children’s Hospital. Dr. Kaye earned his B.S. in biology from Loyola University and earned his M.D. at the Loyola University Stritch School of Medicine.

“Avidity has developed a powerful approach to overcome a central challenge with oligonucleotide therapeutics – namely, delivery,” added Dr. Kaye. “AOCs have the potential to transform the field of oligo-based therapeutics, and I look forward to working with the leadership team and the board to continue to build the company.”

About Avidity Biosciences, Inc.
Avidity Biosciences, Inc. is a privately-held biotech company pioneering Antibody-Oligonucleotide Conjugates (AOC™). AOCs combine the tissue selectivity of monoclonal antibodies and the precision of oligonucleotide-based therapeutics to overcome barriers to the delivery of oligonucleotides and target genetic drivers of disease. Avidity has raised $30 million in venture financing from a top-tier group of investors, including Takeda Ventures, Alethea Capital, Alexandria Real Estate Equities, Brace Pharma, EcoR1 Capital, F-Prime Capital, Moore Venture Partners, and Boxer Capital of Tavistock Group. In April 2019, Avidity and Eli Lilly announced a strategic partnership to utilize Avidity’s AOC™ technology to pursue therapeutic targets initially focused on immunology and other indications. For more information, please visit www.aviditybio.com.

Contacts:
Company:
Leslie Ann Kerins
(858) 401-7900
leslie@aviditybio.com

Media:
Ian Stone
Senior Vice President
Canale Communications
(619) 849-5388
ian@canalecomm.com

• Collaboration will pursue therapeutic targets focused on immunology and other indications
• Avidity to receive an upfront payment of $20 million and an investment of $15 million
• Avidity eligible to receive up to approximately $405 million per target in development and commercialization milestones, plus royalties
  

INDIANAPOLIS, IN, and LA JOLLA, CA — April 22, 2019 /PRNewswire/ — Eli Lilly and Company (NYSE: LLY) and Avidity Biosciences, Inc. today announced a global licensing and research collaboration focused on the discovery, development and commercialization of potential new medicines in immunology and other select indications.

The companies will utilize Avidity’s technology platform to progress new therapeutic approaches toward clinical development and commercialization. Avidity’s platform seeks to combine the tissue selectivity of monoclonal antibodies and the precision of oligonucleotide-based therapeutics to potentially overcome barriers to the delivery of oligonucleotides and target genetic drivers of disease.

“We are excited to expand our oligonucleotide research and development efforts through this strategic collaboration with Avidity,” said Andrew C. Adams, Ph.D., chief scientific officer for RNA therapeutics at Lilly. “Their expertise in studying the combination of monoclonal antibodies and oligonucleotide-based therapies represent a promising avenue of research toward development of new RNA-based medicines.”

“This collaboration with Lilly provides an exceptional opportunity to leverage Avidity’s proprietary AOC platform in order to generate new therapeutic targets in disease areas that have been challenging to pursue using oligonucleotide-based approaches,” said Kent Hawryluk, Avidity’s chief business officer. “Lilly’s extensive research, development, regulatory, and commercial capabilities make them an ideal partner, and we look forward to a long and productive relationship.”

Under the terms of the agreement, Avidity will receive an upfront payment of $20 million, as well as an investment of $15 million. Avidity is also eligible to receive up to approximately $405 million per target for development, regulatory and commercialization milestones, as well as tiered royalties ranging from the mid-single to low-double digits on product sales.

This transaction is subject to clearance under customary closing conditions. The transaction will be reflected in Lilly’s reported results and financial guidance according to Generally Accepted Accounting Principles (GAAP). There will be no change to Lilly’s 2019 non-GAAP earnings per share guidance as a result of this transaction.

About Avidity Biosciences Inc.
Avidity Biosciences Inc. is a privately-held biotech company pioneering Antibody Oligonucleotide Conjugates (AOC™). AOCs combine the tissue selectivity of monoclonal antibodies and the precision of oligonucleotide-based therapeutics to overcome barriers to the delivery of oligonucleotides and target genetic drivers of disease. Avidity has raised $30 million in venture financing from a top-tier group of investors, including Takeda Ventures, Alethea Capital, Alexandria Real Estate Equities, Brace Pharma, EcoR1 Capital, F-Prime Capital, Moore Venture Partners, and Boxer Capital of Tavistock Group.

About Eli Lilly and Company
Lilly is a global healthcare leader that unites caring with discovery to create medicines that make life better for people around the world. We were founded more than a century ago by a man committed to creating high-quality medicines that meet real needs, and today we remain true to that mission in all our work. Across the globe, Lilly employees work to discover and bring life-changing medicines to those who need them, improve the understanding and management of disease, and give back to communities through philanthropy and volunteerism. To learn more about Lilly, please visit us at www.lilly.com and http://newsroom.lilly.com/social-channels.  C-LLY

Lilly Forward-Looking Statement
This press release contains forward-looking statements (as that term is defined in the Private Securities Litigation Reform Act of 1995) about the benefits of a collaboration between Lilly and Avidity, and reflects Lilly’s current beliefs.  However, as with any such undertaking, there are substantial risks and uncertainties in the process of drug development and commercialization. Among other things, there can be no guarantee that Lilly will realize the expected benefits of the collaboration, or that the collaboration will yield commercially successful products. For a further discussion of these and other risks and uncertainties that could cause actual results to differ from Lilly’s expectations, please see Lilly’s most recent Forms 10-K and 10-Q filed with the U.S. Securities and Exchange Commission. Lilly undertakes no duty to update forward-looking statements.

Refer to:
Mark Taylor; mark.taylor@lilly.com; (317) 276-5795 (Lilly Media)
Kevin Hern; hern_kevin_r@lilly.com; (317) 277-1838 (Lilly Investors)
Leslie Ann Kerins; leslie@aviditybio.com; (858) 401-7900 (Avidity)
Ian Stone; ian@canalecomm.com; (619) 849-5388 (Avidity Media)

LA JOLLA, CA — January 3, 2019 – Arthur A. Levin, Ph.D, executive vice president of research and development at Avidity Biosciences LLC, a biotechnology company advancing Antibody- Oligonucleotide Conjugates (AOCs) as a new class of precision medicines, today published a review titled, “Treating Disease at the RNA Level with Oligonucleotides” in the January 3, 2018, issue of the New England Journal of Medicine^[1].

“Although the potential of modulating RNA function to treat diseases and disorders has been long recognized, decades of research have been required to realize the promise of oligonucleotide-based therapeutics,” said Dr. Levin. “With these advances in hand, and multiple oligonucleotide therapeutics now marketed and more advancing toward FDA approval, this is an important time to assess the landscape and how we can realize the full promise of this therapeutic class.”

In the review, Dr. Levin discusses multiple therapeutic approaches with distinct methods of targeting RNA to treat human health conditions such as hemophilia, amyloidosis, hemostasis, and hyperlipidemias. He evaluates the promise of four therapeutic approaches that have shown distinct advances in recent years: splicing to induce changes in function, exon skipping in Duchene’s muscular dystrophy, exon inclusion in spinal muscular atrophy, and cleavage-based mechanisms for the destruction of mRNA. Dr. Levin discusses in depth the therapeutic advantages, as well as challenges in delivery and safety, for oligonucleotide therapeutic agents. He describes how maturing technologies can help overcome these challenges and create new technologies and methods to advance the field.

Dr. Levin has decades of experience in the field of nucleic acid-based therapeutics and is well respected in the field. Prior to his role at Avidity Biosciences, he held senior drug development roles at miRagen Therapeutics, Ionis Pharmaceuticals and Santaris Pharma. He has played key roles in the development of numerous of oligonucleotides including the first approved antisense NDAs, and the first microRNA-targeted therapeutic in clinical trials. He has a combined three decades of experience in all aspects of drug development from discovery through drug registration, both in large pharma and smaller biotech companies. Dr. Levin has published over 60 scientific articles and several of the most cited reviews in the field. He served as a director of the Oligonucleotide Therapeutics Society and holds several additional scientific organization affiliations and honors. He received a doctorate in toxicology from the University of Rochester and a bachelor’s degree in biology from Muhlenberg College.

About Avidity Biosciences

Avidity Biosciences is a privately held biotech company pioneering Antibody Oligonucleotide Conjugates (AOC™). AOCs combine the tissue selectivity of monoclonal antibodies and the precision of oligonucleotide-based therapeutics to overcome barriers to the delivery of oligonucleotides and target genetic drivers of disease. Avidity is advancing a pipeline of therapeutic programs focused on rare muscle disorders and other serious diseases. Avidity has raised $30 million in venture financing from a top-tier group of sophisticated healthcare investors. More information about Avidity can be found on the company’s website at www.aviditybiosciences.com.

Media Contact

Canale Communications Inc.
Ian Stone
(619) 849-5388
ian@canalecomm.com

[1]  Levin, A, Treating Disease at the RNA Level with Oligonucleotides. N Engl J Med 2019;380: 57-70.

Investment advances pre-clinical development of novel exon skipping therapies for treatment of patients with Duchenne muscular dystrophy

LA JOLLA, Calif. (October 17, 2018) — Avidity Biosciences, a privately held biotech company pioneering a new class of precision medicines based upon antibody-oligonucleotide conjugates, announced today that CureDuchenne has made an equity investment in the company. Funding will help to advance pre-clinical development of potential therapies to treat patients with Duchenne muscular dystrophy (DMD).

“We are grateful for an investment from CureDuchenne Ventures to advance our antibody-oligonucleotide technology for treatment of patients with Duchenne muscular dystrophy,” said Arthur A. Levin, Ph.D., EVP of research and development, Avidity Biosciences. “Cure Duchenne has a history of supporting groundbreaking research in Duchenne, and we look forward to working with them, as well as their community of patients, parents, scientists and scientific experts to advance our pipeline of new therapies for boys with this serious disease.”

The AOC™ technology being advanced by Avidity addresses a key issue of oligonucleotide therapeutics for Duchenne: namely, delivery of these powerful therapeutic agents for exon skipping to muscle, diaphragm and heart. Using antibody-mediated uptake in muscle cells, AOCs increase the activity of splice skipping oligonucleotides dramatically (a 100-fold increase in potency in the MDX mouse model), which should allow for reduced dose levels and reduced dosing frequency. This ground-breaking science provides a great opportunity to address Duchenne muscular dystrophy, and other muscle diseases.

“CureDuchenne is excited about the future of Avidity’s science and committed to supporting Exon skipping development to treat the entire Duchene population,“ said Debra Miller, founder and CEO, CureDuchenne.  “Currently only 13% of Duchenne patients are served with an approved treatment for Exon 51.   Avidity’s programs have the potential to enable effective therapies to treat a broad set of Duchenne patients. We hope it won’t be long before every Duchenne patient has a viable therapy.”

Avidity will present its Duchenne specific research during a webinar hosted by CureDuchenne on November 16, 2018 at 1:00 PT/4:00 ET.   To register for the webinar, please click here:  https://bit.ly/2OryjSI.

About Avidity Biosciences

Avidity Biosciences is a privately held biotech company pioneering Antibody Oligonucleotide Conjugates (AOC™), which combine the tissue selectivity of monoclonal antibodies and the precision of oligonucleotide-based therapeutics to overcome barriers to the delivery of oligonucleotides and target genetic drivers of disease. Avidity is advancing a pipeline of therapeutic programs focused on rare muscle disorders and other serious diseases. Avidity has raised $30 million in venture financing from a top-tier group of sophisticated healthcare investors. More information about Avidity can be found on the company’s website at www.aviditybio.com.

About CureDuchenne

CureDuchenne is the nation’s leading nonprofit organization dedicated to finding a cure for Duchenne, the most common and lethal form of muscular dystrophy. As the leading genetic killer of young boys, Duchenne affects more than 300,000 boys living today. CureDuchenne has garnered international attention for its efforts to raise funds and awareness for Duchenne through venture philanthropy. For more information on how to help raise awareness and funds needed for research, please visit www.cureduchenne.org.

For more details about CureDuchenne’s philanthropic investments, go to: https://www.cureduchenne.org/ventures/.

CORPORATE COMMUNICATIONS CONTACT:
Ian Stone
Vice President
Canale Communications Inc.
(619) 849-5388
ian@canalecomm.com

LA JOLLA, CA — January 5, 2017 – Avidity Biosciences today announced the completion of a $16 million Series B financing round to support the development of its Antibody-siRNA Conjugate (ASC) platform. The Series B round includes investment of $10 million in new capital and conversion of $6 million in convertible debt.  Takeda Pharmaceuticals, through its venture group, led the Series B round, and both new and existing investors participated, including Alethea Capital, Alexandria Real Estate Equities, Brace Pharma, EcoR1 Capital, F-Prime Capital, Moore Venture Partners and Tavistock Life Sciences.

“Our ASC platform unites two of the most impactful innovations over the past twenty years – monoclonal antibodies and oligonucleotides – to create a new class of precision medicines,” said Troy Wilson, Ph.D., J.D., president and chief executive officer of Avidity Biosciences. “Although siRNA-based therapeutics have demonstrated significant clinical and commercial promise, conventional approaches are limited to targeting diseases of the liver. Because ASCs use antibodies to overcome barriers of delivering siRNA, they have the potential to impact a broader range of therapeutic areas. Our goal is to partner with leading pharmaceutical and biotechnology companies to deliver a pipeline of ASCs targeting genetic drivers of disease.”

ASCs link a monoclonal antibody—designed against a specific molecular target—with a siRNA therapeutic payload, allowing the conjugate to have unmatched specificity and selectivity. In preclinical models, ASCs have shown potential to knockdown messenger RNA levels in multiple important cell types and tissues, including tumor, muscle, heart, lung, liver and B cells. In addition, ASCs have drug-like properties comparable to antibodies and antibody-drug conjugates.

In connection with the financing, Avidity Biosciences annouced that Michael Martin, Ph.D., global head of Takeda Ventures Inc., and Todd Brady, director of finance and investments of Brace Pharma Capital, will join its board of directors. Tony Hsu, founder and chief investment officer of Alethea Capital, will also join the board as a non-voting member.

“We believe Avidity’s ASC platform offers a compelling approach that builds on successes with antibodies, ADCs and oligonucleotide-based therapeutics,” said Dr. Martin. “Avidity has recruited a top-notch team and  made significant progress against its scientific and business goals.  We look forward to working with the company to realize the promise of ASCs as a new class of precision medicines.”

About Avidity Biosciences

Avidity Biosciences is a privately held biotech company pioneering a new class of precision medicines – Antibody-siRNA Conjugates (ASC™) – which combine the strengths of monoclonal antibodies and siRNA-based therapeutics. Avidity Biosciences is working with partners to discover best-in-class drug candidates against important undrugged therapeutic targets.  Avidity has entered research collaborations with leading biopharma companies and are actively seeking additional partnerships. Avidity has raised $25 million in venture financing from a top-tier group of sophisticated healthcare investors.  More information about Avidity can be found on the company’s website at www.aviditybiosciences.com.

CORPORATE COMMUNICATIONS CONTACT:
Ian Stone
Vice President
Canale Communications Inc.
(619) 849-5388
ian@canalecomm.com

LA JOLLA, CA — January 5, 2017 – Avidity Biosciences LLC, a biotechnology company advancing antibody-siRNA conjugates (ASCs) as a new class of precision medicines, today announced a publication entitled, “Targeting Therapeutic Oligonucleotides” in the most recent issue of the New England Journal of Medicine.¹  The paper was authored by Arthur A. Levin, Ph.D., Executive Vice President of Research and Development at Avidity.

Dr. Levin summarized the advantages of targeted strategies for the delivery of therapeutic oligonucleotides.  “Recent work indicates that the usefulness of the technology can be increased by conjugating oligonucleotides with targeting moieties so that they home to specific cell types,” said Dr. Levin, citing examples of enhanced delivery of antisense oligonucleotides (ASO) and small interfering ribonucleotides (siRNAs) conjugated to triantennary N-acetylgalactosamine (GalNAc) moieties. Citing enhanced potency and potentially lower toxicity of conjugates, Dr. Levin states, “targeted delivery was determined to result in a median effective dose that was one thirtieth of that associated with nontargeted delivery, which clearly underscored the potential advantage of the approach.”  Moreover, he suggests the potential to move beyond targeting hepatic tissue to oligonucleotide delivery in other cell types: “there remains a need to target nonhepatic cells specifically and efficiently: a means to achieve this goal could lie in the exploitation of the natural heterogeneity of cell surface receptors.”

The article appeared in the Clinical Implications of Basic Research section of the January 5, 2017 issue of the New England Journal of Medicine.

Arthur A. Levin has an unparalleled track record and reputation in the field of nucleic acid-based therapeutics. Prior to his role at Avidity Biosciences, he held senior drug development roles at miRagen Therapeutics, Ionis Pharmaceuticals and Santaris Pharma. He has played key roles in the development of numerous of oligonucleotides including the first approved antisense NDAs, and the first microRNA-taregeted therapeutic in clinical trials. He has a combined three decades of experience in all aspects of drug development from discovery through drug registration, both in large pharma and biotech companies. Dr. Levin has published over 60 scientific articles and several of the most cited reviews in the field. He serves as a director of the Oligonucleotide Therapeutics Society and holds several additional scientific organization affiliations and honors. He received a doctorate in toxicology from the University of Rochester, and a bachelor’s degree in biology from Muhlenberg College.

About Avidity Biosciences

Avidity Biosciences is a privately held biotech company pioneering a new class of precision medicines – antibody-siRNA conjugates (ASCs^™) – which combine the strengths of monoclonal antibodies and siRNA-based therapeutics. Avidity is collaborating with partners to discover and develop best-in-class drug candidates against important undrugged therapeutic targets. The company has entered research collaborations with leading biopharma companies and is actively seeking additional partnerships. Avidity has raised $25 million in venture financing from a top-tier group of sophisticated healthcare investors.  More information about Avidity Biosciences can be found on the company’s website at www.aviditybiosciences.com.

¹  Levin, A, Targeting therapeutic oligonucleotides. N Engl J Med 2016;376: 86-88.