Kriya Expands Gene Therapy Pipeline and Establishes its Rare Disease Therapeutic Area Division with the Acquisition of Warden Bio

On January 7, 2022 Kriya Therapeutics, Inc., a fully integrated company pioneering novel technologies and therapeutics in gene therapy, reported that it has acquired Warden Bio, a company developing novel AAV-mediated gene therapies for glycogen storage disorders (GSDs) (Press release, Kriya Therapeutics, JAN 7, 2022, View Source [SID1234598419]). Under the agreement, Kriya has obtained exclusive rights to Warden Bio’s five preclinical gene therapy programs. This acquisition serves as the foundation for Kriya’s Rare Disease Division focused on the discovery and development of gene therapies for rare diseases.

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GSDs are a group of rare disorders caused by distinct single enzyme deficiencies involved in the synthesis or breakdown of glycogen. Warden Bio was established with a goal of comprehensively addressing multiple GSDs without approved therapies based on pioneering gene therapy technologies originally developed at Duke University. Currently, approved therapies are only available for one type of GSD, Pompe disease (GSD II).

"We are incredibly enthusiastic about the compelling research that has led to the development of Warden Bio’s pioneering gene therapies for GSDs," said Shankar Ramaswamy, M.D., Co-Founder and Chief Executive Officer of Kriya. "This acquisition and the establishment of our Rare Disease Division align with our commitment to accelerating the advancement of best-in-class gene therapies. Kriya’s technology and manufacturing platforms uniquely position our company to drive meaningful progress to address a group of diseases in desperate need of new and better treatments."

Together with the acquisition, Warden Bio co-founder Kunal Kishnani is joining Kriya as President of its Rare Disease Division, where he will lead overall strategic, development, and partnership activities. Prior to founding Warden Bio, Mr. Kishnani was a member of the corporate development team at Roivant Sciences, where he was responsible for helping to evaluate and negotiate new investment opportunities and strategic alliances. Mr. Kishnani also previously served as Co-Founder and Head of Operations at Genevant, where he was instrumental in driving key milestones for the company, including its launch, establishment of business operations, and execution of multiple collaborations with major pharmaceutical companies. Mr. Kishnani has degrees in finance and accounting from Elon University.

"I am excited to join Kriya and help advance its mission of expanding the reach of gene therapy to new frontiers," said Mr. Kishnani. "Despite recent advancements in the rare disease field, many conditions remain significantly undertreated. There is an urgent need to address rare diseases such as GSDs, and we are committed to making the treatment of these patients our priority – both previously at Warden Bio and now at Kriya."

The new GSD gene therapy programs Warden Bio brings to Kriya are based on research led by the lab of Dr. Priya Kishnani, Chen Family Distinguished Professor & Division Chief for Medical Genetics at Duke University, in close collaboration with Dr. Aravind Asokan, Professor & Director of Gene Therapy at Duke University and Co-Founder of StrideBio. Over the past three decades, Dr. Kishnani has built an interdisciplinary team with world-leading GSD expertise whose efforts are most notably demonstrated by the long-standing research and clinical experience that resulted in FDA approval of alglucosidase alfa (Myozyme/Lumizyme) as the first available treatments for Pompe disease. Dr. Kishnani and Dr. Asokan will become Scientific Advisors to Kriya as the company advances its portfolio of gene therapies for GSDs and other diseases.

Cytokinetics and Royalty Pharma Announce Funding Agreements Totaling up to $450 Million

On January 7, 2022 Cytokinetics, Incorporated (Nasdaq: CYTK) and Royalty Pharma plc (Nasdaq: RPRX) reported that Cytokinetics has secured long-term capital from Royalty Pharma to support the potential commercialization of omecamtiv mecarbil and the further development of aficamten (Press release, Cytokinetics, JAN 7, 2022, View Source [SID1234598418]). In addition, Cytokinetics sold to Royalty Pharma royalties on future worldwide sales of aficamten .

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Omecamtiv mecarbil is an investigational, selective, small molecule cardiac myosin activator, that was granted Fast Track Designation by the U.S. Food and Drug Administration (FDA) and was the subject of GALACTIC-HF, a positive Phase 3 clinical trial in patients with heart failure with reduced ejection fraction. Aficamten is an investigational selective, small molecule cardiac myosin inhibitor that is under investigation for hypertrophic cardiomyopathy and was granted Breakthrough Therapy Designation by the U.S. FDA.

"These corporate development transactions continue our history of monetizing our leadership in muscle biology through creative deal making and provide further optionality, capital efficiencies and financial resources to expand our cardiovascular development and commercialization programs," said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. "We are pleased to enter into these additional transactions with Royalty Pharma, underscoring our shared commitment to build an industry leading cardiovascular franchise in cardiac myosin modulation."

"We are pleased to expand our partnership with Cytokinetics," said Pablo Legorreta, Royalty Pharma’s Founder and Chief Executive Officer. "Royalty Pharma is excited to support the company at this important stage of transformation towards a commercial organization with an exciting late stage pipeline addressing unmet medical needs in multiple cardiovascular diseases."

Royalty Pharma will provide Cytokinetics long-term capital of up to $300 million to support the potential commercialization of omecamtiv mecarbil and the further development of aficamten, and other general corporate purposes. The capital is available in five tranches, including an initial tranche of $50 million upon closing and four additional tranches in the aggregate amount of $250 million upon the occurrence of certain regulatory and clinical development milestones related to omecamtiv mecarbil and aficamten. Each tranche has an interest-free and payment-free period of six calendar quarters, followed by 34 calendar quarters of installment re-payments totaling 1.9 times the amount drawn.

In addition, Royalty Pharma has purchased from Cytokinetics a royalty on aficamten of 4.5% on sales up to $1 billion and 3.5% on sales above $1 billion, subject to certain potential step-downs, in exchange for payments of up to $150 million, comprised of $50 million at closing and two additional $50 million payments, conditional upon the initiation of potential pivotal clinical trials for oHCM and nHCM, respectively.

From these transactions, Cytokinetics anticipates receipt of up to $150 million in near-term funding. Together with its proforma cash at the end of 2021 which was bolstered by transactions executed by Cytokinetics last year, this funding from Royalty Pharma enables Cytokinetics to have at least two years of cash runway based on expected 2022 expenditures, inclusive of planned commercialization activities and expanded pipeline development programs.

Cooley LLP and Morrison Foerster acted as legal advisors to Cytokinetics on the transactions. Goodwin Procter, Fenwick & West LLP, Dechert and Maiwald acted as legal advisors to Royalty Pharma.

Conference Call and Webcast Information

Members of Cytokinetics’ senior management team will host a conference call and webcast today at 8:30 AM Eastern Time. The webcast can be accessed through the Investors & Media section of the Cytokinetics website at www.cytokinetics.com. The live audio of the conference call can also be accessed by telephone by dialing either (866) 999-CYTK (2985) (United States and Canada) or (706) 679-3078 (international) and typing in the passcode 5478696.

An archived replay of the webcast will be available via Cytokinetics’ website until January 21, 2022. The replay will also be available via telephone by dialing (855) 859-2056 (United States and Canada) or (404) 537-3406 (international) and typing in the passcode 5478696 from January 7, 2022 at 11:30 AM Eastern Time until January 21, 2022.

About Omecamtiv Mecarbil

Omecamtiv mecarbil is an investigational, selective, small molecule cardiac myosin activator, the first of a novel class of myotropes1 designed to directly target the contractile mechanisms of the heart, binding to and recruiting more cardiac myosin heads to interact with actin during systole. Omecamtiv mecarbil is designed to increase the number of active actin-myosin cross bridges during each cardiac cycle and consequently augment the impaired contractility that is associated with heart failure with reduced ejection fraction (HFrEF). Preclinical research has shown that omecamtiv mecarbil increases cardiac contractility without increasing intracellular myocyte calcium concentrations or myocardial oxygen consumption.2-4

The development program for omecamtiv mecarbil is assessing its potential for the treatment of HFrEF. Positive results from GALACTIC-HF, the first Phase 3 clinical trial of omecamtiv mecarbil demonstrated a statistically significant effect of treatment with omecamtiv mecarbil to reduce risk of the primary composite endpoint of cardiovascular (CV) death or heart failure events (heart failure hospitalization and other urgent treatment for heart failure) compared to placebo in patients treated with standard of care. No reduction in the secondary endpoint of time to CV death was observed. Adverse events and treatment discontinuation of study drug were balanced between treatment arms. METEORIC-HF, a second Phase 3 clinical trial of omecamtiv mecarbil is designed to evaluate the effect of treatment with omecamtiv mecarbil compared to placebo on exercise capacity. Results from METEORIC-HF are expected in early 2022.

About Aficamten

Aficamten is an investigational selective, small molecule cardiac myosin inhibitor discovered following an extensive chemical optimization program that was conducted with careful attention to therapeutic index and pharmacokinetic properties and as may translate into next-in-class potential in clinical development. Aficamten was designed to reduce the number of active actin-myosin cross bridges during each cardiac cycle and consequently suppress the myocardial hypercontractility that is associated with hypertrophic cardiomyopathy (HCM). In preclinical models, aficamten reduced myocardial contractility by binding directly to cardiac myosin at a distinct and selective allosteric binding site, thereby preventing myosin from entering a force producing state.

The development program for aficamten is assessing its potential as a treatment that improves exercise capacity and relieves symptoms in patients with HCM as well as its long-term effects on cardiac structure and function. Cytokinetics is currently conducting start-up activities for SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM), the Phase 3 clinical trial of aficamten in patients with symptomatic obstructive HCM. The company is also planning for an expanded development program that may evaluate aficamten in indications such as non-obstructive HCM and heart failure with preserved ejection fraction (HFpEF).

Monopar to Present at the H.C. Wainwright BioConnect Conference

On January 7, 2022 Monopar Therapeutics Inc. (Nasdaq: MNPR), a clinical-stage biopharmaceutical company focused on developing proprietary therapeutics designed to extend life or improve the quality of life for cancer patients, reported that Chandler Robinson, MD, Monopar’s Chief Executive Officer, will present at the H.C. Wainwright BioConnect Conference (Press release, Monopar Therapeutics, JAN 7, 2022, https://ir.monopartx.com/news/detail/48/monopar-to-present-at-the-h-c-wainwright-bioconnect-conference [SID1234598417]). The presentation will be available for on-demand viewing starting on Monday, January 10, 2022 at 7:00am ET.

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Presentation Details:

Date: Monday, January 10, 2022

Time: 7:00am ET

Location: Virtual

View Source

Plus Therapeutics to Participate in Upcoming January Conferences

On January 7, 2022 Plus Therapeutics, Inc. (Nasdaq: PSTV) (the "Company"), a clinical-stage pharmaceutical company developing innovative, targeted radiotherapeutics for rare and difficult-to-treat cancers, reported that Marc H. Hedrick M.D., President and Chief Executive Officer of Plus Therapeutics, will participate at two virtual conferences in January (Press release, Cytori Therapeutics, JAN 7, 2022, View Source [SID1234598416]).

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Event H.C. Wainwright BioConnect Conference
Date January 10-13, 2022
Time Presentation available on demand starting January 10 at 7:00 a.m. ET

Event Biotech Showcase 2022
Date January 10-12 & 17-19, 2022
Time Presentation available on demand
A webcast of the H.C. Wainwright presentation will be available under the ‘Events’ tab of the Investor Relations section of the Plus Therapeutics website at www.plustherapeutics.com. A webcast replay will be accessible for 90 days following the event.

Investors interested in arranging a virtual meeting with the Company’s management during the Biotech Showcase 2022 conference should contact the conference coordinator. The Company’s Biotech Showcase presentation can be accessed on demand to qualified investors through a complimentary registration on the conference website here: Biotech Showcase Registration.

PureTech to Present at 40th Annual J.P. Morgan Healthcare Conference

On January 7, 2022 PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) ("PureTech" or the "Company"), a clinical-stage biotherapeutics company dedicated to discovering, developing and commercializing highly differentiated medicines for devastating diseases, reported that Daphne Zohar, Founder and Chief Executive Officer, will present at the 40th Annual J.P. Morgan Healthcare Conference on Wednesday, January 12, 2022, at 7:30am EST (Press release, PureTech Health, JAN 7, 2022, View Source [SID1234598415]). A webcast of the presentation will be available at View Source

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