Market Newsdesk

WOBURN, Mass., May 05, 2021 (GLOBE NEWSWIRE) — Replimune Group, Inc. (Nasdaq: REPL), a biotechnology company developing oncolytic immuno-gene therapies derived from its Immulytic® platform, today announced a clinical development update for CERPASS, its registration-directed global randomized Phase 2 clinical trial of RP1 in combination with Libtayo® (cemiplimab) versus Libtayo for the treatment of cutaneous squamous cell carcinoma (CSCC), and for IGNYTE, its registration-directed single arm clinical trial in combination with Opdivo® (nivolumab) for the treatment of anti-PD1 failed melanoma.

CERPASS

“Based on the depth and durability of responses and the manageable safety profile we have seen in patients with non-melanoma skin cancers treated with RP1 in combination with Opdivo to date, we are amending the clinical trial protocol for our Phase 2 CERPASS clinical trial to include complete response (CR) rate as a primary endpoint in addition to overall response rate (ORR), and to reduce target enrollment to 180 patients,” said Robert Coffin, Ph.D., President and Chief Research and Development Officer of Replimune. “Incorporating CR as an independent additional primary endpoint should ensure a robust assessment of the clinical meaningfulness of adding RP1 to Libtayo in the primary data analysis upon which a BLA submission and FDA’s assessment for approval will be based.”

Under the modified clinical trial protocol for CERPASS, Replimune plans to add CR rate as an additional independent primary endpoint, in addition to ORR, and to reduce target enrollment from 240 patients to 180 patients. Secondary endpoints will continue to include duration of response, progression-free survival (PFS), and overall survival (OS). Replimune is maintaining its guidance to expect initial data in 2022.

Replimune plans to submit the amended CERPASS clinical trial protocol to the U.S. Food and Drug Administration (FDA) in the first half of the year.

Libtayo® is a registered trademark of Regeneron.

IGNYTE

Replimune recently held a Type B meeting with the FDA to discuss the design of the 125-patient, single-arm cohort in the IGNYTE clinical trial which is currently enrolling patients with anti-PD1 failed cutaneous melanoma, with registrational intent. The FDA expressed that while a randomized controlled trial would always be preferred to support licensure, that in this population if the data generated were sufficiently compelling, then the data could be considered for submission by the FDA under the accelerated approval pathway. The FDA also confirmed that a confirmatory trial would be needed as is required under the accelerated approval pathway. The design of the confirmatory trial is intended to be discussed with the FDA prior to a Biologics License Application (BLA) submission.

Dr. Coffin commented: “The discussion with the FDA at the Type B meeting provides clarity on the appropriate regulatory pathway to potential approval for RP1 in combination with Opdivo in anti-PD1 failed melanoma based on the data intended to be generated from our ongoing clinical trial. We look forward to releasing initial data from this study during 2022.”

Opdivo® is a registered trademark of Bristol Myers Squibb.

About CERPASS

CERPASS is Replimune’s registration-directed randomized, global Phase 2 clinical study to compare the effects of Libtayo alone versus a combination of Libtayo and Replimune’s investigational oncolytic immunotherapy RP1. The clinical trial will enroll 180 patients with locally advanced or metastatic cutaneous squamous cell carcinoma (CSCC) who are naïve to anti-PD1 therapy. The trial will evaluate complete response (CR) rate and objective response rate (ORR) as its two primary efficacy endpoints as assessed by independent review, as well as duration of response, progression-free survival (PFS), and overall survival (OS) as its secondary endpoints. The study is being run under a clinical trial collaboration agreement with Regeneron in which the costs of the trial are shared and full commercial rights retained by Replimune. Libtayo is being jointly developed by Regeneron and Sanofi.

About IGNYTE

IGNYTE is Replimune’s multi-cohort Phase 1/2 trial of RP1 plus Opdivo®. There are 4 tumor specific cohorts currently enrolling in this trial including a 125-patient extension cohort of RP1 combined with Opdivo in anti-PD-1 failed cutaneous melanoma. This cohort was initiated after completing enrollment in a prior Phase 2 cohort in the same trial of approximately 30 patients with melanoma. The additional thirty patient cohorts are studying RP1 in combination with Opdivo in non-melanoma skin cancers which includes both naïve and anti-PD-1 failed CSCC, in microsatellite instability high, or MSI-H/dMMR tumor types and anti-PD-1 failed non-small cell lung cancer, or NSCLC. This trial is being done under a collaboration and supply agreement with Bristol Myer Squibb.

About RP1

RP1 is Replimune’s lead Immulytic® product candidate and is based on a proprietary new strain of herpes simplex virus engineered to maximize tumor killing potency, the immunogenicity of tumor cell death and the activation of a systemic anti-tumor immune response through the expression of a GALV-GP R- fusogenic protein and GM-CSF.

About Replimune

Replimune Group, Inc., headquartered in Woburn, MA, was founded in 2015 to develop the next generation of oncolytic immune-gene therapies for the treatment of cancer. Replimune is developing novel, proprietary therapeutics intended to improve the direct cancer-killing effects of selective virus replication and the potency of the immune response to the tumor antigens released. Replimune’s Immulytic® platform is designed to maximize systemic immune activation, in particular to tumor neoantigens, through robust viral-mediated immunogenic tumor cell killing and the delivery of optimal combinations of immune-activating proteins to the tumor and draining lymph nodes. The approach is expected to be highly synergistic with immune checkpoint blockade and other approaches to cancer treatment across a broad range of cancers. Replimune intends to progress these therapies rapidly through clinical development in combination with other immuno-oncology products with complementary mechanisms of action as well as in standalone indications. For more information, please visit www.replimune.com.

Forward Looking Statements

This press release contains forward looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, including statements regarding our expectations about the design and advancement of our clinical trials, the sufficiency of our clinical trial outcomes to support potential approval of any of our product candidates, our goals to develop and commercialize our product candidates, patient enrollments in our existing and planned clinical trials and the timing thereof, the potential impact of COVID-19 on our operations and milestones, and other statements identified by words such as “could,” “expects,” “intends,” “may,” “plans,” “potential,” “should,” “will,” “would,” or similar expressions and the negatives of those terms. Forward-looking statements are not promises or guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond our control, and which could cause actual results to differ materially from those contemplated in such forward-looking statements. These factors include risks related to our limited operating history, our ability to generate positive clinical trial results for our product candidates, the costs and timing of operating our in-house manufacturing facility, the timing and scope of regulatory approvals, changes in laws and regulations to which we are subject, competitive pressures, our ability to identify additional product candidates, political and global macro factors including the impact of the coronavirus as a global pandemic and related public health issues, and other risks as may be detailed from time to time in our Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q and other reports we file with the Securities and Exchange Commission. Our actual results could differ materially from the results described in or implied by such forward-looking statements. Forward-looking statements speak only as of the date hereof, and, except as required by law, we undertake no obligation to update or revise these forward-looking statements.

Investor Inquiries

Chris Brinzey
Westwicke, an ICR Company
339.970.2843
[email protected]

Media Inquiries

Lissette Steele
Verge Scientific Communications
202.431.4116
[email protected]

 

• Dosed first patient with infantile GM1 gangliosidosis in Imagine-1 Phase 1/2 trial of PBGM01, initial safety and 30-day biomarker data expected 4Q21
• Received multiple regulatory clearances for clinical trial initiations for three most advanced pipeline programs
• Expected enrollment of first patient in Phase 1/2 FTD-GRN trial in 2Q/3Q21 and first patient in Phase 1/2 Krabbe trial in 3Q21
• Continued to advance pipeline under leadership of newly appointed Chief R&D Officer Eliseo Salinas, M.D., MSc.
• Appointed Maxine Gowen, Ph.D., an experienced biotech leader, to Passage Bio Board of Directors
• Raised $166 million, further strengthening company’s cash position
• Management to host conference call today at 8:30 a.m. ET
  

PHILADELPHIA, May 05, 2021 (GLOBE NEWSWIRE) — Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today reported financial results for the first quarter ended March 31, 2021 and provided recent business highlights.

“We are particularly proud to have recently dosed our first patient in our global Imagine-1 trial of PBGM01 for infantile GM1 gangliosidosis,” said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio. “This accomplishment speaks to the strength of our collaboration with the University of Pennsylvania’s Gene Therapy Program. By raising an additional $166 million in the first quarter, we are well funded to advance both our clinical- and research-stage programs.

“Our primary focus in 2021 remains advancing and expanding our differentiated pipeline for patients with rare CNS disorders,” Dr. Goldsmith added. “We are pleased to have successfully received a number of clinical trial regulatory approvals in a period of several months for our three most advanced programs. While some impact on clinical site initiations from the Covid-19 pandemic has occurred, which is reflected in adjusted study timelines, we look forward to reporting on several meaningful milestones throughout 2021 as we diligently work toward delivering transformative CNS therapies for patients.”

Recent Highlights:

• First patient dosed in Imagine-1 study of PBGM01 for infantile GM1 gangliosidosis: The company announced in April 2021 that the first patient had been dosed in its Imagine-1 global Phase 1/2 trial of PBGM01 for the treatment of infantile GM1 gangliosidosis (GM1). Imagine-1 is a global open-label study of PBGM01 administered by a single injection into the cisterna magna in pediatric subjects with early and late infantile GM1.

• Several recent regulatory milestones achieved in a period of three months for three most advanced pipeline programs:
  • PBGM01 for GM1 and PBKR03 for Krabbe disease received regulatory clearances for clinical trial initiations from U.S. Food and Drug Administration (FDA), UK MHRA, and Health Canada
  • PBGM01 also received regulatory clearance for clinical trial initiation from the Brazilian Regulatory Health Agency
  • PBFT02 for frontotemporal dementia with granulin mutations (FTD-GRN) received regulatory clearances for clinical trial initiation from FDA and Health Canada
  • The European Commission granted Orphan designation for PBKR03 for the treatment of early infantile Krabbe disease.
• Announced collaboration with InformedDNA, the nation’s leading genetics services organization: The collaboration will provide no-cost genetic counseling and testing for adults who have been diagnosed with FTD. The testing program will facilitate identification of FTD patients with certain inherited genetic mutations as well as support for clinical trial recruitment and enrollment. It is estimated that approximately 5 to 10 percent of FTD is caused by a GRN gene mutation.

• Timelines adjusted for global Phase 1/2 clinical trials for PBGM01, PBFT02 and PBKR03 due primarily to impacts associated with Covid-19: The initial safety and 30-day biomarker data for Imagine-1 for PBGM01 for GM1 is expected to read out in 4Q21. The global clinical trial upliFT-D for PBFT02 for FTD-GRN is expected to initiate in 2Q/3Q 2021 with initial safety and 30-day biomarker data to be reported in 1H22. The global clinical trial GALax-C for PBKR03 for Krabbe disease is expected to initiate in 3Q21 with initial safety and 30-day biomarker data to be reported in 1H22.

• Eliseo O. Salinas, M.D., MSc, appointed as chief research & development officer: In March 2021, the company appointed Dr. Salinas as the company’s chief research and development (R&D) officer. Dr. Salinas has extensive experience in the development of small molecules, biologics and cell therapy and has been directly involved with numerous investigational new drug (IND) application submissions and regulatory approvals in the United States and globally.

• Maxine Gowen, Ph.D., appointed to Passage Bio board of directors: In February 2021, the company announced the appointment of Dr. Gowen to its board of directors. She brings significant public company leadership and clinical development expertise, having co-founded and led a start-up biotech company and served in a variety of leadership roles at GlaxoSmithKline over a period of 15 years.

• Raised $166 million in public offering of common stock: In January 2021, the company announced a public offering of 7,000,000 shares of its common stock at a price of $22 per share. The underwriters also exercised their option to purchase an additional 1,050,000 shares of common stock for total offering net proceeds of $166 million.

Anticipated Upcoming Milestones

• Host R&D day focused on FTD-GRN, May 17.
• Open CMC research and development site in Hopewell, NJ, by the end of the second quarter of 2021.
• Report initial safety and 30-day biomarker data from Phase 1/2 PBGM01 trial in 4Q21.
• Dose first patient in Phase 1/2 FTD-GRN trial in 2Q/3Q21; report initial safety and 30-day biomarker data in 1H22.
• Dose first patient in Phase 1/2 Krabbe trial in 3Q21; report initial safety and 30-day biomarker data in 1H22.
• Continue to advance preclinical programs for PBML04 (Metachromatic leukodystrophy), PBLA05 (Amyotrophic lateral sclerosis) and PBCM06 (Charcot-Marie-Tooth Disease Type 2A), and an undisclosed adult CNS program.

First Quarter 2021 Financial Results

• Cash Position: Cash, cash equivalents and marketable securities were $437.6 million as of March 31, 2021 as compared to $304.8 million as of December 31, 2020. This includes $166 million in net proceeds from the company’s public offering in January 2021.
• Research and Development (R&D) Expenses: R&D expenses were $25.0 million for the first quarter ended March 31, 2021, compared to $13.1 million for the same quarter in 2020. Acquired in-process R&D expenses were $1.5 million for the first quarter ended March 31, 2021, compared to none in the same quarter of 2020.
• General and Administrative (G&A) Expenses: G&A expenses were $12.5 million for the first quarter ended March 31, 2021, compared to $4.8 million for the same quarter in 2020. 
• Net Loss: Net loss was $38.9 million, or a net loss of $0.76 per basic and diluted share, for the quarter ended March 31, 2021 compared to $17.6 million, or a net loss of $1.00 per basic and diluted share, for the same quarter in 2020.

Conference Call Details

Passage Bio will host a conference call and webcast today at 8:30 a.m. ET. To access the live conference call, please dial 833-528-0605 (domestic) or 830-221-9711 (international) and reference conference ID number 6366724. A live audio webcast of the event will be available on the Investors & Media section of Passage Bio’s website at investors.passagebio.com. The archived webcast will be available on Passage Bio’s website approximately two hours after the completion of the event and for 30 days following the call.

About Passage Bio

At Passage Bio (Nasdaq: PASG), we are on a mission to provide life-transforming gene therapies for patients with rare, monogenic CNS diseases that replace their suffering with boundless possibility, all while building lasting relationships with the communities we serve. Based in Philadelphia, PA, our company has established a strategic collaboration and licensing agreement with the renowned University of Pennsylvania’s Gene Therapy Program to conduct our discovery and IND-enabling preclinical work. This provides our team with enhanced access to a broad portfolio of gene therapy candidates and future gene therapy innovations that we then pair with our deep clinical, regulatory, manufacturing and commercial expertise to rapidly advance our robust pipeline of optimized gene therapies into clinical testing. As we work with speed and tenacity, we are always mindful of patients who may be able to benefit from our therapies. More information is available at www.passagebio.com.

Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about timing and execution of anticipated milestones, including initiation of clinical trials and the availability of clinical data from such trials; our expectations about our collaborators’ and partners’ ability to execute key initiatives; our expectations about manufacturing plans and strategies; our expectations about cash runway; and the ability of our lead product candidates to treat their respective target monogenic CNS disorders. These forward-looking statements may be accompanied by such words as “aim,” “anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,” “might,” “plan,” “potential,” “possible,” “will,” “would,” and other words and terms of similar meaning. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop and obtain regulatory approval for our product candidates; the timing and results of preclinical studies and clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; the risk that positive results in a preclinical study or clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; failure to protect and enforce our intellectual property, and other proprietary rights; our dependence on collaborators and other third parties for the development and manufacture of product candidates and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; and the other risks and uncertainties that are described in the Risk Factors section in documents the company files from time to time with the Securities and Exchange Commission (SEC), and other reports as filed with the SEC. Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

For further information, please contact:

Investors:

Stuart Henderson
Passage Bio
267.866.0114
[email protected] 

Media:

Gwen Fisher
Passage Bio
215.407.1548
[email protected]

Passage Bio, Inc.

Balance Sheets

(Unaudited)

(in thousands, except share data)		March 31, 2021				December 31, 2020
Assets
Current assets:
Cash and cash equivalents		$	249,521			$	135,002
Marketable securities			188,068				169,815
Prepaid expenses			3,374				1,405
Prepaid research and development			9,986				10,961
Total current assets			450,949				317,183
Property and equipment, net			6,574				2,795
Other assets			7,649				8,029
Total assets		$	465,172			$	328,007
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable		$	6,248			$	5,265
Accrued expenses and other current liabilities			13,221				15,910
Total current liabilities			19,469				21,175
Deferred rent			4,199				2,077
Other liabilities			—				41
Total liabilities			23,668				23,293
Commitments and Contingencies
Stockholders’ equity:
Common stock, $0.0001 par value: 300,000,000 shares authorized; 53,977,484 shares issued and 53,848,324 shares outstanding at March 31, 2021 and 45,917,084 shares issued and 45,614,807 shares outstanding at December 31, 2020			5				4
Additional paid‑in capital			651,283				475,617
Accumulated other comprehensive loss			(7	)			(12	)
Accumulated deficit			(209,777	)			(170,895	)
Total stockholders’ equity			441,504				304,714
Total liabilities and stockholders’ equity		$	465,172			$	328,007

.

Passage Bio, Inc.

Statements of Operations and Comprehensive Loss

(Unaudited)

		Three Months Ended March 31,
(in thousands, except share and per share data)		2021				2020
Operating expenses:
Research and development		$	24,970			$	13,117
Acquired in‑process research and development			1,500				—
General and administrative			12,464				4,795
Loss from operations			(38,934	)			(17,912	)
Interest income, net			52				327
Net loss		$	(38,882	)		$	(17,585	)
Per share information:
Net loss per share of common stock, basic and diluted		$	(0.76	)		$	(1.00	)
Weighted average common shares outstanding, basic and diluted			51,331,449				17,624,011
Comprehensive loss:
Net loss		$	(38,882	)		$	(17,585	)
Unrealized gain on marketable securities			5				—
Comprehensive loss		$	(38,877	)		$	(17,585	)

 

Strengthening Leadership by Adding Mark S. Shearman, Ph.D., as Chief Scientific Officer

Advancing BRILLIANCE trial of EDIT-101 for LCA10; clinical data expected by year-end

RUBY trial of EDIT-301 for sickle cell disease active and recruiting

Preclinical ocular data presented at ARVO supports in vivo gene editing

Cash, cash equivalents, and marketable securities of
$723 million
as of March 31, 2021

CAMBRIDGE, Mass., May 05, 2021 (GLOBE NEWSWIRE) — Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today reported business highlights and financial results for the first quarter of 2021.

“Our team is making tremendous progress towards discovering, developing, and manufacturing novel genome editing medicines, including excellent forward momentum this quarter for our two clinical-stage medicines,” said James C. Mullen, Chairman, President and Chief Executive Officer, Editas Medicine. “We are pleased to announce Dr. Mark Shearman will be joining Editas as Chief Scientific Officer in June 2021. Mark has an outstanding track record of drug discovery and development, and we are confident that his expertise will help us achieve our near-term milestones and continue to realize the promise of bringing transformative medicines to patients.”

Mr. Mullen continued, “In ocular, we advanced the BRILLIANCE trial of EDIT-101 for LCA10 and have line of sight for initiating the next cohort. We also presented preclinical data at ARVO that further validates our in vivo gene editing platform in USH2A and RP4. In ex vivo, we advanced the RUBY trial of EDIT-301 for sickle cell disease, including activating sites for recruitment and finalizing preparations to produce study drug material for dosing patients in the trial. We look forward to maintaining the momentum through the remainder of the year and expect to share Editas’ first clinical data for EDIT-101 by year-end.”

Recent Achievements and Outlook

In Vivo

 Gene Edited Medicines 

• EDIT-101 for Leber Congenital Amaurosis 10 (LCA10)
  
  Initial clinical data expected by year-end
  Editas Medicine is progressing the Phase 1/2 BRILLIANCE trial for the treatment of LCA10. The Company is continuing to dose patients in the second cohort, the adult mid-dose, and results of this cohort will inform the path to initiate dosing of pediatric patients. Clinical data from the first two cohorts is expected to be presented by the end of 2021.

• Other Ocular Programs
  
  Preclinical data for USH2A and RP4 presented at ARVO
  Editas Medicine presented preclinical data at the Association for Research in Vision and Ophthalmology (ARVO) 2021 Annual Meeting for Usher Syndrome 2A (USH2A) and retinitis pigmentosa 4 (RP4). For USH2A, the Company demonstrated that gene editing restored protein expression and rescued deficits in photoreceptor morphology in human retinal organoids. For RP4, the Company demonstrated that a dual adeno-associated virus editing system, which simultaneously knocks out and replaces the rhodopsin mutation of RP4, produced clinically relevant editing levels with no off-target editing. The Company expects to declare a development candidate for its RP4 program by the end of 2021.
  

Ex Vivo

Gene Edited Medicines

• EDIT-301 for Sickle Cell Disease
  
  On track to dose first patient in RUBY trial by year-end
  Editas Medicine is developing EDIT-301 using Cas12a (Cpf1), a proprietary enzyme, as a potentially best-in-class medicine to treat sickle cell disease. The Phase 1/2 RUBY trial for the treatment of sickle cell disease is active and recruitment of patients has started. The Company remains on track to begin patient dosing in the RUBY trial by the end of 2021.

• EDIT-301 for Beta-Thalassemia
  
  On track to file IND by year-end
  Editas Medicine is confirming the sustained effects of EDIT-301 on cells derived from beta-thalassemia patients. The Company remains on track to file an investigational new drug application for EDIT-301 for the treatment of beta-thalassemia by the end of 2021.

Cellular Therapy

• Edited iPSC NK (iNK) Cell Medicines to Treat Solid Tumors
  
  Preclinical data presented at AACR
  Editas Medicine presented preclinical data at the American Association for Cancer Research (AACR) 2021 Annual Meeting for its iNK program to treat solid tumors. The data showed that iPSC-derived natural killer (NK) cells, edited with CRISPR/Cas12a to knockout CISH and TGFβR2, demonstrated superior cytotoxicity and enhanced metabolic function in a tumor microenvironment, as well as improved serial killing capacity of certain tumors. These data confirm the expectation of the CISH and TGFβR2 double knockout as a potential superior treatment for solid tumors.

• Alpha-Beta T Cells for Oncology
  
  Bristol Myers Squibb opting into additional program
  Editas Medicine and Bristol Myers Squibb continue to advance alpha-beta T cell medicines for the treatment of solid and liquid tumors. Bristol Myers Squibb recently opted into an additional gene editing program, further validating the Company’s technology and expertise in engineered cell medicines.

Corporate

• Leadership
  Today, the Company announced Mark S. Shearman, Ph.D., will be joining Editas as Chief Scientific Officer, in June 2021. Dr. Shearman has more than 30 years of experience in drug discovery and development across multiple therapeutic modalities, and his expertise in neurology, ophthalmology, and immunology will strengthen the Company’s existing pipeline. Dr. Shearman will join Editas Medicine from Applied Genetic Technologies Corporation (AGTC), where he is currently serving as Chief Scientific Officer and is responsible for leading the company’s product candidate selection process, pre-clinical and translational research, and long-term research and development planning. Prior to AGTC, Dr. Shearman served as Senior Vice President of Research & Early Development at EMD Serono, Inc., the U.S. and Canadian subsidiary of Merck KGaA. Previously, Dr. Shearman was Executive Director of Merck & Co. Research Laboratories in Boston, and Senior Director at the Merck Sharp & Dohme Research Laboratories Neuroscience Research Centre in the United Kingdom. Dr. Shearman earned a B.Sc. from the University of Bristol, U.K., and a Ph.D. from the University of Nottingham, U.K. He also conducted academic research at institutes in Japan and Germany.

• Manufacturing
  Editas Medicine continues to advance internal and external manufacturing capabilities for the Company’s portfolio of in vivo gene edited medicines, ex vivo gene edited cell medicines, and cell therapy medicines. The Company is finalizing the preparation of internal manufacturing capabilities for EDIT-301. This capability will provide early phase product for the RUBY trial. Concurrently, the Company has initiated the technology transfer process of EDIT-301 to its strategic CDMO partner Catalent for the later stages of the RUBY trial.

• Offering of Common Stock
  
  Strengthened balance sheet with net proceeds of approximately $250 million
  Editas Medicine closed an underwritten offering of 4,025,000 shares of its common stock at a public offering price of $66.00 per share, before deducting underwriter discounts and commissions and estimated offering expenses. This included 525,000 shares issued upon exercise in full by the underwriters of their option to purchase additional shares. All shares in the offering were sold by Editas Medicine.

• Balance Sheet
  The Company expects that its existing cash, cash equivalents and marketable securities of $723.2 million as of March 31, 2021, and anticipated interest income will enable it to fund its operating expenses and capital expenditures well into 2023.

First Quarter for 2021 Financial Results

Cash, cash equivalents, and marketable securities as of March 31, 2021, were $723.2 million, compared to $511.8 million as of December 31, 2020.

For the three months ended March 31, 2021, net loss attributable to common stockholders was $56.7 million, or $0.86 per share, compared to $37.7 million, or $0.69 per share, for the same period in 2020.

• Collaboration and other research and development revenues were $6.5 million for the three months ended March 31, 2021, compared to $5.7 million for the same period in 2020. The majority of the revenue recognized this quarter was attributable to the Company’s strategic alliance with Bristol Myers Squibb.

• Research and development expenses increased by $7.3 million, to $41.9 million for the three months ended March 31, 2021, from $34.6 million for the same period in 2020. The $7.3 million increase was primarily attributable to sublicense and license fees primarily related to success payments under certain of our license agreements, as well as increased manufacturing and clinical related costs, during the first quarter of 2021.
• General and administrative expenses increased by $3.6 million to $21.4 million for the three months ended March 31, 2021, from $17.8 million for the same period in 2020. The $3.6 million increase was primarily attributable to increased employee-related expenses.

Upcoming Events

Editas Medicine plans to participate in the following scientific and medical conferences:

• 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), May 11-14, Virtual
• 2021 World Medical Innovation Forum, May 20, Virtual
• European Hematology Association (EHA) 2021 Virtual Congress, June 11, Virtual

Editas Medicine plans to participate in the following investor event:

• Raymond James Human Health Innovation Conference, June 20, Virtual

Conference Call

The Editas Medicine management team will host a conference call and webcast today at 8:00 a.m. ET to provide and discuss a corporate update and financial results for the first quarter 2021. To access the call, please dial 844-348-3801 (domestic) or 213-358-0955 (international) and provide the passcode 7587147.  A live webcast of the call will be available on the Investors & Media section of the Editas Medicine website at www.editasmedicine.com and a replay will be available approximately two hours after its completion.

About Editas Medicine

As a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cas12a (also known as Cpf1) genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit www.editasmedicine.com.

About EDIT-101

EDIT-101 is a CRISPR-based experimental medicine under investigation for the treatment of Leber congenital amaurosis 10 (LCA10). EDIT-101 is administered via a subretinal injection to reach and deliver the gene editing machinery directly to photoreceptor cells.

About BRILLIANCE

The BRILLIANCE Phase 1/2 clinical trial of EDIT-101 for the treatment of Leber congenital amaurosis 10 (LCA10) is designed to assess the safety, tolerability, and efficacy of EDIT-101 in up to 18 patients with this disorder. Clinical trial sites are enrolling up to five cohorts testing up to three dose levels in this open label, multi-center study. Both adult and pediatric patients (3 – 17 years old) with a range of baseline visual acuity assessments are eligible for enrollment. Patients receive a single administration of EDIT-101 via subretinal injection in one eye. Additional details are available on www.clinicaltrials.gov (NCT#03872479).

About EDIT-301

EDIT-301 is an experimental, autologous cell therapy medicine under investigation for the treatment of sickle cell disease. EDIT-301 is comprised of sickle patient CD34+ cells genetically modified using a highly specific and efficient CRISPR/Cas12a (also known as Cpf1) ribonucleoprotein (RNP) that targets the HBG1 and HBG2 promoters in the beta-globin locus where naturally occurring fetal hemoglobin (HbF) inducing mutations reside. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in HbF production, which has the potential to provide a durable treatment benefit for people living with sickle cell disease.

About RUBY

The RUBY Trial is a single-arm, open-label, multi-center Phase 1/2 study designed to assess the safety and efficacy of EDIT-301 in patients with severe sickle cell disease. Enrolled patients will receive a single administration of EDIT-301. Additional details are available on www.clinicaltrials.gov (NCT#04853576).

Forward-Looking Statements

This press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘target,’’ ‘‘should,’’ ‘‘would,’’ and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the initiation, timing, progress and results of the Company’s preclinical and clinical studies and its research and development programs, including beginning patient dosing in the RUBY trial by the end of 2021, the timing for the Company’s receipt and presentation of data from its clinical trials and preclinical studies, including presenting data from the first two cohorts of the BRILLIANCE trial by the end of 2021, and the timing or likelihood of regulatory filings and approvals, including filing an IND for EDIT-301 for the treatment of beta-thalassemia by the end of 2021. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of pre-clinical studies and clinical trials and clinical development of the Company’s product candidates; availability and timing of results from pre-clinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption “Risk Factors” included in the Company’s most recent Annual Report on Form 10-K, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release represent Company’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, the Company explicitly disclaims any obligation to update any forward-looking statements.

EDITAS MEDICINE, INC.
Condensed Consolidated Statements of Operations
(unaudited)
(amounts in thousands, except per share and share data)
		Three Months Ended
		March 31,
		2021				2020
Collaboration and other research and development revenues		$	6,499			$	5,723
Operating expenses:
Research and development			41,937				34,570
General and administrative			21,445				17,769
Total operating expenses			63,382				52,339
Operating loss			(56,883	)			(46,616	)
Other income, net:
Other income (expense), net			21				7,333
Interest income, net			134				1,559
Total other income, net			155				8,892
Net loss		$	(56,728	)		$	(37,724	)
Net loss per share attributable to common stockholders, basic and diluted		$	(0.86	)		$	(0.69	)
Weighted-average common shares outstanding, basic and diluted			65,992,395				54,590,194

EDITAS MEDICINE, INC.
Selected Condensed Consolidated Balance Sheet Items
(unaudited)
(amounts in thousands)
			March 31,			December  31,
			2021			2020
Cash, cash equivalents, and marketable securities			$	723,223		$	511,774
Working capital				568,305			358,894
Total assets				780,294			572,602
Deferred revenue, net of current portion				56,667			73,984
Total stockholders’ equity				637,996			393,586

Contacts:
Media
Cristi Barnett
(617) 401-0113
[email protected]

Investors
Ron Moldaver
(617) 401-9052
[email protected]

NEW YORK, May 05, 2021 (GLOBE NEWSWIRE) — Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) diseases, today announced the appointment of Dr. Adebayo (Bayo) Laniyonu, as Senior Vice President for Nonclinical Development. Dr. Laniyonu has over 24 years’ experience with the US Food and Drug Administration (FDA).

“Dr. Laniyonu has an accomplished track record of working within the FDA,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex. “His depth of experience makes him an excellent choice to provide objective perspective and manage the continuous nonclinical projects at Anavex.”

Prior to joining Anavex, Dr. Laniyonu served as Supervisory Pharmacologist/Toxicologist at the FDA Center for Drug Evaluation and Research (CDER). He has reviewed hundreds of NDAs, BLAs (including first in class), sNDAs, ANDAs and INDs and provided high impact regulatory and strategic recommendations to the Sponsors of these products. He organized, chaired and presented at FDA workshops and at scientific sessions of professional societies where he presented on FDA current thinking on regulatory issues, he has also presented at FDA Advisory Committee meetings. His review and Supervisory experience span several therapeutics areas including Rare Diseases and Medical Genetics, hematology, Medical Imaging, Radiation Medicine and Medical Counter Measures products. Dr. Laniyonu played pivotal roles in the development of several FDA regulatory guidance documents.

“I am very pleased to be joining Anavex at this important and exciting time for the company,” stated Dr. Laniyonu. “Anavex deep scientific base, innovative and novel approaches to discovery, including use of precision medicine and molecular biology to target a breath of rare diseases, and challenging therapeutic areas such as Rett syndrome, Parkinson’s disease, Parkinson’s disease dementia, Alzheimer’s disease, and other CNS diseases is exciting given the unmet medical needs or orphan nature of some of these diseases. I believe there are tremendous opportunities ahead, and I look forward to working with the Anavex team to deliver on the corporate objectives for 2021 and beyond.”

Dr. Laniyonu holds a PhD in Pharmacology from the University of Glasgow, UK where he was a Commonwealth Scholar. He was also a Canadian Cystic Fibrosis Post-Doctoral Fellow. Dr. Laniyonu was a recipient of many FDA distinguished awards including Department of Health and Human Services Secretary’s Award for Distinguished Service, FDA Award of Merit, FDA Outstanding Service Award, FDA Group Recognition Award, CDER Excellence in Communication Award, CDER Excellence in Mentoring Award.

About Anavex Life Sciences Corp.

Anavex Life Sciences Corp. (Nasdaq: AVXL) is a publicly traded biopharmaceutical company dedicated to the development of differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) diseases, pain and various types of cancer. Anavex’s lead drug candidate, ANAVEX^®2-73 (blarcamesine), completed successfully a Phase 2a clinical trials for Alzheimer’s disease and recently a Phase 2 proof-of-concept study in Parkinson’s disease dementia and a Phase 2 study in adult patients with Rett syndrome. ANAVEX^®2-73 is an orally available drug candidate that restores cellular homeostasis by targeting sigma-1 and muscarinic receptors. Preclinical studies demonstrated its potential to halt and/or reverse the course of Alzheimer’s disease. ANAVEX^®2-73 also exhibited anticonvulsant, anti-amnesic, neuroprotective and anti-depressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy. The Michael J. Fox Foundation for Parkinson’s Research previously awarded Anavex a research grant, which fully funded a preclinical study to develop ANAVEX^®2-73 for the treatment of Parkinson’s disease. ANAVEX^®3-71, which targets sigma-1 and muscarinic receptors, is a promising clinical stage drug candidate demonstrating disease-modifying activity against the major hallmarks of Alzheimer’s disease in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid and tau pathologies. In preclinical trials, ANAVEX^®3-71 has shown beneficial effects on mitochondrial dysfunction and neuroinflammation. Further information is available at www.anavex.com. You can also connect with the company on Twitter,Facebook and LinkedIn.

Forward-Looking Statements

Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties. Actual events or results may differ materially from those projected in any of such statements due to various factors, including the risks set forth in the Company’s most recent Annual Report on Form 10-K filed with the SEC. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Anavex Life Sciences Corp. undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof.

For Further Information:

Anavex Life Sciences Corp.
Research & Business Development
Toll-free: 1-844-689-3939
Email: [email protected]

Investors:

Andrew J. Barwicki
Investor Relations
Tel: 516-662-9461
Email: [email protected]

WATERTOWN, Mass., May 05, 2021 (GLOBE NEWSWIRE) — Kymera Therapeutics, Inc. (NASDAQ: KYMR), a clinical-stage biopharmaceutical company advancing targeted protein degradation to deliver novel small molecule protein degrader medicines, today announced the appointment of Juliet Williams, PhD, as Senior Vice President, Head of Biology. Dr. Williams joins Kymera with 20 years of experience in the biopharmaceutical industry, including leadership roles in drug discovery and translational development.

“I’m very excited to welcome Juliet to the leadership team at Kymera,” said Nello Mainolfi, PhD, Co-Founder, President and CEO, Kymera Therapeutics. “Juliet has been a successful drug hunter in both biotech and large pharma. Her expertise and leadership in target identification, drug discovery, and translational drug development across small molecule and innovative drug classes will be invaluable as we continue to expand Kymera’s capabilities to build a best-in-class degrader medicines company.”

“I am excited to join Kymera during a transformational time as the Company has initiated a first-in-class development program in immunology and inflammation and expects to advance its two lead oncology degrader programs into clinical development later this year,” said Dr. Williams. “I look forward to working with the team to continue to advance our platform and science and I am inspired by the opportunity to invent and deliver new medicines for patients in need.”

Dr. Williams joins Kymera with 20 years of drug development experience, including service at Novartis, Sanofi, Millennium, and Curis. Prior to Kymera, she led oncology small molecule drug discovery at Novartis Institutes for Biomedical Research and served in Head of Oncology Pharmacology roles at both Novartis and Sanofi. She holds a degree in Natural Sciences (Biochemistry) from the University of Cambridge and a PhD in Developmental Biology from University College London. Dr. Williams completed a Wellcome Postdoctoral Fellowship at University College London in Developmental Biology.

About Kymera Therapeutics


Kymera Therapeutics (Nasdaq: KYMR) is a clinical-stage biopharmaceutical company founded with the mission to discover, develop, and commercialize transformative therapies while leading the evolution of targeted protein degradation, a transformative new approach to address previously intractable disease targets. Kymera’s Pegasus™ platform enables the discovery of novel small molecule degraders designed to harness the body’s natural protein recycling machinery to degrade disease-causing proteins, with a focus on undrugged nodes in validated pathways currently inaccessible with conventional therapeutics. Kymera’s initial programs are IRAK4, IRAKIMiD, and STAT3, which each address high impact targets within the IL-1R/TLR or JAK/STAT pathways, providing the opportunity to treat a broad range of immune-inflammatory diseases, hematologic malignancies, and solid tumors. Kymera’s goal is to be a fully integrated biopharmaceutical company at the forefront of this new class of protein degrader medicines, with a pipeline of novel degrader medicines targeting disease-causing proteins that were previously intractable.

Founded in 2016, Kymera is headquartered in Watertown, Mass. Kymera has been named a “Fierce 15” biotechnology company by FierceBiotech and has been recognized by the Boston Business Journal as one of Boston’s “Best Places to Work.” For more information about our people, science, and pipeline, please visit www.kymeratx.com or follow us on Twitter or LinkedIn.

Cautionary Note Regarding Forward-Looking Statements


This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, implied and express statements regarding its: beliefs regarding its program in immunology and inflammation and that it is first-in-class; plans to continue to expand capabilities to build a best-in-class degrader medicines company; expectations for Dr. Williams; strategy, business plans and objectives for the IRAK4,
IRAKIMiD and STAT3 degrader programs
and the clinical development of Kymera Therapeutics’ product candidates, including the therapeutic potential and clinical benefits thereof. The words “may,” “might,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “expect,” “estimate,” “seek,” “predict,” “future,” “project,” “potential,” “continue,” “target” and similar words or expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks associated with: the impact of COVID-19 on countries or regions in which Kymera Therapeutics has operations or does business, as well as on the timing and anticipated results of its current preclinical studies and future clinical trials, strategy and future operations; the delay of any current preclinical studies or future clinical trials or the development of Kymera Therapeutics’ drug candidates; the risk that the results of current preclinical studies may not be predictive of future results in connection with future clinical trials; Kymera Therapeutics’ ability to successfully demonstrate the safety and efficacy of its drug candidates; the timing and outcome of Kymera Therapeutics’ planned interactions with regulatory authorities, including the resolution of the current partial clinical hold for KT-474; and obtaining, maintaining and protecting its intellectual property. These and other risks and uncertainties are described in greater detail in the section entitled “Risk Factors” in the Annual Report on Form 10-K for the period ended December 31, 2020, filed on March 11, 2021, as well as discussions of potential risks, uncertainties, and other important factors in Kymera Therapeutics’ subsequent filings with the Securities and Exchange Commission. In addition, any forward-looking statements represent Kymera Therapeutics’ views only as of today and should not be relied upon as representing its views as of any subsequent date. Kymera Therapeutics explicitly disclaims any obligation to update any forward-looking statements. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements.

Investor Contact:

Paul Cox
VP, Investor Relations and Communications
[email protected]
917-754-0207

Media Contact:

Lissette L. Steele
Verge Scientific Communications for Kymera Therapeutics
[email protected]
202-930-4762