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Morphic Announces Corporate Highlights and First Quarter 2020 Financial Results

On May 6, 2020 Morphic Therapeutic (NASDAQ: MORF), a biopharmaceutical company developing a new generation of oral integrin therapies for the treatment of serious chronic diseases, reported corporate highlights and first quarter 2020 financial results (Press release, Morphic Therapeutic, MAY 6, 2020, View Source [SID1234557143]).

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"During the first quarter of 2020 Morphic made critical steps toward our goal of creating oral drugs targeting integrins with major advances in our scientific and operational groups," commented Praveen Tipirneni, M.D., president and chief executive officer of Morphic Therapeutic. "At the ECCO meeting, we presented preclinical data that strongly support the development of our candidate MORF-057, an oral inhibitor of α4β7, which is on track to enter clinical studies for inflammatory bowel disease in the second half of this year. These data demonstrated high specificity for its intended biologic target and mechanistic equivalence to the approved intravenous therapeutic against α4β7, vedolizumab. We also bolstered our executive management team with the appointments of two industry veterans as chief medical officer and chief financial and operating officer. We are now focused on completing the necessary preparations for Phase 1 studies of MORF-057 as Morphic’s lead oral integrin inhibitor."

Recent Corporate Highlights:

Presented new preclinical data in both murine and non-human primate models at the ECCO Annual Congress supporting MORF-057, a novel small molecule inhibitor of the α4β7 integrin in development for inflammatory bowel disease

In these data, MORF-057 demonstrated high specificity for the α4β7 integrin and clear biologic activity through its intended mechanism of action, blocking the migration of α4β7-high-expressing lymphocytes

Made substantial progress in our collaboration with AbbVie on the development of integrin inhibitors, including αvβ6 and other integrin targets

Appointed Peter G. Linde, M.D., as chief medical officer; Dr. Linde was formerly vice president of medical research at Acceleron Pharma, and additionally has significant prior clinical program development experience with agents across multiple therapeutic areas at AbbVie and Johnson & Johnson

Appointed Marc Schegerin, M.D., as chief financial officer and chief operating officer; Dr. Schegerin was previously chief financial officer of ArQule until its acquisition by Merck. His prior roles include senior positions in finance, business development and healthcare investment banking

COVID-19 Preparedness

Morphic is not currently aware of any significant delay to timelines due to the COVID-19 pandemic. In light of the evolving circumstances, Morphic will continue to assess any potential impact of the COVID-19 pandemic in dialogue with regulators, partners and vendors.

Financial Results for First Quarter 2020

Net loss for the quarter ended March 31, 2020 was $16.7 million, or $0.55 per share, compared to a net loss of $5.2 million, or $2.77 per share, for the same quarter last year.

Revenue was $5.6 million for the quarter ended March 31, 2020 compared to $6.1 million for the same quarter last year. The decrease was due to lower revenue recognized on the AbbVie collaboration offset by an increase in revenue recognized on the company’s collaboration with Janssen as a result of higher FTE reimbursements in the quarter ended March 31, 2020.

Research and development expenses were $19.0 million for the quarter ended March 31, 2020, compared to $10.4 million in the same quarter last year. The $8.6 million increase year-over-year reflects higher development and manufacturing costs associated with lead product candidate, MORF-057, along with increased research costs associated with other early development candidates, as well as increased personnel-related costs to support continued progress with the company’s pipeline.

General and administrative expenses were $4.4 million for the quarter ended March 31, 2020, compared to $1.8 million in the same quarter last year. The $2.6 million increase year-over-year was primarily attributable to increased headcount and higher professional and consulting fees associated with ongoing business activities and Morphic’s operating as a public company.

As of March 31, 2020, Morphic had cash, cash equivalents, and marketable securities of $219 million, compared to $237 million as of December 31, 2019. Morphic believes its cash, cash equivalents, and marketable securities as of March 31, 2020, will be sufficient to fund operating expenses and capital expenditure requirements at least through the end of 2022.

Abeona Therapeutics Reports First Quarter Financial Results and Business Updates

On May 6, 2020 Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, reported first quarter 2020 financial results, which will be discussed on a conference call scheduled for Thursday, May 7, 2020 at 8:30 a.m. ET (Press release, Abeona Therapeutics, MAY 6, 2020, View Source [SID1234557142]). Interested parties are invited to participate in the call by dialing 844-455-1352 (U.S. toll-free) or 509-844-0155 (international), and reference conference ID 5352629, or via webcast at View Source

"During these challenging times, our priority remains to ensure the safety of our employees and patients, while supporting continuity of our business and clinical operations," said João Siffert, M.D., Chief Executive Officer of Abeona. "We have made considerable progress in our clinical programs in the first quarter of 2020, and are working with our investigators to minimize the impact of the COVID-19 pandemic. It is our intention to restore full patient access to our clinical programs as soon as possible. Our gene and cell therapies in development aim at addressing urgent unmet needs, and have the potential to provide durable benefit to patients who have no approved treatments."

First Quarter and Recent Highlights

●First patient treated in pivotal Phase 3 VIITALTM study evaluating EB-101 for recessive dystrophic epidermolysis bullosa (RDEB). An additional 10 patients have been prescreened for this study.
●Additional patients treated in dose cohort 3 of the Transpher A study and the Transpher B study.
●Presented positive interim data from the Transpher A study of ABO-102 at WORLDSymposium demonstrating improved neurocognitive skills 18 months to two years post-treatment in MPS IIIA patients younger than 30 months, sustained, dose-related biomarker improvements, and a favorable safety profile.
●Presented positive interim data from the Transpher B study of ABO-101 at WORLDSymposium demonstrating initial improvement in multiple disease-specific biomarkers, denoting clear biologic effects, and a favorable safety profile among MPS IIIB patients.
●Updated interim results from the Transpher A and Transpher B studies to be presented during the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 23rd Annual Meeting, which will take place online May 12-15, 2020.
●Two U.S. patents issued for adeno-associated virus (AAV) capsids exclusively licensed by Abeona from the University of North Carolina ("UNC"), generated using UNC’s AIM vector platform.

●Announced key appointments of industry leaders to its Board of Directors in April. Dr. Brian J. G. Pereira was appointed as Executive Chairman and Ms. Shawn Tomasello as an Independent Board Member. Dr. Pereira is a seasoned biopharmaceutical and healthcare leader with experience in financing and growing companies, including the clinical development and commercialization of innovative drug products. Ms. Tomasello has substantial commercial and strategic experience, including serving as Chief Commercial Officer at cell therapy pioneer Kite Pharma, which was acquired by Gilead Sciences.
●Strengthened its leadership team with the appointments of Gregory Gin as Vice President, Investor Relations and Dr. Dan Rudin as Vice President, Clinical Development, focusing on the EB-101 program. Mr. Gin brings more than 25 years of investor relations, communications, and capital markets experience with small- and mid-cap biotechnology and specialty pharmaceutical companies developing novel treatments for orphan diseases and areas of high unmet medical need. Dr. Rudin has substantial research and development experience gained in industry and academia with focus on rare diseases, including lysosomal storage diseases. He has led several programs through the lifecycle of clinical development supporting multiple product approvals.

Dr. Siffert continued, "We look forward to working with our new Executive Chairman, Dr. Brian Pereira and Independent Board Member, Ms. Shawn Tomasello. Both bring invaluable experience guiding biotech companies from clinical development through commercial launch. In addition, with the appointments of Greg and Dan, we have strengthened our leadership in investor relations and clinical development, respectively."

COVID-19 Impact Mitigation

The ongoing COVID-19 pandemic has caused meaningful disruptions to the global healthcare system, including the conduct of clinical trials as healthcare institutions shift their focus and resources to treating COVID-19 patients. In response to the unprecedented challenges related to the COVID-19 pandemic, Abeona has taken several measures to protect and support the health of its employees and their families, healthcare partners and patients participating in its clinical trials. At the same time, the Company has implemented measures to maintain continuity of its operations and to preserve financial flexibility for the future.

First Quarter Financial Results

Cash, cash equivalents and marketable securities as of March 31, 2020, were $116 million compared to $129 million as of December 31, 2019. The decrease in cash of $13 million was driven by R&D expenses across our programs along with supporting administrative costs.

The net loss was $0.52 per share for the first quarter of 2020, compared to $0.39 per share in the comparable period in 2019. The increase in the net loss per share results primarily from the non-cash impairment charge on the termination of the REGENXBIO license of $32.9 million, or $0.36 per share.

Entry into a Material Definitive Agreement

On May 6, 2020 On May 6, 2020, Amgen Inc. (the "Company") reported that issued and sold $1,000,000,000 aggregate principal amount of its 2.200% Senior Notes due 2027 (the "2027 Notes"), $1,250,000,000 aggregate principal amount of its 2.300% Senior Notes due 2031 (the "2031 Notes"), $750,000,000 aggregate principal amount of its 3.150% Senior Notes due 2040 (the "2040 Notes") and $1,000,000,000 aggregate principal amount of its 3.375% Senior Notes due 2050 (the "2050 Notes" and, together with the 2027 Notes, the 2031 Notes and the 2040 Notes, the "Notes") (Filing, 8-K, Amgen, MAY 6, 2020, View Source [SID1234557141]). The Notes are registered under an effective Registration Statement on Form S-3 (Registration No. 333-236351) (the "Registration Statement"), filed on February 10, 2020, and were issued pursuant to an indenture, dated as of May 22, 2014 (the "Indenture"), between the Company and The Bank of New York Mellon Trust Company, N.A., as trustee, and (i) as to the 2027 Notes, the 2040 Notes and the 2050 Notes, an officer’s certificate, dated as of February 21, 2020 (the "Existing Officer’s Certificate") and (ii) as to the 2031 Notes, an officer’s certificate, dated as of May 6, 2020 (the "2031 Notes Officer’s Certificate" and, together with the Existing Officer’s Certificate, the "Officer’s Certificates"), setting forth the terms of the Notes. The 2027 Notes form a single series with, and have the same terms as, the Company’s 2.200% senior notes due 2027, issued on February 21, 2020, the 2040 Notes form a single series with, and have the same terms as, the Company’s 3.150% senior notes due 2040, issued on February 21, 2020, and the 2050 Notes form a single series with, and have the same terms as, the Company’s 3.375% senior notes due 2050, issued on February 21, 2020. Net proceeds to the Company from the offering were approximately $ 4,034,736,618, after deducting underwriters’ discounts and estimated offering expenses payable by the Company, plus, in the case of the 2027 Notes, the 2040 Notes, and the 2050 Notes, interest deemed to have accrued from February 21, 2020 to, but excluding, the settlement date.

The relevant terms of the Notes are set forth in the Indenture, included as Exhibit 4.1 of the Company’s Current Report on Form 8-K, filed on May 22, 2014, and incorporated herein by reference, the Existing Officer’s Certificate (including the forms of the 2027 Notes, the 2040 Notes and the 2050 Notes) included as Exhibit 4.2 of the Company’s Current Report on Form 8-K, filed on February 21, 2020, and incorporated herein by reference, and the 2031 Notes Officer’s Certificate (including the form of the 2031 Notes) attached hereto as Exhibit 4.3 and incorporated herein by reference.

The 2027 Notes will pay interest at the rate of 2.200% per annum, the 2031 Notes will pay interest at the rate of 2.300% per annum, the 2040 Notes will pay interest at the rate of 3.150% per annum and the 2050 Notes will pay interest at the rate of 3.375% per annum, which, as to the 2027 Notes, the 2040 Notes and the 2050 Notes, shall be payable in cash semi-annually in arrears on February 21 and August 21 of each year, beginning on August 21, 2020, and as to the 2031 Notes, shall be payable in cash semi-annually in arrears on February 25 and August 25 of each year, beginning on February 25, 2021. The interest payment to be made with respect to the 2027 Notes, the 2040 Notes and the 2050 Notes on August 21, 2020 will include interest deemed to have accrued from and including February 21, 2020 to, but excluding, the settlement date. Such accrued interest was paid by the purchasers of the additional notes of each series on the settlement date. The 2027 Notes will mature on February 21, 2027, the 2031 Notes will mature on February 25, 2031, the 2040 Notes will mature on February 21, 2040 and the 2050 Notes will mature on February 21, 2050.

In the event of a change in control triggering event, as defined in the Officer’s Certificates, the holders of the Notes may require the Company to purchase for cash all or a portion of their Notes at a purchase price equal to 101% of the principal amount of Notes, plus accrued and unpaid interest, if any. The descriptions of the Indenture, the Officer’s Certificates and the Notes in this report are summaries and are qualified in their entirety by the terms of the Indenture, the Officer’s Certificates and the Notes, respectively.

The Notes will rank equal in right of payment to all of the Company’s other existing and future senior unsecured indebtedness, senior in right of payment to all of the Company’s existing and future subordinated indebtedness, effectively subordinated in right of payment to all of the Company’s subsidiaries’ obligations (including secured and unsecured obligations) and subordinated in right of payment to the Company’s secured obligations, to the extent of the assets securing such obligations.

Principia Biopharma Reports First Quarter 2020 Financial Results

On May 6, 2020 Principia Biopharma Inc. (Nasdaq: PRNB), a late-stage biopharmaceutical company focused on developing treatments for immune-mediated diseases, reported financial results for the first quarter ended March 31, 2020 (Press release, Principia Biopharma, MAY 6, 2020, View Source [SID1234557140]).

"Despite these challenging times of a COVID-19 pandemic, our focus remains on our commitment to delivering for patients with immune-mediated diseases through continued enrollment in our clinical trials and broadening our BTK footprint. We are also excited about the positive Phase 2 data in multiple sclerosis that Sanofi presented studying our partnered investigational brain penetrant BTK inhibitor, PRN2246/SAR442168, which is further validation of our drug discovery platform," said Martin Babler, president and chief executive officer of Principia.

Principia remains focused on executing its business plan in the midst of this global pandemic, including advancing its ongoing clinical trials. In considering the current impacts of the COVID-19 pandemic, Principia is not changing previously communicated guidance except in the case of its Phase 2 trial of rilzabrutinib in patients with IgG4-RD, which now will begin in the second half of 2020 rather than the first half as originally planned.

First Quarter 2020 Financial Results

Cash Position: Cash, cash equivalents, and marketable securities were $341.1 million as of March 31, 2020, compared to $367.8 million as of December 31, 2019.

Revenues: We did not recognize any collaboration revenue for the three months ended March 31, 2020, compared to $5.2 million for the same period in 2019. Revenue in the first quarter of 2019 was related to the recognition of a portion of an upfront payment received in 2017 from AbbVie Biotechnology Limited.

R&D Expenses: Total research and development expenses were $26.7 million for the three months ended March 31, 2020, including stock-based compensation expense of $2.3 million, compared to $15.5 million for the same period in 2019, including stock-based compensation expense of $1.2 million. The increase in total research and development expenses was mainly driven by an increase in rilzabrutinib program costs, due to the progression of our global Phase 3 trial in pemphigus, ongoing Phase 2 trial in ITP and certain manufacturing campaigns to supply drug products for our rilzabrutinib

clinical trials, the initiation of our Phase 1 trial for PRN473 Topical and an increase in employee-related expenses.

G&A Expenses: General and administrative expenses were $7.4 million for the three months ended March 31, 2020, including stock-based compensation expense of $2.0 million, compared to $4.5 million for the same period in 2019, including stock-based compensation expense of $1.1 million. The increase in total general and administrative expenses was primarily driven by increased employee-related expenses.

Net Income (Loss): For the three months ended March 31, 2020, net loss was $32.5 million compared to a net loss of $13.7 million for the same period in 2019.

Atara Biotherapeutics Announces First Quarter 2020 Financial Results and Operational Progress

On May 6, 2020 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a pioneer in T-cell immunotherapy, leveraging its novel allogeneic EBV T-cell platform to develop transformative therapies for patients with severe diseases including solid tumors, hematologic cancers and autoimmune disease, reported financial results for the first quarter ended March 31, 2020 and recent business highlights (Press release, Atara Biotherapeutics, MAY 6, 2020, View Source [SID1234557139]).

"It is with tremendous pride that I acknowledge the commitment and resiliency of our entire Atara team. We have remained focused on our mission to serve patients and implemented industry-leading practices to ensure safety while mitigating the impact of COVID-19 on our business," said Pascal Touchon, President and Chief Executive Officer of Atara. "We have made great progress in Q1 toward accomplishing our key objectives and are well-poised to achieve significant milestones throughout the year as we expect to initiate the tab-cel biologics license application (BLA) submission in the second half of 2020 and are eager to present further results from the ATA188 Phase 1a study in progressive multiple sclerosis in Q2. Such momentum in developing innovative off-the-shelf allogeneic T-cell immunotherapies has also supported our ability to attract top talent including a non-executive board chair and general counsel."

Recent Highlights and Anticipated Upcoming Milestones

Operational

COVID-19 Response and Actions

Atara continues to deliver product to patients from our inventory of off-the-shelf, allogeneic tab-cel and ATA188.

Prior to the COVID-19 outbreak, as part of our routine supply planning and operational risk management strategies, the Company had already manufactured significant inventories of tab-cel and process intermediates and procured the required starting materials needed to maintain long-term product supply across tab-cel, ATA188 and other programs.

The Atara clinical study and operational teams have been working closely with sites to ensure the safety of site staff and patients as well as preserve data integrity and access to treatment as appropriate. Where needed, they have established remote study visits, leveraged tele-medicine, home health care, and other methods to ensure continuity of care for patients while preserving key endpoint data.

Atara is closely monitoring the evolving COVID-19 pandemic and continues to assess potential impact on the business and operations, including the timing and execution of clinical and preclinical studies.

Board and Executive Appointments

Atara recently announced the appointment of a Chair of the Board and executive with extensive leadership and management experience in the life sciences industry:

Atara appointed Ron Renaud as Chairman of the Board of Directors. Mr. Renaud has served as Translate Bio’s chief executive officer since 2014 and brings deep and broad experience in strategic and corporate development, partnering, financing, and industry and Wall Street relationships. He has significant prior board experience having served on the boards of both public and private companies.

K. Amar Murugan was named Senior Vice President, General Counsel. Mr. Murugan brings significant expertise in M&A, corporate finance, securities, life science transactions and corporate governance. Mr. Murugan was most recently Senior Vice President and General Counsel of Assertio Therapeutics.

Tab-cel (tabelecleucel)

Atara continues to progress tab-cel Phase 3 development for patients with EBV-associated post-transplant lymphoproliferative disease (EBV+ PTLD).

Atara remains on track to initiate a tab-cel BLA submission for patients with EBV+ PTLD in the second half of 2020.

Atara plans to discuss the totality of tab-cel data with the U.S. Food and Drug Administration (FDA) in a pre-BLA meeting prior to initiating the BLA submission.

In the U.S. and Australia, most of the current 40 clinical study sites are available for enrollment and the Company is preparing to open additional sites in the U.S., Canada and Europe.

The Company’s clinical trial applications (CTAs) in the United Kingdom, Spain, France, and Austria were recently approved, the first European clinical site has opened for enrollment, and we expect to open additional European Phase 3 clinical sites in 2020.

Atara has submitted a Pediatric Investigation Plan (PIP) to the European Medicines Agency (EMA).

Following EMA approval of the PIP, Atara plans to submit a tab-cel EU marketing authorization application (MAA) for patients with EBV+ PTLD in 2021.

Atara continues to see strong tab-cel investigator, physician and patient interest and, for cases in which the Company is not able to enroll patients in its EBV+ PTLD Phase 3 clinical study, Atara is providing tab-cel to patients in need under its expanded access protocol (EAP) and single patient use (SPU) programs.

Studies supporting potential additional tab-cel indications are also advancing.

Based on clinical data from treating a variety of ultra-rare EBV+ diseases, Atara expects to initiate enrollment in the second half of 2020 in a tab-cel Phase 2 multi-cohort study including up to six additional ultra-rare EBV+ patient populations.

ATA188 for Progressive Multiple Sclerosis (MS)

A Phase 1a clinical study of off-the-shelf, allogeneic ATA188 in patients with progressive MS is ongoing across clinical sites in the U.S. and Australia.

Atara expects to present ATA188 Phase 1a six-month clinical results for the dose-escalating cohorts 1-4 and 12-month results for cohorts 1-3 in an appropriate forum in the second quarter of 2020 and expects to present 12-month cohort 4 data in the second half of 2020.

Atara is re-treating patients in the open-label extension (OLE) of the Phase 1a study in an appropriate setting and as determined by the treating physician and patient.

Atara has temporarily paused the screening and enrollment of patients in the Phase 1b randomized placebo-controlled study to ensure sites can focus on meeting the needs of patients with COVID-19 and to protect the safety of study participants, investigators and staff.

This action will help to preserve study and data integrity as there are numerous assessments that require a specific clinical setting.

Atara expects this pause to be limited and plans to initiate enrollment in this study in the second or third quarter of 2020.

ATA2271/ATA3271 and ATA3219 CAR T Programs

Atara is developing a next generation mesothelin-targeted autologous CAR T immunotherapy (ATA2271) and expects collaborators at Memorial Sloan Kettering Cancer Center (MSK) to submit an Investigational New Drug (IND) application to the FDA for patients with advanced mesothelioma in the second or third quarter of 2020.

ATA2271 is designed to improve efficacy, persistence, and durability of response using a novel 1XX CAR co-stimulatory domain and cell intrinsic checkpoint inhibition technology with a PD-1 dominant negative receptor (DNR).

Data from IND-enabling studies for ATA2271 have been accepted as a late-breaking e-poster at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting II in June, and the abstract will be released on May 15.

Additional clinical data for the first-generation academic program from MSK are expected to be presented in an upcoming forum in the second half of this year.

Atara is also developing off-the-shelf, allogeneic CAR T immunotherapies targeting mesothelin (ATA3271) and CD19 (ATA3219) using its next-generation technologies and EBV T-cell platform.

The Company has initiated preclinical IND-enabling studies for ATA3271 and ATA3219.

First Quarter 2020 Financial Results

Atara believes that its cash, cash equivalents and short-term investments as of March 31, 2020, are sufficient to fund planned operations into the second quarter of 2021.

Cash, cash equivalents and short-term investments as of March 31, 2020 totaled $214.6 million, as compared to $259.1 million as of December 31, 2019.

The decrease of $44.5 million includes the benefit of the sale of 1,528,216 shares of common stock pursuant to the Company’s at-the-market (ATM) facility in the first quarter of 2020 for net proceeds of $23.1 million.

Net cash used in operating activities was $67.0 million for the first quarter of 2020, as compared to $70.2 million for the same period in 2019.

The number of outstanding shares of common stock and pre-funded common stock warrants as of April 30, 2020 was 58,952,045 shares and 2,888,526 warrants, respectively.

Atara reported net losses of $73.5 million, or $1.20 per share, for the first quarter of 2020 as compared to $66.3 million, or $1.44 per share, for the same period in 2019.

Total operating expenses include non-cash expenses of $14.5 million for the first quarter 2020, as compared to $13.9 million for the same period in 2019.

Research and development expenses were $57.7 million for the first quarter of 2020, as compared to $48.7 million for the same period in 2019. The increase in the first quarter of 2020 was due to costs associated with the Company’s continuing expansion of research and development activities, including:

Clinical study, manufacturing and process performance qualification activities related to tab-cel.

Higher employee-related costs from increased headcount.

Increased facilities and information technology expenses allocated to our research and development function.

Research and development expenses include $7.7 million of non-cash stock-based compensation expenses for the first quarter of 2020, as compared to $6.1 million for the same period in 2019.

General and administrative expenses were $17.0 million for the first quarter of 2020, as compared to $19.2 million for the same period in 2019. The decrease in the first quarter 2020 was primarily due to a decrease in outside services costs and non-cash stock-based compensation expenses, partially offset by an increase in payroll-related costs driven by increased headcount.

General and administrative expenses include $5.0 million of non-cash stock-based compensation expenses for the first quarter of 2020, as compared to $6.2 million for the same period in 2019.