On May 6, 2020 Horizon Therapeutics plc (Nasdaq: HZNP) reported its first-quarter 2020 financial results (Press release, Horizon Pharma, MAY 6, 2020, View Source [SID1234557145]). The Company increased its full-year 2020 net sales guidance and revised its adjusted EBITDA guidance.

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"We had a very strong start to 2020, highlighted by the early approval and rapid uptake of TEPEZZA, which significantly exceeded expectations, excellent KRYSTEXXA growth and our recent acquisition of HZN-825," said Timothy Walbert, chairman, president and chief executive officer, Horizon. "We are increasing our full-year net sales guidance to account for significantly higher TEPEZZA net sales that more than offset the expected impact from COVID-19 this year, and we are widening both our net sales and adjusted EBITDA guidance ranges to account for future uncertainty. The fundamentals of our business are strong, including a robust cash position, and we continue to be very well positioned for the long term."

Walbert continued, "Given the current environment, our priority is to safeguard the health, safety and welfare of patients and our employees, as well as to support our communities in their COVID-19 response efforts. I would like to recognize and applaud the tireless efforts of our employees who are helping patients access and maintain therapy, as well as all the healthcare professionals showing such incredible dedication and effort during this challenging time."

Horizon is closely monitoring the COVID-19 pandemic and its impact on the patients who are treated with the Company’s medicines, the communities in which the Company operates and its business. Horizon is making every effort as a company to help minimize the spread of COVID-19 and at the same time working to ensure continued patient access to Horizon’s medicines. The Company is maintaining its business operations remotely to protect the health and safety of Horizon employees who are ensuring business continuity and providing support to patients, physicians and partners virtually. The Company continues to closely monitor its supply chain and expects no impact at this time on the supply of its medicines. Horizon has provided more than $1.5 million in financial support for COVID-19 response efforts in Illinois, Dublin, South San Francisco, Washington D.C. and Canada. Many Horizon employees are also contributing their personal time and financial support to response efforts in line with Horizon’s mission to go to incredible lengths to improve people’s lives.

TEPEZZA Launch Progress

On Jan. 21, 2020, the Company received U.S. Food and Drug Administration (FDA) approval for TEPEZZA for the treatment of thyroid eye disease (TED). TEPEZZA, a fully human monoclonal antibody insulin-like growth factor-1 receptor (IGF-1R) inhibitor, is the first and only FDA-approved medicine for the treatment of TED. The Company initiated the commercial launch of TEPEZZA shortly after obtaining FDA approval. The first-quarter results for TEPEZZA greatly exceeded the Company’s expectations, generating net sales of $23.5 million.

Several factors contributed to the strong TEPEZZA first-quarter results and the Company’s increased full-year expectations:

Severity of Disease: TED is a rare, serious, progressive and vision-threatening autoimmune disease, and is associated with proptosis (eye bulging), diplopia (double vision), blurred vision, pain and facial disfigurement that can significantly impact patients’ quality of life. The severity of the disease is a motivating factor for patients seeking therapy.

Pre-Launch Market Education Efforts: In early 2019, the Company initiated its pre-launch disease awareness, market development and market access efforts with the multi-functional field-based teams beginning to engage with key stakeholders in July of 2019. The pre-launch preparation included educating physicians, payors, sites of care and patients about the acute and debilitating nature of the disease and the urgency to treat. It also involved facilitating post-approval access to TEPEZZA by identifying an infusion site-of-care referral network and establishing a supportive patient services organization.

High Volume of Patient and Physician Interest Driven by Commercial Execution: The Company’s pre-launch efforts generated significant awareness and interest among physicians and TED patients, which resulted in a significantly higher number of patients beginning therapy than initially expected. Approximately 200 patients started TEPEZZA treatment in the first quarter.

"The tremendous success of the TEPEZZA launch reflects the potential of TEPEZZA and is a tribute to the strong execution and dedication of our TEPEZZA commercial team," said Walbert. "Since the beginning of last year, the team has paved the way for launch, establishing a pathway for treatment and driving awareness of TED, the urgency to treat it and its highly debilitating symptoms – symptoms with which patients previously had to live, often for years. This has allowed many more patients to seek and start treatment, which epitomizes Horizon’s commitment to patients and the strength of our commercial strategy and execution."

Walbert continued, "To maximize the future and long-term potential of TEPEZZA for TED patients, we announced today two new TEPEZZA development programs – one to evaluate TEPEZZA in the fibrotic phase of the disease and another to assess the potential for subcutaneous administration."

First-Quarter and Recent Company Highlights

Announced New TEPEZZA Pooled Efficacy Data: In March 2020, new pooled efficacy data from the Phase 2 and 3 clinical trials of TEPEZZA were presented at a virtual ENDO 2020 news conference in lieu of the ENDO 2020, the Endocrine Society’s annual meeting, which was cancelled due to COVID-19. The analysis was conducted to determine if there were any differences in proptosis response to TEPEZZA based on patient demographic characteristics. The results showed that the medicine effectively reduces proptosis in TED patients, regardless of age, gender and smoking status, which are among several risk factors of the disease.

Expanded the Company’s Pipeline with Acquisition of Development-Stage Candidate HZN-825: On April 1, 2020, the Company completed the acquisition of Curzion Pharmaceuticals, Inc. and its lysophosphatidic acid 1 receptor (LPAR1) antagonist candidate (renamed HZN-825) for the treatment of diffuse cutaneous systemic sclerosis (dcSSc). A positive signal was observed in the 8-week placebo-controlled Phase 2a trial and data from the subsequent 24-week open-label extension period suggests that longer duration of treatment may demonstrate a meaningful benefit.

Launched PROCYSBI Delayed-Release Oral Granules in Packets: In April 2020, the Company launched PROCYSBI Delayed-Release Oral Granules in Packets after receiving FDA approval in February 2020. The new dosage form provides another administration option in addition to the PROCYSBI capsules for adults and children one year of age and older living with nephropathic cystinosis. The tear-open packets offer a convenient option for cystinosis patients who may have difficulty swallowing or have to administer medication through a gastrostomy tube (G-tube). Additionally, being able to access PROCYSBI granules in tear-open packets may help reduce the burden of managing multiple daily medications often faced by families living with cystinosis.

Acquired Certain Rights to Proceeds from Future Milestones and Royalties Related to TEPEZZA: In April 2020, in two separate transactions, the Company acquired the rights to proceeds from the future milestones and royalties related to TEPEZZA net sales from each of S.R. One and Lundbeckfond in exchange for an aggregate of $110 million. These transactions relate to the rights to approximately 71 percent of the $225 million in milestone payments due upon achievement of certain TEPEZZA annual worldwide net sales thresholds and approximately 71 percent of the 3 percent royalty tied to the portion of TEPEZZA annual worldwide net sales exceeding $300 million.
Award: In April 2020, Great Place to Work and Fortune selected Horizon as one of the 2020 Best Workplaces in Health Care and Biopharma for the third consecutive year. The ranking is based on input from nearly 800,000 employees in the industry and evaluates multiple elements, including the extent to which employees trust leaders, the respect with which people are treated and the fairness of workplace decisions.

Research and Development Programs

HZN-825 dcSSc Program: HZN-825 is the Company’s LPAR1 antagonist in development for the treatment of dcSSc, a rare, chronic autoimmune disease marked by fibrosis, or skin thickening, in areas including hands, forearms, upper arms and thighs with no FDA-approved treatment options. The Company expects to begin a Phase 2b pivotal trial in the first half of 2021.

New TEPEZZA Trial in Fibrotic TED: The Company is planning to initiate a single-arm, open-label trial of TEPEZZA in patients with fibrotic TED (previously referred to as inactive TED). In fibrotic TED, the disease is no longer progressive or inflammatory; however, significant disease manifestations such as proptosis (eye bulging) and diplopia (double vision) remain.

New Potential TEPEZZA Subcutaneous Administration Program: The Company is planning to initiate a pharmacokinetic trial to explore subcutaneous dosing of TEPEZZA, which is currently administered by infusion. The objective of the trial is to inform the potential for additional administration options for TEPEZZA, which could provide greater flexibility for patients and physicians.

TEPEZZA dcSSc Exploratory Trial: As part of its evaluation of additional indications for TEPEZZA, the Company is planning to initiate an exploratory trial in dcSSc. The Company expects to initiate the trial by the end of 2020.

KRYSTEXXA MIRROR Randomized Clinical Trial: The Company is currently evaluating the coadministration of KRYSTEXXA with methotrexate to increase the complete response rate of KRYSTEXXA in the MIRROR placebo-controlled randomized clinical trial (RCT), initiated in June 2019. The registrational trial is designed to enable the potential submission of results to the FDA to update the prescribing information. The MIRROR RCT follows the initial MIRROR open-label trial completed in 2019 that demonstrated a 79 percent complete response rate for patients using KRYSTEXXA with methotrexate. The 79 percent response rate is nearly double the 42 percent response rate in the KRYSTEXXA Phase 3 clinical program, which evaluated KRYSTEXXA alone. The combination was also well tolerated in the MIRROR open-label trial. Methotrexate is the immunomodulator most used by rheumatologists and has been shown to reduce anti-drug antibody formation to biologic therapies when used in conjunction with these therapies. The MIRROR RCT is approximately 80 percent enrolled and the Company expects to complete enrollment in the second half of 2020 with data available in 2021.

KRYSTEXXA PROTECT Trial in Kidney Transplant Patients with Uncontrolled Gout:

The Company is evaluating the effect of KRYSTEXXA on serum uric acid levels in kidney transplant patients with uncontrolled gout in its PROTECT open-label clinical trial, initiated in October 2019. Kidney transplant patients have more than a tenfold increase in the prevalence of gout when compared to the general population, and literature suggests that persistently high serum uric acid levels can be associated with organ rejection. Managing uncontrolled gout is one of the most common and significant unmet needs of kidney transplant patients.

KRYSTEXXA Shorter-Infusion Duration Trial: The Company is planning to initiate an open-label trial to evaluate the impact of administering KRYSTEXXA over a significantly shorter infusion duration. Currently, KRYSTEXXA is infused over a two-hour or longer timeframe. A shorter infusion duration could meaningfully improve the experience and convenience for patients, physicians and sites of care. The Company expects to initiate the trial by the end of 2020.

Next-Generation Programs for Uncontrolled Gout: The Company is pursuing early-stage development programs for next-generation biologics for uncontrolled gout to support and sustain the Company’s market leadership in this area. These include HZN-003 and HZN-007, as well as a collaboration with HemoShear Therapeutics, LLC, to discover new targets for gout.

First-Quarter Financial Results

Note: For additional detail and reconciliation of non-GAAP financial measures to the most directly comparable GAAP financial measures, please refer to the tables at the end of this release.

Net Sales: First-quarter 2020 net sales were $355.9 million, an increase of 27 percent.

Gross Profit: Under U.S. GAAP, the first-quarter 2020 gross profit ratio was 72.6 percent compared to 68.6 percent in the first quarter of 2019. The first-quarter 2020 non-GAAP gross profit ratio was 90.0 percent compared to 89.8 percent in the first quarter of 2019.

Operating Expenses: First-quarter 2020 research and development (R&D) expenses were 7.6 percent of net sales and selling, general and administrative (SG&A) expenses were 69.6 percent of net sales. Non-GAAP R&D expenses were 5.8 percent of net sales, and non-GAAP SG&A expenses were 54.2 percent of net sales.

Income Tax Rate: The first-quarter 2020 income tax rate on a GAAP basis was 58.3 percent and the income tax expense rate on a non-GAAP basis was 12.8 percent.

Net Income (Loss): On a GAAP basis, first-quarter 2020 net loss was $13.6 million. First-quarter 2020 non-GAAP net income was $83.2 million.

Adjusted EBITDA: First-quarter 2020 adjusted EBITDA was $107.2 million.

Earnings (Loss) per Share: On a GAAP basis, diluted loss per share (EPS) in the first quarter of 2020 and 2019 were $0.07 and $0.19, respectively. Non-GAAP diluted earnings per share in the first quarter of 2020 and 2019 were $0.40 and $0.30, respectively. Weighted average shares outstanding used for calculating GAAP and non-GAAP diluted earnings per share in the first quarter of 2020 were 190.1 million and 213.1 million, respectively.

First-Quarter Segment Results

Management uses net sales and segment operating income to evaluate the performance of the Company’s two segments, the orphan segment and the inflammation segment. While segment operating income contains certain adjustments to the directly comparable GAAP figures in the Company’s consolidated financial results, it is considered to be prepared in accordance with GAAP for purposes of presenting the Company’s segment operating results. Prior to the first quarter of 2020, the two operating segments were the orphan and rheumatology segment, which included RAYOS, and the inflammation segment. Beginning with the first quarter of 2020, RAYOS was moved to the inflammation segment and the orphan and rheumatology segment was renamed the orphan segment.

First-quarter 2020 net sales of the orphan segment, the Company’s strategic growth segment, were $245.4 million, an increase of 47 percent over the prior year’s quarter, driven by and strong continued growth of KRYSTEXXA and RAVICTI, as well as the launch of TEPEZZA.

First-quarter 2020 orphan segment operating income was $54.4 million, which includes significant investment spend associated with the commercial launch of TEPEZZA.

In June 2019, the Company divested the rights to MIGERGOT.

First-quarter 2020 net sales of the inflammation segment were $110.5 million and segment operating income was $51.9 million.

Cash Flow Statement and Balance Sheet Highlights

On a GAAP basis in the first quarter of 2020, cash used in operating activities was $62.6 million. On a non-GAAP basis, cash used in operating activities was $62.4 million.

The Company had cash and cash equivalents of $754.6 million as of March 31, 2020.

As of March 31, 2020, the total principal amount of debt outstanding was $1.418 billion, consisting of $418 million in senior secured term loans due 2026, $600 million of senior notes due 2027 and $400 million of exchangeable senior notes due 2022. As of March 31, 2020, net debt was $663.4 million and the net-debt-to-last-12-months adjusted EBITDA leverage (net leverage) ratio was 1.3 times. The Company has no maintenance covenants on its debt.

Revised 2020 Guidance

The Company now expects full-year 2020 net sales to range between $1.40 billion and $1.45 billion, an increase from the previous range of $1.40 billion to $1.42 billion, reflecting the significant increase in full-year TEPEZZA net sales guidance, offset by the current estimated impact of COVID-19 on its medicines. The Company now expects TEPEZZA full-year net sales of greater than $200 million, compared to the previous guidance of $30 million to $40 million. Full-year 2020 adjusted EBITDA is now expected to range between $450 million and $500 million, revised from the previous guidance range of $485 million to $500 million and reflects increased investment in TEPEZZA to support higher-than-expected demand, new TEPEZZA clinical programs and HZN-825. The updated guidance assumes that healthcare activity begins to return in the second half of 2020.

Webcast

At 8 a.m. EST / 1 p.m. IST today, the Company will host a live webcast to review its financial and operating results and provide a general business update. The live webcast and a replay may be accessed at View Source Please connect to the Company’s website at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast. A replay of the webcast will be available approximately two hours after the live webcast.

On May 6, 2020 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported first quarter financial results for 2020 (Press release, Precigen, MAY 6, 2020, View Source [SID1234557144]).

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First Quarter Business Highlights:

PRGN-2009 AdenoVerse Immunotherapy: Precigen announced that the US Food and Drug Administration (FDA) cleared the Investigational New Drug (IND) application to initiate a Phase 1/2 trial for PRGN-2009, a first-in-class, off-the-shelf investigational immunotherapy utilizing the AdenoVerse platform and designed to activate the immune system to recognize and target HPV-positive solid tumors. The Phase 1 portion of the study will follow a 3+3 dose escalation design to evaluate the safety of PRGN-2009 administered as a monotherapy and to determine the recommended Phase 2 dose (R2PD) followed by an evaluation of the safety of the combination of PRGN-2009 at the R2PD and bintrafusp alfa (M7824), an investigational bifunctional fusion protein, in patients with recurrent or metastatic HPV-associated cancers;

PRGN-3005 UltraCAR-T: Dosing in the second dose level of the intraperitoneal (IP) arm of the Phase 1 trial of PRGN-3005 UltraCAR-T was completed;

PRGN-3006 UltraCAR-T: Enrollment of patients in the non-lymphodepletion and lymphodepletion arms of the Phase 1 trial of PRGN-3006 UltraCAR-T, has been unaffected by the COVID-19 pandemic to date. The IND has been amended, and the FDA has allowed for concurrent dosing of patients in both arms; and

In order to further Precigen’s efforts to focus resources on its healthcare programs and as a result of market uncertainty driven by the COVID-19 pandemic and the current state of the energy sector, MBP Titan LLC, a wholly-owned subsidiary of Precigen focused on methane bioconversion, has significantly reduced its resource requirements through a workforce reduction. These actions will significantly decrease cash burn while maintaining intellectual property.

First Quarter 2020 Financial Highlights:

Total revenues of $29.8 million;

Net loss from continuing operations attributable to Precigen of $29.9 million, or $(0.19) per basic share, of which $8.7 million was for non-cash charges; and

Cash, cash equivalents, and short-term investments totaled $149.2 million at March 31, 2020.

"This is the first full quarter operating as the new Precigen, and we have made tremendous progress in consolidating operations and adhering to our operating priniciples to deliver value to all stakeholders," said Helen Sabzevari, PhD, President and CEO of Precigen. "From a clinical perspective, we are incredibly pleased to receive the third IND clearance for a Precigen asset in just over one year. From an operational perspective, we’ve achieved significant progress in streamlining our healthcare operations. This helps us focus our capital allocation to ensure that we have a solid runway for maximum value creation."

First Quarter 2020 Financial Results Compared to Prior Year Period

Total revenues increased $7.3 million over the quarter ended March 31, 2019. Collaboration and licensing revenues increased $4.8 million, or 80%, over the quarter ended March 31, 2019 primarily due to the accelerated recognition of previously deferred revenue upon the mutual termination of a collaboration with Fibrocell Science, Inc., in February 2020. This increase was partially offset by a decrease in collaboration revenues related to programs that were paused in 2019. Service revenues increased $2.6 million, or 23%, over the quarter ended March 31, 2019 primarily due to increased service revenues at Precigen’s subsidiary, Trans Ova Genetics L.C., due to an increase in services performed for new and existing customers and the expansion of its commercial dairy business.

Research and development expenses decreased $8.0 million, or 30%. Salaries, benefits and other personnel costs decreased $2.1 million, and contract research organization costs and lab supplies decreased $5.1 million as Precigen narrowed its focus on its primary healthcare programs. Selling, general and administrative expenses decreased $8.0 million, or 26%. Salaries, benefits and other personnel costs decreased $4.8 million primarily due to a reduction of corporate employees in the first quarter of 2020 as Precigen scaled down its corporate functions. Additionally, professional fees decreased $3.6 million primarily due to the expiration of the services agreement with Third Security, LLC on December 31, 2019.

More information on Precigen’s first quarter financial results will be available in our Quarterly Report on Form 10-Q, which we expect to file by May 11, 2020.

Conference Call and Webcast

Precigen will host a conference call today Wednesday, May 6th at 4:15 PM ET to discuss the results and provide a general business update. The conference call may be accessed by dialing 1-833-646-0488 (US/Canada toll-free) or 1-918-922-6615 to join the Precigen Conference Call. Participants are asked to dial in 10-15 minutes in advance of the scheduled call time to facilitate timely connection to the call. Participants may also access the live webcast through Precigen’s website in the Events section at View Source

Precigen: Advancing Medicine with Precision

Precigen (Nasdaq: PGEN) is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cell therapies using precision technology to target urgent and intractable diseases in our core therapeutic areas of immuno-oncology, autoimmune disorders, and infectious diseases. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well-differentiated unique therapies toward clinical proof-of-concept and commercialization. For more information about Precigen, visit www.precigen.com or follow us on Twitter @Precigen and LinkedIn.

Trademarks

Precigen, AdenoVerse, UltraCAR-T, and Advancing Medicine with Precision are trademarks of Precigen and/or its affiliates. Other names may be trademarks of their respective owners.

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.

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

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"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.

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.

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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.