On May 6, 2020 Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported financial results and provided a business update for the first quarter ended March 31, 2020 (Press release, Blueprint Medicines, MAY 6, 2020, View Source [SID1234557148]).

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"Our progress in recent months is a testament to the agility of the Blueprint Medicines team and the strength of our fully-integrated business," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "Despite challenges related to the COVID-19 pandemic and top-line results from the VOYAGER trial, we broadly executed against our planned milestones, including the submission of marketing applications for pralsetinib for RET fusion-positive non-small cell lung cancer in the U.S. and Europe and the reporting of transformative datasets for avapritinib in systemic mastocytosis and pralsetinib in RET-altered cancers, paving the way for multiple planned launches through 2021."

First Quarter 2020 Highlights and Recent Updates

Avapritinib: systemic mastocytosis (SM)

Announced results from Part 1 of the Phase 2 PIONEER trial in patients with indolent SM, which showed treatment with avapritinib at the recommended Part 2 dose of 25 mg once daily showed improvements in symptom scores, measures of mast cell burden and patient-reported quality of life. Avapritinib was well-tolerated, and no patients discontinued treatment due to an adverse event (AE). Read the press release here.
Avapritinib: gastrointestinal stromal tumors (GIST)

Launched AYVAKIT in the U.S. for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutations, including PDGFRA D842V mutations, and achieved net product revenue of $3.5 million in the first partial quarter of the launch.
Announced top-line data from the Phase 3 VOYAGER trial of avapritinib versus regorafenib in third- or fourth-line GIST. The VOYAGER trial did not meet its primary endpoint of an improvement in progression-free survival for avapritinib versus regorafenib. Based on the top-line results, Blueprint Medicines plans to discontinue further development of avapritinib in GIST indications other than PDGFRA exon 18 mutant GIST. Read the press release here.
Pralsetinib: RET-altered cancers

Announced top-line data from the Phase 1/2 ARROW trial of pralsetinib in patients with RET fusion-positive non-small cell lung cancer (NSCLC). The top-line data showed overall response rates (ORRs) of 61 percent in patients previously treated with platinum-based chemotherapy and 73 percent in patients naïve to prior systemic therapy. In both populations, the median duration of response (DOR) was not reached. Pralsetinib was well-tolerated, and most AEs were grade 1 or 2. Read the press release here.
Completed the submission of a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) and a marketing authorization application to the European Medicines Agency for pralsetinib for the treatment of patients with RET fusion-positive NSCLC. In connection with the NDA submission, Blueprint Medicines plans to participate in the FDA’sProject Orbis initiative, which provides a framework for concurrent submission and review of marketing applications for oncology drugs across participating global health authorities. Additional information about the FDA’sProject Orbis initiative can be found here.
Announced top-line data from the Phase 1/2 ARROW trial of pralsetinib in patients with RET mutant medullary thyroid cancer (MTC). The top-line data showed ORRs of 60 percent in patients previously treated with a multi-kinase inhibitor and 74 percent in patients naïve to prior systemic treatment. In both populations, the median DOR was not reached. Pralsetinib was well-tolerated, and most AEs were grade 1 or 2. Read the press release here.
BLU-263: SM

Received clearance from the FDA for an investigational new drug (IND) application for BLU-263 for the treatment of patients with indolent SM.
Key Upcoming Milestones

The company expects to achieve the following near-term milestones:

Submit an NDA to the FDA for pralsetinib for the treatment of patients with MTC previously treated with an approved multi-kinase inhibitor in the second quarter of 2020 under the FDA’sOncology Center of Excellence Real-Time Oncology Review pilot program (RTOR program). The FDA’s RTOR program aims to explore a more efficient review process to ensure that safe and effective treatments are available to patients as early as possible, while maintaining and improving review quality by the FDA. Additional information about the FDA’s RTOR program can be found here.
Present updated data from the ARROW trial of pralsetinib in RET-altered cancers at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
Present additional data from Part 1 of the PIONEER trial of avapritinib in indolent SM at the European Academy of Allergy and Clinical Immunology (EAACI) 2020 Congress.
Present updated data from the EXPLORER trial of avapritinib in advanced SM at the European Hematology Association (EHA) (Free EHA Whitepaper) 25th Annual Congress.
Initiate patient screening for the registration-enabling Part 2 of the PIONEER trial of avapritinib in indolent SM in June 2020.
Initiate a Phase 1 trial of BLU-263 in healthy volunteers in June 2020.
Report top-line data from the EXPLORER and PATHFINDER trials of avapritinib in advanced SM in the third quarter of 2020.
Response to the COVID-19 Pandemic and Potential Business Impacts

Due to the evolving and uncertain global impacts of the COVID-19 pandemic, Blueprint Medicines cannot precisely determine or quantify the impact this pandemic will have on its business, operations (including clinical trials) and financial performance for the remainder of our fiscal year ending December 31, 2020 and beyond.

For ongoing and planned clinical trials, while the company anticipates and has experienced some temporary delays or disruptions due to the COVID-19 pandemic, Blueprint Medicines is working with any impacted clinical trial sites to ensure study continuity.
Blueprint Medicines currently has sufficient supply to meet anticipated global commercial and clinical development needs for avapritinib, pralsetinib, fisogatinib and BLU-263 through 2021. However, the COVID-19 pandemic could adversely impact Blueprint Medicines’ suppliers and result in delays or disruptions in the company’s current or future supply chain.
Blueprint Medicines has shifted commercial and medical affairs field activities across its portfolio toward virtual formats where possible in order to allow the company to continue to serve the needs of healthcare providers, patients and other stakeholders during this critical time. As the pandemic evolves, the company anticipates utilizing a mix of in-person and virtual engagement formats, as is locally and regionally appropriate.
Blueprint Medicines will continue to assess potential impacts of the COVID-19 pandemic and seek to advance the company’s pipeline of targeted therapies as quickly as possible, while making the health and safety of the company’s employees and their families, healthcare providers, patients and communities a top priority.

First Quarter 2020 Financial Results

Cash Position: As of March 31, 2020, cash, cash equivalents and investments were $750.4 million, as compared to $548.0 million as of December 31, 2019. This increase was primarily related to $308.4 million in estimated net proceeds received from the company’s January 2020 follow-on underwritten public offering, partially offset by cash used in operating activities.
Revenues: Revenues were $6.2 million for the first quarter of 2020, including $3.5 million of net product revenues from sales of AYVAKIT and $2.7 million in collaboration revenues under the collaboration agreements with CStone and Roche. Blueprint Medicines recorded $0.7 million in collaboration revenues for the first quarter of 2019.
Cost of Sales: Cost of sales was less than $0.1 million for the first quarter of 2020. Blueprint Medicines did not incur cost of sales in the first quarter of 2019 as no product sales were generated during that period.
R&D Expenses: Research and development expenses were $84.1 million for the first quarter of 2020, as compared to $74.3 million for the first quarter of 2019. This increase was primarily due to increased clinical and manufacturing costs and personnel expenses driven by the progression of Blueprint Medicines’ lead programs. Research and development expenses included $7.8 million in stock-based compensation expenses for the first quarter of 2020.
SG&A Expenses: Selling, general and administrative expenses were $35.7 million for the first quarter of 2020, as compared to $16.6 million for the first quarter of 2019. This increase was primarily due to increased costs and personnel expenses associated with building Blueprint Medicines’ commercial infrastructure for the commercialization of AYVAKIT and to support the overall growth of the company’s business. General and administrative expenses included $9.1 million in stock-based compensation expenses for the first quarter of 2020.
Net Loss: Net loss was $111.0 million for the first quarter of 2020, or a net loss per share of $2.11, as compared to a net loss of $87.4 million for the first quarter of 2019, or a net loss per share of $1.98.
Financial Guidance

Based on its current operating plans, Blueprint Medicines expects that its existing cash, cash equivalents and investments, together with anticipated product revenues but excluding any potential option fees, milestone payments or other payments under its collaboration or license agreements, will be sufficient to enable it to fund its operating expenses and capital expenditure requirements into the second half of 2022.

Conference Call Information

Blueprint Medicines will host a live conference call and webcast at 8:30 a.m. ET today to discuss first quarter 2020 financial results and recent business activities. The conference call may be accessed by dialing (855) 728-4793 (domestic) or (503) 343-6666 (international), and referring to conference ID 7980947. A webcast of the call will be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.

On May 6, 2020 Saniona reported that its interim report for the first quarter 2020 (Press release, Saniona, MAY 6, 2020, View Source [SID1234557146])

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Financial highlights

Q1 2020 (Q1 2019)
· Net revenues were SEK 0.5 M (1.7 M)
· EBIT was SEK -30.6 M (-29.1 M)
· Net profit/loss was SEK -28.2 M (-24.8)
· Earnings per share were SEK -0.96 (-1.06)
· Diluted earnings per share were SEK -0.96 (-1.06)

Business highlights in Q1 2020

In January, Saniona appointed Rami Levin as its President and Chief Executive Officer. Rami Levin will oversee the transformation of Saniona to a fully integrated rare disease biopharmaceutical company. He has extensive commercial experience in CNS and rare diseases, both in U.S. and globally. Jørgen Drejer, previous CEO, was appointed Chief Scientific Officer.
In January, Saniona completed a private placement of SEK 25 million and proposed a financing of up to SEK 158 million comprising a combination of the directed issue and rights issue of warrants totaling SEK 111 million – 133 million at a strike price of SEK 25 – 30 per share as well as a loan facility of SEK 25 million.
On February 18, 2020, Saniona co-founded new migraine therapy company Cephagenix.
CFO Thomas Feldthus has left the company and Saniona has initiated a search for a new, U.S.-based CFO. Anita Milland, the current VP of Finance and Administration, was appointed interim CFO and Head of IR, and Jørgen Drejer assumed the role of Deputy CEO.
On 18 March 2020, Saniona announced the completion of its the six-month double-blind Phase 2 trial of Tesomet in hypothalamic obesity.
In March, Saniona signed a second research collaboration agreement with Boehringer Ingelheim in schizophrenia.

Significant events after the reporting period

In April, Saniona reported positive topline results from its Phase 2 Trial of Tesomet in Hypothalamic Obesity. The double-blind placebo controlled results showed that Tesomet was safe and well tolerated and on its main efficacy end points it showed that Tesomet was statistically significantly different than placebo.
Saniona reported the appointment of Rudolf Baumgartner, M.D., as Chief Medical Officer and Head of Clinical Development. Dr. Baumgartner will be based in the US, along with CEO Rami Levin.

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.