On May 6, 2020 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, reported its financial results for the quarter ended March 31, 2020 and maintained its full year 2020 financial guidance (Press release, Ultragenyx Pharmaceutical, MAY 6, 2020, View Source [SID1234557128]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The global coronavirus pandemic has created unprecedented challenges for the world at large, and I am proud of the company’s continued efforts to assure an uninterrupted supply of our therapies" said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "We delivered strong financial results in the first quarter, and our commercial business so far has been manageably impacted and resilient, though the global crisis continues. The FDA reviews for Crysvita for TIO and UX007 for LC-FAOD are on track to be completed in the coming months. Our gene therapy franchise has recently been bolstered by our recent manufacturing collaboration with Daiichi Sankyo, and our two clinical-stage gene therapy programs continue to proceed."

COVID-19 Update
As the COVID-19 pandemic continues to evolve, the top priority for Ultragenyx is the health and safety of its extended community, including patients, caregivers, healthcare providers, partners, and employees. To date, the company has been able to maintain an uninterrupted supply of medicine to patients around the world as no significant disruptions to manufacturing or distribution activities have been experienced. Even if there were an interruption in the supply chain, the company does not anticipate a shortage of medicine in the near term due to ample levels of inventory currently available.

First Quarter 2020 Financial Results

Net Revenues
For the first quarter of 2020, Ultragenyx reported $36.3 million in total revenue. Ultragenyx recognized $28.8 million in total Crysvita revenue for Ultragenyx territories, which includes $27.2 million in collaboration revenue in the North American profit share territory and net product sales in other regions of $1.6 million. Non-cash royalty revenue related to European Crysvita royalties were $2.6 million. Mepsevii (vestronidase alfa) product revenue for the first quarter of 2020 was $3.4 million, and UX007 named patient revenue was $1.4 million.

Operating Expenses
Total operating expenses for the first quarter of 2020 were $157.0 million, which includes $25.0 million to maintain the option to acquire GeneTx after the acceptance of the investigational new drug application (IND) for GTX-102, $7.0 million for an exclusive, sublicensable, worldwide license to NAV AAV8 and AAV9 Vectors from REGENXBIO, Inc and non-cash stock-based compensation of $20.2 million. The cost of sales for the three months ended March 31, 2020 was negative $3.5 million due to a credit with an estimated value of $4.6 million that was agreed to during the quarter with a contract manufacturer in exchange for certain Mepsevii inventory batches that did not meet specified quality standards in prior quarters. Total operating expenses in the first quarter of 2019 were $117.4 million, which includes non-cash stock-based compensation of $20.2 million. The increase in total operating expenses is due to the increase in commercial, development, and general and administrative costs as the company commercializes, grows, and advances its portfolio.

For the first quarter of 2020, Ultragenyx reported a net loss of $119.0 million, or $2.05 per share, basic and diluted, compared with a net loss for the first quarter of 2019 of $96.8 million, or $1.82 per share, basic and diluted. The loss for the first quarter of 2020 includes unrealized gains of $7.7 million from the fair value adjustments on the investment in Arcturus equity securities and non-cash interest expense on the liability related to the sale of future royalties of $8.1 million. The net loss for the first quarter of 2020 reflected cash used in operations of $95.2 million compared to $95.8 million for the same period in 2019.

Cash, Cash Equivalents and Investments
Cash, cash equivalents, and investments were $705.0 million as of March 31, 2020. This includes $75.0 million from the Daiichi Sankyo common stock purchase which closed in March 2020, but excludes the $125.0 million upfront license payment from the Daiichi Sankyo strategic manufacturing partnership which was received in April 2020.

2020 Guidance

Crysvita Guidance in Ultragenyx Territories
The company currently maintains the guidance range for 2020 Crysvita revenue in the Ultragenyx territories to be between $125.0 million and $140.0 million, although it continues to monitor the COVID-19 pandemic situation. Ultragenyx territories include the collaboration revenue from the North American profit share territory (U.S. and Canada) and other regions where revenue from product sales are recognized by Ultragenyx (Latin America, Turkey). This excludes the European territory revenue, which is recognized as non-cash royalty revenue since the rights were sold to Royalty Pharma in December 2019.

2020 Expected Net Cash Burn Rate
The company also expects a more than 20 percent reduction in net cash burn (net cash used in operations plus capital expenditures) in 2020 compared to 2019 due to a combination of financial discipline in spending with flattening operating expense growth, combined with a significant anticipated growth in revenue.

Corporate Update

Gene Therapy Manufacturing: Strategic partnership and non-exclusive license and technology access agreement with Daiichi Sankyo

Ultragenyx granted Daiichi Sankyo a non-exclusive license on March 31, 2020 to intellectual property, including know-how and patent applications, with respect to its HeLa PCL and HEK293 transient transfection manufacturing technology platforms for AAV-based gene therapy products. Daiichi Sankyo made an upfront payment in April 2020 of $125.0 million pursuant to the license agreement and in March 2020 closed on the purchase of 1,243,913 shares of Ultragenyx common stock in exchange for $75.0 million, or an effective purchase price of approximately $60 per share. Daiichi Sankyo will pay an additional $25.0 million upon completion of the technology transfer of the HeLa PCL and HEK293 platforms, as well as single-digit royalties on net sales of products manufactured in either system.
Program Updates and Upcoming Milestones

UX007 for Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD): New Drug Application (NDA) under review by U.S. FDA

The U.S. FDA is currently reviewing the NDA and has set a Prescription Drug User Fee Act (PDUFA) date of July 31, 2020. The review process is currently on track for a decision by the PDUFA date. The anticipated clinical and manufacturing inspections for UX007 have been successfully completed and, at this time, the FDA has not indicated that any additional inspections would be required for completion of their review.
Crysvita for Tumor-Induced Osteomalacia (TIO): Supplemental Biologics License Application (sBLA) under review by U.S. FDA

The U.S. FDA accepted for review the sBLA and has set a PDUFA date of June 18, 2020. The review process is currently on track for a decision by the PDUFA date and the FDA has not indicated, at this time, that any inspections would be required for completion of their review.
DTX301 for Ornithine Transcarbamylase (OTC) Deficiency: Positive data from higher dose cohort of Phase 1/2 study; prophylactic steroid cohort planned and Phase 3 study planning underway

Positive data from this study indicated two confirmed responders and a potential third responder out of three total patients in Cohort 3, as well as a new responder in Cohort 2. There are currently up to six responders of the nine dosed to date with a more consistent response at higher doses. An update with currently available data from the first three cohorts of the study will be presented at the virtual American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) on May 13.
A fourth cohort of three patients at the same 1.0 × 10^13 GC/kg dose is planned, using prophylactic steroids. Dosing in this cohort is currently on hold, but data are still expected in the second half of 2020, barring significant further delays related to COVID-19. Ultragenyx is simultaneously planning the Phase 3 study and is currently planning to hold an end-of-phase 2 meeting with the FDA based on the first three cohorts. The Phase 3 study is currently expected to begin enrollment in the first half of 2021.
DTX401 for Glycogen Storage Disease Type Ia (GSDIa): Positive data from Phase 1/2 study; enrollment complete in confirmatory cohort and data expected in second quarter 2020

Enrollment is complete in the confirmatory cohort of three patients at the second dose level (6.0 x 10^12 GC/kg). An update with currently available data from the confirmatory cohort will be presented at the ASGCT (Free ASGCT Whitepaper) meeting on May 15. Certain data may not be available for later patient site visits due to COVID-19 related delays. Depending on the results of the confirmatory cohort, a Phase 3 study could begin in late 2020.
GTX-102 for Angelman Syndrome (AS): Partnered program with GeneTx; First two patients enrolled

The two patients in the first cohort of the Phase 1/2 study of GTX-102 have been enrolled and continue through dose escalation. Preliminary data are expected in first half of 2021.
In February 2020, Ultragenyx paid $25.0 million after the acceptance of the IND to maintain its option to acquire GeneTx until the earlier of 30 months from the first dosing of a patient in the Phase 1/2 study (subject to extensions) or 90 days after results are available from that study.
In May 2020, the U.S. FDA granted Fast Track designation to GTX-102 for the treatment of Angelman syndrome.
UX701 for Wilson Disease: IND anticipated by the end of the year

An IND application for UX701 gene therapy for Wilson disease, a larger rare metabolic disease, is expected by the end of 2020 barring any delays in nonclinical or manufacturing activities. UX701 will be the second clinical program to utilize the company’s HeLa manufacturing system, in addition to the hemophilia A program being developed by our collaborative partner, Bayer.
Conference Call and Webcast Information

Ultragenyx will host a conference call today, Wednesday, May 6, 2020, at 2 p.m. PT/ 5 p.m. ET to discuss first quarter 2020 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 9455159. The replay of the call will be available for one year.

On May 6, 2020 Celldex Therapeutics, Inc. (NASDAQ:CLDX) reported business and financial highlights for the first quarter ended March 31, 2020 (Press release, Celldex Therapeutics, MAY 6, 2020, View Source [SID1234557127]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Despite the ongoing challenges associated with the COVID-19 pandemic, Celldex continued to make considerable progress advancing our pipeline in the first quarter of 2020," said Anthony Marucci, Co-founder, President and Chief Executive Officer of Celldex Therapeutics. "Notably, the Company completed enrollment and treatment of all subjects in the Phase 1 healthy volunteer study of our KIT inhibitor, CDX-0159, which we intend to study in mast cell driven disorders. Results from this study have been accepted as a late-breaking poster presentation with voice over at the EAACI Annual Congress 2020 and will be presented in early June by Dr. Marcus Maurer, a leading medical expert in urticaria. We are excited about the promising data observed to date and, based on these results, have expanded development of CDX-0159. We are planning to initiate studies in chronic urticaria later this year.

"We are also completing preparations to advance CDX-527, the first candidate from our bispecific platform, into a Phase 1 study in refractory, advanced cancers and look forward to initiating this study in the second half of 2020. Finally, we continue to enroll patients in the ongoing studies of CDX-1140 and CDX-3379 and plan to report data updates from these programs later this year," concluded Marucci.

Recent Pipeline Highlights:

CDX-0159—a monoclonal antibody that specifically binds the KIT receptor and potently inhibits its activity. The KIT receptor tyrosine kinase is expressed in a variety of cells, including mast cells. In certain inflammatory diseases, such as chronic urticarias, mast cell degranulation plays a central role in the onset and progression of the disease.

Enrollment and treatment was recently completed in the ongoing Phase 1 randomized, double-blind, placebo-controlled, single ascending dose escalation study of CDX-0159 in healthy subjects. Subjects received a single intravenous infusion of CDX-0159 at 0.3, 1, 3, or 9 mg/kg or placebo. This study is designed to evaluate the safety profile, pharmacokinetics and pharmacodynamics of CDX-0159 and to select a dose for further study in mast cell driven diseases. The Phase 1 study also evaluates plasma tryptase levels in healthy subjects. Tryptase is an enzyme synthesized and secreted by mast cells and decreases in plasma tryptase levels reflect a systemic reduction in mast cell burden, even in healthy volunteers. If CDX-0159 is able to decrease systemic mast cell load in healthy volunteers, Celldex believes the drug candidate could have significant potential in mast cell driven diseases. Based on promising results observed to date, Celldex is expanding development of CDX-0159 into both chronic spontaneous urticaria (CSU) and chronic inducible urticaria (CINDU).

— Results from the Phase 1 study of CDX-0159 have been accepted as a late-breaking poster presentation with voice over at the European Academy of Allergy and Clinical Immunology (EAACI) Annual Congress 2020, which this year will be held digitally June 6-8, 2020. Following the press release issued this morning announcing the presentation, Celldex became aware that the full abstracts had been temporarily published on the EAACI website ahead of the lifting of the embargo. Information included in the published abstract was current as of the time of submission in March and did not include the full results that will be presented in June. Results published in the abstract outlined that mild infusion reactions were the most common adverse event occurring in 50% of subjects and did not appear dose related. The infusion reactions spontaneously resolved shortly after the infusions were completed. Transient, mild decreases in hemoglobin and white blood cell count were observed, which spontaneously resolved without intervention. Remarkably, patients who received a single 0.3 mg/kg dose of CDX-0159 decreased serum tryptase—a specific marker of mast cell load—by approximately 50% from baseline for a week, while a single dose at 1 or 3 mg/kg rapidly reduced serum tryptase below the levels of assay detection for at least 3 weeks and 6 weeks, respectively. The poster presentation with voice over in June will include complete, unblinded results from the study, including the 9 mg/kg cohort and pharmacokinetic, immunogenicity and stem cell factor data. The study will be presented by Dr. Marcus Maurer, Professor of Dermatology and Allergy and Director of Research at the Department of Dermatology and Allergy at the Allergie-Centrum-Charité of the Charité – Universitätsmedizin in Berlin. Dr. Maurer is also head of the Specialty Clinics for Urticaria, Mastocytosis, Pruritus and Angioedema and the Dermatological Allergology Lab. Dr. Maurer´s research focuses on the physiological and pathological functions of mast cells.

— The Company plans to initiate Phase 1b studies of CDX-0159 in CSU and CINDU, both mast cell-related diseases, by year end. CSU presents as itchy hives, angioedema or both for at least six weeks without a specific trigger; multiple episodes can play out over years or even decades. About 50% of patients with CSU achieve symptomatic control with antihistamines or leukotriene receptor antagonists. Omalizumab, an IgE inhibitor, provides relief for roughly half of the remaining antihistamine/leukotriene refractory patients. Consequently, there is a need for more effective later line therapies. CINDUs are forms of urticaria that have an attributable cause or trigger associated with them, typically resulting in hives or wheals. Celldex is exploring cold-induced and dermographism (scratch-induced) urticarias.
CDX-1140—a potent CD40 agonist that Celldex believes has the potential to successfully balance systemic doses for good tissue and tumor penetration with an acceptable safety profile.

In the Phase 1 dose-escalation study of CDX-1140 in patients with recurrent, locally advanced or metastatic solid tumors and B cell lymphomas both the monotherapy and combination with CDX-301 dose escalation portions of the trial are complete with an identified maximum tolerated dose (MTD) and recommended Phase 2 dose of CDX-1140 at 1.5 mg/kg—one of the highest systemic dose levels in the CD40 agonist class. Expansion cohorts are actively recruiting including:

— CDX-1140 with KEYTRUDA (pembrolizumab) in patients who have progressed on checkpoint therapy; and,
— CDX-1140 with CDX-301 in patients with head and neck squamous cell carcinoma (HNSCC).

In addition, a combination of CDX-1140 with chemotherapy in first line metastatic pancreatic cancer is planned.
CDX-3379—a differentiated human monoclonal antibody designed to block the activity of ErbB3 (HER3). ErbB3 is expressed in many cancers, including HNSCC and is believed to be an important receptor regulating cancer cell growth and survival as well as resistance to targeted therapies.

Enrollment continues in the Phase 2 study of CDX-3379 in advanced HNSCC in combination with Erbitux (cetuximab) in Erbitux-resistant patients who have been previously treated with or are ineligible for checkpoint therapy. Data reported at the 2019 American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June suggested that observed antitumor activity with CDX-3379 might be associated with somatic mutations in the FAT1 and NOTCH1-3 genes—genes associated with tumor suppression. Based on these biomarker observations and the clinical activity observed in the ongoing Phase 2 study, the study was expanded (n= ~45 patients, including at least 15 patients with FAT1 mutations) to allow for an evaluation of the utility of biomarkers for patient selection.
Celldex continues to advance a robust preclinical portfolio and is preparing to advance the first candidate from the Company’s bispecific platform, CDX-527, into the clinic in the second half of 2020. CDX-527 uses Celldex’s proprietary highly active anti-PD-L1 and CD27 human antibodies to couple CD27 co-stimulation with blockade of the PD-L1/PD-1 pathway. The Phase 1 dose escalation study will enroll up to 90 patients with advanced or metastatic solid tumors that have progressed during or after standard of care therapy, followed by tumor specific expansion cohorts to further evaluate the tolerability, biologic and anti-tumor effects of selected dose level(s) of CDX 527 in specific tumor types.

Recent Business Highlights:

In March 2020, Celldex announced a milestone payment related to an existing 2013 agreement with Rockefeller University under which Celldex performed manufacturing and development services for Rockefeller University’s portfolio of broadly neutralizing antibodies (bNAbs) against HIV, including two clinical-stage candidates 3BNC117 and 10-1074. These investigational agents have potential for use in HIV long-acting therapies for treatment and prevention, as well as cure strategies. This portfolio was licensed by Gilead Sciences in January of 2020 from Rockefeller University and pursuant to Celldex’s agreement with Rockefeller, Celldex received an upfront payment of $1.8 million as a result of this transaction and is eligible to receive additional payments from Rockefeller if this portfolio progresses through clinical and commercial development. Under the terms of the agreement, Celldex utilized internal capabilities in production cell line development, process development and GMP manufacturing to support the rapid and successful execution of Rockefeller University’s clinical development of 3BNC117 and 10-1074 and contributed to the IND and clinical studies by managing key IND-enabling studies and clinical sample testing for pharmacokinetics and anti-drug antibodies. The close collaboration between Celldex and Rockefeller University allowed for efficient program development and the ability to rapidly overcome challenges.
First Quarter 2020 Financial Highlights and 2020 Guidance

Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2020 were $53.7 million compared to $64.4 million as of December 31, 2019. The decrease was primarily driven by first quarter cash used in operating activities of $12.1 million, partially offset by $1.6 million in net proceeds from sales of common stock under the Cantor agreement. At March 31, 2020, Celldex had 17.7 million shares outstanding.

Revenues: Total revenue was $2.7 million in the first quarter of 2020 compared to $1.4 million for the comparable period in 2019. The increase in revenue was primarily due to the $1.8 million milestone payment from Rockefeller University related to our manufacturing and development services agreement.

R&D Expenses: Research and development (R&D) expenses were $11.7 million in the first quarter of 2020 compared to $11.2 million for the comparable period in 2019. The increase in R&D expenses was primarily due to higher laboratory supply and clinical trial costs, offset by lower contract research costs.

G&A Expenses: General and administrative (G&A) expenses were $3.7 million in the first quarter of 2020 compared to $4.9 million for the comparable period in 2019. The decrease in G&A expenses was primarily due to lower stock-based compensation expense and professional service costs.

Changes in Fair Value Remeasurement of Contingent Consideration: During the quarter ended March 31, 2020, the Company recorded a $0.2 million loss on fair value remeasurement of contingent consideration primarily due to the passage of time. During the quarter ended March 31, 2019, the Company recorded a $1.5 million loss on the fair value remeasurement of contingent consideration primarily due to changes in discount rates and the passage of time.

Net Loss: Net loss was $12.6 million, or ($0.73) per share, for the first quarter of 2020 compared to a net loss of $17.2 million, or ($1.40) per share, for the comparable period in 2019.

Financial Guidance: Celldex believes that the cash, cash equivalents and marketable securities at March 31, 2020 are sufficient to meet estimated working capital requirements and fund planned operations into the second quarter of 2021. This guidance excludes anticipated proceeds from future sales of common stock under the Cantor agreement or other potential fundraising.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ USA. Erbitux is a registered trademark of Eli Lilly & Co.

On May 6, 2020 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported financial results for the three months ended March 31, 2020 and provided an operating forecast and program updates (Press release, Ligand, MAY 6, 2020, View Source [SID1234557126]). Ligand management will host a conference call with slides today beginning at 4:30 p.m. Eastern time to discuss this announcement and answer questions.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Our first quarter financial results feature strong sales of Captisol to partners evaluating remdesivir in multiple clinical trials and scaling-up for potential treatment courses for COVID-19. We continue to meet Captisol requirements to support these trials as well as manufacturing scale-up, and are proud to play a role in developing potential treatments to address the pandemic both with Captisol and with various OmniAb and Vernalis-derived product candidates," said John Higgins, Chief Executive Officer of Ligand. "We recently closed a strategic acquisition, and throughout the first quarter we added a number of Shots on Goal with new agreements for various technologies, in particular our OmniAb platform. Overall, our business is performing very well, especially given the difficult business environment due to the pandemic. As such, we are pleased to be raising our 2020 financial guidance."

COVID-19 Impact

As the COVID-19 pandemic continues to evolve, our primary concern remains the health and safety of our employees and partners globally, while we continue to take actions to help address the pandemic.

We are supporting our employees through a range of programs, have enabled working from home and staggered operations in our labs and other critical work spaces. Importantly, Ligand is committed that there will be no COVID-19 related layoffs. Our corporate structure is spread across five sites in the U.S. and England, which positions us well to operate effectively in the current remote working environment. The Ligand team is ready to support a return to full operations with appropriate social distancing measures in place, following the lifting of shelter-in-place restrictions.

We have a very strong balance sheet, and we anticipate no material operational impacts for the rest of the year due to COVID-19. During the first quarter, COVID-19 did not have a material negative impact on our underlying business, financial condition, cash collections or liquidity. During the first quarter we reduced our outstanding convertible debt by approximately one-third given the favorable pricing on the bonds and the lower interest rate environment.

Our partnership with Gilead Sciences for remdesivir resulted in an increase in sales of Captisol to supply the scale up and manufacturing of that medicine as the first new treatment for COVID-19 available under an Emergency Use Authorization. We do not anticipate supply chain disruption for Captisol production at this time given inventory levels, risk management measures and operations at multiple sites throughout the world. We believe we are well positioned to meet Captisol requirements and are planning to make further capital investments in plant and operational capacity.

We have a large portfolio of more than 200 programs fully funded by more than 125 different pharmaceutical and biotechnology companies. We recently surveyed all our partners and found that the majority of them are generally in a relatively strong capital and operating condition with limited expected long-term impact on our partnered programs. Looking ahead to the remainder of the year and after thoroughly analyzing our business, we anticipate royalty and contract revenue will be lower than originally forecasted. We believe that patient access to certain medicines around the world will be disrupted over several months, which may decrease revenue for products from which we earn royalties. In addition, we anticipate that some partners will delay trial initiations or experience a slowdown in patient enrollment. These delays and slowdowns will likely reduce milestone payments for contract revenue due to Ligand. In addition, some smaller partners may face cash constraints or difficulty raising new capital, which could impact their ability to make payments to Ligand. Nonetheless, as the economy begins to reopen we expect our partners will resume important clinical and regulatory work on a wide array of partnered programs. Additionally, to date the increase in sales of Captisol has more than offset our projected decline in royalty and contract revenues. While the mix of revenue will be different than our original outlook, we anticipate total revenue and earnings to be higher in 2020 compared to our previous guidance.

Ligand has multiple programs relating to potential treatments for COVID-19. One is with Gilead for remdesivir, a nucleotide analogue issued an Emergency Use Authorization by the U.S. Food and Drug Administration on May 1, 2020. We also have two OmniAb partners with antibody programs in discovery stage, and a heat shock protein program that was added to our R&D programs when we acquired Vernalis.

First Quarter 2020 Financial Results

Total revenues for the first quarter of 2020 were $33.2 million, compared with $43.5 million for the same period in 2019. Royalties for the first quarter of 2020 were $6.6 million and primarily consisted of royalties from Kyprolis and EVOMELA. Royalties for the first quarter of 2019 were $19.5 million and included $14.2 million in royalties from Promacta; Ligand sold its Promacta license to Royalty Pharma as of March 6, 2019. Captisol sales were $21.1 million for the first quarter of 2020, compared with $9.0 million for the same period in 2019, primarily reflecting higher sales of Captisol for remdesivir. Effective this quarter, Ligand is presenting service revenue as a separate line item. Service revenue includes revenue generated from our Vernalis, Icagen and OmniChicken businesses as we collaborate with partners on early stage discovery and development work, and was $3.4 million for the first quarter of 2020, compared with $3.9 million for the same period in 2019. Contract revenue was $2.1 million for the first quarter of 2020, compared with $11.1 million for the same period in 2019, with the change driven by the timing of partner events.

Cost of Captisol was $4.7 million for the first quarter of 2020, compared with $3.9 million for the same period in 2019. Amortization of intangibles was $3.5 million for the first quarter of both 2020 and 2019. Research and development expense was $11.9 million for the first quarter of 2020, compared with $11.3 million for the same period of 2019. General and administrative expense was $9.3 million for the first quarter of 2020, compared with $11.1 million for the same period in 2019, with the decrease primarily attributable to lower legal expenses.

Net loss for the first quarter of 2020 was $(24.1) million, or $(1.46) per diluted share, compared with net income of $666.3 million, or $31.32 per diluted share, for the same period in 2019. The net loss for the first quarter of 2020 includes a non-cash change in the value of Ligand’s investments of $(25.5) million, while net income for the first quarter of 2019 includes a $17.3 million net non-cash gain from the value of Ligand’s investments as well as a $640.3 million gain, net of taxes, from the sale of the Promacta license. Adjusted net income for the first quarter of 2020 was $15.3 million, or $0.89 per diluted share, compared with $24.7 million, or $1.16 per diluted share, for the same period in 2019. Please see the table below for a reconciliation of net income/(loss) to adjusted net income.

As of March 31, 2020, Ligand had cash, cash equivalents and short-term investments of $738.8 million. During the first quarter of 2020, Ligand repurchased $234 million in principal amount of its convertibles notes at a price of $203 million, and repurchased 878,525 common shares for $73.3 million.

2020 Financial Guidance

Ligand is raising its 2020 financial guidance. Ligand now expects 2020 total revenues to be approximately $140 million and diluted EPS to be $3.65, up from previous guidance for total revenues of approximately $133 million and diluted EPS of $3.62. This increase reflects Ligand’s revised view on the business incorporating an estimated impact from COVID-19. The revised estimate of approximately $140 million in total revenues includes higher sales of Captisol, partially offset by reductions in royalties and contract revenue.

First Quarter 2020 and Recent Business Highlights

Kyprolis (carfilzomib), an Amgen Product Utilizing Captisol

Amgen reported first quarter Kyprolis sales of $280 million. Ono Pharmaceutical Co. will report first quarter Kyprolis sales in Japan on May 11, 2020.
OmniAb Platform Updates

There are now more than 80 OmniAb-related Shots on Goal in Ligand’s partnered portfolio, representing over 40% of the Ligand pipeline.
OmniAb partners have filed or been issued more than 30 U.S. and international patent applications or patents, claiming OmniAb-derived antibodies as the primary invention.
The number of active or recently completed clinical trials that include an OmniAb-derived antibody reached 43, with new clinical trial starts in Q1 that included:
two new Phase 1/1b trials;
three new Phase 2 trials; and
two new Phase 3 trials.
Two COVID-19 antibody programs are being pursued by Ligand partners. One is a multinational Big Pharma partner that has initiated a program using OmniChicken, and the other is focused on antibodies derived from OmniRat.
Immunovant announced positive results from its Phase 2a proof-of-concept study of OmniAb-derived IMVT-1401 in thyroid eye disease. IMVT-1401 is a novel investigational anti-FcRn antibody delivered by subcutaneous injection. The results showed a 65% mean reduction in total IgG observed from baseline to end of treatment, with a pharmacodynamic response nearly identical to modeled predictions for the dosing regimen tested in the trial. IMVT-1401 was generally well-tolerated.
Janssen initiated a Phase 1b trial of OmniAb-derived teclistamab (also known as JNJ-64007957) in combination with subcutaneous daratumumab in patients with multiple myeloma to identify a Phase 2 dose regimen and assess safety of the combination.
Gloria Biosciences submitted an application for marketing approval in China for OmniAb-derived zimberelimab for the treatment of classical Hodgkin lymphoma, marking multiple OmniAb drug applications filed seeking approval.
Arcus Biosciences and Taiho Pharmaceutical announced Taiho’s exercise of its option for an exclusive license to zimberelimab (also known as AB122) for Japan and other Asian countries, excluding China.
Ligand entered into an OmniAb Platform agreement with Pandion Therapeutics, and Pandion subsequently closed an $80 million financing and announced that proceeds will support the advancement of their pipeline of modular proteins and bi-functional antibodies for the treatment of autoimmune diseases.
CStone Pharmaceuticals and Blueprint Medicines initiated a Phase 1b/2 clinical trial of fisogatinib in combination with OmniAb-derived CS1001 for patients with hepatocellular carcinoma. CStone also announced that the first patient was dosed in the global proof-of-concept study of OmniAb-derived CS1001 in combination with Bayer’s regorafenib in patients with advanced solid tumors.
Harbour BioMed raised $75 million to fund the clinical development of an OmniAb-derived anti-FcRn antibody batoclimab, among other uses. Harbour also announced first patient dosing of Phase 1b/2a study of batoclimab for treating neuromyelitis optica spectrum disorder.
OmniAb partner GenMab highlighted DuoBody-PD-L1x4-1BB (GEN1046) at the J.P. Morgan investor conference in January; the GEN1046 anti-PD-L1 is derived from OmniRat.
Captisol Business Update

Ligand’s Captisol is a patented ultra-high pure form of sulfobutylether beta-cyclodextrin (SBECD) that is built upon drug master files maintained in multiple countries and has an extensive safety record for use in intravenous, inhaled, subcutaneous, oral, and ophthalmic formulations, among others. Ligand is the sole supplier of Captisol globally.
Ligand announced that it is supplying Captisol to partners for remdesivir in clinical trials and for manufacturing scale-up as a treatment for COVID-19.
On May 1, 2020 remdesivir was the first new treatment for COVID-19 to be issued an Emergency Use Authorization. Gilead announced that it intends to donate 1.5 million doses of remdesivir. Gilead has also stated that it is expanding manufacturing to increase product supply to over 1 million treatment courses by December 2020 ahead of potential increased demand.
On April 29, 2020 Gilead announced topline results from the open-label, Phase 3 SIMPLE trial evaluating 5-day and 10-day dosing durations of the investigational antiviral remdesivir in hospitalized patients with severe manifestations of COVID-19 disease. The study demonstrated that patients receiving a 5-day treatment course of remdesivir achieved similar improvement in clinical status compared with those taking a 10-day treatment course. The study demonstrated the potential for some patients to be treated with a 5-day regimen, which could significantly expand the number of patients who could be treated with Gilead’s current supply of remdesivir. The study results complement positive data from a placebo-controlled Phase 3 study of remdesivir conducted by the National Institute for Allergy and Infectious Diseases also reported on April 29, 2020, and may help to determine the optimal duration of treatment with remdesivir.
Ligand’s Captisol network is served by manufacturing plants in two European countries and five distribution facilities around the globe, all of which remain fully operational. Ligand has substantial capacity to supply Captisol manufactured according to cGMP and its focus is to ensure sufficient supply to meet all existing and future partner needs, and to supply Gilead. Ligand is also evaluating plans with its supply partners to further increase capacity by bringing additional sites online, if needed.
During the first quarter, Ligand entered into Captisol clinical use agreements with Double-Crane Pharmaceuticals and OnKure Therapeutics.
Vernalis Business Update

Ligand entered into an exclusive worldwide license agreement with Neuritek Therapeutics to develop and commercialize V158866, a novel oral, selective fatty acid amide hydrolase inhibitor that was discovered using the Vernalis Design Platform. Neuritek plans to develop V158866 for post-traumatic stress disorder and other CNS diseases. Under the terms of the agreement, Ligand will receive an upfront license fee and is eligible to receive over $240 million in milestones and tiered royalties on net sales six to eight percent. On March 31, 2020 Neuritek announced it had secured approximately $27 million in a capital commitment from GEM Global Yield LLC SCS.
Third-party academic drug analyses suggest a potential role for heat shock protein 90 (Hsp90) inhibitors in treating COVID-19 infection. Based on these studies, Ligand is evaluating potential collaborations or partnerships relating to intravenous luminespib (AUY-922) as a potential treatment for patients with COVID-19. Luminespib is a Phase 2-ready Hsp90 inhibitor, previously investigated in clinical trials for cancer.
Additional Pipeline and Partner Developments

Retrophin announced that the first 190 patients have been enrolled in its pivotal Phase 3 DUPLEX Study evaluating the safety and efficacy of sparsentan in focal segmental glomerulosclerosis.
Nucorion Pharmaceuticals initiated a Phase 1 clinical trial of NCO-48 Fumarate (NCO-1010), an oral prodrug of the nucleotide tenofovir utilizing Ligand’s LTP Platform technology for the potential treatment of hepatitis B. The first-in-human, double-blind, placebo-controlled, randomized, ascending single oral dose study will evaluate the safety and tolerability of NCO-48 Fumarate in healthy subjects. Topline results are expected in August 2020.
Palvella Therapeutics announced the completion of enrollment in its seamless Phase 2/3 VALO Study of PTX-022 (QTORIN 3.9% rapamycin anhydrous gel) for the treatment of adults with pachyonchia congenita.
Verona Pharma reported positive topline data in its 4-week Phase 2b COPD study with nebulized ensifentrine on top of tiotropium therapy, and also reported positive efficacy and safety data with a single dose of the pressurized metered-dose inhaler (pMDI) formulation of ensifentrine in a Phase 2 COPD study. Ensifentrine has demonstrated statistically significant and clinically meaningful improvements in lung function in Phase 2 studies in COPD patients when delivered via all three widely used inhaled formulations: nebulizer, DPI and pMDI.
Corvus Pharmaceuticals presented clinical data from its Phase 1b/2 trial of ciforadenant at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s Genitourinary Cancers Symposium.
Ligand regained global rights to its novel glucagon receptor antagonist (formerly known as LGD-6972 and RVT-1502) from Metavant Sciences.
Business Development and Corporate Highlights

Following the close of the first quarter, Ligand completed its acquisition of the core assets of Icagen, Inc.’s North Carolina operations, including partnered programs, proprietary ion channel screening and assay platforms, x-ray fluorescence capabilities, custom screening technologies and novel unpartnered preclinical-stage molecules for $15 million in cash.
Ligand announced the launch of its Environmental, Social and Corporate Responsibility Governance section of its corporate website. Please visit this link for current disclosures.
Adjusted Financial Measures

The Company reports adjusted net income and adjusted net income per diluted share in addition to, and not as a substitute for, or superior to, financial measures calculated in accordance with GAAP. The Company’s financial measures under GAAP include share-based compensation expense, amortization of debt-related costs, amortization related to acquisitions and intangible assets, changes in contingent liabilities, mark-to-market adjustments for amounts relating to its equity investments in public companies, excess tax benefit from share-based compensation, gain on the sale of Promacta and others that are listed in the itemized reconciliations between GAAP and adjusted financial measures included at the end of this press release. However, other than with respect to total revenues, the Company only provides financial guidance on an adjusted basis and does not provide reconciliations of such forward-looking adjusted measures to GAAP due to the inherent difficulty in forecasting and quantifying certain amounts that are necessary for such reconciliation, including adjustments that could be made for changes in contingent liabilities, changes in the market value of its investments in public companies, stock-based compensation expense and effects of any discrete income tax items. Management has excluded the effects of these items in its adjusted measures to assist investors in analyzing and assessing the Company’s past and future core operating performance. Additionally, adjusted earnings per diluted share is a key component of the financial metrics utilized by the Company’s board of directors to measure, in part, management’s performance and determine significant elements of management’s compensation.

Conference Call

Ligand management will host a conference call with slides today beginning at 4:30 p.m. Eastern time (1:30 p.m. Pacific time) to discuss this announcement and answer questions. To participate via telephone, please dial (833) 325-0071 from the U.S. or (720) 405-1612 from outside the U.S., using the conference ID 5190682. To participate via live or replay webcast, a link is available at www.ligand.com. Slides to accompany the conference call are available here.

On May 6, 2020 MannKind Corporation (NASDAQ:MNKD) reported financial results for the quarter ended March 31, 2020 (Press release, Mannkind, MAY 6, 2020, View Source [SID1234557125]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are pleased to report first quarter Afrezza net revenue of $8.0 million, which is 58% higher than the same quarter in 2019," said Michael Castagna, Chief Executive Officer of MannKind Corporation. "We believe that some of the increased revenue this quarter reflects the impact of patients stocking up on extra refills in advance of the stay-at-home orders that have since been implemented across the country. In response to the COVID-19 pandemic, we have rapidly implemented digital tools and programs to help our sales force and our Afrezza prescribers navigate this challenging time for our healthcare system. In addition, our manufacturing and development team in Connecticut remain focused on maintaining supply of Afrezza and meeting our obligations under our collaboration with United Therapeutics."

Total revenues were $16.2 million for the first quarter of 2020, reflecting Afrezza net revenue of $8.0 million and collaboration and services revenue of $8.2 million. Afrezza net revenue increased 58% compared to $5.1 million in the first quarter of 2019, primarily driven by higher product demand as well as a price increase and a more favorable mix of cartridges. Collaboration and services revenue for the first quarter of 2020 decreased $4.2 million compared to the first quarter of 2019, primarily due to the substantial completion of the research agreement with United Therapeutics in the second quarter of 2019.

Afrezza gross profit for the first quarter of 2020 was $3.8 million vs. $1.1 million in the same period of 2019, a 263% increase that was driven primarily by higher Afrezza revenue. Cost of goods sold increased by $0.1 million which included an increase related to Afrezza unit sales growth and an inventory write-off offset by a greater amount of costs capitalized to inventory due to a higher volume of manufacturing activities in the first quarter of 2020. Gross margin in the first quarter of 2020 increased to 48%, our highest gross margin to date, from 21% for the same quarter in 2019, primarily due to higher Afrezza revenue.

Selling, general and administrative expenses for the first quarter of 2020 were $14.4 million compared to $25.7 million for the first quarter of 2019. This 44% decrease was primarily due to $9.3 million spent on direct-to-consumer television advertising in 2019, which was not repeated in 2020, a $1.1 million decrease in promotional and marketing activities and $0.8 million decrease in personnel and employee related costs.

Net interest expense for the first quarter of 2020 was $2.2 million compared to $1.4 million for the first quarter of 2019. This $0.8 million increase was due to a higher balance of outstanding principal and an increase in the interest rate of certain promissory notes.

The net loss for the first quarter of 2020 was $9.3 million, or $0.04 per share, compared to a $14.9 million net loss in the first quarter of 2019, or $0.08 per share. The lower net loss is mainly attributable to a decrease in operating expenses of $7.6 million. The reduction in the net loss per share was impacted by the lower operating expenses and a greater number of outstanding shares.

Cash, cash equivalents and restricted cash at March 31, 2020 was $39.2 million compared to $50.2 million at December 31, 2019, which also included short-term investments of $20.0 million. The decrease was primarily due to net cash used in operating activities of $11.2 million in the first quarter of 2020.

Non-GAAP Measures

Certain financial information contained in this press release is presented on both a reported basis (GAAP) and a non-GAAP basis. Reported results were prepared in accordance with GAAP whereas non-GAAP measures exclude items described in the reconciliation tables below. Non-GAAP financial information is intended to portray the results of our baseline performance, supplement or enhance management, analysts and investors overall understanding of our underlying financial performance and facilitate comparisons among current and past periods. The non-GAAP financial measures are in addition to, not a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP.

MannKind will host a conference call and presentation webcast to discuss these results today at 5:00 p.m. Eastern Time. Those interested in listening to the conference call live via the Internet may do so by visiting the Company’s website at View Source under News & Events.

A telephone replay of the call will be accessible for approximately 14 days following completion of the call by dialing (844) 512-2921 or (412) 317-6671 and use the participant passcode: 7127207#. A replay will also be available on MannKind’s website for 14 days.

On May 6, 2020 Incyte (Nasdaq:INCY) reported that the U.S. Food and Drug Administration (FDA) has approved TabrectaTM (capmatinib) for treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have a mutation that leads to MET exon 14 skipping (METex14) as detected by an FDA-approved test (Press release, Incyte, MAY 6, 2020, View Source [SID1234557123]). This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). Tabrecta, the first and only treatment approved to specifically target NSCLC with this driver mutation, is approved for first-line and previously treated patients regardless of prior treatment type.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Novartis has exclusive worldwide development and commercialization rights to Tabrecta, and the FDA approval of Tabrecta triggers $70 million in milestone payments from Novartis to Incyte. Incyte is also eligible to receive 12-14 % royalties on net sales of Tabrecta by Novartis.

NSCLC accounts for approximately 85% of lung cancer diagnoses1. METex14 occurs in 3-4% of newly-diagnosed metastatic NSCLC cases2 and is a recognized oncogenic driver3,4.

"We are pleased that the FDA has approved Tabrecta for patients with METex14 NSCLC," said Steven Stein, M.D., Chief Medical Officer, Incyte. "Having a therapy that targets the recognized oncogenic driver will provide a much needed treatment option for patients with METex14 NSCLC who currently have limited treatment options. Tabrecta is the fourth Incyte molecule to be approved by the FDA, highlighting our world-class discovery program and commitment to bringing innovative medicines to patients in need."

The approval of Tabrecta is based on results5 from the pivotal GEOMETRY mono-1 Study. In the METex14 population (n=97), the confirmed overall response rate was 68% (95% CI, 48-84) and 41% (95% CI, 29-53) among treatment-naive (n=28) and previously treated patients (n=69), respectively, based on the Blinded Independent Review Committee (BIRC) assessment per RECIST v1.1. In patients taking Tabrecta, the study also demonstrated a median duration of response of 12.6 months (95% CI, 5.5–25.3) in treatment-naive patients (19 responders) and 9.7 months (95% CI, 5.5-13.0) in previously treated patients (28 responders). The most common treatment-related adverse events (AEs) (incidence ≥20%) are peripheral edema, nausea, fatigue, vomiting, dyspnea, and decreased appetite.

Capmatinib was previously granted Breakthrough Therapy designation by the FDA, which is designed to expedite the development and review of drugs for serious conditions that have shown encouraging early clinical results and may demonstrate substantial improvements over available medicines.

Full prescribing information for Tabrecta can be found at: View Source

About GEOMETRY mono-1

The Novartis-sponsored GEOMETRY mono-1 trial is a multi-center, non-randomized, open-label, multi-cohort Phase 2 study to evaluate the efficacy and safety of single-agent capmatinib in adult patients with EGFR wild-type, metastatic NSCLC as measured by ORR.

Patients (n=97) with metastatic NSCLC harboring mutations that lead to METex14 (centrally confirmed) were assigned to Cohorts 4 (n=69, previously treated patients) or 5B (n=28, treatment-naïve), and received 400 mg capmatinib tablets orally twice daily. The major efficacy outcome was ORR based on BIRC assessment per RECIST v1.1. An additional efficacy outcome was DOR by BIRC.

About Tabrecta

Tabrecta (capmatinib; formerly INC280) is a kinase inhibitor that targets MET discovered by Incyte and licensed to Novartis in 2009. Under the terms of the Agreement, Incyte granted Novartis exclusive worldwide development and commercialization rights to capmatinib and certain back-up compounds in all indications. Incyte is eligible for over $500 million in future milestones as well as royalties of between 12 and 14 percent on global sales by Novartis.