On February 27, 2020 Jounce Therapeutics, Inc. (NASDAQ: JNCE), a clinical-stage company focused on the discovery and development of novel cancer immunotherapies and predictive biomarkers, reported financial results for the fourth quarter and year ended December 31, 2019 and provided a corporate update (Press release, Jounce Therapeutics, FEB 27, 2020, View Source [SID1234554864]).

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!

"On the heels of a year of significant clinical progress in 2019, we have set the pace for a robust 2020 with the advancement of our vopratelimab Phase 2 studies, the ongoing EMERGE trial and the upcoming SELECT trial. The recent announcement of the TISvopra biomarker for patient selection in SELECT is a testament to our Translational Science Platform and further emphasizes our vision of bringing the right immunotherapy to the right patients," said Richard Murray, Ph.D., chief executive officer and president of Jounce Therapeutics. "With two of our programs, vopratelimab and JTX-4014, in clinical trials, along with the advancement of our potential first-in-class monoclonal antibody, JTX-1811, into IND enabling activities, we are continuing to establish our differentiated approach to immunotherapy and clinical trial design. We look forward to executing on several key milestones in 2020 across our growing pipeline."

Pipeline Update and Highlights:
Vopratelimab

Established two development paths for vopratelimab program: In early 2019, Jounce announced two development paths for vopratelimab, which are now represented by two distinct and independent Phase 2 clinical trials, EMERGE and SELECT. Both trials are based on the biology related to the pharmacodynamic biomarker, ICOS hi CD4 T cells, which emerge in the blood of patients due to vopratelimab and not PD-1 inhibitors and are associated with clinical benefit.
— Enrollment on track in Phase 2 EMERGE trial: EMERGE trial enrollment in non-small cell lung cancer (NSCLC) remains on track, and Jounce expects to report preliminary efficacy and related biomarker data in the second half of 2020.
— Identified the baseline TISvopra biomarker: In February 2020, Jounce presented new data announcing the identification of the predictive biomarker associated with vopratelimab patient selection, known as TISvopra, which will be used in the upcoming SELECT trial to select patients more likely to develop ICOS hi CD4 T cells and experience clinical benefit in the presence of vopratelimab. The TISvopra biomarker is a baseline RNA signature with a threshold optimized for the prediction of the emergence of ICOS hi CD4 T cells and is expected to predict for both vopratelimab and PD-1 activity.
— Announced trial design for upcoming Phase 2 SELECT trial: In January 2020, Jounce announced the trial design for SELECT, a randomized, ex-U.S. trial that will evaluate vopratelimab and JTX-4014, a PD-1 inhibitor, in patients with second line, PD-1 inhibitor naive NSCLC. Jounce expects to enroll approximately 75 patients, who will be selected using the TISvopra biomarker. Jounce expects to initiate the Phase 2 SELECT trial in mid-2020 and report interim clinical data in 2021.
JTX-4014

Established JTX-4014 as combination agent for Phase 2 SELECT trial: Based on the results of the Phase 1 JTX-4014 trial announced in November 2019, Jounce plans to use JTX-4014 as the PD-1 inhibitor in combination with its other product candidates beginning with the upcoming Phase 2 SELECT trial.
Presented safety and preliminary efficacy data from Phase 1 trial at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 34th Annual Meeting: In November 2019, Jounce presented safety and preliminary efficacy data from the Phase 1 trial of JTX-4014 at SITC (Free SITC Whitepaper) demonstrating anti-tumor activity with an overall RECIST 1.1 response rate of 16.7% (3/18), one complete response, two partial responses and a disease control rate of 44.4% (8/18) in patients with advanced refractory solid tumor malignancies and an average of over four lines of prior therapies. JTX-4014 was found to have an acceptable safety profile in this trial.
JTX-1811

Named JTX-1811 as next development candidate from Translational Science Platform: In December 2019, JTX-1811 was selected as the next development candidate to emerge from Jounce’s Translational Science Platform. JTX-1811 is a monoclonal antibody designed to selectively deplete immuno-suppressive T regulatory cells. Jounce will present additional preclinical data at the 2020 American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting and expects to file an Investigational New Drug (IND) application in the first half of 2021.
Research Collaborations and Partnerships:

Established research collaboration with NanoString Technologies to support the application of TISvopra in Phase 2 SELECT trial: In January 2020, Jounce entered into a new research collaboration with NanoString Technologies for the application of the TISvopra biomarker, including the optimized selection threshold in the SELECT trial. The TISvopra clinical trial assay will be implemented on the nCounter Dx Analysis System.
Updated strategic collaboration with Celgene Corporation: In July 2019, Jounce and Celgene entered into a mutual agreement to terminate their broad strategic collaboration established in 2016. As a result, Jounce owns all global rights to its current pipeline, including vopratelimab, JTX-4014, JTX-1811 and all discovery-stage assets. Separately, Celgene obtained exclusive worldwide licensing rights to JTX-8064, a potential first-in-class antibody that targets the LILRB2 receptor on macrophages. Under the terms of the new license agreement, Jounce received a $50.0 million non-refundable license fee and is eligible to receive up to $480.0 million from Celgene in development, regulatory and commercial milestone payments, as well as royalties from potential worldwide sales.
Fourth Quarter and Full Year 2019 Financial Results:

Cash position: As of December 31, 2019, cash, cash equivalents and investments were $170.4 million, compared to $195.9 million as of December 31, 2018. The decrease in cash, cash equivalents and investments was primarily due to operating costs incurred during the year, offset by the $50.0 million license fee received in July 2019 pursuant to Jounce’s JTX-8064 license agreement with Celgene.
License and collaboration revenue: No license and collaboration revenue was recognized during the fourth quarter of 2019, compared to $20.1 million for the same period in 2018. License and collaboration revenue was $147.9 million for the full year 2019, compared to $65.2 million for the full year 2018. License and collaboration revenue recognized during 2019 was comprised of $50.0 million of cash revenue related to Jounce’s JTX-8064 license agreement with Celgene and $97.9 million of non-cash revenue recognition relating to the $225.0 million upfront payment received from Celgene in July 2016. License and collaboration revenue recognized during 2018 was comprised solely of non-cash revenue recognition related to the July 2016 upfront payment.
Research and development expenses: Research and development expenses were $16.6 million for both the fourth quarter of 2019 and 2018. Research and development expenses were $67.1 million for the full year 2019, compared to $70.1 million for the full year 2018. The decrease in research and development expenses for the full year 2019 was primarily due to $6.0 million of decreased manufacturing and IND-enabling expenses as well as $0.9 million of decreased lab consumable costs. These decreases were partially offset by $3.1 million of increased employee compensation costs.
General and administrative expenses: General and administrative expenses were $6.9 million for the fourth quarter of 2019, compared to $6.6 million for the same period in 2018 and $27.9 million for the full year 2019, compared to $26.4 million for the full year 2018. The increase in general and administrative expenses for both the fourth quarter of 2019 and the full year 2019 was primarily attributable to increased employee compensation costs.
Net (loss) income: Net loss was $22.7 million for the fourth quarter of 2019, or a basic and diluted net loss per share of $0.68. Net loss was $2.0 million for the same period in 2018, or a basic and diluted net loss per share of $0.06. Net income was $56.8 million for the full year 2019, resulting in basic net income per share of $1.72 and diluted net income per share of $1.66. Net loss was $27.4 million for the full year 2018, or a basic and diluted net loss per share of $0.84. Net income recognized for the full year 2019 was primarily attributable to $147.9 million of license and collaboration revenue recognized under Jounce’s agreements with Celgene.
Financial Guidance:
Based on its current operating and development plans, Jounce expects gross cash burn on operating expenses and capital expenditures for the full year 2020 to be approximately $80.0 million to $95.0 million. Jounce will no longer provide license and collaboration revenue guidance as potential future payments under the JTX-8064 license agreement with Celgene are royalty- and milestone-based.

Given the strength of its balance sheet, Jounce expects its existing cash, cash equivalents and investments to be sufficient to enable the funding of its operating expenses and capital expenditure requirements through the end of 2021.

Conference Call and Webcast Information:
Jounce Therapeutics will host a live conference call and webcast today at 8:00 a.m. ET. To access the conference call, please dial (866) 916-3380 (domestic) or (210) 874-7772 (international) and refer to conference ID 7889239. The live webcast can be accessed under "Events & Presentations" in the Investors and Media section of Jounce’s website at www.jouncetx.com. The webcast will be archived and made available for replay on Jounce’s website approximately two hours after the call and will be available for 30 days.

On February 27, 2020 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151), a global specialty pharmaceutical company creating innovative medical solutions using the latest biotechnology, reported that the U.S. Food and Drug Administration (FDA) has accepted for review the supplemental Biologics License Application (sBLA) for Crysvita (burosumab) for the treatment of FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors (tumor-induced osteomalacia; TIO) that cannot be curatively resected or localized (Press release, Kyowa Hakko Kirin, FEB 27, 2020, View Source [SID1234554891]). The FDA has assigned priority review designation with a Prescription Drug User Fee Act (PDUFA) target date of June 18, 2020.

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 appreciate the FDA’s collaboration in evaluating the data, and as a result, we are another step closer to bringing the first treatment to patients with this devastating disease in the setting of an unresectable tumor," said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. "We look forward to continuing to work closely with the FDA with the goal of bringing Crysvita to patients with TIO as quickly as possible."

"The discovery and submission of Crysvita has meant a great deal to patients and families that previously had no other treatment options," said Tomohiro Sudo, Head of Global Product Management Office of Kyowa Kirin. " "If approved, we believe Crysvita may also become a meaningful treatment option for many patients with TIO in the U.S."

The sBLA package includes data from two single-arm Phase 2 studies, a 144-week study in 14 adult patients conducted by Ultragenyx in the U.S. and an 88-week study in 13 adult patients conducted by Kyowa Kirin in Japan and South Korea.

Crysvita is approved by the U.S. FDA for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients six months of age and older, and by Health Canada and Brazil’s National Health Surveillance Agency (ANVISA) for the treatment of XLH in adult and pediatric patients one year of age and older. In Japan, it is approved by the Ministry of Health, Labor and Welfare (MHLW) for the treatment of FGF23-related hypophosphatemic rickets and osteomalacia. In Europe, Crysvita has received European conditional marketing authorization for the treatment of XLH with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons, and an application for the expanded use in adults with XLH is currently under review by the European Medicines Agency.

See below for Important Safety Information for Crysvita in X-linked hypophosphatemia.

About Tumor-Induced Osteomalacia (TIO)
TIO is caused by typically benign tumors that produce excess levels of fibroblast growth factor 23 (FGF23), causing phosphate wasting in the urine that leads to severe hypophosphatemia, osteomalacia, muscle weakness, fatigue, bone pain and fractures. The symptoms rapidly resolve if the causal tumors or lesion can be resected; however, there are cases in which resection is not feasible or recurrence of the tumor occurs after resection. In patients for whom the tumor or lesion is inoperable, the current treatment consists of oral phosphate and/or vitamin D replacement. Efficacy of this management is often limited, as it does not treat the underlying disease and its benefits must be balanced with monitoring for potential risks such as nephrocalcinosis, hypercalciuria and hyperparathyroidism. An estimated 500-1,000 people in the United States have TIO, and approximately half of all cases are inoperable.

About Crysvita
Crysvita (burosumab-twza) is a recombinant fully human monoclonal IgG1 antibody, discovered by Kyowa Kirin, against the phosphaturic hormone FGF23. FGF23 is a hormone that reduces serum levels of phosphorus and active vitamin D by regulating phosphate excretion and active vitamin D production by the kidney. Phosphate wasting in TIO and other hypophosphatemic conditions, including XLH, is caused by excessive levels and activity of FGF23. Crysvita is designed to bind to and thereby inhibit the biological activity of FGF23. By blocking excess FGF23 in patients with TIO and XLH, Crysvita is intended to increase phosphate reabsorption from the kidney and increase the production of vitamin D, which enhances intestinal absorption of phosphate and calcium.

Kyowa Kirin and Ultragenyx have been collaborating in the development and commercialization of Crysvita globally based on the collaboration and license agreement between the parties.

INDICATION (IN THE U.S.)

Crysvita is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older.

IMPORTANT SAFETY INFORMATION Crysvita should not be taken if:

• An oral phosphate supplement and/or a specific form of vitamin D supplement are taken (such as calcitriol, paricalcitol, doxercalciferol, calcifediol).

• Phosphorus levels from a blood sample are within or above the normal range for age.

• Kidney problems are present. What is the most important information to know about Crysvita?

• Some patients developed allergic reactions (rash and hives) while taking Crysvita. Doctors will monitor for symptoms of an allergic reaction while Crysvita is taken.

• High levels of phosphorus in the blood have been reported in some patients taking Crysvita. This may be related to a risk of high calcium levels in the kidneys. Doctors will collect samples to monitor levels.

• Administration of Crysvita may result in reactions at the injection site, such as hives, reddening of the skin, rash, swelling, bruising, pain, severe itching of the skin, and collection of blood outside of a blood vessel (hematoma). What are the possible side effects of Crysvita?

• The most common adverse reactions that were seen in children with XLH are: o Fever o Injection site reaction o Cough o Vomiting o Pain in arms and legs o Headache o Tooth infection o Dental cavities o Diarrhea o Decreased vitamin D levels o Toothache o Constipation o Muscle pain o Rash o Dizziness o Nausea

• The most common adverse reactions that were seen in adults with XLH are: o Back pain o Headache o Tooth infection o Restless leg syndrome o Decreased vitamin D levels o Dizziness o Muscle spasms o Constipation o Phosphorus levels increased in the blood

• Narrowing of the spaces within the spine is common in adults with XLH and pressure on the spinal cord has been reported in adults taking Crysvita. It is not known if taking Crysvita worsens the narrowing of the spaces within the spine or the pressure on the spinal cord. Before taking Crysvita, doctors should be informed about all medications (including supplements) and medical conditions, including if:

• One is taking oral phosphate and/or active vitamin D (such as calcitriol, paricalcitol, doxercalciferol, calcifediol).

• One is pregnant, thinks she may be pregnant, or plans to become pregnant. There is not enough experience to know if Crysvita may harm an unborn baby. Report pregnancies to the Kyowa Kirin, Inc. Adverse Event reporting line at 1-888-756-8657.

• One is breastfeeding or plans to breastfeed. There is not enough experience to know if Crysvita passes into breast milk. Women should talk with their doctors about the best way to feed their babies while taking Crysvita. While taking Crysvita, doctors should be informed if one experiences:

• An allergic reaction such as rash or hives

• A rash, swelling, bruising or other reaction at the injection site

• New or worsening restless leg syndrome

These are not all the possible side effects of Crysvita. Doctors should be contacted for medical advice about side effects.

Side effects may be reported to the FDA at (800) FDA-1088 or www.fda.gov/medwatch.

Side effects may also be reported to Kyowa Kirin, Inc. at 1-888-756-8657.

Please see full Prescribing Information for additional Important Safety Information.

On February 27, 2020 Nektar Therapeutics (Nasdaq: NKTR) reported financial results for the fourth quarter and full year ended December 31, 2019 (Press release, Nektar Therapeutics, FEB 27, 2020, View Source [SID1234554908]).

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!

Cash and investments in marketable securities at December 31, 2019 were approximately $1.6 billion as compared to $1.9 billion at December 31, 2018.

"Nektar’s progress over the past year has established a strong foundation for growth, with a robust portfolio of clinical-stage immuno-oncology and immunology candidates addressing multiple therapeutic areas," said Howard W. Robin, President and CEO of Nektar. "Our amended joint development plan with Bristol-Myers Squibb for bempegaldesleukin in combination with Opdivo expands the active registrational program for the doublet to five indications, including new Phase 3 studies in the adjuvant melanoma setting and muscle invasive bladder cancer. It also provides a path forward in first-line lung cancer and enhances our ability to pursue new combinations in additional indications."

Mr. Robin continued, "We also advanced NKTR-255, a novel IL-15 agonist that stimulates NK cells and memory T cells, into the clinic in combination with ADCC therapies. With NKTR-358, we have an opportunity to address the underlying immune imbalance associated with multiple autoimmune and chronic inflammatory diseases. Our partner Eli Lilly is on track to initiate a Phase 2 study in lupus, advance ongoing Phase 1b clinical trials in psoriasis and atopic dermatitis, and start an additional Phase 2 study in a new autoimmune indication this year."

Summary of Financial Results

Revenue in the fourth quarter of 2019 was $33.9 million as compared to $39.8 million in the fourth quarter of 2018. Revenue for the year ended December 31, 2019 was $114.6 million as compared to $1.2 billion in 2018 and was lower primarily due to the recognition of $1.06 billion of license revenue from the Bristol-Myers Squibb collaboration agreement in the second quarter of 2018.

Total operating costs and expenses in the fourth quarter of 2019 were $143.5 million as compared to $140.1 million in the fourth quarter of 2018. Total operating costs and expenses for 2019 were $554.7 million as compared to $505.4 million in 2018. Total operating costs and expenses increased primarily as a result of increases in research and development (R&D) expense and general and administrative (G&A) expense.

R&D expense in the fourth quarter of 2019 was $110.4 million as compared to $108.9 million for the fourth quarter of 2018. R&D expense for the year ended December 31, 2019 was $434.6 million as compared to $399.5 million in 2018. R&D expense was higher in 2019 as compared to 2018 primarily because of the continued clinical development of bempegaldesleukin, including the registrational studies in melanoma, bladder cancer and renal cell carcinoma, and manufacture of Phase 2 drug supply for NKTR-358, which were partially offset by lower bempegaldesleukin and NKTR-181 manufacturing costs.

G&A expense was $27.1 million in the fourth quarter of 2019 as compared to $23.8 million in the fourth quarter of 2018. G&A expense for 2019 was $98.7 million as compared to $81.4 million in 2018. G&A expense was higher in the fourth quarter and full year 2019 as compared to the same periods in 2018 primarily due to non-cash stock based compensation expense, limited commercialization readiness activities for NKTR-181, as well as other costs related to personnel, facilities and outside services.

Net loss for the fourth quarter of 2019 was $112.2 million or $0.64 basic and diluted loss per share as compared to a net loss of $98.2 million or $0.57 basic and diluted loss per share in the fourth quarter of 2018. Net loss for the year ended December 31, 2019 was $440.7 million or $2.52 diluted loss per share as compared to net income of $681.3 million or $3.78 diluted earnings per share in 2018.

2019 and Year-to-Date Business Highlights:

·In February 2020, Nektar announced the publication of preclinical bempegaldesleukin data in two manuscripts in Nature Communications showing how bempegaldesleukin works synergistically with multiple immune-based therapies to enhance T-cell-mediated tumor control.

·In January 2020, Nektar and Bristol-Myers Squibb announced a new joint development plan that expands the ongoing registrational program for bempegaldesleukin plus Opdivo (nivolumab) from three ongoing registrational trials in first-line metastatic melanoma, first-line cisplatin-ineligible metastatic urothelial cancer and first-line metastatic renal cell carcinoma (RCC) to include two additional registrational trials in adjuvant melanoma and muscle-invasive bladder cancer. In addition, a Phase 1/2 study will be initiated to evaluate bempegaldesleukin plus nivolumab in combination with axitinib in first-line RCC in order to support a future registrational trial. Bristol-Myers Squibb will also independently conduct and fund a Phase 1/2 study in first-line non-small-cell lung cancer with bempegaldesleukin and nivolumab.

●In January 2020, Nektar made the strategic business decision to withdraw its New Drug Application (NDA) for NKTR-181, an investigational medicine in development for chronic pain and make no further investment into the program.

●In December 2019, Nektar presented results from preclinical studies of NKTR-255, its IL-15 agonist, at the 61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting highlighting the candidate’s potential in the treatment of hematological malignancies by restoring both NK cell and memory CD8 T cell compartments in patients.

In November 2019, Nektar presented updated results from the first-in-human Phase 1a study of NKTR-358 at the 2019 Annual Meeting of the American College of Rheumatology supporting development of the candidate as a first-in-class T regulatory cell stimulator for the treatment of autoimmune and other chronic inflammatory conditions.

●In November 2019, Nektar presented new data from the Stage IV front-line melanoma cohort in the PIVOT-02 study at the 2019 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting. At a median time of follow-up of 18.6 months, median progression free survival had not yet been reached.

●In October 2019, Nektar announced that its partner Eli Lilly initiated two Phase 1b studies of NKTR-358, one in patients with psoriasis and one in patients with atopic dermatitis.

●In October 2019, Nektar announced the initiation of a first-in-human, Phase 1 clinical study evaluating NKTR-255 as monotherapy for patients with relapsed or refractory non-Hodgkin lymphoma or multiple myeloma.

·In September 2019, Nektar presented clinical data from its PIVOT-02 study for bempegaldesleukin in combination with Opdivo (nivolumab) at the 2019 CRI-CIMT-EATI-AACR International Cancer Immunotherapy Conference (CIMT) (Free CIMT Whitepaper) demonstrating the promising clinical activity of the combination in patients with advanced or metastatic triple-negative breast cancer, particularly in patients with PD-L1 negative baseline tumors.

●In August 2019, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for bempegaldesleukin in combination with Opdivo (nivolumab) for the treatment of patients with previously untreated unresectable or metastatic melanoma.

●In June 2019, Nektar presented biomarker and clinical data from the ongoing PIVOT-02 study for bempegaldesleukin in combination with Opdivo (nivolumab) at the 2019 ASCO (Free ASCO Whitepaper) Annual Meeting. Clinical data presented included 12-month follow-up for the Stage IV first-line melanoma patient cohort and showed a deepening and durability of response over time.

●In April 2019, Nektar presented positive preclinical data on its immuno-oncology pipeline candidates, bempegaldesleukin and NKTR-255, at the 2019 AACR (Free AACR Whitepaper) Annual Meeting.

●In March 2019, Nektar presented preliminary immune activation, safety and clinical activity data from the ongoing dose-escalation stage of the REVEAL study at the 2019 ASCO (Free ASCO Whitepaper)-SITC Meeting. The REVEAL Phase 1/2 study is evaluating the safety and efficacy of NKTR-262, a novel TLR agonist, in combination with bempegaldesleukin.

●In February 2019, Nektar presented clinical data from first-line Stage IV urothelial carcinoma patients enrolled in the PIVOT-02 study of bempegaldesleukin with Opdivo (nivolumab) at the 2019 ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium.

The company also announced upcoming presentations at the following scientific congresses:

Society of Toxicology (SOT) 59th Annual Meeting, Anaheim, CA

●Presentation: "Bempegaldesleukin (NKTR-214), a novel IL-2 based immunotherapy, demonstrates superior nonclinical safety compared to that reported for recombinant human IL-2 (rhIL-2)", Leung, S., et al.

oSession: Safety Assessment: Pharmaceutical—Drug Development
oDate: Wednesday, March 18th, 10:45 a.m. – 12:30 p.m.

●Presentation: "Toxicology Species Selection for Preclinical Safety Assessment of TLR7/8 Prodrug Agonist", Gunther, J., et al.

oSession: Safety Assessment: Pharmaceutical—Drug Development
oDate: Wednesday, March 18th, 10:45 a.m. – 12:30 p.m.

American Chemical Society National Meeting

●Presentation: "NKTR-262: Discovery of a novel TLR 7/8 agonist prodrug that demonstrates synergistic anti-tumor effect in combination with NKTR-214, a CD-122 preferential IL-2 pathway agonist", Anand, N., et al.

oSession: MEDI: Tissue Specific Delivery: TLR Agonists
oDate: Tuesday, March 24th, 10:10 a.m. – 10:45 a.m.

Conference Call to Discuss Fourth Quarter and Year-End 2019 Financial Results

Nektar management will host a conference call to review the results beginning at 5:00 p.m. Eastern Time/2:00 p.m. Pacific Time, Thursday, February 27, 2020.

This press release and a live audio-only Webcast of the conference call can be accessed through a link that is posted on the home page and Investors section of the Nektar website: View Source The web broadcast of the conference call will be available for replay through March 27, 2020.

To access the conference call, follow these instructions:

Dial: (877) 881-2183 (U.S.); (970) 315-0453 (international)

Passcode: 2507828 (Nektar Therapeutics is the host)

In the event that any non-GAAP financial measure is discussed on the conference call that is not described in the press release, or explained on the conference call, related information will be made available on the Investors page at the Nektar website as soon as practical after the conclusion of the conference call.

On February 27, 2020 -NantHealth, Inc. (NASDAQ: NH), a next-generation, evidence-based, personalized healthcare company, reported two expanded programs with Eviti Connect, NantHealth’s healthcare payer solution and provider decision support software (Press release, NantHealth, FEB 27, 2020, View Source [SID1234554927]). A leading U.S. Medicaid health plan expanded its coverage and one of the largest non-profit, rural health plans in the country offering coverage for 165,000 members will continue to drive high-value oncological care with Eviti Connect.

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!

Eviti Connect streamlined the pre-authorization process for cancer treatments and reduced denials and appeals for this leading U.S. Medicaid health plan, while providing oncologists in their network with access to a comprehensive library of evidence-based treatment options for optimal decision making. For one of the largest non-profit rural health plans in the country, Eviti Connect improved consistency in the use of evidence-based medicine, simplified processes, and streamlined treatment approvals that enable cancer patients to begin treatment quickly and efficiently.

"Implementation of the Affordable Care Act has resulted in a much more diverse Medicaid population," said Dr. William Flood, Chief Medical Officer for Eviti at NantHealth. "Medicaid plans now must address more members and a higher prevalence of cancer. Eviti’s comprehensive evidence-based medical library and experienced oncology medical staff enables Medicaid programs to evolve and meet these new challenges quickly."

The Medicaid health plan experienced compelling results in their two-state pilot program. During the two-year pilot, which covered approximately 250,000 members, the insurer noted a significant improvement in the use of evidence-based medicine for member oncology care as a result of providers submitting treatment requests via Eviti Connect. The successful pilot led the insurer to expand the program from two to 10 U.S. states.

For Eviti Connect to continue to help assure quality, high-value care for members with cancer, the non-profit, rural health plan signed a three-year renewal agreement. Under terms of the agreement, the health plan’s provider network will continue to benefit from Eviti Connect decision support solution, which includes evidence-based treatment regimens, condition-specific clinical trial options, and the potential for more efficient preauthorization and expedited reimbursement.

"This renewal of Eviti Connect by a large health plan demonstrates our continued leadership in delivering solutions that align the priorities of physicians, patients, and payers at the moment of clinical prescribing to promote the best care for patients," said Mark Mozley, Senior Vice President, Global Sales & Marketing, NantHealth. "We are pleased to continue this relationship momentum and support our clients’ goals to exceed industry standards for the authorization of oncology care."

NantHealth’s Eviti Connect is a web-based healthcare payer solution that electronically connects health plans and oncology practices for the authorization of treatment plans in near real-time. Eviti Connect reduces the administrative time involved in obtaining authorizations at the drug level, assures appropriate reimbursement at the regimen level, and validates cancer treatment options overall. It also provides the ability to have peer-to-peer conversations between prescribing oncologists and NantHealth oncologists.

On February 27, 2020 ANI Pharmaceuticals, Inc. ("ANI") (NASDAQ: ANIP) reported its financial results for the three and twelve months ended December 31, 2019 and provided its 2020 financial guidance (Press release, ANI Pharmaceuticals, FEB 27, 2020, https://www.prnewswire.com/news-releases/ani-pharmaceuticals-reports-full-year-and-fourth-quarter-2019-results-provides-2020-guidance-and-remains-on-track-to-submit-cortrophin-gel-snda-to-fda-in-march-2020-301012068.html [SID1234554944]). The Company will host its earnings conference call this morning, February 27, 2020, at 10:30 AM ET. Investors and other interested parties can join the call by dialing (866) 776-8875. The conference ID is 4783883.

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!

"ANI generated record net revenues and non-GAAP gross profit in 2019 while continuing to diversify its commercial product offering and pipeline opportunities. While revenues and non-GAAP gross profit for three of our important generic products were negatively impacted in recent periods, overall our business remains resilient, and we look forward to leveraging important recent launches such as Vancomycin Oral Solution and Bretylium Tosylate Injection as well as the recently acquired Amerigen portfolio in 2020. During 2019, we launched several new products, entered into collaborative arrangements with strategic partners and acquired both previously approved and development stage products we plan to launch in the future. We have utilized strong operating cash flows for asset acquisitions and investment into our portfolio of pipeline products and expect to continue to do so in the future.

During the fourth quarter, we successfully completed our fourth commercial scale batch of both Corticotropin API and the Cortrophin Gel drug product, and importantly, remain on track to submit our supplemental NDA filing in March 2020."

2020 Financial Guidance

For the twelve months ending December 31, 2020, ANI is providing guidance on net revenue, adjusted non-GAAP EBITDA, and adjusted non-GAAP diluted earnings per share. The following table summarizes 2020 guidance as compared to 2019 actual results:

In addition, we expect that adjusted non-GAAP gross margin, defined as the result of Net Revenues less Cost of Sales (excluding depreciation and amortization) as a percentage of Net Revenues, will decline from approximately 71% in 2019 to the mid 60% range in 2020, driven by the impact of negative price and product mix. Year over year changes of our Adjusted non-GAAP diluted earnings per share metric differ from our Adjusted non-GAAP EBITDA metric due to a projected increase in cash interest expense resulting from the refinancing of our convertible debt to bank debt.

ANI’s full year 2020 financial guidance reflects management’s current assumptions regarding customer relationships, product pricing, prescription trends, competition, inventory levels, cost of sales, operating costs, timing of research and development spend, taxes, and the anticipated timing of future product launches and other key events.

Cortrophin Gel Re-commercialization Update

Product

Required Filing

Expected Filing Date

Total Annual Market(d)

Cortrophin Gel

sNDA

March 2020

$1.0 billion

(d) Based on data from IQVIA

We continue to successfully progress our Cortrophin Gel re-commercialization program. Significant accomplishments since the third quarter 2019 press release (dated November 6, 2019) include:

ANI successfully completed API process validation by completing the fourth commercial scale batch of Corticotropin API. ANI also completed manufacturing for a fifth commercial scale batch of Corticotropin API. All five commercial scale batches were analytically consistent with each other and met all API release specifications. ANI expects to have 6 months stability on all API registration batches prior to the sNDA filing and by the end of first quarter 2020.
ANI successfully completed drug product process validation in the fourth quarter of 2019. ANI also completed manufacturing of a fourth commercial scale batch of Cortrophin Gel. This batch was analytically consistent with previously manufactured batches and met all drug product release specifications. ANI had already completed manufacturing for three commercial scale registration stability batches of Cortrophin Gel and expects to have 6 months stability on each prior to the sNDA filing and by the end of first quarter 2020.
ANI is on track to file the sNDA as planned by March 2020.

For further details, please see ANI’s Cortrophin Gel Re-commercialization Milestone Update in Table 5.

Fourth Quarter Results

Generic Pharmaceutical Products

Fourth Quarter Net Revenues – Results and Update

Net revenues from sales of generic pharmaceuticals decreased 14% to $29.1 million from $33.7 million in the prior period, primarily due to decreases in sales of Esterified Estrogen with Methlytestosterone ("EEMT"), Erythromycin Ethylsuccinate ("EES"), Propafenone, and Fenofibrate. These decreases were tempered by the September 2019 launch of Vancomycin HCl for Oral Solution as well as increased unit sales of Vancomycin tablets.

Branded Pharmaceutical Products

Fourth Quarter Net Revenues – Results and Update

Net revenues from sales of branded pharmaceuticals decreased 17% to $15.6 million from $18.8 million in the prior period, primarily due to decrease in sales of Inderal LA, Atacand and Atacand HCT. These decreases were tempered by increased sales of InnoPran XL.

Contract Manufacturing

Fourth Quarter Net Revenues – Results and Update

Contract manufacturing revenues decreased 28% to $2.6 million from $3.7 million in the prior year period, due to the timing and volume of orders from contract manufacturing customers in the period.

Royalty and Other

Fourth Quarter Net Revenues – Result and Update

Royalty and Other decreased 34% to $0.6 million from $0.9 million, primarily due to the timing and volume of product development and laboratory services revenue earned by ANI Canada.

Operating Expenses

Operating expenses increased to $52.6 million for the three months ended December 31, 2019, from $45.7 million in the prior year period. The increase was primarily due to the following:

$6.5 million in the build of Cortrophin pre-launch commercial inventories (which are expensed for US GAAP); there were no such activities in 2018,
$1.2 million increase in research and development expense, primarily due to Q4 2019 development-based milestone payments earned by collaborative partners, and
$0.9 million increase in depreciation and amortization expense, primarily due to additional amortization expense associated with a March 2019 asset acquisition and a January 2019 royalty buyout payment related to a prior period asset acquisition.
These increases were partially offset by a $2.3 million decrease in cost of sales as a result of the previously mentioned royalty buyout in January 2019 and a decrease in sales over the comparable periods, tempered by Q4 2019 inventory reserve charges of $4.6 million primarily related to the Company’s exit from the Methylphenidate Extended Release market.

Cost of sales as a percentage of net revenues increased to 37% during the three months ended December 31, 2019, from 35% during same period in 2018. The increase was primarily due to the inventory reserve charges recognized in the fourth quarter 2019 and negative price, which were tempered by reductions related to the 2019 royalty buyout.

Net Loss and Diluted Loss per Share

Net loss was $4.8 million for the three months ended December 31, 2019, as compared to net income of $5.4 million in the prior year period. The net loss was driven by the previously mentioned $6.5 million build of Cortrophin pre-launch commercial inventories and $4.6 million of inventory reserve charges. The effective consolidated tax benefit rate for the three months ended December 31, 2019 was 37%.

Diluted loss per share for the three months ended December 31, 2019 was $0.41, based on 11,886 thousand diluted shares outstanding, as compared to diluted earnings per share of $0.46 in the prior year period. Adjusted non-GAAP diluted earnings per share was $1.08, as compared to adjusted non-GAAP diluted earnings per share of $1.32 in the prior year period. For a reconciliation of adjusted non-GAAP diluted earnings per share to the most directly comparable GAAP financial measure, please see Table 4.

ANI Product Development Pipeline

ANI’s pipeline consists of 118 products, addressing a total annual market size of $7.0 billion, based on data from IQVIA. Of these, ANI expects that at least 52 can be commercialized based on either CBE-30s or prior approval supplements filed with the FDA.

Non-GAAP Financial Measures

The Company’s fiscal 2019 guidance for adjusted non-GAAP EBITDA and adjusted non-GAAP diluted earnings per share is not reconciled to the most comparable GAAP measure. This is due to the inherent difficulty of forecasting the timing or amount of items that would be included in a reconciliation to the most directly comparable forward-looking GAAP financial measures. Because a reconciliation is not available without unreasonable effort, it is not included in this release.

Adjusted non-GAAP EBITDA

ANI’s management considers adjusted non-GAAP EBITDA to be an important financial indicator of ANI’s operating performance, providing investors and analysts with a useful measure of operating results unaffected by non-cash stock-based compensation and differences in capital structures, tax structures, capital investment cycles, ages of related assets, and compensation structures among otherwise comparable companies. Management uses adjusted non-GAAP EBITDA when analyzing Company performance.

Adjusted non-GAAP EBITDA is defined as net income/(loss), excluding tax expense, interest expense, depreciation, amortization, the excess of fair value over cost of acquired inventory, stock-based compensation expense, expense from acquired in-process research and development, gains, losses, and expenses related to the repurchase of convertible debt, expenses related to debt financing, transaction and integration expenses, non-cash impairment charges, Cortrophin pre-launch charges, other income / expense and certain other items, including expensed milestone payments and inventory reserve charges, that vary in frequency and impact on ANI’s results of operations. Adjusted non-GAAP EBITDA should be considered in addition to, but not in lieu of, net income or loss reported under GAAP. A reconciliation of adjusted non-GAAP EBITDA to the most directly comparable GAAP financial measure is provided in Table 3.

Adjusted non-GAAP Net Income

ANI’s management considers adjusted non-GAAP net income to be an important financial indicator of ANI’s operating performance, providing investors and analysts with a useful measure of operating results unaffected by purchase accounting adjustments, non-cash stock-based compensation, non-cash interest expense, depreciation and amortization, non-cash impairment charges, Cortrophin pre-launch charges and certain other items, including expensed milestone payments and inventory reserve charges, that vary in frequency and impact on ANI’s results of operations. Management uses adjusted non-GAAP net income when analyzing Company performance.

Adjusted non-GAAP net income is defined as net income/(loss), plus the excess of fair value over cost of acquired inventory, stock-based compensation expense, transaction and integration expenses, gains, losses, and expenses related to the repurchase of convertible debt, expenses related to debt financing, non-cash interest expense, depreciation and amortization expense, expense from acquired in-process research and development, non-cash impairment charges, Cortrophin pre-launch charges and certain other items, including expensed milestone payments and inventory reserve charges, that vary in frequency and impact on ANI’s results of operations, less the tax impact of these adjustments calculated using an estimated statutory tax rate. Management will continually analyze this metric and may include additional adjustments in the calculation in order to provide further understanding of ANI’s results. Adjusted non-GAAP net income should be considered in addition to, but not in lieu of, net income reported under GAAP. A reconciliation of adjusted non-GAAP net income to the most directly comparable GAAP financial measure is provided in Table 4.

Adjusted non-GAAP Diluted Earnings per Share

ANI’s management considers adjusted non-GAAP diluted earnings per share to be an important financial indicator of ANI’s operating performance, providing investors and analysts with a useful measure of operating results unaffected by purchase accounting adjustments, non-cash stock-based compensation, non-cash interest expense, depreciation and amortization, non-cash impairment charges, Cortrophin pre-launch charges and certain other items, including expensed milestone payments and inventory reserve charges, that vary in frequency and impact on ANI’s results of operations.

Management uses adjusted non-GAAP diluted earnings per share when analyzing Company performance.

Adjusted non-GAAP diluted earnings per share is defined as adjusted non-GAAP net income, as defined above, divided by the diluted weighted average shares outstanding during the period, as adjusted for the dilutive effect of the convertible debt notes (in 2019), when applicable. Management will continually analyze this metric and may include additional adjustments in the calculation in order to provide further understanding of ANI’s results. Adjusted non-GAAP diluted earnings per share should be considered in addition to, but not in lieu of, diluted earnings or loss per share reported under GAAP. A reconciliation of adjusted non-GAAP diluted earnings per share to the most directly comparable GAAP financial measure is provided in Table 4.