On November 6, 2019 Precision BioSciences, Inc. (Nasdaq: DTIL), a genome editing company dedicated to improving life through the application of its pioneering, proprietary ARCUS platform, reported that initial results from the ongoing Phase 1/2a trial of its lead investigational off-the-shelf (allogeneic) chimeric antigen receptor (CAR) T cell therapy candidate, PBCAR0191, will be presented during the 61st Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in Orlando, Florida, December 7-10, 2019 (Press release, Precision Biosciences, NOV 6, 2019, View Source [SID1234550485]).

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!

PBCAR0191 is Precision’s first allogeneic CAR T therapy candidate in clinical trials and targets the well characterized cancer cell surface protein CD19. It is being developed in collaboration with Servier, an international pharmaceutical company. The Phase 1/2a trial includes adult patients with relapsed or refractory (R/R) non-Hodgkin lymphoma (NHL) or R/R B-cell precursor acute lymphoblastic leukemia (B-ALL). The abstract outlining initial data from patients treated with PBCAR0191 at Dose Level 1 is available on the ASH (Free ASH Whitepaper) conference website (View Source). This trial is ongoing and updated results, including from patients treated at Dose Level 2, will be presented at the ASH (Free ASH Whitepaper) Annual Meeting on December 9, 2019 starting at 6:00 p.m. ET.

"We are excited to share initial clinical data from Precision’s PBCAR0191 program at ASH (Free ASH Whitepaper), which we believe demonstrate the potential of our differentiated approach to the development of allogeneic CAR T therapies," said Chris Heery, MD, Chief Medical Officer of Precision BioSciences. "These data bring the reality of a true off-the-shelf CAR T therapy a step closer for patients in need of new and improved treatment options. We remain committed to the wider goal of improving access to cellular therapies for patients with advanced NHL and ALL, and we are optimistic, based on these initial findings, that we may be able to help meet this need. While preliminary and from a limited number of patients, the safety profile, in vivo cell expansion and early evidence of clinical activity we have demonstrated at our lowest dose level with PBCAR0191 in the absence of biologic lymphodepletion is very encouraging. We look forward to sharing updated results from patients treated at Dose Levels 1 and 2 at ASH (Free ASH Whitepaper)."

Investigator Update & Webcast Information
Precision will host a live webcast of an investigator update event during the ASH (Free ASH Whitepaper) Annual Meeting to discuss the presented data, beginning at 8:15 p.m. ET on Monday, December 9, 2019. To access the webcast, please visit the "Events & Presentations" page within the Investors & Media section of the Precision BioSciences website at View Source A replay of the webcast will be available on the Precision website for 30 days following the call.

First Clinical Data from Precision’s PBCAR0191 Program
Title: Initial findings of the Phase 1 trial of PBCAR0191
Presenter: Bijal Shah, MD, Moffitt Cancer Center
Session: 627. Aggressive Lymphoma (Diffuse Large B-Cell and Other Aggressive B-Cell Non-Hodgkin Lymphomas)—Results from Retrospective/Observational Studies
Poster/Presentation Number: Poster III
Date and Time: December 9, 2019, 6:00-8:00 p.m. ET
Location: Orange County Convention Center, Hall B

PBCAR0191 is Precision’s first allogeneic CAR T therapy candidate in clinical trials. This presentation will include initial data from the Phase 1 portion of the ongoing Phase 1/2a trial of PBCAR0191, which is designed to assess safety, identify an optimal dose of PBCAR0191 and evaluate preliminary clinical activity in patients with R/R NHL and B-ALL. The trial is a 3+3 dose escalation study (at dose levels of 3×105, 1×106, and 3×106 CAR T+ cells/kg); in each of the three dose levels up to six patients may be enrolled in each of the two cohorts (NHL and B-ALL). Lymphodepletion is achieved using fludarabine 30mg/m2/day and cyclophosphamide 500mg/m2/day.

Data in the abstract include results as of the data cutoff date of August 1, 2019 for three patients with advanced NHL treated at Dose Level 1, one with mantle cell lymphoma (MCL) and two with diffuse large B cell lymphoma (DLBCL). No significant toxicity was observed, including no serious adverse events and no dose-limiting toxicities. All patients had a minimum follow-up of 28 days (median 60 days).

Findings indicate preliminary evidence of cell-mediated anti-tumor activity, which will be evaluated more fully at subsequent dose levels. Two of the three patients experienced an objective tumor response by Lugano criteria, at day 14 and day 28, respectively. Both patients progressed due to new lesions (on day 28 and day 60, respectively). The third patient, who had previously progressed following treatment with axicabtagene ciloleucel (Yescarta), an approved anti-CD19 autologous CAR T therapy, had not met the definition of response, but had shown evidence of central necrosis, decreased tumor size, and decreased PET-avidity at day 28, in the context of post-infusion tumor site pain and mild CRS symptoms.

Peripheral blood analysis for CAR T cell expansion has identified preliminary evidence of cell expansion.

This trial is ongoing and updated results, including from patients treated at Dose Level 2, will be shared at the ASH (Free ASH Whitepaper) conference.

Precision’s Off-The-Shelf CAR T Platform
Precision is advancing a pipeline of cell-phenotype optimized allogeneic CAR T therapies, leveraging fully scaled, proprietary manufacturing processes. The platform is designed to maximize the number of patients who can potentially benefit from CAR T therapy by improving access to care through a well-tolerated lymphodepletion regimen. Precision carefully selects high-quality T cells derived from healthy donors as starting material, then utilizes its unique ARCUS genome editing technology to modify the cells via a single-step engineering process. By inserting the CAR gene at the T cell receptor (TCR) locus, this process knocks in the CAR while knocking out the TCR, creating a consistent product that can be reliably and rapidly manufactured and is designed to prevent graft-versus-host disease. Precision optimizes its CAR T therapy candidates for immune cell expansion in the body by maintaining a high proportion of naïve and central memory CAR T cells throughout the manufacturing process and in the final product.

On November 6, 2019 Pfenex Inc. (NYSE American: PFNX) reported that Eef Schimmelpennink, President and Chief Executive Officer, will be presenting at the Jefferies 2019 London Healthcare Conference on Wednesday, November 20th, taking place at the Waldorf Hilton in London (Press release, Pfenex, NOV 6, 2019, View Source [SID1234550484]).

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!

Conference: Jefferies 2019 London Healthcare Conference

Date: Wednesday, November 20, 2019

Time: 4:00 p.m. GMT (8:00 a.m. PST)

Webcast: Interested parties can access the live audio webcast and archive from the Investors Section of Pfenex’s website at www.pfenex.com

On November 6, 2019 PDL BioPharma, Inc. ("PDL" or "the Company") (Nasdaq: PDLI) reported financial results for the three and nine months ended September 30, 2019 (Press release, PDL BioPharma, NOV 6, 2019, View Source [SID1234550483]):

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!

Third Quarter Financial Highlights

Total revenues were $44.2 million, including $20.3 million in product revenue and $23.9 million in revenue from royalty rights – change in fair value.

LENSAR revenues were $8.1 million, up 22% over the prior-year period.

U.S. market share for branded Tekturna and authorized generic of Tekturna remained steady at approximately 73%.

Net cash royalties from all royalty rights were $25.6 million, up from $19.1 million in the prior-year period.

GAAP net loss was $17.8 million. Non-GAAP net income was $10.4 million. A reconciliation of GAAP to non-GAAP financial results can be found in Table 3 at the end of this news release.

Completed $100 million share repurchase program by repurchasing 1.3 million shares of common stock in the open market for $4.1 million.

"Third quarter revenues exceeded $44 million driven by continued growth from the LENSAR Laser System and strong contributions from our royalty assets, both of which are tracking ahead of our previous guidance range," said Dominique Monnet, president and CEO of PDL. "LENSAR’s quarterly revenues reached a record $8 million, up 22% over the prior-year period and up 27% year-to-date. Through PDL-funded R&D investments, LENSAR has continued to advance its best-in-class technology for the treatment of cataracts and the management of astigmatism. LENSAR’s technology benefits have been increasingly recognized by ophthalmic surgeons, as indicated by steady and robust year-over-year procedure volume growth since the product was launched in mid-2012. Procedure volume for the first nine months of 2019 is on pace to continue this trend.

"We are also pleased with progress at Evofem Biosciences, which is on track to resubmit the Amphora NDA in the fourth quarter," he added. "Given this timeframe, we anticipate Amphora’s commercial launch for the prevention of pregnancy in 2020, subject to FDA approval. We believe our strategic investment in Evofem allows our shareholders to benefit from the significant near-term and longer-term commercial potential of Amphora. It may also present a strategic position for PDL’s potential expansion into the underserved women’s health market.

"We completed our $100 million share buy-back program during the third quarter, which represents the Company’s largest single investment year-to-date. We are also continuing our efforts to reduce our operating expenses, with general and administrative expenses (G&A) down 16% year-to-date."

Revenue Highlights

Total third quarter revenues were $44.2 million and included $20.3 million in product revenue and $23.9 million in revenue from royalty rights – change in fair value.

Product revenue from the LENSAR Laser System was $8.1 million, a 22% increase from the third quarter of 2018. Revenue generated outside the U.S. accounted for the majority of the revenue increase. LENSAR procedure volume for the third quarter of 2019 increased 28% from the prior-year period.

Product revenue from Noden was $12.2 million compared with $17.8 million in the prior-year period. Revenue was split evenly between the U.S. and rest of the world at $6.1 million, compared with $9.7 million and $8.0 million, respectively, in the prior-year period. The U.S. market share for branded Tekturna and the authorized generic of Tekturna was 73%, relatively unchanged from the second quarter of 2019.
◦
Net royalty revenues from acquired royalty rights, which include cash royalties received and a change in fair value of the royalty rights assets, were $23.9 million compared with $42.2 million in the prior-year period. The decrease in royalty revenue is primarily related to the increase in fair value in the prior year period that resulted from the acquisition of additional Glumetza royalty rights from Assertio Therapeutics in that period. PDL received $25.6 million in net cash royalties from all of its royalty rights in the third quarter of 2019, up from $19.1 million in the year-ago quarter.

Total revenues for the first nine months of 2019 were $60.6 million and included $64.9 million in product revenue and negative $4.3 million in revenue from royalty rights – change in fair value.

Product revenue from the LENSAR Laser System was $22.2 million, a 27% increase from the prior-year period. Revenue generated outside of the U.S. accounted for the majority of the increase. LENSAR procedure volume for the first nine months of 2019 increased 30% from the prior-year period.

Product revenue from the Noden Products was $42.6 million compared with $62.0 million for the prior-year period. Sales for the first nine months of 2019 were comprised of $21.0 million in the U.S. and $21.6 million in the rest of the world, compared with $30.6 million and $31.4 million, respectively, in the prior-year period. The decline in sales of branded Tekturna in the U.S. is due primarily to the launch of an authorized generic of Tekturna in the U.S. and the launch of a third-party generic of aliskiren late in the first quarter of 2019. The decline in sales in the rest of the world is due to lower sales volume of Rasilez in certain territories, in part reflecting additional measures to maximize product profitability

Revenue from royalty rights – change in fair value was negative $4.3 million for the first nine months of 2019, compared with $66.1 million in the prior-year period. The decrease is primarily related to a non-cash adjustment to the AcelRx royalty asset fair value of negative $60.0 million in the second quarter of 2019. PDL received $58.3 million in net cash royalties from its royalty rights in the first nine months of 2019.

Interest revenue decreased by $2.3 million from the prior-year period due to modifications to the Company’s agreement with CareView Communications, which deferred interest payments for the first nine months of 2019.

Royalties from PDL’s licensees to the Queen et al. patents were less than $0.1 million for the first nine months of 2019, compared with $4.5 million for the prior-year period, reflecting the runout of the royalties on the sales of Tysabri.

Operating Expense Highlights

Operating expenses for the third quarter of 2019 were $34.7 million, a $3.6 million increase from the third quarter of 2018. The increase was primarily due to a $3.6 million increase in research and development (R&D) expenses associated with product development and patent licensing for LENSAR, and a $3.1 million, or 26%, increase in cost of product revenue, $2.4 million of which related to a termination provision in a Noden supply agreement amended in June 2019 involving end of contract fees, most of which were incurred in the third quarter of 2019. These increases were partially offset by a $1.1 million, or 8%, decline in G&A expenses, primarily due to lower professional fees, and a $1.8 million, or 51%, decline in sales and marketing expenses reflecting savings from the change in the Company’s marketing strategy for the Noden Products.

Operating expenses for the first nine months of 2019 were $90.6 million, a $146.5 million decrease from the prior-year period. The decrease was primarily a result of the net impact of: the $152.3 million impairment of the Noden Products intangible assets in the second quarter of 2018 and related reductions to the Noden Products contingent liability and amortization expense associated with those intangible assets which, in aggregate, accounted for

$139.1 million of the decrease; a $6.4 million, or 16%, decline in G&A expenses primarily due to lower professional and asset management fees; and a $7.9 million, or 55%, decline in sales and marketing expenses reflecting savings from the change in the Company’s marketing strategy for the Noden Products. These decreases were partially offset by an increase in R&D expenses of $3.9 million associated with product development and patent licensing for LENSAR.

Other Financial Highlights

On a GAAP basis, the net loss attributable to PDL’s shareholders for the third quarter of 2019 was $17.8 million, or $0.16 per share, compared with GAAP net income attributable to PDL’s shareholders of $25.6 million, or $0.18 per share on a diluted basis, for the prior-year period. Noteworthy items reflected in the third quarter net loss include pre-tax charges of $3.9 million for the convertible debt exchange, a $3.6 million increase to R&D expense, primarily the result of the acquisition of LENSAR intellectual property, a $2.4 million manufacturing charge for our Noden products and a $27.4 million loss due to the decrease in fair value of our investment in Evofem, partially offset by a $3.5 million gain recognized for the sale of intangible assets. Non-GAAP net income attributable to PDL’s shareholders was $10.4 million for the third quarter of 2019, compared with non-GAAP net income of $13.1 million for the third quarter of 2018.

The GAAP net loss attributable to PDL’s shareholders for the first nine months of 2019 was $15.5 million, or $0.13 per share, compared with a GAAP net loss attributable to PDL’s shareholders of $85.1 million or $0.58 per share, for the prior-year period. Non-GAAP net income attributable to PDL’s shareholders was $34.9 million for the first nine months of 2019, compared with non-GAAP net income of $44.2 million for the prior-year period.

PDL had cash and cash equivalents of $294.3 million as of September 30, 2019, compared with cash and cash equivalents of $394.6 million as of December 31, 2018.

The $100.3 million reduction in cash and cash equivalents during the first nine months of 2019 was primarily the result of common stock repurchases of $75.9 million, the Company’s investment in Evofem Biosciences of $60.0 million and costs incurred in the exchange of convertible debt of $11.1 million, which extended the maturity date of $86.1 million of our notes to December 2024, and net cash used in operations of $13.3 million. This was partially offset by the proceeds from royalty rights of $58.1 million and cash proceeds from the sale of intangible assets of $5.0 million.

Stock Repurchase Programs

In November 2018 PDL began repurchasing shares of its common stock in the open market pursuant to the
$100.0 million share repurchase program authorized by the Company’s board of directors in September 2018.
During the third quarter of 2019, the Company completed the stock repurchase program by repurchasing 1.3 million shares for an aggregate purchase price of $4.1 million.

Since initiating its first stock repurchase program in March 2017, the Company has repurchased 53.1 million shares for $155.0 million, at an average cost of $2.92 per share.

As of October 31, 2019, the Company had approximately 114.2 million shares of common stock outstanding.

Financial Guidance

PDL is affirming 2019 financial guidance for Noden product revenue, which is expected to be in the range of
$50 million to $55 million.

PDL now expects 2019 LENSAR product revenue to exceed $29 million and 2019 cash royalties to exceed
$65 million. This compares with previous guidance for LENSAR product revenue, which was expected to be in
the range of $27 million to $29 million, and cash royalties expected to be in the range of $60 million to $65 million.

Conference Call and Webcast

PDL will hold a conference call to discuss financial results and provide a business update at 4:30 p.m. Eastern time today. Slides to accompany the conference call will be available in the Investor Relations section of www.pdl.com.

To access the live conference call via phone, please dial 844-535-4071 from the U.S. and Canada or 706-679-2458 internationally. The conference ID is 3195828. A telephone replay will be available beginning approximately one hour after the call through one week following the call, and can be accessed by dialing 855-859-2056 from the U.S. and Canada or 404-537-3406 internationally. The replay passcode is 3195828.

To access the live and subsequently archived webcast of the conference call, go to the Investor Relations section of www.pdl.com and select "Events & Presentations."

On November 6, 2019 Oncopeptides AB (Nasdaq Stockholm: ONCO) reported that data from the clinical program evaluating its lead candidate melflufen in multiple myeloma and Light Chain (AL) Amyloidosis have been selected for six poster presentations to be featured at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2019 Annual Meeting taking place December 7-10 in Orlando, Florida (Press release, Oncopeptides, NOV 6, 2019, View Source [SID1234550482]). Melflufen is a peptide-conjugated alkylator belonging to the novel class of Peptidase Enhanced Cytotoxics (PEnC).

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!

A wide range of clinical and preclinical data for melflufen in both multiple myeloma and AL amyloidosis will be presented at the ASH (Free ASH Whitepaper) annual meeting. Two key data sets that will be presented are;

Updated efficacy and safety data from pivotal phase 2 HORIZON trial evaluating melflufen in patients with relapsed/refractory multiple myeloma (RRMM). The data will support the New Drug Application filing with U.S. FDA late in the first quarter of 2020.
First presentation of Progression-Free Survival (PFS) data from the ANCHOR phase 2 combination trial in RRMM patients.
"The acceptance of six abstracts from our robust clinical development program evaluating melflufen in multiple myeloma and AL amyloidosis is a significant validation of Oncopeptides’ progress in advancing our pipeline and conducting thorough scientific exploration of the compound. Of particular interest will be long-term follow up data from the HORIZON Phase 2 pivotal trial which is evaluating melflufen in RRMM. These data will form the basis of our New Drug Application for submission to the FDA late in the first quarter of 2020. The update on ANCHOR data will be of high importance as this will be the first time that we present progression free survival (PFS) data with longer term follow up from the combination study," said Jakob Lindberg, CEO of Oncopeptides. "This ASH (Free ASH Whitepaper) Annual Meeting also represents the first time in which we will be presenting data from our early-stage development of melflufen in the treatment of AL Amyloidosis, which is an area of significant unmet need as there are currently no approved therapies available to patients."

The full ASH (Free ASH Whitepaper) Annual Meeting 2019 abstract book can be found at:

View Source

The six abstracts detailing melflufen clinical program data and updates that have been accepted for poster presentations at this year’s ASH (Free ASH Whitepaper) Annual Meeting and can be found at:

www.oncopeptides/investor&media/presentations/ASH Abstracts 2019

Data included in the abstracts currently available on the ASH (Free ASH Whitepaper) Annual Meeting website is based on data cut-off dates in second quarter 2019. The final conference presentations will include additional data collected between the abstract submission cutoff and the presentation itself.

Details about the upcoming poster presentations.

Title: Clinical Activity of Melflufen in Patients with Triple-Class Refractory Multiple Myeloma and Poor-Risk Features in an Updated Analysis of HORIZON (OP-106), a Phase 2 Study in Patients with Relapsed/Refractory Multiple Myeloma Refractory to Pomalidomide and/or Daratumumab
Presenter: Maria-Victoria Mateos, Hospital Clínico Universitario de Salamanca, Salamanca, Spain
Session: 653. Myeloma: Therapy, excluding Transplantation: Poster I
Date/Time: Saturday, December 7 at 5:30 – 7:30 p.m.
Location: Hall B

Title: Updated Progression-Free Survival (PFS) and Overall Survival (OS) with Melflufen and Dexamethasone in Patients with Relapsed/Refractory Multiple Myeloma (RRMM): Results from the Phase 2 Study O-12-M1
Presenter: Sara Bringhen, MD, Division of Hematology University of Torino, Torino, Italy
Session: 653. Myeloma: Therapy, excluding Transplantation: Poster I
Date/Time: Saturday, December 7 at 5:30 – 7:30 p.m.
Location: Hall B

Title: OP201: A Phase 1/2 Study of Melflufen and Dexamethasone in Patients with Immunoglobulin Light Chain (AL) Amyloidosis
Presenter: Stefan Schönland, MD, Medizinische Klinik V and Amyloidosis Center, Universitätsklinikum Heidelberg, Heidelberg, Germany
Session: 653. Myeloma: Therapy, excluding Transplantation: Poster II
Date/Time: Sunday, December 8 at 6:00 – 8:00 p.m.
Location: Hall B

Title: ANCHOR (OP-104): Updated Efficacy and Safety from a Phase 1/2 Study of Melflufen and Dexamethasone Plus Bortezomib or Daratumumab in Patients with Relapsed/Refractory Multiple Myeloma (RRMM) Refractory to an IMiD or a Proteasome Inhibitor (PI)
Presenter: Enrique M. Ocio, MD, University Hospital Marqués de Valdecilla (IDIVAL), University of Cantabria, Santander, Spain
Session: 653. Myeloma: Therapy, excluding Transplantation: Poster II
Date/Time: Sunday, December 8 at 6:00 – 8:00 p.m.
Location: Hall B

Title: In Vitro and in Vivo Activity of Melflufen in Amyloidosis
Presenter: Kenneth Flanagan, PhD, Oncopeptides AB, Stockholm, Sweden
Session: 652. Myeloma: Pathophysiology and Pre-Clinical Studies, excluding Therapy: Poster II Date/Time: Sunday, December 8 at 6:00 – 8:00 p.m.
Location: Hall B

Title: The Burden of Relapsed/Refractory Multiple Myeloma: An Indirect Comparison of Health-Related Quality of Life Burden across Different Types of Advanced Cancers at Baseline and after Treatment Based on HORIZON (OP-106) Study of Melflufen Plus Dexamethasone
Presenter: Paul G. Richardson, MD, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA
Session: 905. Outcomes Research—Malignant Conditions (Lymphoid Disease): Poster II
Date/Time: Sunday, December 8 at 6:00 – 8:00 p.m.
Location: Hall B

For more information, please contact:
Jakob Lindberg, CEO of Oncopeptides
E-mail: [email protected]
Telephone: +46 8 615 20 40

Rein Piir, Head of Investor Relations at Oncopeptides
E-mail: [email protected]
Cell phone: +46 70 853 72 92

This information was submitted for publication at 15.00 CET on November 6, 2019.

On November 6, 2019 Nektar Therapeutics (Nasdaq: NKTR) reported its financial results for the third quarter ended September 30, 2019 (Press release, Nektar Therapeutics, NOV 6, 2019, View Source [SID1234550481]).

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 September 30, 2019 were approximately $1.7 billion as compared to $1.9 billion at December 31, 2018.

"We continue to make steady progress with our diverse portfolio of immuno-oncology and immunology programs," said Howard W. Robin, President and CEO of Nektar. "With our partner Bristol-Myers Squibb, we are conducting registrational trials evaluating the combination of bempegaldesleukin with nivolumab in melanoma, urothelial cancer and renal cell carcinoma. We are also working collaboratively with BMS to finalize the next set of registrational studies. This weekend at the SITC (Free SITC Whitepaper) Annual Meeting, we are excited to present an 18-month follow-up for patients with metastatic melanoma enrolled in our PIVOT-02 study. This follows the recent announcement of our Breakthrough Therapy Designation for the doublet which was granted by FDA in August for patients with previously untreated metastatic melanoma. For NKTR-358, we now have three separate Phase 1b clinical trials ongoing in lupus, psoriasis and atopic dermatitis with our partner Eli Lilly, with plans to add an additional autoimmune indication to the development program in 2020. And importantly, we initiated our first clinical trial for NKTR-255, our novel IL-15 agonist, in patients with non-Hodgkin lymphoma and multiple myeloma."

Revenue in the third quarter of 2019 was $29.2 million as compared to $27.8 million in the third quarter of 2018. Year-to-date revenue for 2019 was $80.8 million as compared to $1.15 billion in the first nine months of 2018. Revenue was higher in the third quarter of 2019 as compared to the same period in 2018 primarily due to non-cash royalty revenue and an increase in product sales. Revenue was lower in the first nine months of 2019 as compared to the same period in 2018 primarily because of 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 third quarter of 2019 were $128.0 million as compared to $126.4 million in the third quarter of 2018. Total operating costs and expenses in the first nine months of 2019 were $411.2 million as compared to $365.3 million in the same period of 2018. Total operating costs and expenses increased marginally in the third quarter of 2019 as compared to the third quarter of 2018 due to a decrease in research and development (R&D) expense, offset by an increase in general and administrative (G&A) expense. Total operating costs and expenses increased in the first nine months of 2019 as compared to the same period in 2018 due to increases in both R&D and G&A expense.

R&D expense in the third quarter of 2019 was $99.0 million as compared to $102.9 million for the third quarter of 2018. For the first nine months of 2019, R&D expense was $324.2 million as compared to $290.7 million in the first nine months of 2018. R&D expense was lower in the third quarter of 2019 as compared to the same period in 2018 due to decreased expense for the bempegaldesleukin program. R&D expense was higher in the first nine months as compared to the same period in 2018 primarily because of expenses for our pipeline programs, including the continued development of bempegaldesleukin in Phase 2 and registrational studies and related manufacturing costs, costs related to Phase 1 clinical studies of NKTR-358 and IND-enabling activities for NKTR-255.

G&A expense was $24.0 million in the third quarter of 2019 as compared to $18.7 million in the third quarter of 2018. G&A expense in the first nine months of 2019 was $71.6 million as compared to $57.7 million in the first nine months of 2018. G&A expense was higher in the third quarter and first nine months of 2019 as compared to the same periods in 2018 due to costs related to commercialization readiness activities for NKTR-181 and bempegaldesleukin, and increased non-cash stock-based compensation.

Net loss in the third quarter of 2019 was $98.8 million or $0.56 basic and diluted loss per share as compared to net loss of $96.1 million or $0.56 basic and diluted loss per share in the third quarter of 2018. Net loss in the first nine months of 2019 was $327.2 million or $1.87 basic and diluted loss per share as compared to net income of $779.5 million or $4.34 diluted income per share in the first nine months of 2018.

Third Quarter 2019 and Recent Business Highlights

·In September, 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 October, Nektar announced that its partner Eli Lilly initiated two Phase 1b studies of NKTR-358, a novel T regulatory (Treg) cell stimulator, one in patients with psoriasis and one in patients with atopic dermatitis. NKTR-358 is designed to treat autoimmune and inflammatory conditions by correcting the immune system imbalance that results from reduced numbers and impaired function of immune regulating Treg cells.

·In October, Nektar announced the initiation of a first-in-human, Phase 1 clinical study evaluating NKTR-255, an interleukin-15 (IL-15) receptor agonist, as monotherapy for patients with relapsed or refractory non-Hodgkin lymphoma or multiple myeloma (MM). The study will also combine NKTR-255 with multiple targeted antibodies that function through an antibody-dependent cell-mediated cytotoxicity mechanism to evaluate the safety and efficacy in adults with relapsed or refractory MM.

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

2019 Society for Immunotherapy and Cancer (SITC) (Free SITC Whitepaper) Annual Meeting, National Harbor, MD:

·Oral Presentation: "Clinical activity of BEMPEG plus NIVO in previously untreated patients with metastatic melanoma: updated results from the phase 1/2 PIVOT-02 study"
oPresenter: Dr. Adi Diab, MD Anderson Cancer Center
oSession: Concurrent Session 310: Combination Phase 1-2 Clinical Trials
oDate: Saturday, November 9, 2019, 5:15 p.m. – 5:30 p.m. Eastern Standard Time

·Poster: "NKTR-255, a polymer-conjugated IL-15 receptor agonist, enhances efficacy of therapeutic monoclonal antibodies with ADCC activity in solid tumor models", Kivimäe, S., et al.
oSession Date and Time: Friday, November 8, 2019, 7:00 a.m. – 8:00 p.m. Eastern Standard Time

·Poster: "Bempegaldesleukin in combination with local radiation and systemic checkpoint blockade induces a robust systemic anti-tumor immunity", Pieper, A., et al.
oSession Date and Time: Friday, November 8, 2019, 7:00 a.m. – 8:00 p.m. Eastern Standard Time

·Poster: "Characterization and comparison of NKTR-255, a polymer-conjugated IL-15 versus IL-15 superagonist", Miyazaki, T., et al.
oSession Date and Time: Saturday, November 9, 2019, 7:00 a.m. – 8:00 p.m. Eastern Standard Time

·Trials in Progress Poster: "A multicenter, open-label, exploratory platform study to evaluate biomarkers and immunotherapy combinations for the treatment of patients with metastatic castration-resistant prostate cancer (PORTER)", Nissola, L., et al.
oSession Date and Time: Friday, November 8, 2019, 7:00 a.m. – 8:00 p.m. Eastern Standard Time

ACR 2019 American College of Rheumatology Annual Meeting, Atlanta, GA:

·Poster: "Selective induction of functional regulatory T-cells in healthy volunteers by NKTR-358, a novel IL-2 conjugate Treg stimulator, in development for the treatment of autoimmune diseases", Fanton, C., et al.
oSession: T Cell Biology & Targets in Autoimmune & Inflammatory Disease Poster
oDate: Sunday, November 10, 2019, 9:00 a.m. – 11:00 a.m. Eastern Standard Time

The Promise of Interleukin-2 Therapy 2019, Paris, France:

·Presentation: "NKTR-358: A polymer-conjugated IL-2 that drives the selective expansion of endogenous Tregs for the treatment of autoimmune diseases"
oPresenter: Christine Fanton, Ph.D., Nektar Therapeutics
oSession: Session V.a – Novel IL-2s
oDate: Friday, November 15, 2019, 2:00 p.m. Central European Time

·Presentation: "Bempegaldesleukin: A polymer-conjugated IL-2 prodrug for multiple immune oncology applications"
oPresenter: Loui Madakamutil, Ph.D., Nektar Therapeutics
oSession: Session VI – IL-2 in cancer therapy
oDate: Friday, November 15, 2019, 4:30 p.m. Central European Time

11th Annual PEGs Europe, Lisbon, Portugal:

·Presentation: "TLR Agonist NKTR-262 Immunotherapy Combination with Bempegaldesleukin (NKTR-214) Harnessing Innate and Adaptive Immune System for the Treatment of Solid Tumors"
oPresenter: Saul Kivimäe, Ph.D., Nektar Therapeutics
oSession: Reprogramming the Microenvironment/The Innate Immune System/Glyco-immune Checkpoints
oDate: Tuesday, November 19, 2019, 5:30 p.m. Western European Time

·Presentation: "NKTR-255: A Polymer-Conjugated IL-15 that Enhances CAR T Efficacy in Murine Models"
oPresenter: Loui Madakamutil, Ph.D., Nektar Therapeutics
oSession: TILs and Gamma Delta Therapy
oDate: Thursday, November 21, 2019, 11:15 a.m. Western European Time

Melanoma Bridge 2019, Naples, Italy:

·Presentation: "Clinical activity of BEMPEG plus NIVO in previously untreated patients with metastatic melanoma: updated results from the phase 1/2 PIVOT-02 study"- [Encore Presentation]
oPresenter: Igor Puzanov, M.D., Roswell Park Comprehensive Cancer Center, Melanoma Bridge Co-chair
oSession: Emergent Strategies Session
oDate: Saturday, December 7, 2019, 12:00 p.m. – 12:15 p.m. Central European Time

61st American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, Orlando, FL:

·Poster: "Combination of NKTR-255, a Polymer Conjugated Human IL-15, with CD19 CAR T Cell Immunotherapy in a Preclinical Lymphoma Model", Chou, C., et al.
oSession: 625. Lymphoma: Pre-Clinical—Chemotherapy and Biologic Agents: Poster II
oDate: Sunday, December 8, 2019, 6:00 p.m. – 8:00 p.m. Eastern Standard Time

·Poster: "Restoring Innate and Adaptive Immune Repertoire in Multiple Myeloma for Therapeutic Application", Fernandez, R., et al.
oSession: 652. Myeloma: Pathophysiology and Pre-Clinical Studies, Excluding Therapy: Poster III
oDate: Monday, December 9, 2019, 6:00 p.m. – 8:00 p.m. Eastern Standard Time

·Trials in Progress Poster: "A Phase 1, Open-Label, Multi-Center, Dose Escalation and Dose Expansion Study of NKTR-255 As a Single Agent in Relapsed or Refractory Hematologic Malignancies and in Combination with Daratumumab As a Salvage Regimen for Multiple Myeloma", Shah, N. et al.
oSession: 704. Immunotherapies: Poster III
oDate: Monday, December 9, 2019, 6:00 p.m. – 8:00 p.m. Eastern Standard Time

Conference Call to Discuss First Quarter 2019 Financial Results

Nektar management will host a conference call to review the results beginning at 5:00 p.m. Eastern Standard Time/2:00 p.m. Pacific Standard Time, Wednesday, November 6, 2019.

This press release and a 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 Monday, December 9, 2019.

To access the conference call, follow these instructions:

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

Conference ID: 3079832 (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.