On November 2, 2021 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on the discovery and development of targeted drugs for the treatment of cancer, reported Laurence Reilly, M.D., LL.M as its interim chief medical officer (Press release, Cellectar Biosciences, NOV 2, 2021, View Source [SID1234594106]). Dr. Reilly will oversee the company’s clinical development programs and report to Chief Executive Officer James Caruso. He succeeds John Friend, MD, who is stepping down for personal reasons but will stay on through mid-November to finalize the ongoing transition.

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"We are delighted to welcome Laurence to our executive team and look forward to his leadership of our clinical development programs. His deep background in hematological oncology and experience leading global clinical development teams to successfully bring new therapies to patients will support Cellectar’s next phase of growth. Laurence will be a tremendous asset as we execute our registrational trial for iopofosine I-131 (iopofosine) in Waldenstrom’s macroglobulinemia (WM) and continue to develop our hematologic cancer and pediatric solid tumor programs as well as further advance our other pipeline assets," said James Caruso, president and CEO of Cellectar.

"We thank John for his outstanding work and collaboration with the FDA that established a defined registrational pathway for iopofosine in WM, and for securing the world-class global trial sites and thought leadership support critical for our pivotal WM study. We are now focused on executing and completing our pivotal WM trial, enriching our Phase 2 CLOVER-1 trial with additional multiple myeloma patients, and enrolling in our pediatric trial. Our balance sheet remains strong with sufficient capital to achieve our strategic goals into the second half of 2023, and we look forward to continued execution across our programs."

Dr. Laurence Reilly has consulted for the company since early this year. Prior to joining Cellectar, he provided strategic consulting and due-diligence services to biotech companies, life science venture capital and private equity clients, alongside serving as chief strategy & development officer to a European-based medical device company. Prior to founding his consulting practice, Dr. Reilly served as chief scientific officer and vice president at Avillion, a drug development company focused on the co-development and financing of pharmaceutical candidates, where he was responsible for clinical and strategic oversight of co-development programs and partnering with both large pharma and biotech, including Pfizer and AstraZeneca. Dr. Reilly previously served as a clinician at Pfizer and began his industry career at Lundbeck where he served as medical and scientific advisor overseeing investigator-initiated research, opinion leader interaction and new compound presentation. Dr. Reilly earned his medical degree from the University of Liverpool Medical School, U.K., and a Masters Degree in Law from De Montfort University, U.K.

Dr. Reilly added, "I am excited to join the Cellectar team and drive the clinical development and success of iopofosine. The data in WM and multiple myeloma are extremely encouraging and with a defined registrational path, iopofosine is poised to become a meaningful treatment option for these patients. I look forward to bringing iopofosine to market and continuing to develop the exciting pipeline of candidates from the PDC platform."

On November 2, 2021 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported consolidated financial results for the third quarter ended September 30, 2021 and raised full-year 2021 product revenue guidance to $7.4 to $7.5 billion (Press release, Vertex Pharmaceuticals, NOV 2, 2021, View Source [SID1234594105]).

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"Our financial performance in the third quarter was outstanding, marked by the exceptional continued growth of TRIKAFTA/KAFTRIO. Based on these results, we are again raising our 2021 product revenue guidance, and we see significant additional growth ahead as we continue to deliver this transformational medicine to more people with CF," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. "During the last quarter, we also significantly expanded and advanced our pipeline. We announced unprecedented data from the first type 1 diabetes patient dosed with our stem cell-derived, allogeneic islet cells (VX-880). We completed enrollment in the Phase 2 proof-of-concept study of VX-147 in APOL1-mediated FSGS and will report results later this quarter. We also achieved target enrollment in both pivotal CTX001 studies to support our regulatory submissions next year. Finally, we made important advancements in our earlier stage pipeline and expect to submit INDs for both our CF mRNA program and type 1 diabetes cells and device program in 2022."

Product revenues increased 29% compared to the third quarter of 2020, primarily driven by the strong launches of KAFTRIO in Europe and performance of TRIKAFTA in the U.S., including the rapid uptake of TRIKAFTA to children 6-11 years old. Net product revenues in the third quarter of 2021 increased 13% to $1.38 billion in the U.S. and increased 92% to $601 million outside the U.S., compared to the third quarter of 2020.

GAAP and Non-GAAP net income increased compared to the third quarter of 2020, driven by strong product revenue growth.

Cash, cash equivalents and marketable securities were $7.0 billion, an increase of $0.3 billion compared to $6.7 billion as of December 31, 2020. The increase was primarily driven by strong operating cash flow partially offset by repurchases of our common stock authorized under our share repurchase programs and a $900 million payment in the second quarter of 2021 in connection with the amendment of Vertex’s collaboration with CRISPR Therapeutics.

Combined GAAP and Non-GAAP R&D and SG&A expenses increased compared to the third quarter of 2020, primarily due to the expansion of Vertex’s pipeline in cystic fibrosis and other disease areas and incremental investment to support the launches of Vertex’s medicines globally.

GAAP income taxes increased compared to the third quarter of 2020, primarily due to Vertex’s increased operating income and the impact of certain discrete tax events (Note 1).

Non-GAAP income taxes increased compared to the third quarter of 2020, primarily due to Vertex’s increased operating income.

Full-Year 2021 Financial Guidance

Vertex today increased its full-year 2021 product revenue guidance based on strong year-to-date performance. Vertex’s guidance is summarized below:

Key Business Highlights

Cystic Fibrosis (CF) Marketed Products

Vertex anticipates the number of CF patients treated with our medicines will continue to grow as we enter into additional reimbursement agreements, achieve new approvals for the treatment of younger patients, and expand treatment options for the approximately 10 percent of patients who do not benefit from cystic fibrosis transmembrane conductance regulator (CFTR) modulators, all of which will lead to continued growth of our CF business in the years ahead. Recent progress includes:

Vertex has signed a Letter of Intent (LOI) with the pan-Canadian Pharmaceutical Alliance (pCPA) regarding the public reimbursement of TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for eligible patients with CF and has also reached multiple provincial agreements across Canada providing approximately 90% of Canadian patients 12 years of age and older and covered by government insurance with reimbursed access to TRIKAFTA.
Data are being presented at the North American CF Conference (NACFC) in November from the ongoing 192-week TRIKAFTA open-label extension study which show there has been no loss in mean lung function during long term follow-up at 96 weeks, a first for any CFTR modulator to date. Data from a retrospective study of KALYDECO (ivacaftor) will also be presented at the conference. The results showed that people with CF over approximately 6 years old had significantly lower rates of mortality, lung transplant and pulmonary exacerbations compared to those not on treatment.
Vertex’s application for approval of TRIKAFTA in children 6 through 11 years of age has been accepted for priority review by Health Canada.
TRIKAFTA/KAFTRIO is now approved and reimbursed or accessible in more than 20 countries outside the U.S., including Italy, France and Canada.

R&D pipeline

Vertex is delivering on a diversified pipeline of potentially transformative small molecule, cell and genetic therapies aimed at serious diseases. Recent and anticipated progress for key pipeline programs is noted below:

Cystic Fibrosis

The Phase 3 studies evaluating the new once-daily investigational triple combination of VX-121/tezacaftor/VX-561 (deutivacaftor) are underway.
In collaboration with Moderna, Vertex is evaluating CF mRNA therapeutics designed to treat the underlying cause of CF by enabling cells in the lungs to produce functional CFTR protein for the treatment of the 10% of patients who do not produce any CFTR protein. IND-enabling studies are underway and Vertex expects to submit an IND for this program in 2022.
Beta Thalassemia and Sickle Cell Disease (SCD)

The CTX001 program employs a non-viral ex vivo CRISPR gene-editing therapy for the treatment of transfusion-dependent thalassemia (TDT) and severe SCD.
Data presented to date support the profile of CTX001 as a one-time functional cure for patients with TDT and severe SCD, showing consistent and durable benefit across all treated patients.
Target enrollment has been achieved in the ongoing clinical trials in TDT and SCD to support the planned regulatory submissions in late 2022.
Type 1 Diabetes (T1D)

Vertex is evaluating cell therapies designed to replace insulin-producing islet cells that are destroyed in people with T1D with the goal of delivering a potential functional cure.
VX-880 is a stem cell-derived, allogeneic, fully differentiated, insulin-secreting islet cell replacement therapy, using standard immunosuppression to protect the implanted cells. VX-880 is being evaluated in a Phase 1/2 clinical trial for the treatment of T1D.
In October, Vertex announced positive Day 90 data for the first T1D patient in the Phase 1/2 clinical trial of VX-880. The patient was treated with a single infusion of VX-880 at half the target dose in conjunction with standard immunosuppressive therapy. The patient demonstrated rapid and robust improvements in multiple measures, including significant increases in fasting and stimulated C-peptide, improvements in glycemic control as measured by HbA1c, and a 91% decrease in exogenous insulin requirements.
The VX-880 Phase 1/2 study is ongoing at multiple clinical sites in the U.S. and the Clinical Trial Application (CTA) has been approved in Canada.
Vertex is pursuing a second program in which these stem cell-derived, fully differentiated, insulin-secreting islet cells are encapsulated and implanted in an immunoprotective device. IND-enabling studies are underway and Vertex expects to submit an IND for the cells and device program in 2022.
APOL1-mediated Kidney Diseases (AMKD)

Vertex is evaluating the potential of oral, small molecule inhibitors of APOL1 function to treat people with AMKD.
Enrollment is complete in the Phase 2 proof-of-concept study evaluating VX-147 for the treatment of people with APOL1-mediated focal segmental glomerulosclerosis (FSGS) with reduction of proteinuria as the primary endpoint. Results from this study will be reported in the fourth quarter of 2021 and will inform the potential progression of VX-147 into pivotal studies in the broader population of people with APOL1-mediated non-diabetic proteinuric kidney diseases.
Preclinical data for VX-147 will be presented at the American Society of Nephrology Annual Meeting, November 4-7, 2021.
Pain (NaV1.8)

Vertex has discovered multiple selective small molecule inhibitors of NaV1.8 with the objective of creating a new class of medicines that have the potential to be highly effective for both acute and chronic pain, without the limitations of opioids and other existing pain medications.
Two Phase 2 dose ranging acute pain studies with VX-548 are underway, one following bunionectomy surgery and the other following abdominoplasty surgery. Data from the acute pain studies are expected in Q1 2022.
Alpha-1 Antitrypsin (AAT) Deficiency

Vertex plans to advance one or more novel small molecule zAAT correctors into the clinic in 2022.
Investments in External Innovation

To further expand our capabilities in gene editing, Vertex recently announced two new collaborations.

In August, Vertex announced a new collaboration with Arbor Biotechnologies Inc. to enhance efforts in developing ex vivo engineered cell therapies for multiple serious diseases using Arbor’s proprietary CRISPR gene-editing technology.
In October, Vertex announced a new collaboration with Mammoth Biosciences to develop in vivo gene-editing therapies for two diseases using Mammoth’s next-generation CRISPR systems.

On November 2, 2021 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, reported its financial results for the third quarter 2021 (Press release, Ultragenyx Pharmaceutical, NOV 2, 2021, View Source [SID1234594104]).

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"In the third quarter we executed on key commercial and clinical milestones, including the resumption of the GTX-102 study for the treatment of Angelman syndrome and the initiation of our seamless Phase 1/2/3 study of UX701 for the treatment of Wilson disease. Looking ahead, we are preparing to initiate four additional studies across multiple modalities, including two Phase 3 gene therapies, a Phase 2/3 anti-sclerostin monoclonal antibody, and a Phase 1/2 leveraging our first mRNA program," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "The breadth of our clinical programs with the strength of our balance sheet put us in position to deliver disease-modifying therapies across a spectrum of rare diseases."

Third Quarter 2021 Financials and Full Year Crysvita Guidance Update

In the third quarter 2021, Crysvita revenue in Ultragenyx territories1 increased 35% versus the third quarter 2020. For the full year 2021, the company now expects the 2021 Crysvita revenue to be towards the upper end of the guidance range of $180 million to $190 million that was previously provided.

Dojolvi revenue in the third quarter 2021 grew 176% versus the third quarter of 2020, which was the first quarter following FDA approval. As of the end of the third quarter 2021, the company had received approximately 310 completed start forms from approximately 145 unique prescribers. This has led to approximately 250 patients on reimbursed therapy as of the end of September 2021.

Third quarter 2021 revenue included $12.1 million related to the technology transfer as part of the Daiichi Sankyo strategic manufacturing partnership around the company’s producer cell line and HEK293 technologies. The technology transfer activities and resulting revenue is expected to be substantially complete in the fourth quarter 2021.

Total operating expenses of $171.5 million in the third quarter 2021 increased 30% or $39.7 million versus the third quarter 2020, primarily driven by pipeline advancements. For the year, total operating expenses are expected to increase modestly as the company continues the commercial launch of Dojolvi and supports six clinical programs, including four pivotal studies.

Net cash used in operations for the nine months ended September 30, 2021 was $284.4 million, compared to net cash used of $69.8 million for the same period in 2020 which included approximately $154 million of operating cash received in 2020 from Daiichi Sankyo related to the collaboration and license agreement. Cash, cash equivalents, and marketable debt securities were $941.4 million as of September 30, 2021.

Program Updates and Upcoming Milestones

GTX-102 for the treatment of Angelman Syndrome, partnered with GeneTx

The U.S. FDA removed the clinical hold, allowing GeneTx to begin dosing naïve pediatric patients in the Phase 1/2 study of GTX-102 in patients with Angelman syndrome.
The first patients in Canada have been dosed, with dosing in the U.K. and U.S. currently expected to begin in the fourth quarter of 2021.
A preliminary update on the first four patients in the study is expected to be available around the end of the year, with complete data from the full cohort of 12 patients anticipated in mid-2022.
Gene Therapy Clinical Program Updates

UX701 for the treatment of Wilson disease: Multiple patients with Wilson disease have been successfully screened and are enrolled in the baseline monitoring period prior to dosing in the seamless Phase 1/2/3 study. Following the initial screening that includes testing for pre-existing antibodies to the AAV9 capsid, patients will be evaluated to ensure stable measures of disease during a 6-to 12-week baseline monitoring period after which they will then be dosed with either UX701 or placebo.
DTX401 for the treatment of Glycogen Storage Disease Type Ia: The first patients in the U.S. and Canada are expected to enter a 4- to 8-week baseline screening period around the end of 2021 after which they would receive a single dose of DTX401 or placebo.
DTX301 for the treatment of Ornithine Transcarbamylase Deficiency: The first patients in the U.S. are expected to enter a 4- to 8- week baseline screening period around the end of 2021 after which they would receive a single dose of DTX301 or placebo.
14th International Congress of Inborn Errors of Metabolism (ICIEM): Ten abstracts have been accepted for this hybrid conference that will be held November 21-24, 2021. Of the five oral presentations accepted, two will include longer-term follow-up data from the Phase 1/2 clinical studies of DTX301 for OTC deficiency and DTX401 for GSDIa.
UX143 for the treatment of Osteogenesis Imperfecta, or OI

At the 2021 American Society for Bone and Mineral Research (ASBMR) annual meeting, additional secondary endpoint data from the Phase 2b ASTEROID study demonstrated that treatment with UX143 resulted in dose-dependent increase in P1NP serum levels, a marker of bone formation, and decrease in CTx serum levels, a marker of bone resorption confirming the mechanism of action of sclerostin inhibition over the 12-month treatment period. Observed improvements in bone mineral density were continuous over the 12 months of the study, with comparable gains achieved in the first and second 6 months of treatment in the high dose group despite temporal changes in biomarkers.
A Phase 2/3 study in children and young adults is expected to initiate around the end of 2021, with additional studies in other age groups to follow.
1: Ultragenyx territories include the collaboration revenue from the North American profit share territory and other regions where revenue from product sales are recognized by Ultragenyx. 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.

Conference Call and Webcast Information

Ultragenyx will host a conference call today, Tuesday, November 2, 2021, at 2 p.m. PT/ 5 p.m. ET to discuss the third quarter 2021 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 1098326. The replay of the call will be available for one year.

On November 2, 2021 Exelixis, Inc. (Nasdaq: EXEL) reported financial results for the third quarter of 2021 and provided an update on progress toward achieving key corporate objectives, as well as commercial, clinical and pipeline development milestones (Press release, Exelixis, NOV 2, 2021, View Source [SID1234594103]).

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"Exelixis continued to execute across all facets of our business in the third quarter of 2021 with significant progress across our commercial, clinical development and pipeline activities," said Michael M. Morrissey, Ph.D., President and Chief Executive Officer, Exelixis. "We are pleased with the growth of CABOMETYX prescriptions in the third quarter in the face of increasing competition, driven by the continued broad adoption of the CABOMETYX and OPDIVO combination regimen in first-line renal cell carcinoma. Additionally, in September, CABOMETYX was approved by the U.S. Food and Drug Administration for a differentiated thyroid cancer indication, further expanding the CABOMETYX label to add an important new treatment option for a patient population with significant unmet need. We also made important strides across our clinical pipeline during the quarter, including expanding our XL092 development program and progressing enrollment for our XB002 and XL102 phase 1 studies. More recently, following the FDA’s acceptance of the Investigational New Drug application for XL114, we in-licensed the compound from Aurigene. Additionally, we signed a new collaboration agreement with STORM Therapeutics for a novel therapeutic approach to treat cancer. I’d like to thank the Exelixis team for their collective hard work and execution in the third quarter and look forward to providing further updates on our business in the remainder of the year."

Third Quarter 2021 Financial Results

Total revenues for the quarter ended September 30, 2021 were $328.4 million, compared to $231.1 million for the comparable period in 2020.

Total revenues for the quarter ended September 30, 2021 included net product revenues of $263.1 million, compared to $168.6 million for the comparable period in 2020. The increase in net product revenues was primarily related to an increase in sales volume that was driven by strong uptake for the combination therapy of CABOMETYX (cabozantinib) and OPDIVO (nivolumab) following approval by the U.S. Food and Drug Administration (FDA) in January 2021.

Collaboration revenues, composed of license revenues and collaboration services revenues, were $65.3 million for the quarter ended September 30, 2021, compared to $62.5 million for the comparable period in 2020. The increase in collaboration revenues was primarily related to an increase in recognition of milestone-related revenues and higher royalty revenues for the sales of cabozantinib outside of the U.S. generated by Exelixis’ collaboration partners, Ipsen Pharma SAS (Ipsen) and Takeda Pharmaceutical Company Limited (Takeda), which was partially offset by a decrease in development costs reimbursements earned.

Research and development expenses for the quarter ended September 30, 2021 were $163.4 million, compared to $176.8 million for the comparable period in 2020. The decrease in research and development expenses was primarily related to decreases in clinical trial costs, license and other collaboration costs and stock-based compensation expense, which was partially offset by an increase in personnel expenses.

Selling, general and administrative expenses for the quarter ended September 30, 2021 were $101.6 million, compared to $88.2 million for the comparable period in 2020. The increase in selling, general and administrative expenses was primarily related to increases in personnel expenses, marketing costs and corporate giving, which was partially offset by a decrease in stock-based compensation expense.

Provision for (benefit from) income taxes for the quarter ended September 30, 2021 was $15.1 million, compared to $(6.0) million for the comparable period in 2020, primarily due to the change in pre-tax income (loss).

GAAP net income (loss) for the quarter ended September 30, 2021 was $38.2 million, or $0.12 per share, basic and diluted, compared to GAAP net loss of $(32.0) million, or $(0.10) per share, basic and diluted, for the comparable period in 2020.

Non-GAAP net income for the quarter ended September 30, 2021 was $64.5 million, or $0.20 per share, basic and diluted, compared to non-GAAP net income of $11.2 million, or $0.04 per share, basic and diluted, for the comparable period in 2020. Non-GAAP net income excludes stock-based compensation, adjusted for the related income tax effect.

Cash, cash equivalents, restricted cash equivalents and investments were $1.8 billion at September 30, 2021, compared to $1.5 billion at December 31, 2020.

Non-GAAP Financial Measures

To supplement Exelixis’ financial results presented in accordance with U.S. Generally Accepted Accounting Principles (GAAP), Exelixis presents non-GAAP net income (and the related per share measures), which excludes from GAAP net income (loss) (and the related per share measures) stock-based compensation expense, adjusted for the related income tax effect for all periods presented.

Exelixis believes that the presentation of these non-GAAP financial measures provides useful supplementary information to, and facilitates additional analysis by, investors. In particular, Exelixis believes that these non-GAAP financial measures, when considered together with its financial information prepared in accordance with GAAP, can enhance investors’ and analysts’ ability to meaningfully compare Exelixis’ results from period to period, and to identify operating trends in Exelixis’ business. Exelixis has excluded stock-based compensation expense, adjusted for the related income tax effect, because it is a non-cash item that may vary significantly from period to period as a result of changes not directly or immediately related to the operational performance for the periods presented. Exelixis also regularly uses these non-GAAP financial measures internally to understand, manage and evaluate its business and to make operating decisions.

These non-GAAP financial measures are in addition to, not a substitute for, or superior to, measures of financial performance prepared in accordance with GAAP. Exelixis encourages investors to carefully consider its results under GAAP, as well as its supplemental non-GAAP financial information and the reconciliation between these presentations, to more fully understand Exelixis’ business. Reconciliations between GAAP and non-GAAP results are presented in the tables of this release.

2021 Financial Guidance

Guidance updated on November 2, 2021 from previously provided guidance on August 5, 2021.

(2)

Includes $50 million of non-cash stock-based compensation expense.

(3)

Includes $70 million of non-cash stock-based compensation expense.

(4)

This cash and investments guidance does not include any potential new business development activity.

(5)

Cash and investments is composed of cash, cash equivalents, restricted cash equivalents and investments.

Cabozantinib Highlights

Cabozantinib Franchise Net Product Revenues and Royalties. Net product revenues generated by the cabozantinib franchise in the U.S. were $263.1 million during the third quarter of 2021, with net product revenues of $259.8 million from CABOMETYX and $3.3 million from COMETRIQ (cabozantinib). Exelixis earned $27.1 million in royalty revenues during the quarter ended September 30, 2021, pursuant to collaboration agreements with our partners, Ipsen and Takeda.

Exelixis Partner Takeda and Ono Pharmaceutical Co., Ltd. (Ono) Receive Approval in Japan for CABOMETYX in Combination with OPDIVO for the Treatment of Unresectable or Metastatic Renal Cell Carcinoma (RCC). In August, Exelixis announced that Takeda, its partner responsible for the clinical development and commercialization of CABOMETYX in Japan, and Ono, Bristol Myers Squibb’s partner responsible for the clinical development and commercialization of OPDIVO in Japan, received approval from the Japanese Ministry of Health, Labour and Welfare to manufacture and market CABOMETYX in combination with OPDIVO as a treatment for unresectable or metastatic RCC. The approval was based on the results of CheckMate -9ER, a phase 3 pivotal trial evaluating CABOMETYX in combination with OPDIVO in previously untreated patients with advanced or metastatic RCC compared with sunitinib. Upon the first commercial sale of the combination in Japan, Exelixis received a milestone payment of $20.0 million from Takeda in the third quarter of 2021.

FDA Approval of CABOMETYX for Patients with Previously Treated Radioactive Iodine (RAI)-Refractory Differentiated Thyroid Cancer (DTC). In September, Exelixis announced that the FDA approved CABOMETYX for the treatment of adult and pediatric patients 12 years of age and older with locally advanced or metastatic DTC that has progressed following prior vascular endothelial growth factor receptor-targeted therapy and who are RAI-refractory or ineligible. The FDA granted Breakthrough Therapy designation and Priority Review to CABOMETYX for this indication, and its approval came more than two months ahead of the Prescription Drug User Fee Act target action date of December 4, 2021.

Cabozantinib Presentations at the 2021 European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress. In September, cabozantinib was the subject of multiple clinical data presentations at the 2021 ESMO (Free ESMO Whitepaper) Congress, including: a post-hoc exploratory analysis of the phase 3 CheckMate -9ER pivotal trial demonstrating efficacy benefits of the combination of CABOMETYX and OPDIVO compared with sunitinib as a first-line treatment in advanced RCC patients regardless of prior nephrectomy status; detailed results from the expanded cohort 6 of the phase 1b COSMIC-021 trial of cabozantinib in combination with atezolizumab in patients with metastatic castration-resistant prostate cancer; and final results from the phase 3 COSMIC-311 pivotal trial of CABOMETYX in patients with previously treated RAI-refractory DTC.

Pipeline Highlights

Exelixis and Invenra, Inc. (Invenra) Expand Collaboration to Discover and Develop Novel Biologics in Oncology. In August, Exelixis and Invenra announced an expansion to their discovery and licensing collaboration to include an additional 20 oncology targets. The augmented partnership builds on the two companies’ ongoing collaboration and license agreement to discover and develop mono-specific and multi-specific antibodies for incorporation into novel biologics to treat cancer, which was initially announced in May 2018 and expanded in October 2019.

Exelixis In-Licenses Second Anti-Cancer Compound from Aurigene Discovery Technologies Limited (Aurigene) Following FDA Acceptance of Investigational New Drug (IND) Application. In October, Exelixis and Aurigene announced that Exelixis exercised its exclusive option to in-license XL114 (formerly AUR104) under the companies’ July 2019 collaboration, option and license agreement. As a result, Exelixis assumed responsibility for all subsequent clinical development, manufacturing and commercialization of the compound, which inhibits the CARD11-BCL10-MALT1 signaling pathway that promotes lymphocyte survival and proliferation. Following the FDA’s recent acceptance of Exelixis’ IND, the company plans to initiate a phase 1 trial of XL114 as a monotherapy in patients with non-Hodgkin’s lymphoma.

Corporate Updates

Exelixis and STORM Therapeutics (STORM) Enter Exclusive Collaboration and License Agreement to Discover and Develop Inhibitors of Novel RNA Modifying Enzymes. In October, Exelixis and STORM entered into an exclusive collaboration and license agreement under which the companies will discover and advance novel drug leads intended for the treatment of cancer. The collaboration will focus initially on ADAR1, advancing early work by STORM applying its proprietary RNA epigenetic platform, as well as explore an additional undisclosed target.

Basis of Presentation

Exelixis has adopted a 52- or 53-week fiscal year that generally ends on the Friday closest to December 31st. For convenience, references in this press release as of and for the fiscal periods ended October 1, 2021, January 1, 2021 and October 2, 2020 are indicated as being as of and for the periods ended September 30, 2021, December 31, 2020, and September 30, 2020, respectively.

Conference Call and Webcast

Exelixis management will discuss the company’s financial results for the third quarter of 2021 and provide a general business update during a conference call beginning at 5:00 p.m. EDT / 2:00 p.m. PDT today, Tuesday, November 2, 2021.

To access the webcast link, log onto www.exelixis.com and proceed to the News & Events / Event Calendar page under the Investors & Media heading. Please connect to the company’s website at least 15 minutes prior to the conference call to ensure adequate time for any software download that may be required to listen to the webcast. Alternatively, please call 855-793-2457 (domestic) or 631-485-4921 (international) and provide the conference call passcode 9563879 to join by phone.

A telephone replay will be available until 8:00 p.m. EDT on November 4, 2021. Access numbers for the telephone replay are: 855-859-2056 (domestic) and 404-537-3406 (international); the passcode is 9563879. A webcast replay will also be archived on www.exelixis.com for one year.

On November 2, 2021 Corvus Pharmaceuticals, Inc. (NASDAQ: CRVS), a clinical-stage biopharmaceutical company, reported that it will present data from its Phase 1/1b trial of mupadolimab at the 2021 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting, taking place on November 10-14, 2021 (Press release, Corvus Pharmaceuticals, NOV 2, 2021, View Source [SID1234594102]).

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The updated data from the mupadolimab study will be presented in a poster at SITC (Free SITC Whitepaper). Details of the poster presentation are as follows:

TITLE: Activating CD73 on B cells as a target for immunotherapy of COVID-19 and viral associated cancers: Clinical activity in human papilloma virus positive (HPV) head and neck squamous cell cancers (HNSCC)
PRESENTER: Jason J. Luke, M.D, University of Pittsburgh School of Medicine
CATEGORY: Immune-stimulants and immune modulators
POSTER #: 701
DATE & TIME: Friday, November 12, 2021, 7:00 a.m. – 8:30 p.m. ET
LOCATION: Walter E. Washington Convention Center, Hall E, or View Source

Conference Call, Webcast and Presentation Slides
Corvus will host a conference call and webcast on November 12, 2021 at 9:00 am ET (6:00 am PT) to discuss the update on mupadolimab and other topics. The conference call can be accessed by dialing 1-877-407-0784 (toll-free domestic) or 1-201-689-8560 (international) and using the conference ID 13724618. The live webcast, which will include presentation slides, may be accessed via the investor relations section of the Corvus website. A replay of the webcast will be available on Corvus’ website for 90 days.