On November 9, 2020 Castle Biosciences, Inc. (Nasdaq: CSTL), a skin cancer diagnostics company providing personalized genomic information to improve cancer treatment decisions, reported its financial results for the third quarter and nine months ended Sept. 30, 2020 (Press release, Castle Biosciences, NOV 9, 2020, View Source [SID1234570371]).

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"The Castle team made significant progress on our strategic growth initiatives in the third quarter," said Derek Maetzold, president and chief executive officer of Castle Biosciences. "We also returned to year-over-year volume growth in DecisionDx-Melanoma report volume, our current lead revenue driver, despite a reduction in melanoma diagnoses, which we believe is due to COVID-19. We are pleased to see, in October, the posting of the expanded local coverage determination (LCD) and accompanying billing and coding article for our DecisionDx-Melanoma test, with an effective date of November 22, 2020.

"As laid out at the beginning of 2020, we planned to launch our two near-term pipeline tests in the second half of the year. We successfully launched DecisionDx-SCC, our test for patients diagnosed with cutaneous squamous cell carcinoma (SCC) with one or more high-risk factors, on August 31, leveraging our strong, existing dermatologic sales channels. Through October, we have received 282 orders by 193 clinicians for this prognostic test, of which most were existing customers. Additionally, last week we launched our second new test of 2020, DecisionDx DiffDx-Melanoma, for use in patients with suspicious pigmented lesions. We estimate that combined, our three commercially available skin cancer products, DecisionDx-Melanoma, DecisionDx-SCC and DecisionDx DiffDx-Melanoma, have a total addressable U.S. market of approximately $2.0 billion.

"We believe evidence development is key to further support clinician adoption and commercial coverage of our tests. During the quarter, we advanced our clinical studies and saw continued progress with publications of peer-reviewed articles.

"As we look ahead to the remainder of 2020 and into 2021, with our team’s continued commitment to improve patient outcomes, we believe we remain well-positioned for long-term growth and value creation."

Third Quarter Ended Sept. 30, 2020, Selected Results

Revenues were $15.2 million in the third quarter of 2020, a 3% increase compared to $14.8 million in the third quarter of 2019. Included in revenue for the quarter were positive revenue adjustments related to tests delivered in prior periods of $1.5 million, compared to positive adjustments of $3.2 million for the quarter ended Sept. 30, 2019.
Delivered 4,404 DecisionDx-Melanoma test reports in the third quarter of 2020, a 7% increase compared to the 4,126 reports delivered in the third quarter of 2019, despite a reduction in melanoma diagnoses, which the Company believes is due to the ongoing impacts of COVID-19.
Delivered 318 DecisionDx-UM test reports in the third quarter of 2020, compared to 356 reports in the third quarter of 2019. The Company believes this decrease is the result of fewer patient visits to physicians during the quarter due to the COVID-19 pandemic.
The Company’s DecisionDx-SCC test became commercially available on Aug. 31, 2020. For the period of Aug. 31, 2020 through Sept. 30, 2020, the Company delivered 57 DecisionDx-SCC test reports.
Gross margin in the third quarter of 2020 was 84%.
Operating cash flow was $(3.0) million in the third quarter of 2020, compared to $0.8 million in the third quarter of 2019.
Nine Months Ended Sept. 30, 2020, Selected Results

Revenues were $45.4 million, a 32% increase compared to $34.2 million during the same period in 2019. Included in revenue for the period were positive revenue adjustments related to tests delivered in prior periods. For the nine months ended Sept. 30, 2020 and 2019, these amounts totaled $0.2 million and $2.4 million, respectively.
Delivered 11,986 DecisionDx-Melanoma test reports, an increase of 8% over the same period in 2019.
Delivered 985 DecisionDx-UM test reports, a decrease of 10% over the same period in 2019.
Gross margin for the nine months ended Sept. 30, 2020, was 85%.
Operating cash flow was $10.3 million, compared to $2.5 million for the same period in 2019.
Adjusted operating cash flow, excluding the effects of certain relief payments described below, was $0.1 million, compared to $2.5 million for the same period in 2019.
Cash and Cash Equivalents

As of Sept. 30, 2020, the Company’s cash and cash equivalents totaled $183.1 million, and the outstanding principal balance on the Company’s bank term loan was $23.4 million.

Third Quarter and Recent Business and Clinical Evidence Highlights

Medicare Administrative Contractor (MAC), Palmetto GBA MolDx, issued a final expanded local coverage determination (LCD) and an accompanying billing and coding article for the company’s DecisionDx-Melanoma test. The effective date is Nov. 22, 2020. Additionally, Noridian, the MAC that oversees Castle’s laboratory in Arizona, issued an identical LCD to the Palmetto LCD, effective Dec. 6, 2020. The expanded LCD provides reimbursement for the significant majority of Medicare beneficiaries whose clinicians order DecisionDx-Melanoma as part of their melanoma management plan. Details on the LCD and the billing and coding article are posted to the Medicare Coverage Database on the Centers for Medicare & Medicaid Services (CMS) website.
The Company launched its DecisionDx-SCC test, and it was made commercially available on Aug. 31, 2020. DecisionDx-SCC is a 40-gene expression profile test that uses an individual patient’s tumor biology to predict individual risk of squamous cell carcinoma metastasis for patients with one or more risk factors. The test result, in which patients are stratified into a Class 1, 2A or 2B risk category, is designed to predict individual metastatic risk to inform risk-appropriate management. The Company has four peer-reviewed publications to date, demonstrating that DecisionDx-SCC is an independent predictor of metastatic risk and that integrating DecisionDx-SCC with current prognostic methods can add positive predictive value to clinician decisions regarding staging and management.
The Company launched its DecisionDx DiffDx-Melanoma test, and it was made commercially available on Nov. 2, 2020. DecisionDx DiffDx-Melanoma is designed to aid dermatopathologists in characterizing difficult-to-diagnose melanocytic lesions. In the third quarter of 2020, in preparation for the commercial launch, the Company hired and trained a dedicated DiffDx-Melanoma commercial team, which includes outside sales representatives, medical sales liaisons and internal sales staff.
Data from a systematic review and meta-analysis of the DecisionDx-Melanoma test was published, in print, in the September 2020 issue of the Journal of the American Academy of Dermatology (JAAD). Under multi-variate analysis, the DecisionDx-Melanoma test was shown to be independent from other clinical factors (age, Breslow tumor thickness, ulceration and node status) and improve upon risk assessment performed with staging factors alone. Under the Strength of Recommendation Taxonomy (SORT) system, a systematic review and meta-analysis provides for the highest level of evidence for a prognostic biomarker (Level 1 evidence). The SORT system is used by the American Academy of Dermatology and other organizations to evaluate the quality, quantity and consistency of evidence supporting tests, such as DecisionDx-Melanoma. For details, see the Company’s news release from April 1, 2020.
The publication of a retrospective study, titled "Integrating the melanoma 31-gene expression profile test to surgical oncology practice within national guideline and staging recommendations," showing that DecisionDx-Melanoma impacted management decisions for patients diagnosed with American Joint Committee on Cancer (AJCC) 7th edition stage I – III melanoma, appeared in Future Oncology. Study authors developed a recommended melanoma patient care algorithm that incorporates DecisionDx-Melanoma to help inform frequency and duration of follow-up visits, blood work and surveillance imaging in line with predicted metastatic risk. Patients’ DecisionDx-Melanoma test result was found to have an impact on the number and duration of follow-up and surveillance visits, and patients assessed as having a high risk of metastasis (designated by a DecisionDx-Melanoma Class 2 test result) received more intensive management than patients assessed as having a low risk (designated by a DecisionDx-Melanoma Class 1 test result). Clinicians using the test were shown to adjust patient management in a risk-appropriate direction, within recommendations of national guidelines.
Conference Call and Webcast Details

Castle Biosciences will hold a conference call on Monday, Nov. 9, 2020, at 4:30 p.m. Eastern time to discuss its third quarter 2020 results and provide a corporate update.

A live webcast of the conference call can be accessed here: View Source or via the webcast link on the Investor Relations page of the Company’s website (www.castlebiosciences.com). Please access the webcast at least 10 minutes before the conference call start time. An archive of the webcast will be available on the Company’s website until Nov. 30, 2020.

To access the live conference call via phone, please dial 877-282-2581 from the United States and Canada, or +1 470-495-9479 internationally, at least 10 minutes prior to the start of the call, using the conference ID 3586364.

There will be a brief Question & Answer session following management commentary.

Use of Non-GAAP Financial Measures (UNAUDITED)

In this release, we use the metric of Adjusted Operating Cash Flow, which is a non-GAAP financial measure and is not calculated in accordance with generally accepted accounting principles in the United States (GAAP). This non-GAAP financial measure reflects adjustments to net cash provided by operating activities to remove the effects of two payments we received associated with government aid to healthcare providers due to COVID-19, which we believe are not indicative of our ongoing operations.

We use Adjusted Operating Cash Flow internally because we believe this metric provides useful supplemental information in assessing our cash flow performance from our core ongoing business activities by removing the effects of these items on our operating cash flows. We believe this metric is also useful to investors as a supplement to GAAP measures in analyzing the performance of our business. However, this non-GAAP financial measure may be different from non-GAAP financial measures used by other companies, even when the same or similarly titled terms are used to identify such measures, limiting their usefulness for comparative purposes. This non-GAAP financial measure is not meant to be a substitute for net cash provided by operating activities reported in accordance with GAAP and should be considered in conjunction with our financial information presented on GAAP basis. Accordingly, investors should not place undue reliance on non-GAAP financial measures. Reconciliations of this non-GAAP financial measure to the most directly comparable GAAP financial measure are presented in the table at the end of this press release.

On November 9, 2020 Omeros Corporation (Nasdaq: OMER), a commercial-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting inflammation, complement-mediated diseases, disorders of the central nervous system and immune-related diseases, including cancers, reported recent highlights and developments as well as financial results for the third quarter ended September 30, 2020, which include (Press release, Omeros, NOV 9, 2020, View Source [SID1234570370]):

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Revenues for the third quarter of 2020 were $26.1 million following an $8.7 million deduction as a return reserve associated with the expiration of pass-through reimbursement for OMIDRIA on October 1, 2020. Omeros believes that it qualifies for and is pursuing separate payment from the Centers for Medicare and Medicaid Services (CMS) for OMIDRIA. For comparison, third quarter 2019 and second quarter 2020 revenues were $29.9 million and $13.5 million, respectively.
Net loss in the third quarter of 2020 was $38.5 million, or $0.66 per share, of which $13.6 million, or $0.23 per share, were non-cash expenses. This compares to a net loss of $16.5 million, or $0.33 per share, in the third quarter of 2019. On a non-GAAP basis, adjusted net loss for the third quarter of 2020 was $19.9 million, or $0.34 per share, after excluding non-cash expenses and a $5.0 million technology access fee. Net loss and adjusted net loss include the $8.7 million deduction in third quarter 2020 revenues for the return reserve.
At September 30, 2020, the company had cash, cash equivalents and short-term investments available for operations of $153.5 million. This includes $93.7 million in proceeds from a common stock offering and $76.9 million in proceeds from the issuance of convertible notes, following the use of a portion of the proceeds to repurchase a portion of our previously outstanding convertible notes and enter into certain derivative transactions, all of which took place during the third quarter.
Omeros will complete submission next week to the U.S. Food and Drug Administration (FDA) of its rolling Biologics License Application (BLA) for narsoplimab for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA or TA-TMA). Final clinical data from the BLA were presented in a webcast last month, as described below.
In August, Omeros reported results from a study evaluating narsoplimab for treatment of COVID-19-associated acute respiratory distress syndrome (ARDS). Six COVID-19 patients in Bergamo, Italy were treated with narsoplimab. All six patients required mechanical ventilation prior to narsoplimab treatment, and each recovered, survived and was discharged from the hospital following treatment. Two historical control groups that had similar baseline characteristics showed mortality rates of 32 percent and 53 percent. The results of the trial were published in the peer-reviewed journal Immunobiology. Five to six months after cessation of narsoplimab dosing, all patients were doing well and none showed clinical or laboratory evidence of longer-term effects from COVID-19.
In September, Omeros initiated its Phase 1 clinical trial for OMS906, the company’s MASP-3 inhibitor targeting the alternative pathway, and has completed dosing in the first patient cohort.
"The final clinical study results for narsoplimab in the treatment of transplant-associated TMA speak for themselves. The non-clinical and CMC sections of the BLA are under review by FDA, and the clinical sections, which will be submitted next week, are complete, comprehensive and compelling," said Gregory A. Demopulos, M.D., Omeros’ chairman and chief executive officer. "In anticipation of priority review, our team is readying for a successful commercial launch. Beyond TA-TMA and its Phase 3 trials in IgA nephropathy and aHUS, narsoplimab is increasingly recognized as a likely answer to severe COVID-19. Our complement franchise continues expanding with our MASP-3 inhibitor OMS906 on course and marching through its Phase 1 program. Confident that OMIDRIA qualifies for separate payment from CMS, we expect that the drug will increasingly support our unique portfolio of complement inhibitors and the rest of our exciting pipeline programs. Developing a life-saving drug is a rare opportunity, and all of us at Omeros are energized and inspired by the patients – children and adults – who are alive today because of our team’s efforts."

Third Quarter and Recent Developments

Recent developments regarding narsoplimab, Omeros’ lead fully human monoclonal antibody targeting MASP-2 in Phase 3 clinical programs for the treatment of HSCT-TMA, Immunoglobulin A (IgA) nephropathy, and atypical hemolytic uremic syndrome (aHUS), include the following:
In October, Professor Alessandro Rambaldi of the University of Milan and Papa Giovanni XXIII Hospital and Dr. Miguel Perales of Memorial Sloan Kettering Cancer Center presented the final efficacy and safety data from the pivotal trial of narsoplimab in the treatment of HSCT-TMA, which form the basis of the clinical sections of the rolling BLA.
The complete response rates of 61 percent in patients receiving at least one dose of narsoplimab (the full analysis set) and 74 percent in patients receiving the protocol-specified narsoplimab treatment of at least four weeks (the per-protocol population) are higher than what was previously reported.
Median overall survival was 274 days in the full analysis set, 361 days in the per-protocol population and could not be estimated for complete responders because more than half of the responders were still alive at last follow-up, out to as long as roughly four years following treatment.
Omeros applied to the Centers for Disease Control and Prevention (CDC) for an International Classification of Diseases (ICD-10) diagnosis code for HSCT-TMA, and CDC has preliminarily indicated its support for the diagnosis code. Omeros also applied to CMS for an ICD-10 procedure code for the administration of narsoplimab, and CMS has indicated its support for issuance of the procedure code.
In addition to the six COVID-19 patients who were treated with narsoplimab, Omeros has continued to treat critically ill COVID-19 patients under compassionate use. Omeros has also received requests and is in discussions to include narsoplimab in platform trials for COVID-19.
Omeros’ discussions regarding the use of narsoplimab in COVID-19 have progressed with leadership across BARDA, NIAID, NCAT and NIH regarding narsoplimab for the treatment of critically ill COVID-19 patients. With COVID-19 surging again globally and other therapeutics having failed to show benefit in critically ill COVID-19 patients, there is increasing focus on narsoplimab.
Recent developments regarding OMIDRIA include the following:
Pass-through reimbursement status for OMIDRIA expired on October 1, 2020. Omeros met with CMS and the Department of Health and Human Services to assert that OMIDRIA meets the objective criteria specified by CMS and must be paid separately in the ambulatory surgery center (ASC) setting. Omeros also submitted to CMS a comment letter on the proposed 2021 Outpatient Prospective Payment System/ASC Rule along with a legal memorandum from an outside law firm reiterating this position and seeking confirmation of separate payment status for OMIDRIA in the ASC setting for the fourth quarter of 2020 and calendar year 2021.
An article entitled "Real-world opioid prescribing after cataract surgery among patients who received intracameral phenylephrine and ketorolac 1.0%/0.3%" was published in the peer-reviewed journal Current Medical Research and Opinion. The study demonstrates that patients who received OMIDRIA during cataract surgery were prescribed fewer opioid pills following surgery than patients who did not receive OMIDRIA, despite the OMIDRIA-treated group having a greater incidence of preoperative comorbidities and higher risk for surgical complexity.
Updates regarding Omeros’ other development programs and platforms include the following:
Omeros has completed, on schedule, dosing in the first cohort in a Phase 1, placebo-controlled, double-blind, single-ascending-dose and multiple-ascending-dose study for OMS906, the company’s MASP-3 inhibitor. The second cohort in the Phase 1 study has begun dosing. Omeros expects to achieve a once-monthly subcutaneous dosing regimen. Data readout from the Phase 1 study is planned for next year.
Omeros presented data on the OMS906 program at the 4th Complement-based Drug Development Summit in October.
Financial Results

For the third quarter of 2020, OMIDRIA revenues were $26.1 million, down from $29.9 million for the same period in 2019 and up from $13.5 million for the second quarter of 2020. The decrease compared to the third quarter of 2019 was due to an $8.7 million deduction as a reserve for product returns related to the expiration of pass-through reimbursement on October 1, 2020.

Total costs and expenses for the third quarter of 2020 were $51.5 million compared to $41.0 million for the same period in 2019. The increase reflects a fee payable under a technology license agreement related to Omeros’ MASP-3 program and increased pre-commercialization marketing activities for narsoplimab. Selling, general and administrative expenses were $19.8 million in the third quarter of 2020, compared to $16.9 million in the corresponding period in 2019.

For the three months ended September 30, 2020, Omeros reported a net loss of $38.5 million, or $0.66 per share, compared to a net loss of $16.5 million, or $0.33 per share, for the same period in 2019. On a non-GAAP basis, adjusted net loss for the three months ended September 30, 2020 was $19.9 million, or $0.34 per share, after excluding non-cash expenses of $13.6 million, or $0.23 per share, and a technology access fee of $5.0 million, or $0.09 per share. Both net loss and adjusted net loss include the $8.7 million deduction in the third quarter of 2020 for the return reserve.

As of September 30, 2020, Omeros had $153.5 million of cash, cash equivalents and short-term investments available for operations and accounts receivable of $37.4 million.

During the third quarter, Omeros issued approximately $225.0 million aggregate principal amount 5.25% convertible senior notes due February 2026 (the 2026 Notes). Concurrently, Omeros repurchased $115.0 million aggregate principal amount of previously outstanding 6.25% convertible senior notes due November 2023. Omeros recorded a $13.4 million non-cash loss on early extinguishment of debt and a $7.9 million non-cash income tax benefit associated with these transactions. Omeros also entered into capped call contracts associated with the 2026 Notes that cover, subject to anti-dilution adjustments that may not match those applicable to the conversion price of the 2026 Notes, the number of shares of Omeros’ common stock underlying the 2026 Notes when Omeros’ common stock is trading between the initial conversion price of approximately $18.49 and the $26.10 cap price.

In August, Omeros sold 6.9 million shares in an underwritten public offering and received $93.7 million in net proceeds from the offering.

On November 9, 2020 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a pioneer in T-cell immunotherapy, leveraging its novel allogeneic EBV T-cell platform to develop transformative therapies for patients with serious diseases including solid tumors, hematologic cancers and autoimmune disease, reported financial results for the third quarter ended September 30, 2020 and recent business highlights (Press release, Atara Biotherapeutics, NOV 9, 2020, View Source [SID1234570369]).

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"Atara is on track to finish the year strong, meeting each milestone set to date," said Pascal Touchon, President and Chief Executive Officer of Atara. "Following an encouraging interim analysis of the pivotal Phase 3 data for tab-cel in relapsed-refractory EBV+ PTLD and very productive interactions with regulatory authorities, we are preparing for tab-cel submissions, and then approval and launch. We are committed to bringing this potentially life-saving therapy to the patients who acutely need it as they have no other therapeutic solution and face a very limited median life expectancy of only two to three months."

Tab-cel for Post-transplant Lymphoproliferative Disease (PTLD)

An Interim Analysis of the tab-cel ALLELE study showed a 50 percent objective response rate (ORR) to tab-cel with independent oncologic and radiographic assessment (IORA) in patients with relapsed-refractory EBV+ PTLD following hematopoietic cell transplants (HCT) or solid organ transplants (SOT), that had reached at least six months follow-up after achieving a response. This ORR is consistent with previously published investigator assessed data. The tab-cel safety profile is also consistent with previously published data, with no new safety signals.
In October, working under the Breakthrough Therapy Designation (BTD) for tab-cel, Atara presented a comprehensive data package to the FDA. The Company aligned with FDA on several key topics related to the regulatory package, including:
a rolling submission is acceptable for the biologics license application (BLA);
the Company can complete the BLA submission with the ALLELE study’s currently enrolled patients with at least six months follow-up for duration of response; and
the FDA will consider the following as supportive data to the pivotal study in the BLA clinical module: the Phase 2 trials conducted at Memorial Sloan Kettering (MSK); Atara’s Phase 2 multicenter expanded access protocol (201 EAP study); and, the Single Patient Use (SPU) program.
Atara remains on track to initiate a BLA submission for patients with EBV+ PTLD by the end of 2020. The Company will continue engaging with the FDA as part of its rolling BLA and BTD status and expects to finalize the BLA submission in Q3 2021.
Following discussion with the PRIority MEdicines (PRIME) team and after EMA approval of the Pediatric Investigation Plan (PIP) expected in December 2020, Atara is on track to submit an EU marketing authorization application (MAA) for patients with EBV+ PTLD in the second half of 2021.
Data from the ALLELE study will be presented at an appropriate congress in 2021.
Tab-cel for Potential Additional Indications

Atara initiated a tab-cel Phase 2 multi-cohort study in the third quarter of 2020 and expects to enroll the first patient in the fourth quarter of 2020. This study is being initiated concurrently in the U.S. and the EU.
The multi-cohort study is intended to enrich the evidence base with the goal of expanding the potential label for tab-cel in both treatment-naïve and previously treated patients (in six populations, including four within Immunodeficiency-Associated Lymphoproliferative Diseases (IA-LPDs) and two in other EBV-associated diseases).
Data demonstrating tab-cel was well-tolerated and showed encouraging clinical activity in patients with EBV+ AID-LPD and PID-LPD (Acquired and Primary Immunodeficiency LPDs) were featured in an e-poster at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2020 Virtual Congress held in September 2020. Specifically, in patients where previous treatments have failed, the objective response rate, including complete response, were 33.3 percent (three out of nine patients) in AID-LPD and 37.5 percent (three out of eight patients) in PID-LPD groups. Tab-cel was generally well-tolerated with a favorable safety profile consistent with previously published clinical studies.
Data on tab-cel in patients with life-threatening complications stemming from persistent EBV viremia was accepted for presentation at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition being held virtually December 5-8, 2020. These clinical data demonstrate that tab-cel was well-tolerated and showed encouraging clinical activity in this patient population with objective response rate ranging from 50 to 80 percent, warranting further investigation in the Phase 2 multi-cohort study.
ATA188 for Progressive Forms of Multiple Sclerosis (MS)

Atara’s Phase 1 clinical study of off-the-shelf, allogeneic ATA188 in patients with progressive forms of MS is ongoing, with encouraging data recently reported and more data from the Open Label Extension (OLE) to be periodically presented over the next 12 months including at the European Charcot Foundation (ECF) 28th Annual Meeting November 15-19, 2020.
Twelve-month data from all four cohorts in the Phase 1a portion of the study were presented at the MSVirtual2020: 8th Joint ACTRIMS-ECTRIMS Meeting held in September 2020.
These data demonstrated that ATA188 was well-tolerated across all four dose cohorts; patients who demonstrated sustained disability improvements (SDI) at any timepoint maintained it at all future timepoints; and, a higher proportion of patients showed SDI with increasing dose (42 percent in Cohorts 3 and 4 (higher doses) vs 17 percent in Cohorts 1 and 2 (lower doses)).
No dose-limiting toxicities and no fatal adverse events (AEs) have been reported. The safety profile has remained consistent with previously reported data.
Data from the OLE with redosing at 12 months show that of the three patients enrolled in the OLE at that time that had SDI at 12 months, all maintained SDI at 15 months, including one patient evaluated at both 15 and 18 months who maintained SDI at both time points. A fourth patient demonstrated SDI during the OLE at 24 months.
Atara also presented preclinical translational data at ACTRIMS-ECTRIMS that further support the proposed mechanism of action of ATA188 targeting EBV-infected B cells. These combined analyses of T cells comprising ATA188 are consistent with its proposed mechanism of targeting EBV-infected B cells by recognizing MS-relevant EBV antigens on these cells via defined T cell receptors (TCRs).
The double-blind randomized placebo-controlled trial (RCT), which enrolled its first patient in June 2020 continues active recruitment.
Given encouraging clinical results to date in ATA188 studies and the significant unmet medical need in progressive forms of MS, the Company is increasing its investment in the ATA188 program. Atara is expanding the size of the RCT to at least 64 patients, changing the primary endpoint of the study to disability improvement, and maintaining biological and functional endpoints; the design allows for the addition of the cohort 4 dose if desired.
Atara plans to discuss the Phase 1a data with the FDA by the end of this year, as well as the updated RCT study design, and potential opportunities for accelerated development of ATA188 for MS patients.
CAR T Programs

ATA2271/ATA3271 (Solid Tumors Over-Expressing Mesothelin)

The open-label, single-arm Phase 1 clinical study of ATA2271, the Company’s second-generation autologous CAR T therapy targeting mesothelin (MSLN) that incorporates for the first time both a PD-1 DNR (dominant-negative programmed death-1 receptor) for intrinsic check-point inhibition and novel 1XX co-stimulatory domain, has been initiated for the treatment of advanced mesothelioma. Recently presented preclinical data have shown improved functional persistence and enhanced anti-tumor efficacy superior to first-generation MSLN CAR T. MSK is on track to enroll the first patient in the Phase 1 study in Q4 2020.
ATA3271 is an off-the-shelf, allogeneic CAR T therapy targeting MSLN using a PD-1 DNR and 1XX CAR co-stimulatory signaling domain through Atara’s EBV T-cell platform. In new preclinical data, ATA3271 demonstrates potent anti-tumor activity, functional persistence and significant survival benefit with no evidence of allocytotoxicity in vivo, suggesting that allogeneic MSLN-CAR-engineered EBV T cells are a promising approach for the treatment of MSLN-positive cancers. These data will be presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 35th Anniversary Annual Meeting November 11-14, 2020.
ATA3219 (B-cell Malignancies)

A collaborative and successful pre-IND meeting with the FDA in October 2020 provided feedback to guide the IND filing in 2021.
IND-enabling studies are progressing with a package ready to be filed in 2021 for ATA3219, a potent next-generation off-the-shelf allogeneic CD19 CAR T utilizing the 1XX technology without the need for TCR gene editing through Atara’s EBV T-cell platform.
The first abstract to be presented on ATA3219 was accepted for presentation at the 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition being held virtually December 5-8, 2020. These preclinical data for ATA3219 demonstrate persistence, polyfunctional phenotype and efficient targeting of CD19-expressing tumor cells with no evidence of allocytotoxicity in vivo.
Third Quarter 2020 Financial Results

Cash, cash equivalents and short-term investments as of September 30, 2020 totaled $327.2 million, as compared to $347.7 million as of June 30, 2020.
September 30, 2020 cash balance of $327.2 million included $34.1 million net proceeds from at-the-market (ATM) facilities during the third quarter of 2020. Third quarter 2020 cash burn was $20.5 million, net of ATM proceeds.
Atara believes that its cash, cash equivalents and short-term investments as of September 30, 2020 are sufficient to fund planned operations into 2022.
Net cash used in operating activities was $53.0 million for the third quarter of 2020, as compared to $52.1 million for the same period in 2019.
The number of outstanding shares of common stock and pre-funded common stock warrants as of September 30, 2020 was 77,220,159 shares and warrants to purchase 5,755,487 shares, respectively.
Atara reported net losses of $74.3 million, or $0.92 per share, for the third quarter of 2020, as compared to $71.9 million, or $1.31 per share, for the same period in 2019.
Total operating expenses include non-cash expenses of $15.4 million for the third quarter 2020, as compared to $13.9 million for the same period in 2019.
Research and development expenses were $59.9 million for the third quarter of 2020, as compared to $53.5 million for the same period in 2019. The increase in the 2020 period was primarily due to costs associated with the Company’s continuing expansion of research and development activities, including:
Clinical trial, manufacturing and process performance qualification activities related to tab-cel.
Higher employee-related costs from increased headcount.
Research and development expenses include $8.2 million of non-cash stock-based compensation expenses for the third quarter of 2020, as compared to $7.0 million for the same period in 2019.
General and administrative expenses were $14.8 million for the third quarter of 2020, as compared to $19.0 million for the same period in 2019. The decrease in the 2020 period was primarily due to decrease in outside services costs and employee-related costs.
General and administrative expenses include $5.1 million of non-cash stock-based compensation expenses for the third quarter of 2020, as compared to $5.1 million for the same period in 2019.

On November 9, 2020 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported its third quarter 2020 financial results and provided an update on the Company, including its third quarter progress with eganelisib (IPI-549), the Company’s first-in-class, oral immuno-oncology product candidate targeting immune-suppressive tumor-associated myeloid cells through selective phosphoinositide-3-kinase-gamma (PI3K-gamma) inhibition (Press release, Infinity Pharmaceuticals, NOV 9, 2020, View Source [SID1234570368]).

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"We are approaching an important inflection point at Infinity, with expected data readouts across our clinical programs in the next few months that demonstrate the benefit of eganelisib across multiple indications, patient populations and treatment settings." said Adelene Perkins, Chief Executive Officer and Chair of Infinity Pharmaceuticals. "We are pleased that the IDMC supports the further exploration of eganelisib in second-line metastatic urothelial cancer. We will continue to follow the forty-nine patients previously enrolled through the remainder of the year and use these data to determine the best path forward, which may include the re-opening of enrollment of MARIO-275 or the initiation of a new study that leverages our clinical and translational insights from the patients enrolled to date."

Ms. Perkins continued, "In addition, encouraging data from the MARIO-3 TNBC cohort suggest that eganelisib has the potential to be an important component of a new treatment regimen in the front-line setting, and we look forward to presenting these data at SABCS next month. This week we also shared data from the melanoma and SCCHN cohorts of MARIO-1 at SITC (Free SITC Whitepaper)."

Key Q3 2020 Updates:

Clinical and Regulatory:

MARIO-275 is the Company’s ongoing controlled, randomized Phase 2 study evaluating eganelisib in combination with Opdivo in platinum-refractory, I/O naïve patients with advanced urothelial cancer (aUC), in collaboration with Bristol Myers Squibb.
The MARIO-275 Independent Data Monitoring Committee (IDMC) determined that the risk/benefit for patients warrants resumption of enrollment after the successful implementation of a dose reduction from 40mg QD to 30mg QD to reduce the reversible liver enzyme elevations, which were reported after the first scheduled MARIO-275 IDMC meeting.
Infinity is continuing to evaluate the forty-nine patients previously enrolled across safety and time-to-event measures including progression free survival and overall survival and will determine next steps by year end. This may include the re-opening of enrollment of MARIO-275 or the initiation of a new study which leverages our findings from the patients enrolled to date.
MARIO-3 is the Company’s ongoing Phase 2 study in collaboration with Roche/Genentech to evaluate eganelisib in a novel triple combination in the front-line setting with Tecentriq and Abraxane in triple negative breast cancer (TNBC) and with Tecentriq and Avastin in renal cell cancer (RCC).
Encouraging data from the TNBC cohort of MARIO-3 will be presented at the San Antonio Breast Cancer Symposium (SABCS) Annual Meeting, December 8-11, 2020.
Fast Track Designation: Infinity received Fast Track Designation for eganelisib in combination with a checkpoint inhibitor and chemotherapy for first-line treatment of advanced TNBC.
Enrollment has been completed in the RCC cohort. In TNBC, we have implemented a number of enrollment initiatives and expect to provide an update on enrollment expectations at SABCS.
MARIO-1-is the Company’s ongoing Phase 1/1b study evaluating eganelisib as a monotherapy and in combination with Opdivo in patients with solid tumors in collaboration with Bristol Myers Squibb.
Presented data from the MARIO-1 melanoma and squamous cell carcinoma of the head and neck (SCCHN) cohorts, which were designed to isolate the clinical benefit of eganelisib by examining clinical activity in patients not expected to respond to checkpoint inhibitor monotherapy due progression on an immediate prior checkpoint inhibitor, at SITC (Free SITC Whitepaper). Both presentations demonstrate that eganelisib had a manageable safety and tolerability profile, provide further validation of the eganelisib mechanism of action of immune modulation, and support the Company’s strategy of moving eganelisib into earlier treatment settings.
Arcus Collaboration: A Phase 1b collaboration study being conducted by Arcus Biosciences is evaluating a checkpoint-inhibitor free, novel triple-combination regimen of eganelisib + etrumadenant (AB928, dual adenosine receptor antagonist) + Doxil in up to approximately 40 advanced TNBC patients.
Data from the study will be presented at SABCS in December 2020.
Third Quarter 2020 Financial Results:

At September 30, 2020, Infinity had total cash, cash equivalents and available-for-sale securities of $41.3 million, compared to $42.7 million at June 30, 2020.
Research and development expense for the third quarter of 2020 was $6.1 million, compared to $7.1 million for the same period in 2019. The decrease is primarily related to a combination drug purchase during the third quarter of 2019.
General and administrative expense was $2.9 million for the third quarter of 2020, compared to $3.6 million for the same period in 2019. The decrease is primarily related to a reduction in professional services and consulting.
Net loss for the third quarter of 2020 was $9.5 million, or a basic and diluted loss per common share of $0.16, compared to a net loss of $11.4 million, or a basic and diluted loss per common share of $0.20 for the same period in 2019.
2020 Financial Outlook:

Net Loss: Infinity expects net loss for 2020 to range from $35 million to $45 million.

Cash and Investments: Infinity expects to end 2020 with a year-end cash, cash equivalents and available-for-sale securities balance ranging from $25 million to $35 million.

Cash Runway: Based on its current operational plans, Infinity expects that its existing cash, cash equivalents and available-for-sale securities, will be adequate to satisfy the Company’s capital needs through 2021. Infinity’s financial guidance does not include potential additional funding or business development activities, a potential $5 million milestone payment from BVF based on PellePharm’s ongoing Phase 3 clinical trial of patidegib topical gel in Gorlin Syndrome, or any milestones from, or the sale of the Company’s equity interest in, PellePharm.

Conference Call Information

Infinity will host a conference call today, November 9, 2020, at 4:30 p.m. ET to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the "Investors/Media" section of Infinity’s website at www.infi.com. To participate in the conference call, please dial (877) 316-5293 (domestic) and (631) 291-4526 (international) five minutes prior to start time. The conference ID number is 1575996. An archived version of the webcast will be available on Infinity’s website for 30 days.

On November 9, 2020 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported financial results for the third quarter ended September 30, 2020 and provided a corporate update (Press release, Cellectar Biosciences, NOV 9, 2020, View Source [SID1234570367]).

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Third Quarter and Recent Corporate Highlights

·Held FDA Type B guidance meeting to define the registrational pathway for our priority adult hematology oncology indications and planned initiation of the pivotal study for our lead indication in the fourth quarter

·Announced CLR 131 achieved a 40% overall response rate (ORR) in Triple Class Refractory Multiple Myeloma (MM) patients with an administered total body dose (TBD) of 60mCi or greater from the Phase 2 CLOVER-1 study

·Interim results from the Phase 2 COVER-1 study at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Virtual Meeting: Advances in Malignant Lymphoma demonstrated a 100% ORR and a 75% major response rate (MRR) in LPL/WM. Mean duration of response exceeding 17 months (8.4 – 31.7 months); duration of response continues to increase for all patients

·Strengthened the management team with the appointment of Dr. John Friend, chief medical officer

"We continue to make good progress towards the fourth quarter initiation of the CLR 131 pivotal study in our lead heme-oncology indication. Our recent FDA guidance meeting was most encouraging and we look forward to providing greater details in the near-term," said James Caruso, president and CEO of Cellectar. "Additional data from our Phase 2 CLOVER-1 study remain strong, with patients in WM achieving a 100% ORR and a 75% MRR in patients failing a BTKi and a 40% ORR in the challenging to treat triple class refractory MM patient population."

Third Quarter 2020 Financial Highlights

·Cash and Cash Equivalents: As of September 30, 2020, the company had cash and cash equivalents of $18.8 million compared to $10.6 million at December 31, 2019. Cash used in operating activities was approximately $10.1 million during the nine months ended September 30, 2020 as compared to $9.0 million during the nine months ended September 30, 2019.

·Research and Development Expense: R&D expense for the three months ended September 30, 2020 was $2.7 million, compared to $2.7 million for the three months ended September 30, 2019. The cumulative R&D spending for the first nine months of 2020 was $7.8 million as compared to $6.8 million for the first nine months of 2019. The increase in R&D expense year-to-date in 2020 was primarily a result of higher general research and development costs resulting from increased personnel related costs and clinical study costs. Manufacturing and related costs decreased because of a reduction in materials production processes and related costs.

·General and Administrative Expense: G&A expense for the three months ended September 30, 2020 was $1.2 million compared to $1.3 million for the three months ended September 30, 2019. The cumulative G&A spending for the first nine months of 2020 were of $3.7 million as compared to $4.0 million for the first nine months of 2019. The decrease in G&A expense year-to-date in 2020 was primarily a result of lower stock-based compensation expense.

·Net Loss: The net loss attributable to common stockholders for the three months ended September 30, 2020 was ($3.9) million, or ($0.15) per share, compared to ($3.9) million, or ($0.42) per share, in 2019. Net loss attributable to common stockholders for the nine months ended September 30, 2020 was ($11.5) million, or ($0.69) per share, compared to ($10.7) million, or ($1.51) per share, in 2019.