On November 4, 2021 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported third quarter 2021 financial results and provided a corporate update (Press release, Kura Oncology, NOV 4, 2021, View Source [SID1234594418]).

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"I am pleased by the progress our team has made over the past quarter, highlighted by accelerated enrollment in the Phase 1b expansion cohorts for KO-539," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "We continue to be encouraged by preliminary results from the Phase 1b study, with evidence of activity at both doses and a favorable safety and tolerability profile. Pending determination of a recommended Phase 2 dose, we are designing a development strategy that builds on the potential to register KO-539 as a monotherapy, while giving us the flexibility to get to larger opportunities in combination more quickly, including in earlier lines of therapy. We look forward to providing a more comprehensive update, including results from the Phase 1a and Phase 1b studies, at a future medical meeting."

Recent Highlights

Continued enrollment in Phase 1b expansion cohorts for KO-539 – Kura is currently enrolling two expansion cohorts in the Phase 1b portion of its KOMET-001 trial – a lower dose of 200 mg and a higher dose of 600 mg. Each cohort is comprised of patients with NPM1-mutant or KMT2A-rearranged relapsed or refractory acute myeloid leukemia (AML). Approximately half of an estimated 40 global sites are now actively screening patients for enrollment in the study. The Company maintains its enrollment guidance of 12 evaluable patients in each cohort by the first quarter of 2022. Once enrolled, patients in each cohort will be assessed for safety and tolerability, pharmacokinetics and efficacy to determine the recommended Phase 2 dose for KO-539.

Comprehensive clinical development strategy for KO-539 – Pending determination of a recommended Phase 2 dose, Kura intends to conduct a comprehensive clinical development plan for KO-539, both as a monotherapy and in combination. This development strategy builds on the potential to register KO-539 as a monotherapy while giving the Company the flexibility to address larger opportunities in combination more efficiently, including earlier lines of therapy.

ASH presentation to highlight potential for synergistic activity of KO-539 with venetoclax – Encouraging pre-clinical data has been generated through a research collaboration with Dr. Kapil Bhalla at the MD Anderson Cancer Center highlighting the molecular mechanisms of anti-leukemic activity for KO-539 and its potential for synergistic activity in combination with venetoclax in KMT2A-rearranged and NPM1-mutant AML models. These data have been accepted for presentation at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2021. The abstract was published earlier today and is now available on the ASH (Free ASH Whitepaper) website.

Final results from Phase 2 study of tipifarnib in T-cell lymphoma at ASH (Free ASH Whitepaper) – Final results from a Phase 2 study of tipifarnib in patients with relapsed or refractory peripheral T-cell lymphoma (PTCL) have been accepted for oral presentation by Dr. Thomas Witzig, Hematologist at Mayo Clinic and the study’s lead investigator, at the upcoming ASH (Free ASH Whitepaper) Annual Meeting. The abstract is now available on the ASH (Free ASH Whitepaper) website. The clinical benefit demonstrated in patients with certain subtypes of T-cell lymphoma, including an overall response rate of 56.3% (18/32) and a median overall survival of 32.8 months in patients with angioimmunoblastic T-cell lymphoma (AITL), underscores the potential to target farnesyl transferase to drive clinical benefit in patients with cancer.

First clinical site activated in Phase 1/2 trial of tipifarnib plus alpelisib in HNSCC – In July, Kura announced a clinical collaboration with Novartis to evaluate the combination of tipifarnib and the PI3Kα inhibitor alpelisib in patients with head and neck squamous cell carcinoma (HNSCC). The Company believes this combination has the potential to increase the total addressable population for tipifarnib to as much as 50% of patients with HNSCC and is now beginning a Phase 1/2 clinical trial (KURRENT) of tipifarnib in combination with alpelisib in patients who have HRAS- and/or PIK3CA-dependent HNSCC. Kura has now activated the first clinical site and expects to dose the first patient in the trial by the end of 2021.

Next-generation FTI program focused on delaying onset of drug resistance in large solid tumor indications – Last quarter, Kura nominated KO-2806 as the lead development candidate in its next-generation farnesyl transferase inhibitor (FTI) program. The Company’s next-generation FTI program is designed to target innovative biology and address large solid tumor indications of high unmet need through rational combinations, with a focus on delaying the onset of drug resistance. IND-enabling studies of KO-2806 are ongoing.
Financial Results and Guidance

Research and development expenses for the third quarter of 2021 were $22.4 million, compared to $16.6 million for the third quarter of 2020.

General and administrative expenses for the third quarter of 2021 were $11.3 million, compared to $7.6 million for the third quarter of 2020.

Net loss for the third quarter of 2021 was $33.4 million, compared to a net loss of $23.8 million for the third quarter of 2020. This included non-cash share-based compensation expense for the third quarter of 2021 of $6.1 million, compared to $3.4 million for the same period in 2020.

Cash, cash equivalents and short-term investments totaled $543.4 million as of September 30, 2021, compared with $633.3 million as of December 31, 2020.

Operating expenses for the full year 2021 are expected to be in the range of $130 million to $140 million.

Net cash used in operating activities for the full year 2021 is expected to be $105 million to $115 million.

Management expects that current cash, cash equivalents and short-term investments will be sufficient to fund current operations into 2024.
Upcoming Milestones

Dose first patient in the KURRENT Phase 1/2 study of tipifarnib in combination with alpelisib by the end of 2021.

Complete enrollment of 24 evaluable patients in the KOMET-001 Phase 1b expansion cohorts by the first quarter of 2022.

Determine the recommended Phase 2 dose of KO-539 by the first quarter of 2022.

Submit an IND application for KO-2806 by the end of 2022.
Conference Call and Webcast

Kura’s management will host a webcast and conference call at 4:30 p.m. ET / 1:30 p.m. PT today, November 4, 2021, to discuss the financial results for the third quarter 2021 and provide a corporate update. The live call may be accessed by dialing (866) 269-4260 for domestic callers and (323) 347-3277 for international callers and entering the conference code: 9395801. A live webcast and archive of the call will be available online from the investor relations section of the company website at www.kuraoncology.com.

On November 4, 2021 Quanterix Corporation (NASDAQ: QTRX), a company digitizing biomarker analysis with the goal of advancing the science of precision health, reported financial results for the three months ending September 30, 2021 (Press release, Quanterix, NOV 4, 2021, View Source [SID1234594435]).

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"We are making critical advances across neurology and infectious disease with recent breakthrough device designation for an Alzheimer’s blood test and expanded EUA label for a COVID-19 test by the U.S. FDA," said Kevin Hrusovsky, Chairman and Chief Executive Officer, Quanterix. "Our Simoa phospho-Tau 181 (pTau-181) blood test is a critical potential aid in the diagnostic evaluation of Alzheimer’s disease, in particular by unlocking the possibility for earlier, more accessible, higher-throughput, non-invasive diagnosis to enable advances in neuro-diagnostic therapies. Furthermore, our label expansion for our Simoa SARS-CoV-2 N Protein Antigen Test demonstrates yet another example of Simoa’s potential for achieving asymptomatic and low-invasive clinical testing."

Third Quarter 2021 Financial Highlights

Key financial results for the third quarter of 2021 are shown below:

Q3 GAAP total revenue, which includes grant revenue of $1.0M, was $27.7M versus prior year Q3 of $31.4M; prior year Q3 included one-time license revenue of $11.2M and grant revenue of $1.9M;
Q3 non-GAAP total revenue was $26.7M versus prior year Q3 non-GAAP total revenue of $18.3M, an increase of 46%;
Q3 GAAP product revenue was $20.7M versus prior year Q3 of $11.7M, an increase of 77%;
Q3 GAAP service and other revenue was $5.9M versus prior year Q3 of $6.6M, a decrease of 10% over a strong 2020 driven by one time COVID-related services; our two year CAGR (Q319 – Q321) for services is 18%; and
Q3 GAAP gross margin was 55.1% versus prior year Q3 GAAP gross margin of 67.2%; Q3 non-GAAP gross margin was 54.8% versus prior year Q3 non-GAAP gross margin of 51.5%.
YTD 2021 Financial Highlights

Key financial results for YTD 2021 are shown below:

YTD GAAP total revenue, which includes grant revenue of $4.2M, was $80.3M versus prior year YTD of $60.2M; prior year YTD included one-time license revenue of $11.2M and grant revenue of $1.9M;
YTD non-GAAP total revenue was $76.0M versus prior year YTD non-GAAP total revenue of $47.1M, an increase of 61%;
YTD GAAP product revenue was $57.6M versus prior year YTD of $28.3M, an increase of 104%;
YTD GAAP service and other revenue was $18.0M, versus prior year YTD of $18.6M, a decrease of 4% over a strong 2020 driven by one time COVID-related services; our two year CAGR (YTD19 – YTD21) for services is 24%; and
YTD GAAP gross margin was 56.6% versus prior year YTD GAAP gross margin of 55.0%; YTD non-GAAP gross margin was 56.1% versus prior year YTD non-GAAP gross margin of 48.4%.
For additional information on the non-GAAP financial measures included in this press release, please see "Use of Non-GAAP Financial Measures" below.

Third Quarter 2021 Business Highlights

Quanterix’ Simoa phospho-Tau 181 (pTau-181) blood test was granted Breakthrough Device designation by the U.S. Food and Drug Administration (FDA) as an aid in diagnostic evaluation of Alzheimer’s disease;
The FDA expanded the Emergency Use Authorization (EUA) label for Quanterix’ Simoa SARS-CoV-2 N Protein Antigen Test to include testing with nasal swab and saliva samples, and for asymptomatic serial testing with nasal swab samples. The expanded label established this test as the first antigen test authorized for use with saliva samples;
Quanterix’ Simoa HD-X technology and assays were used to measure pTau-217 using antibodies developed by Eli Lilly and Company for its Phase 2 TRAILBLAZER-ALZ study, which was presented by Lilly at the Alzheimer’s Association International Conference (AAIC) 2021;
Hosted the webinar: COVID-19 Testing in a Residential University Setting with leading pathologist Dr. John Roback, M.D., Ph.D., Professor of Pathology and Laboratory Medicine, Executive Vice-Chair for Clinical Operations, and Medical Director for Emory Medical Laboratories, that discussed implementation of a broad SARS-CoV-2 screening program for Emory students, faculty and staff, enabling a safe return to campus-based learning. To date, Emory has run over 120,000 tests using the Simoa SARS-CoV-2 N Protein Antigen Test;
Co-hosted the webinar, Cytokine Profiles and Personalized Therapeutics in COVID-19 Patients with Guy Gorochov, M.D., Ph.D., Professor of Medicine, Head of the Dept. of Immunology and Co-Director CIMI Research Centre, Sorbonne University/INSERM and Laurel Provencher, Ph.D., Sr. Director of Strategic Collaborations, Quanterix, who discussed recently published observations that describe distinct cytokine profiles and COVID-19 severity and mortality;
Kevin Hrusovsky delivered a presentation at the Second Annual Biomarkers for Alzheimer’s Disease Conference on Aug. 26 with Nicholas Ashton, Ph.D., Assistant Professor, Department of Psychiatry & Neurochemistry, University of Gothenburg. The presentation discussed key features of assay development of plasma pTau-181 and pTau-231 on the Simoa platform, provided an overview of plasma pTau across the AD continuum and featured head-to-head comparisons of pTau plasma biomarkers;
Delivered a presentation at the Precision Medicine Leaders’ Summit on transforming cancer detection with biomarker technology; and
Quanterix Simoa technology was highlighted in 125 new publications, bringing total Simoa-specific inclusions to more than 1,480 publications.
Conference Call

In conjunction with this announcement, Quanterix Corporation will host a conference call on November 4, 2021 at 4:30 p.m. EDT. Individuals interested in listening to the conference call may do so by dialing (833) 686-9351 for domestic callers, or (612) 979-9890 for international callers. Please reference the following conference ID: 3488875.

A live webcast will also be available at: View Source The webcast will be available on the Company’s website, View Source, for one year following completion of the call.

Use of Non-GAAP Financial Measures

To supplement the Company’s financial statements presented on a GAAP basis, the Company has provided certain non-GAAP financial measures, including non-GAAP revenue and non-GAAP gross margin. Management uses these non-GAAP measures to evaluate the Company’s operating performance in a manner that allows for meaningful period-to-period comparison and analysis of trends in its business. Management believes that such measures are important in comparing current results with prior period results and are useful to investors and financial analysts in assessing the Company’s operating performance. The non-GAAP financial information presented here should be considered in conjunction with, and not as a substitute for, the financial information presented in accordance with GAAP. Investors are encouraged to review the reconciliation of these non-GAAP measures to their most directly comparable GAAP financial measures set forth below.

On November 4, 2021 Novartis reported that it has agreed to sell 53.3 million (approximately 33%) Roche bearer shares in a bilateral transaction to Roche for a total consideration of USD 20.7 billion (Press release, Novartis, NOV 4, 2021, View Source [SID1234594451]).

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Vas Narasimhan, CEO of Novartis, said: "After more than 20 years as a shareholder of Roche, we concluded that now is the right time to monetize our investment. Today’s announcement is consistent with our strategic focus and we intend to deploy the proceeds from the transaction in line with our capital allocation priorities to maximize shareholder value and continue to reimagine medicine."

Novartis has been a shareholder of Roche since May, 2001 and currently holds 53.3 million bearer shares of Roche’s common stock, representing approximately 33% of aggregate outstanding bearer shares. Novartis acquired the stake between 2001 and 2003 for a total consideration of approximately USD 5 billion as a long-term financial investment which delivered significant, recurring earnings contribution and cumulative dividends in excess of USD 6 billion. Over the holding period of the stake, this resulted in an annualized return of 10.2 % in USD (and 6.6% in CHF). Today, Novartis does not consider the financial investment in Roche as part of its core business and therefore not a strategic asset.

Novartis will report a gain from the sale of the stake in income from associated companies of approximately USD 14 billion, which will be core adjusted.

The transaction is subject to the approval by the shareholders of Roche, at the Extraordinary General Meeting ("EGM") of Shareholders of Roche to be held on November 26, 2021.

On November 4, 2021 XOMA Corporation (Nasdaq: XOMA) reported its third quarter 2021 financial results and provided a recent operations update (Press release, Xoma, NOV 4, 2021, View Source [SID1234594486]).

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"Our portfolio of potential milestone and royalty assets continues to grow and advance in the clinic. In the third quarter, we acquired an economic interest in Checkmate Pharmaceuticals’ vidotulimod (CMP-001), and we were delighted to learn Compugen’s licensee, AstraZeneca, had dosed the first patient in a Phase 1/2 study with AZD2936, which triggered a $0.5 million milestone payment to XOMA from Compugen. Last month, we announced a significant transaction for XOMA, the purchase of rights to a 0.5% commercial payment on faricimab, a BLA-review-stage asset, for a $6 million upfront plus potential future milestone payments to Affitech SA. Three assets in our portfolio received special designations from the U.S. Food and Drug Administration. Additionally, there was meaningful progress amongst our portfolio as partners expanded their clinical development programs," stated Jim Neal, Chief Executive Officer of XOMA.

"We have further strengthened XOMA’s team in the past few months. Heather L. Franklin, President and Chief Executive Officer of Blaze Bioscience, Inc., joined our Board of Directors this summer. We were very pleased to attract two accomplished talents: Joyce Chan joined as our Vice President, Scientific Analysis, and Christopher Baldwin as our Vice President, Legal. Joyce spent 16 years at Amgen, both as a scientist and in business development, licensing, and alliance management. Chris combines significant experience providing legal counsel with entrepreneurial experience, both of which are important for XOMA’s business operations and our lean operating structure.

"I am pleased with the progress both our team and our partners have made in 2021, and we express our gratitude to all of the patients who participate in our partners’ clinical trials," Mr. Neal concluded.

Financial Results
XOMA recorded total revenues of $0.9 million for the third quarter of 2021, compared with $0.6 million in the third quarter of 2020. The increase for the three months ended September 30, 2021, as compared to the corresponding period of 2020, was primarily due to a $0.5 million milestone earned under our license agreement with Compugen Ltd., which was triggered by the dosing of the first patient in a Phase 1/2 study of AZD2936, a TIGIT/PD-1 bispecific antibody derived from COM902, that is being developed by AstraZeneca.

Research and development expenses were $30,000 and $34,000, respectively, for the third quarters of 2021 and 2020.

General and administrative ("G&A") expenses were $4.3 million for the third quarter of 2021, compared to $3.2 million for the third quarter of 2020. The increase of $1.1 million for the three months ended September 30, 2021, as compared to the corresponding period of 2020, was due primarily to a $0.5 million increase in salaries and related expenses, a $0.3 million increase in consulting costs, and $0.1 million increase in legal and insurance costs.

In the third quarter of 2021, G&A expenses included $0.8 million in non-cash stock-based compensation expense, compared with $0.7 million in the third quarter of 2020. The Company’s net cash used in operations in the third quarter of 2021 was $3.1 million, as compared with $2.4 million during the third quarter of 2020.

In the third quarter of 2020, XOMA recorded $0.4 million in interest expense. In June 2021, the Company repaid its outstanding debt obligations to Silicon Valley Bank and Novartis in full.

Net loss for the third quarter of 2021 was $4.4 million, compared to net loss of $1.1 million for the third quarter of 2020.

On September 30, 2021, XOMA had cash of $68.8 million. The Company ended December 31, 2020, with cash of $84.2 million. The Company continues to believe its current cash position will be sufficient to fund XOMA’s operations for multiple years.

On November 4, 2021 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported an oral presentation related to the industrialization of its manufacturing process and poster presentations related to its obe-cel (AUTO1) and AUTO1/22 programs, the company’s CAR T cell product candidates being investigated in adult Acute Lymphocytic Leukemia (ALL) (the FELIX study) and Pediatric ALL (the CARPALL study), respectively, at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, being held between December 11-14, 2021 (Press release, Autolus, NOV 4, 2021, View Source [SID1234594503]). The data to be presented will demonstrate progress on the Company’s proprietary Miltenyi Prodigy-Based commercial manufacturing process, as well as expand on the positive obe-cel data presented at the European Hematology Association (EHA) (Free EHA Whitepaper) Virtual Congress in June 2021.

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Oral Presentation Title: Industrialization of an Academic Miltenyi Prodigy-Based CAR T process
Session Name: 711. Cell Collection and Processing: Advances in Mobilization, Collection, Manipulation and Engineering of HSCs and T Cells
Abstract: #477
Date: Sunday, December 12, 2021
Session Time: 12:00 PM – 1:30 PM ET; Presentation Time: 12:30 PM ET
Location: Georgia World Congress Center, Hall A1
Presenter: Dr. Claire Roddie MD, PhD, FRCPath, Consultant Haematologist and Honorary Senior Lecturer, Cancer Institute, University College London (UCL)

Poster Presentation Title: Safety and Efficacy of AUTO1, a Fast-Off Rate CD19 CAR in Relapsed/Refractory B-Cell Non-Hodgkin’s Lymphoma (B-NHL) and Chronic Lymphocytic Leukaemia (CLL)
Session Title: 704. Cellular Immunotherapies: Clinical: Poster III
Abstract: #3823
Date: Monday, December 13, 2021
Presentation Time: 6:00 PM – 8:00 PM ET
Location: Georgia World Congress Center, Hall B5
Presenter: Dr. Clare Roddie, MD, PhD, FRCPath, Consultant Haematologist and Honorary Senior Lecturer, Cancer Institute, University College London (UCL)

Poster Presentation Title: A high sensitivity aCD22 CAR combined with aCD19 CAR to generate dual targeting CAR T cells for the treatment of r/r B-ALL
Session Title: 703. Cellular Immunotherapies: Basic and Translational: Poster I
Abstract: #1710
Date: Saturday, December 11, 2021
Presentation Time: 5:30 PM – 7:30 PM ET
Location: Georgia World Congress Center, Hall B5
Presenter: Dr. Sara Ghorashian, MD, PhD, Hon clinical senior lecturer, UCL Great Ormond Street Institute of Child Health

Investor call details
Management will host a conference call and webcast on Tuesday, December 14, 2021 at 8:00 am ET/1:00 pm GMT to discuss the ASH (Free ASH Whitepaper) data. To listen to the webcast and view the accompanying slide presentation, please go to the events section of Autolus’ website.

The call may also be accessed by dialing (866) 679-5407 for U.S. and Canada callers or (409) 217-8320 for international callers. Please reference conference ID 31044873. After the conference call, a replay will be available for one week. To access the replay, please dial (855) 859-2056 for U.S. and Canada callers or (404) 537-3406 for international callers. Please reference conference ID 31044873.