On November 8, 2021 Idera Pharmaceuticals, Inc. ("Idera," the "Company," "we," "us," or "our") (Nasdaq: IDRA) reported its financial and operational results for the third quarter ended September 30, 2021 (Press release, Idera Pharmaceuticals, NOV 8, 2021, View Source [SID1234594796]).

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"We are maintaining our focus on identifying new development or commercial-stage assets for Idera’s portfolio and we are encouraged by the opportunities presented to us," stated Vincent Milano, Idera’s Chief Executive Officer. "As a number of these prospects advance and as we continue to preserve cash, we remain optimistic in Idera’s future potential."

Added Mr. Milano, "Studies also continue in patients with microsatellite-stable colorectal cancer (ILLUMINATE-206) and, in partnership with AbbVie, in patients with head and neck squamous cell carcinoma. We expect to share results from the second stage of ILLUMINATE-206, which involves tilsotolimod in combination with Bristol Myers Squibb’s nivolumab and ipilimumab, by the end of the year."

Third Quarter Financial Results
Our cash position as of September 30, 2021 was $36.6 million. Research and development expenses for the three months ended September 30, 2021 totaled $3.5 million, compared to $4.8 million for the same period in 2020. General and administrative expense for the three months ended September 30, 2021 totaled $2.3 million, compared to $2.7 million for the same period in 2020. Restructuring costs for the three months ended September 30, 2021 totaled approximately $0.1 million and relate to a reduction in force initiated in April 2021 to better align our workforce to our ongoing operational and business development activities. No such costs were incurred during the three months ended September 30, 2020. Additionally, during the three months ended September 30, 2020, we recorded $0.7 million and $12.4 million non-cash warrant revaluation loss and non-cash future tranche right revaluation loss, respectively, related to securities issued in connection with our December 2019 private placement transaction. No such non-cash losses were recognized in the three months ended September 30, 2021.

As a result of the factors above, net loss applicable to common stockholders for the three months ended September 30, 2021 was $6.0 million or $0.11 per basic and diluted share compared to net loss applicable to common stockholders of $20.6 million or $0.59 per basic and diluted share for the same period in 2020. Excluding the non-cash loss of approximately $13.1 million for the three months ended September 30, 2020 related to the securities issued in connection with the December 2019 private placement transaction, net loss applicable to common stockholders was $7.5 million.

On November 8, 2021 Ziopharm Oncology, Inc. ("Ziopharm" or the "Company") (Nasdaq: ZIOP), reported financial results for the third quarter ended September 30, 2021 and provided a business update (Press release, Ziopharm, NOV 8, 2021, View Source [SID1234594795]).

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"I am impressed by the quality and dedication of our team at Ziopharm who all are diligently working to transform our cutting-edge scientific research into meaningful clinical progress. Last quarter we made the decision to restructure the organization and focus our efforts on advancing our differentiated TCR-T library towards the clinic," said Kevin S. Boyle, Sr., Chief Executive Officer at Ziopharm Oncology. "We are making excellent progress to operationalize our in-house cGMP clinical production unit. We completed the necessary engineering and process qualification runs in October and remain committed to begin treating patients in the first half of 2022. Our team is executing on this disciplined strategy to bring value to our stakeholders and improve the lives of cancer patients."

Recent Developments and Upcoming Milestones:

TCR-T Library Clinical Program: Ziopharm’s planned Phase 1/2 TCR-T Library study will be a basket trial and enroll patients with non-small cell lung cancer, colorectal, endometrial, pancreatic, ovarian and cholangiocarcinoma where a matching neoantigen TCR/HLA pairing is available in the Company’s TCR-T library. Using the Company’s Sleeping Beauty transposon/transposase technology, patients will be infused with autologous T-cells that have been engineered to express T-cell receptors that are reactive against mutated neoantigens. Patients will be enrolled in one of three progressing TCR-T cells dose levels. The primary endpoint of the initial phase of the study will be to determine the Maximum Tolerated Dose (MTD) or optimal TCR-T cells dose. Ziopharm continues to expect to dose the first patient in the TCR-T Library Phase 1/2 clinical trial in the first half of 2022.

cGMP clinical production unit (CPU): Ziopharm continues to develop its in-house cGMP CPU in Houston, TX, which is expected to operationalize in the first half of 2022 to support the manufacturing of TCR-T therapies for Ziopharm’s Phase 1/2 TCR-T library trial. In the third quarter, the Company continued to make progress in establishing these manufacturing capabilities, and in October, completed engineering and process qualification runs in the CPU.

Expanding library of TCRs through a robust R&D discovery engine: The Company continues to qualify new TCRs using its Sleeping Beauty technology to further expand the potential utility and applicable patient population for its TCR-T Library. Ziopharm expects to file an amended investigational new drug (IND) application for its TCR-T Library clinical program in the next few months to include four additional TCRs.

Corporate Updates:

Completed strategic restructuring: In September 2021, Ziopharm announced a greater than 50% reduction in personnel, enabling the Company to extend its cash runway and prioritize advancing its lead TCR-T library program towards the clinic.

Leadership Appointments: In October 2021, Ziopharm announced that Michael Wong has been appointed Vice President, Finance and Principal Accounting Officer. Mr. Wong brings over 15 years of experience to Ziopharm. Most recently he was Director of Technical Accounting at a large public company.

Third Quarter Ended September 30, 2021 Financial Results:

Research and Development Expenses: Research and development expenses were $14.5 million for the quarter ended September 30, 2021, as compared to $14.0 million for the same period in 2020, an increase of approximately 4%. The increase in research and development expense was primarily due to a $2.2 million charge recognized during the third quarter of 2021 related to our strategic restructuring announced on September 27. The increase was primarily offset by $1.6 million in reduced trial and consulting costs.

General and Administrative Expenses: General and administrative expenses were $8.2 million for the quarter ended September 30, 2021, as compared to $6.4 million for the same period in 2020, an increase of approximately 29%. The increase in general and administrative expenses was primarily due to $1.3 million in employee related severance charges in association with our September 2021 strategic restructuring recognized during the third quarter of 2021 and an increase of $0.8 million related to consulting service costs. The increases were partially offset by a $0.1 million decrease in salary and employee related costs.

Net Loss: Net loss was $22.7 million, or $0.11 net loss per share, for the quarter ended September 30, 2021, as compared to a net loss of $20.3 million, or $0.10 net loss per share, for the same period in 2020. The increase was primarily due to the severance charge of $3.5 million related to our strategic restructuring.

Cash and Cash Equivalents: As of September 30, 2021, Ziopharm had approximately $91.7 million in cash and cash equivalents. Given its current development plans and continued cost management efforts, the Company anticipates its cash runway will extend into the second quarter of 2023.

Conference Call and Webcast

Ziopharm will host a conference call and webcast today, November 8, 2021 at 4:30pm ET. Participants should dial 877-451-6152 (United States) or 201-389-0879 (International) with the conference code 13724384. A live webcast may be accessed using the link here, or by visiting the "Investors" section of the Ziopharm website at www.ziopharm.com. After the live webcast, the event will be archived on Ziopharm’s website for approximately 90 days after the call.

On November 8, 2021 Mirati Therapeutics, Inc. (NASDAQ: MRTX), a clinical-stage targeted oncology company, reported financial results for the third quarter of 2021 and recent corporate updates (Press release, Mirati, NOV 8, 2021, View Source [SID1234594794]).

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"Mirati is aggressively focused on executing its strategy to deliver targeted cancer medicines to patients, and is well positioned for sustained growth from our differentiated research and development capabilities," said David Meek, chief executive officer, Mirati Therapeutics, Inc. "Our recent positive clinical updates for adagrasib reinforce a best-in-class profile, including topline results from the registration-enabling Phase 2 cohort of the KRYSTAL-1 study in patients with KRASG12C-mutated lung cancer, where we expect to launch in the U.S. next year, as well as encouraging results in KRASG12C-mutated colorectal and pancreatic cancers, which were presented at recent medical congresses. We are also pleased to advance the development of adagrasib in earlier lines of therapy, including in first-line non-small cell lung cancer. The rest of our novel pipeline continues to progress rapidly, which includes sitravatinib, MRTX1719, our MTA-cooperative PRMT5 inhibitor, as well as our mutant KRAS programs beyond KRASG12C, including MRTX1133, our KRASG12D inhibitor, and our SOS1 program."

Pipeline Updates

The U.S. Food and Drug Administration (FDA) will review the Company’s New Drug Application (NDA) for adagrasib for the treatment of patients with previously treated KRASG12C-mutated non-small cell lung cancer (NSCLC) who have received prior systemic therapy under the Real-Time Oncology Review (RTOR) pilot program. The RTOR status follows the previously announced U.S. FDA Breakthrough Therapy Designation for adagrasib in the same indication. The Company expects to complete the adagrasib NDA submission to the FDA by the end of 2021.
Preliminary results from the Phase 1b cohort of the KRYSTAL-1 study evaluating adagrasib plus pembrolizumab1 in 8 patients with KRASG12C-mutated first-line NSCLC support moving forward with a 400 mg BID dose of adagrasib with full dose pembrolizumab, which will be evaluated in the ongoing Phase 2 KRYSTAL-7 study. The Phase 1b data showed adagrasib 400mg BID plus pembrolizumab had a manageable tolerability profile, with no observed Grade 4 or Grade 5 adverse events or treatment-related discontinuations. Of the 7 patients evaluable for a response as of October 21, 2021, 4 had a confirmed RECIST-defined partial response and 1 additional patient, who is still on study, experienced 49% tumor regression in the first scan, which allowed for tumor resection prior to achieving a RECIST-defined confirmed response. The disease control rate was 100%, with all 7 patients exhibiting tumor regression ranging from 37% to 92%. With a median follow up of 9.9 months, 5 of the 7 patients remained on treatment, as of the data cutoff date, and had been on treatment for 8 to 11 months.
Announced positive topline results from the potentially registration-enabling cohort of the Phase 2 KRYSTAL-1 study evaluating adagrasib in patients with advanced NSCLC harboring the KRASG12C mutation following prior systemic therapy, as well as updated findings from the Phase 1/1b KRYSTAL-1 study evaluating adagrasib in all enrolled patients with KRASG12C-mutated advanced NSCLC. The topline results were presented at a virtual Investor Event held during the European Society for Medical Oncology Congress (ESMO) (Free ESMO Whitepaper) congress. The Company plans to submit detailed results for presentation at a medical congress prior to the potential U.S. launch of adagrasib in 2022. (View Release)
Announced a non-exclusive clinical trial collaboration agreement with Sanofi to evaluate adagrasib with Sanofi’s investigational SHP2 inhibitor SAR442720, also known as RMC-4630, in patients with previously-treated NSCLC and KRASG12C mutations. (View Release)
Presented clinical research on adagrasib and sitravatinib at the 2021 ESMO (Free ESMO Whitepaper) Congress, including:
Data from a cohort of the Phase 1/2 KRYSTAL-1 study evaluating adagrasib as a monotherapy or in combination with cetuximab (ERBITUX)2 in patients with KRASG12C -mutated colorectal cancer (CRC). (View Release) The Company has an actively enrolling global registrational Phase 3 clinical trial, KRYSTAL-10, comparing adagrasib plus cetuximab to standard of care chemotherapy in second-line KRASG12C-mutated CRC.
Exploratory results from the Phase 2 MRTX-500 study evaluating sitravatinib plus nivolumab (OPDIVO)3 in patients with advanced NSCLC who had disease progression following treatment with checkpoint inhibitors. The Company has an actively enrolling global registrational Phase 3 study, SAPPHIRE, evaluating sitravatinib plus nivolumab in second or third line non-squamous NSCLC and plans to provide an update based on an interim analysis of overall survival in the second half of 2022. (View Release)
Presented preclinical and clinical data at the 2021 AACR (Free AACR Whitepaper)-NCI-EORTC Virtual AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper), including:
A summary of the discovery and preclinical characterization of MRTX1133, a mutant-selective KRASG12D inhibitor. This presentation also included preliminary new clinical data from a cohort of the KRYSTAL-1 study evaluating adagrasib in previously-treated patients with KRASG12C-mutated pancreatic cancer, as well as proof-of-concept data in non-clinical models for inhibitors of additional KRAS mutations beyond KRASG12C and KRASG12D.
Preclinical results on MRTX1719, the selected development candidate from the Company’s MTA-cooperative PRMT5 inhibitor program, in MTAP-deleted cancer models. The Company expects to file an Investigational New Drug application for MRTX1719 by the end of 2021.
Corporate Updates

Announced the appointment of David Meek as chief executive officer and board member of the Company, Charles M. Baum, M.D., Ph.D. transitioning to the Company’s president, founder and head of research and development, and continuing in his role as a board member; and other previously announced leadership updates.
Third Quarter 2021 Financial Results

Ended the third quarter with approximately $1.2 billion in cash, cash equivalents, and short-term investments, which includes net proceeds of $63.4 million for the upfront fee from Zai Lab pursuant to the collaboration and license agreement executed during the second quarter.
Research and development expenses for the third quarter of 2021 were $116.1 million, compared to $79.9 million for the same period in 2020. Research and development expenses for the nine months ended September 30, 2021 were $354.8 million, compared to $216.6 million for the same period in 2020. The increase in research and development expenses is primarily due to an increase in expense associated with the development of adagrasib, an increase in preclinical and early discovery activities, as well as an increase in salaries and other employee-related expense, which includes an increase in share-based compensation expense. The Company recognized research and development-related share-based compensation expenses of $15.7 million during the third quarter of 2021, compared to $12.6 million for the same period in 2020, and $46.7 million during the nine months ended September 30, 2021, compared to $35.9 million for the same period in 2020.
General and administrative expenses for the third quarter of 2021 were $35.2 million, compared to $20.2 million for the same period in 2020. General and administrative expenses for the nine months ended September 30, 2021 were $93.1 million, compared to $58.1 million for the same period in 2020. The increase is due to an increase in professional services expense primarily associated with commercial scale up, an increase in salaries and other employee-related expenses, an increase in insurance, rent and other facilities-related costs, and an increase in sponsorship agreements expense. The Company recognized general and administrative-related share-based compensation expenses of $11.2 million in the third quarter of 2021, compared to $9.2 million for the same period in 2020, and $32.9 million during the nine months ended September 30, 2021, compared to $28.2 million for the same period in 2020.
Net loss for the third quarter of 2021 was $80.1 million, or $1.55 per share basic and diluted, compared to a net loss of $87.3 million, or $1.96 per share basic and diluted for the same period in 2020. Net loss for the nine months ended September 30, 2021 was $382.2 million, or $7.45 per share basic and diluted, compared to a net loss of $256.9 million, or $5.87 per share basic and diluted for the same period in 2020.
Conference Call Information

Investors and the general public are invited listen to a live webcast of the call at the "Investors and Media" section on Mirati.com or by dialing the U.S. toll free 313-209-7315 or international +1 877-614-0009, confirmation code: 3962567. Materials related to the call will be available at the same website at the time of the conference call. A replay of the call will be available approximately 2 hours after the event has ended at the same website or by dialing in the U.S. toll free 719-457-0820 or international +1 888-203-1112, confirmation code: 3962567.

On November 8, 2021 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on creating and delivering engineered cells as medicines, reported financial results and business highlights for the third quarter of 2021 (Press release, Sana Biotechnology, NOV 8, 2021, View Source [SID1234594793]).

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"We continue to make progress in building the company, progressing our pipeline, and growing our people and capabilities," said Steve Harr, Sana’s President and Chief Executive Officer. "The recent licensing of genome editing technology to enable multiple pipeline programs is an example of our continued focus on augmenting our innovative capacity. As our pipeline progresses, we look forward to presenting data from our in vivo CAR T and ex vivo allogeneic CAR T cell programs at the upcoming 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition in December."

Recent Corporate Highlights

Announced an agreement for non-exclusive commercial rights to Beam’s CRISPR Cas12b nuclease system for certain ex vivo engineered cell therapy programs. Engineering cells for therapeutic applications requires technologies for precise editing of their genome sequence. We plan to use the technology with certain product candidates, including many of our allogeneic CAR T and pluripotent stem cell programs.
Third Quarter 2021 Financial Results

GAAP Results

Cash Position: Cash, cash equivalents, and marketable securities as of September 30, 2021 were $866.1 million compared to $412.0 million as of December 31, 2020, an increase of $454.1 million. The increase was primarily driven by net proceeds of $626.4 million received in Sana’s initial public offering in February 2021, partially offset by cash used in operations of $141.0 million and cash used for the purchase of property and equipment of $24.7 million.

Research and Development Expenses: For the three and nine months ended September 30, 2021, research and development expense, inclusive of non-cash expenses, was $53.2 million and $140.2 million, respectively, compared to $40.1 million and $96.5 million, respectively, for the same periods in 2020. The increases of $13.1 million and $43.6 million, respectively, for the three and nine months ended September 30, 2021 were due to an increase in personnel expenses related to increased headcount to expand Sana’s research and development capabilities, costs for laboratory supplies, costs for preclinical studies and external manufacturing, and facility costs. Research and development expenses include non-cash stock-based compensation of $4.1 million and $9.9 million, respectively, for the three and nine months ended September 30, 2021 and $1.0 million and $2.6 million, respectively, for the same periods in 2020.

Research and Development Related Success Payments and Contingent Consideration: For the three and nine months ended September 30, 2021, we recognized non-cash expenses of $16.8 million and $67.8 million, respectively, in connection with the change in the estimated fair value of the success payment liabilities and contingent consideration, compared to $4.5 million and $57.3 million, respectively, for the same periods in 2020.

General and Administrative Expenses: General and administrative expenses for the three and nine months ended September 30, 2021, inclusive of non-cash expenses, were $13.4 million and $37.7 million, respectively, compared to $7.1 million and $19.1 million, respectively, for the same periods in 2020. The increases of $6.3 million and $18.6 million, respectively, in the three and nine months ended September 30, 2021 were primarily due to increased personnel-related expenses attributable to an increase in headcount to build our infrastructure, legal fees to support our patent portfolio and license arrangements, insurance associated with being a public company, consulting fees, and facility costs. General and administrative expenses include stock-based compensation of $1.9 million and $5.2 million, respectively, for the three and nine months ended September 30, 2021 and $0.2 million and $0.5 million, respectively, for the same periods in 2020.

Net Loss: Net loss for the three and nine months ended September 30, 2021 were $83.3 million, or $0.46 per share, and $245.2 million, or $1.53 per share, respectively, compared to $51.5 million, or $3.76 per share, and $172.1 million, or $14.05 per share, respectively, for the same periods in 2020.
Non-GAAP Measures

Non-GAAP Operating Cash Burn: Non-GAAP operating cash burn for the nine months ended September 30, 2021 was $146.4 million compared to $87.2 million for the nine months September 30, 2020. Non-GAAP operating cash burn is the decrease in cash, cash equivalents, and marketable securities excluding cash inflows from financing activities, cash outflows from business development activities, and the purchase of property and equipment.

Non-GAAP Research and Development Expenses: Non-GAAP research and development expenses for the three and nine months ended September 30, 2021 were $53.2 million and $140.1 million, respectively, compared to $31.6 million and $86.5 million, respectively, for the same periods in 2020. Non-GAAP research and development expenses excludes one-time costs to acquire technology.

Non-GAAP Net Loss: Non-GAAP net loss for the three and nine months ended September 30, 2021 was $66.5 million, or $0.37 per share, and $177.4 million, or $1.11 per share, respectively, compared to $38.5 million, or $2.81 per share, and $104.9 million, or $8.56 per share, respectively, for the same periods in 2020. Non-GAAP net loss exclude one-time costs to acquire technology and non-cash expenses related to the change in the estimated fair value of contingent consideration and success payment liabilities.
A discussion of non-GAAP measures, including a reconciliation of GAAP and non-GAAP measures, is presented below under "Non-GAAP Financial Measures."

On November 8, 2021 Kinnate Biopharma Inc. (Nasdaq: KNTE) ("Kinnate"), a biopharmaceutical company focused on the discovery and development of small molecule kinase inhibitors for difficult-to-treat, genomically defined cancers, reported that its Chief Executive Officer Nima Farzan will provide a company overview at the following upcoming investor conferences in November (Press release, Kinnate Biopharma, NOV 8, 2021, View Source [SID1234594787]):

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Event: Stifel 2021 Virtual Healthcare Conference
Location: Virtual
Date: Wednesday, November 17, 2021
Time: 1:20 PM ET/10:20 AM PT
Event: Piper Sandler 33rd Annual Virtual Healthcare Conference
Location: Virtual
Fireside Chat: A pre-recorded fireside chat will be available on-demand beginning on Monday, November 22nd at 10:00am ET
Members of the Kinnate management team will also host investor meetings during both conferences.

Webcasts of both presentations will be available in the Investors and Media section of the Kinnate website at www.kinnate.com. A webcast replay will also be available on this website shortly after conclusion of the events for 30 days.