On August 14, 2023 Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases, reported the U.S. Food and Drug Administration (FDA) has lifted the partial clinical hold placed on the company’s CART-ddBCMA investigational new drug for the treatment of patients with relapsed or refractory multiple myeloma (rrMM) and reported financial results for the second quarter ended June 30, 2023 (Press release, Arcellx, AUG 14, 2023, View Source [SID1234634362]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We have worked closely with FDA to expeditiously resolve the clinical hold and we thank them for their collaboration and dialogue throughout this process," said Rami Elghandour, Arcellx’s Chairman and Chief Executive Officer. "During the review process, we updated our trial protocol, and were pleased that FDA allowed for expanded bridging therapies, which better aligns our protocol with current clinical practice. As a key step to enhancing protocol adherence related to the prevention and management of the risk of adverse events, we retrained clinical sites. Importantly, during the partial clinical hold, FDA approved dosing of all 17 patients who had been enrolled but not yet dosed prior to the hold, minimizing treatment disruption for patients and clinicians. We and our partners at Kite remain confident in CART-ddBCMA’s potential as a best-in-class therapy for the treatment of patients with rrMM given the totality of data to date across our studies. We have a strong balance sheet funding operations through BLA filing and into 2026. We look forward to presenting data from our Phase 1 study later this year as well as preliminary data from the iMMagine-1 study in the second half of 2024. Additionally, we continue to expect commercial launch of CART-ddBCMA to be in 2026."

Recent Business Progress

Announced clinical hold and clinical hold lifted by FDA for the company’s iMMagine-1 Phase 2 Clinical Program. On June 19, 2023, Arcellx announced that its iMMagine-1 study had been placed on partial clinical hold by FDA following a recent patient death, which involved a patient who was treated with CART-ddBCMA despite becoming ineligible for treatment under the trial protocol prior to CART-ddBCMA infusion. Subsequently, the patient was managed in a manner that conflicted with the trial protocol. On August 14, 2023, Arcellx announced that FDA had lifted the partial clinical hold after aligning with FDA on modifications to the iMMagine-1 trial protocol related to the prevention and management of the risk of adverse events within the trial. As a key effort to enhance protocol adherence, Arcellx retrained clinical sites. Additionally, FDA allowed an expansion of treatment options for therapies that patients in the iMMagine-1 trial are permitted to receive between apheresis and CAR-T infusion (also known as bridging therapies) to better align its protocol with current clinical practice. The company anticipates presenting preliminary data from the iMMagine-1 study in the second half of 2024.

Second Quarter 2023 Financial Highlights

Cash, cash equivalents, and marketable securities:
As of June 30, 2023, Arcellx had cash, cash equivalents, and marketable securities of $506.5 million. Arcellx anticipates that its cash, cash equivalents, and marketable securities will fund its operations into 2026.

Collaboration revenue:
Collaboration revenue was $14.3 million and $0 for the quarters ended June 30, 2023 and 2022, respectively. The revenue results for the three months ended June 30, 2023 is from the recognition of research and development performed under the arrangement described in the recent license and collaboration agreement with Kite Pharma, Inc. (Kite). Revenue is being recognized on a percentage of completion basis over the term of the contract with Kite.

R&D expenses:
Research and development expenses were $28.3 million and $22.1 million for the quarters ended June 30, 2023 and 2022, respectively, an increase of $6.2 million. This increase was primarily driven by higher external costs associated with the advancement of the company’s CART-ddBCMA clinical program and personnel.

G&A expenses:
General and administrative expenses were $15.5 million and $9.2 million for the quarters ended June 30, 2023 and 2022, respectively, an increase of $6.3 million. This increase was driven by primarily by personnel and professional fees.

Net loss:
Net loss was $23.9 million and $30.8 million for the quarters ended June 30, 2023 and 2022, respectively.

On August 14, 2023 CohBar, Inc. (NASDAQ: CWBR) reported its financial results and highlights for the second quarter ended June 30, 2023 (Press release, CohBar, AUG 14, 2023, View Source [SID1234634361]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Second Quarter 2023 Summary and Financial Results

● Entered into Definitive Merger Agreement with Morphogenesis: In May 2023, CohBar announced that the company entered into a definitive merger agreement with a privately held biotechnology company, Morphogenesis, Inc. ("Morphogenesis"), for an all-stock transaction to advance a late-stage oncology pipeline. The combined company is expected to operate under the name "TuHURA Biosciences, Inc." and to trade on The Nasdaq Capital Market ("Nasdaq"). The transaction is expected to close in the fourth quarter of 2023.

● Cash, Cash Equivalents and Investments: The company had cash, cash equivalents and investments of $12.3 million as of June 30, 2023, compared to $15.7 million as of December 31, 2022.

● R&D Expenses: Research and development expenses were $0.2 million for the three months ended June 30, 2023, compared to $1.2 million in the prior year quarter. The lower research and development expenses are due to the suspension of our development activities.

● G&A Expenses: General and administrative expenses were $4.3 million for the three months ended June 30, 2023, compared to $1.6 million in the prior year quarter. The increase in general and administrative expenses was primarily due to costs related to the merger with Morphogenesis and compensation charges incurred related to the retention of our key executives.

● Net Loss: For the three months ended June 30, 2023, net loss, which included $0.3 million of non-cash expenses, was $4.3 million, or $1.49 per basic and diluted share. For the three months ended June 30, 2022, net loss, which included $0.5 million of non-cash expenses, was $2.7 million, or $0.94 per basic and diluted share.

On August 14, 2023 NightHawk Biosciences (NYSE American: NHWK), a fully integrated biopharmaceutical company specializing in the end-to-end development, manufacturing, and commercialization of innovative medical countermeasures that combat unmet and emerging biothreats, reported strategic, financial, and operational updates for the quarter ended June 30, 2023 (Press release, NightHawk Biosciences, AUG 14, 2023, View Source [SID1234634360]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Jeff Wolf, Chief Executive Officer of NightHawk, commented, "Development of our biomanufacturing operations, continues to progress. Specifically, we continue to invest in our San Antonio facilities and are currently evaluating a variety of strategic options to advance these operations. In parallel, we are continuing our R&D activities, and are highly encouraged by the latest preclinical data. Towards this end, our strategy is to further develop these potential therapies through key milestones, with a focus on licensing or partnering these assets to maximize value for shareholders. We look forward to providing updates on these programs."

Second Quarter 2023 Financial Results

For the three months ended June 30, 2023 we recognized $0.7 million of revenue from process development. For the three months ended June 30, 2022 we recognized $0.05 million of service revenue. The increase in process development revenue is attributable to the operations of the San Antonio CDMO facility.
Research and development expenses increased approximately 21.3% to $5.7 million for the three months ended June 30, 2023 compared to $4.7 million for the three months ended June 30, 2022. The components of R&D expense are as follows, in millions: HS-110 expense increased by $0.3 million primarily due to site close out fees; HS-130 expense decreased to $0 from $0.1 million due to the de-prioritization of our oncology assets; PTX-35 expense decreased by $0.4 million primarily due to the discontinued clinical trial and development of the product candidate in the third quarter of 2022; ANTHIM was not acquired until the second quarter of 2022 and the 2023 expense primarily relates to fill finish; other programs expense decreased by $0.3 million primarily due to a decrease in laboratory supplies expense related to preclinical R&D expenses; and unallocated research expenses increased by $0.9 million primarily due to increased personnel costs, including stock-based compensation from stock awards, contractor expense and supplies purchased for discovery projects.

Cost of revenues were $0.4 million for the three months ended June 30, 2023. These expenses primarily reflect direct cost of labor, overhead and material costs. There was no cost of revenues for the three months ended June 30, 2022 as the Scorpius facility was not operational.

Selling, general and administrative expenses were $7.4 million and $4.9 million for the three months ended June 30, 2023 and 2022, respectively. The increase was primarily due to increases in consulting and other professional expenses of $0.7 million, personnel expense of $0.5 million, marketing expense of $0.5 million, facility expense of $0.3 million, rent expense of $0.2 million, depreciation and amortization of $0.3 million, insurance and taxes of $0.2 million, offset by a decrease in supplies expense of $0.2 million.
Net loss attributable to NightHawk Biosciences was approximately $13.9 million, or ($0.53) per basic and diluted share, for the three months ended June 30, 2023, compared to approximately $6.8 million, or ($0.27) per basic and diluted share, for the three months ended June 30, 2022.
As of June 30, 2023, the Company had approximately $18.6 million in cash, cash equivalents, and short-term investments.

On August 14, 2023 TRACON Pharmaceuticals, Inc. (Nasdaq: TCON), a clinical stage biopharmaceutical company utilizing a cost-efficient, CRO-independent product development platform to advance its pipeline of novel targeted cancer therapeutics and to partner with other life science companies, reported financial results for the second quarter ended June 30, 2023 (Press release, Tracon Pharmaceuticals, AUG 14, 2023, View Source [SID1234634359]). The Company will host a conference call and webcast today at 4:30 PM Eastern Time / 1:30 PM Pacific Time.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We were pleased to collect the arbitration award of $22M, the initial net proceeds of which extend our cash runway into early 2024 and past expected full accrual of the ENVASARC pivotal trial," said Charles Theuer, M.D., Ph.D., TRACON’s Chief Executive Officer. "We look forward to reporting the interim efficacy assessment from ENVASARC later this quarter, which includes a futility threshold that has already been achieved based on responses seen to date. In June, we reported that envafolimab treatment continues to generate a double-digit objective response rate (ORR) and has been well tolerated, and as such we believe that we remain on track to achieve the primary endpoint of the study, which is a minimum 11.25% ORR. Achieving a double-digit ORR with a well-tolerated safety profile positions envafolimab to become a potentially compelling treatment option for patients with the refractory sarcoma subtypes of UPS and MFS. The sole approved treatment for these patients is Votrient, which achieved a 4% ORR and carries a black box warning for fatal liver toxicity."

Recent Corporate Highlights

In June, we announced an ongoing double-digit ORR for single agent envafolimab in patients with two on-study scans in the ongoing ENVASARC Phase 2 pivotal trial without any > Grade 2 drug related toxicity. The single agent envafolimab ORR exceeded the futility rule that will be applied at the interim efficacy analysis expected later this quarter, and full ENVASARC accrual is expected in the fourth quarter.

In July, we announced collection of the arbitration award of $22M from I-Mab Biopharma, with the initial net proceeds of $7.1 million expected to fund the Company’s operations, as currently planned, into early 2024.

Expected Upcoming Milestones

Report the second and final interim efficacy analysis from the ENVASARC pivotal trial following the review of more than 12 weeks of efficacy data (including two on-study CT scans) by the IDMC from 46 patients who receive envafolimab as a single agent, which we expect later this quarter as the ENVASARC trial has enrolled 180 patients to date, including 56 of the 80 expected patients in cohort C of single agent envafolimab treatment that is the basis for determination of the primary endpoint of the study, which is a minimum 11.25% ORR.

Complete accrual of the ENVASARC pivotal trial in the fourth quarter of 2023.

Leverage TRACON’s cost-efficient, CRO-independent product development platform to generate non-dilutive capital by the end of 2023.

Final data from ENVASARC pivotal trial in mid-2024.

Second Quarter 2023 Financial Results

Cash, cash equivalents and restricted cash were $1.9 million at June 30, 2023, compared to $17.5 million at December 31, 2022, which does not include the $7.1 million in initial net proceeds from the arbitration award that was received in July. TRACON’s pro forma cash position of $9.0 million is expected to fund the Company into the first quarter of 2024.

An additional $4.4M of the arbitration award remains in a client trust account administered by our law firm at this time, the disbursement of which is predicated on discussions as to the amount of success-based deferred legal fees the firm is due.

Collaboration revenue was $9.0 million for the second quarter of 2023, compared to nil for the second quarter of 2022. The increase was related to the termination of the TJ4309 license.

Research and development expenses for the second quarter of 2023 were $3.5 million, compared to $2.9 million for the second quarter of 2022. The increase was primarily related to increased enrollment into the ENVASARC pivotal trial.

General and administrative expenses for the second quarter of 2023 were $1.9 million, compared to $3.3 million for the second quarter of 2022. The decrease was primarily attributable to lower legal expenses.

Net loss for the second quarter of 2023 was $6.3 million, compared to $6.2 million for the second quarter of 2022. In July we collected the arbitration award from I-Mab which will result in a one-time gain in the third quarter of 2023 of $13.0 million.

Conference Call Details

To access the call by phone, please register using this link and you will be provided with dial-in details.

A live webcast of the conference call will be available online from the Investor/Events and Presentation page of the Company’s website at www.traconpharma.com.

After the live webcast, a replay will remain available on TRACON’s website for 60 days.

About Envafolimab

Envafolimab (KN035), a single-domain antibody against PD-L1 invented by Alphamab Oncology and licensed by TRACON, is the first approved subcutaneously injected PD-(L)1 inhibitor. Envafolimab was approved by the Chinese NMPA in November 2021 in adult patients with MSI-H/dMMR advanced solid tumors who failed systemic treatment and have no satisfactory alternative treatment options. In December 2019, Alphamab Oncology, 3D Medicines and TRACON entered into a collaboration whereby TRACON has the right to develop and commercialize envafolimab in soft tissue sarcoma in North America. Envafolimab is currently being studied in the pivotal ENVASARC Phase 2 trial in the United States sponsored by TRACON and a Phase 3 pivotal trial in combination with gemcitabine and oxaliplatin in advanced biliary tract cancer patients as well as multiple Phase 1 and Phase 2 clinical trials in China sponsored by TRACON’s corporate partners, Alphamab Oncology and 3D Medicines. TRACON has received orphan drug designation from the U.S. Food and Drug Administration for envafolimab for patients with soft tissue sarcoma and fast track designation from the U.S. Food and Drug Administration for envafolimab for patients with locally advanced, unresectable or metastatic undifferentiated pleomorphic sarcoma (UPS) and myxofibrosarcoma (MFS) who have progressed on one or two prior lines of chemotherapy.

About ENVASARC (NCT04480502)

The ENVASARC pivotal trial is a multicenter, open label, randomized, non-comparative, parallel cohort study at 30 top cancer centers in the United States and the United Kingdom that began dosing in December 2020. TRACON expects the trial to enroll more than 160 patients with UPS or MFS who have progressed following one or two lines of prior treatment and have not received an immune checkpoint inhibitor, with 80 patients enrolled into a cohort of treatment with single agent envafolimab at 600 mg every three weeks and 80 patients enrolled into a cohort of treatment with envafolimab at 600 mg every three weeks with Yervoy. The primary endpoint is objective response rate by central review with duration of response a key secondary endpoint.

About YH001

YH001 is an IgG1 antibody against CTLA-4 that has shown enhanced antibody dependent cellular cytotoxicity and complement dependent cytotoxicity in vitro. In preclinical studies YH001 demonstrated superior T cell activation and superior tumor growth inhibition activity compared to ipilimumab. YH001 also demonstrated superior activity compared to ipilimumab in human transgenic mouse tumor models when combined with a PD-(L)1 antibody. In these models, single agent YH001 depleted regulatory T cells and increased CD8+ T cells in tumor tissue. YH001 is being studied with envafolimab and doxorubicin in a Phase 1/2 clinical trial sponsored by TRACON (NCT05448820), and has been studied in multiple Phase 1 trials in China and Australia sponsored by TRACON’s corporate partner Eucure, a division of Biocytogen.

About TRC102

TRC102 (methoxyamine) is a novel small molecule inhibitor of the DNA base excision repair pathway, which is a pathway that causes resistance to alkylating and antimetabolite chemotherapeutics. TRC102 is currently being studied in multiple Phase 1 and Phase 2 clinical trials sponsored by the National Cancer Institute through a Cooperative Research and Development Agreement (CRADA) and has orphan drug designation from the FDA in malignant glioma, including glioblastoma.

On August 14, 2023 Checkpoint Therapeutics, Inc. ("Checkpoint") (Nasdaq: CKPT), a clinical-stage immunotherapy and targeted oncology company, reported financial results for the second quarter ended June 30, 2023, and recent corporate highlights (Press release, Checkpoint Therapeutics, AUG 14, 2023, View Source [SID1234634358]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We continue to work with the U.S. Food and Drug Administration ("FDA") toward the January 3, 2024 action date for our Biologics License Application ("BLA") for cosibelimab. Recently, our mid-cycle communication meeting with the FDA was successfully completed, and the FDA noted that no significant review issues or safety concerns have been identified in their review to date," said James Oliviero, President and Chief Executive Officer of Checkpoint.

"We are also encouraged by the recently announced longer-term data from our pivotal studies of cosibelimab in locally advanced and metastatic cutaneous squamous cell carcinoma ("cSCC"), which demonstrate a deepening of response with cosibelimab treatment over time, resulting in substantially higher complete response rates than previously reported. Specifically, complete response rates more than doubled from 10% to 23% in locally advanced cSCC and nearly doubled, from 8% to 13%, in metastatic cSCC," continued Oliviero. "We believe cosibelimab’s unique dual mechanism of action will benefit not just immunocompetent patients, but also the large number of difficult-to-treat patients with immunosuppressive conditions or taking immunosuppressive medications who are in need of more effective treatment options than available today."

"Equally important, longer-term results continue to confirm cosibelimab’s favorable safety profile, with only 2% of patients experiencing a severe immune-related adverse event ("irAE") and only 1% discontinuing treatment because of an irAE, substantially lower rates than observed with currently approved immunotherapies. We believe cosibelimab’s favorable safety profile should position the product as the preferred immunotherapy of oncologists for high-risk patients, such as those with solid organ transplants or autoimmune disease, upon its potential launch early next year," concluded Oliviero.

Recent Corporate Highlights:

Checkpoint submitted a BLA to the FDA seeking approval of cosibelimab in January 2023. In March 2023, Checkpoint announced the FDA accepted the BLA filing for cosibelimab and set a Prescription Drug User Fee Act ("PDUFA") goal date of January 3, 2024. In its BLA filing acceptance letter, the FDA indicated that no potential filing review issues have been identified, and that an advisory committee meeting to discuss the application is not currently planned.
In April, May and July 2023, Checkpoint completed registered direct offerings priced at-the-market under Nasdaq rules for total gross proceeds of approximately $26.1 million.
In June 2023, Checkpoint announced that new pharmacokinetic modeling data on cosibelimab supporting the extension to an every-three-week dosing regimen were presented at the Population Approach Group Europe 2023 annual meeting. Results support the comparability of cosibelimab 800 mg every-two-week and 1200 mg every-three-week dosing regimens.
In July 2023, Checkpoint announced new, longer-term data for cosibelimab from its pivotal studies in locally advanced and metastatic cSCC. These results demonstrate a deepening of response with cosibelimab treatment over time, resulting in substantially higher complete response rates than previously reported. Furthermore, responses continue to remain durable over time with the median duration of response not yet reached in either the locally advanced or metastatic cSCC group.
Financial Results:

Cash Position: As of June 30, 2023, Checkpoint’s cash and cash equivalents totaled $7.4 million, compared to $4.8 million at March 31, 2023 and $12.1 million at December 31, 2022, an increase of $2.6 million for the quarter and a decrease of $4.7 million for the first half of 2023. Subsequent to the end of the second quarter, Checkpoint raised approximately $10.0 million of gross proceeds in a registered direct offering completed in July 2023.
R&D Expenses: Research and development expenses for the second quarter of 2023 were $13.9 million, compared to $12.1 million for the second quarter of 2022, an increase of $1.8 million. Research and development expenses for the second quarter of 2023 primarily consisted of $9.9 million related to commercial manufacturing costs and inventory build for cosibelimab to support a potential 2024 launch.
G&A Expenses: General and administrative expenses for the second quarter of 2023 were $2.3 million, compared to $2.1 million for the second quarter of 2022, an increase of $0.2 million. General and administrative expenses for the second quarter of 2023 included $0.8 million of non-cash stock expenses, compared to $0.5 million for the second quarter of 2022.

Net Loss: Net loss attributable to common stockholders for the second quarter of 2023 was $16.5 million, or $1.05 per share, compared to a net loss of $14.1 million, or $1.62 per share, in the second quarter of 2022. Net loss for the second quarter of 2023 included $1.0 million of non-cash stock expenses, compared to $0.7 million for the second quarter of 2022.