On May 13, 2024 Turnstone Biologics Corp. ("Turnstone" or the "Company") (Nasdaq: TSBX), a clinical-stage biotechnology company developing a differentiated approach to treat and cure patients with solid tumors by pioneering selected tumor-infiltrating lymphocyte (Selected TIL) therapy, reported financial results for the first quarter ended March 31, 2024, and provided recent business highlights (Press release, Turnstone Biologics, MAY 13, 2024, View Source [SID1234643161]).

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"We are excited about the therapeutic potential of our pipeline of next-generation Selected TIL therapies," said Sammy Farah, M.B.A., Ph.D., Turnstone’s President and Chief Executive Officer. "The next frontier for TIL therapy is to extend its therapeutic activity in additional solid tumor indications beyond melanoma. Our differentiated approach includes a proprietary ‘cell selection’ step which is designed to generate a TIL product that is dominated by tumor-reactive T cells. We believe this process is crucial to develop potent TIL-based therapies such as Turnstone’s Selected TILs to address harder-to-treat, lower mutational burden cancers, such as colorectal cancer and other immunologically cold tumors. This year, we look forward to generating clinical data to highlight the differentiation of our platform and support further advancement of our lead asset, TIDAL-01, which is currently being evaluated in several Phase 1 studies in multiple solid tumor indications. We remain on track and plan to provide a TIDAL-01 clinical update mid-year in connection with our next quarterly financial results"

First Quarter 2024 and Recent Business Highlights

Continued TIDAL-01 Development in Multiple Phase 1 Clinical Trials. The Company is continuing the development of TIDAL-01, Turnstone’s lead Selected TIL therapy. TIDAL-01 is designed to potentially expand the applicability of TIL therapy into solid tumor types where first-generation TILs have not been effective by employing an unbiased identification and functional screening process to isolate and selectively expand the greatest breadth of tumor-reactive TILs from the patient’s tumor. TIDAL-01 is currently being evaluated in colorectal cancer, head and neck squamous cell carcinoma, uveal melanoma, breast cancer, and cutaneous melanoma across Turnstone’s multi-site STARLING trial and investigator-sponsored trials in collaboration with H. Lee Moffitt Cancer Center. Furthermore, Turnstone recently secured additional dedicated cleanroom capacity at Moffit’s on-site cGMP facility for manufacturing of TIDAL-01 for the STARLING trial with IND clearance from the FDA. Turnstone expects to provide an initial/preliminary clinical update from the Phase 1 studies around mid-year.

Executed a $20M Non-Dilutive Revolving Credit Facility. In April, Turnstone secured a revolving credit facility from Banc of California that allows Turnstone to draw on an aggregate amount up to $20 million. The proceeds from the facility, if drawn, will be utilized to support ongoing development of the Company’s pipeline and clinical trials.

Strengthened Company’s Board of Directors. In April, Turnstone announced the appointment of William Waddill to its Board of Directors. Mr. Waddill brings more than three decades of financial and operational expertise in the biotechnology space, and proven leadership in industry organizations. The Company also announced that Patrick Machado has stepped down as a member of its Board of Directors.

First Quarter 2024 Financial Results

Cash, Cash Equivalents and Short-Term Investments: As of March 31, 2024, cash, cash equivalents and short-term investments were $77.8 million. The Company expects that the combined cash, cash equivalents and short-term investments will be sufficient to fund its operations into the third quarter of 2025.

Research and Development (R&D) Expenses: R&D expenses for the three months ended March 31, 2024, were $15.8 million, compared to $15.7 million for the same period in 2023. The increase was due primarily to an increase of $2.4 million in manufacturing expenses and $0.2 million in personnel related costs as we ramp up TIDAL-01 clinical trials offset by a decrease of $0.9 million in clinical and regulatory costs and $1.6 million in pre-clinical research and development costs due to the termination of the Takeda Agreement and winding down activities related to the RIVAL-01 platform during the three months ended March 31, 2023.

General and Administrative (G&A) Expenses: G&A expenses for the three months ended March 31, 2024, were $4.9 million, compared to $4.0 million for the same period in 2023. The increase was due primarily to an increase in professional service costs of $1.1 million related to the increased costs of operating as a public company offset by a decrease in personnel costs of $0.2 million.

Net Loss: Net loss for the three months ended March 31, 2024, was $19.6 million, compared to net income of $0.1 million for the same period in 2023. The decrease was primarily due to the recognition of deferred revenue from the termination of the Takeda Agreement recorded in Collaboration Revenue for the three months ended 2023 compared to no Collaboration Revenue recognized for the same period in 2024.

On May 13, 2024 TScan Therapeutics, Inc. (Nasdaq: TCRX), a clinical-stage biopharmaceutical company focused on the development of T cell receptor (TCR)-engineered T cell (TCR-T) therapies for the treatment of patients with cancer, reported financial results for the first quarter ended March 31, 2024, and provided a corporate update (Press release, TScan Therapeutics, MAY 13, 2024, View Source [SID1234643160]).

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"During the first quarter we advanced our clinical pipeline, most recently marked by the dosing of the first patient in our Phase 1 solid tumor program. This is a significant milestone on the way to developing enhanced, customized, multiplex TCR-T therapy. We continue to prioritize screening patients for this study to enable rapid enrollment, and I am pleased to announce that we are on track to sharing initial data later this year," said Gavin MacBeath, Ph.D., Chief Executive Officer. "At the same time, we remain focused on enrolling and following patients in our heme malignancies study. We plan to complete Phase 1 enrollment and open expansion cohorts at the proposed recommended Phase 2 dose in the third quarter of 2024 and provide a data update near the end of 2024. With the recent closing of our public offering, TScan is well-funded to execute on anticipated milestones into the fourth quarter of 2026."

Recent Corporate Highlights

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The Company recently announced that the first patient has been dosed in its Phase 1 clinical trial evaluating TCR-T therapy for the treatment of various solid tumors. The Company is initially dosing patients with singleplex therapy to establish safety prior to initiating dosing with multiplex TCR-T (T-Plex). Patients are prospectively assigned to a treatment cohort based on expression of cancer-associated antigens and human leukocyte antigens (HLAs) in their tumor samples. The first patient, who has metastatic melanoma, was dosed with TSC-203-A0201, a TCR-T targeting PReferentially expressed Antigen in MElanoma (PRAME) on HLA-A*02:01. The Company expects robust patient enrollment as over 60 patients have completed all biomarker testing in the screening protocol. Of these patients, approximately 55% qualify for at least one TCR-T in the ImmunoBank and approximately 30% could qualify for multiplex therapy.

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The Company recently provided an update on its Phase 1 heme malignancies program designed to treat residual disease and prevent relapse in patients with acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), or myelodysplastic syndromes (MDS). The update included additional follow-up on all eight treatment-arm patients as well as data on two additional control-arm patients. With a median follow-up of >10 months, all eight patients treated with TSC-100 or TSC-101 remain relapse-free with no detectable disease. No dose-limiting toxicities were observed. In contrast, two control-arm patients relapsed approximately six months post-transplant and one of these patients died approximately three months later. A third control-arm patient required clinical intervention because of concerns of impending relapse, and a fourth control-arm patient died post-transplant.

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In April 2024, the Company announced the closing of an upsized $167.8 million underwritten public offering with participation from new and existing high-quality healthcare investors. The financing extends the Company’s cash runaway into the fourth quarter of 2026. The Company intends to use the net proceeds to

advance its heme and solid tumor programs and expand and optimize its manufacturing capabilities, as well as for general corporate purposes.

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The Company announced two upcoming poster presentations at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting being held May 31–June 4 both in Chicago and virtually. Details on the respective presentations can be found here.

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The Company recently presented at two major medical meetings:

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American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 27th Annual Meeting
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American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2024

Presentation materials from the meetings can be found on the "Publications" tab of TScan’s website at tscan.com.

Upcoming Anticipated Milestones

Heme Malignancies Program: TScan’s two lead TCR-T therapy candidates, TSC-100 and TSC-101, are designed to treat residual disease and prevent relapse in patients with AML, ALL, or MDS undergoing allogeneic hematopoietic cell transplantation (HCT) (NCT05473910).

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Opening of expansion cohorts at the proposed recommended Phase 2 dose level to further characterize safety and evaluate translational and efficacy endpoints is planned for the third quarter of 2024.
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Completion of Phase 1 enrollment and reporting of one-year clinical and translational data on initial patients is anticipated in the second half of 2024.
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Expects to initiate registration trial pending feedback from regulatory authorities and report two-year clinical and translational data in 2025.

Solid Tumor Program: TScan continues to expand the ImmunoBank, a collection of therapeutic TCR-Ts that target different cancer-associated antigens presented on diverse HLA types. TScan’s strategy is to treat patients with multiple TCR-Ts to overcome tumor heterogeneity and prevent resistance that may arise from either target or HLA loss (screening protocol: NCT05812027; treatment protocol: NCT05973487).

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Phase 1 solid tumor clinical study has been initiated; first patient dosed in early May, with three additional patients enrolled and manufacturing underway.
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Initial data expected in the second half of 2024.
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Additional investigational new drug (IND) filings planned to continue to expand the ImmunoBank.
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Long-term duration of response data for multiplex therapy anticipated in 2025.

First Quarter 2024 Financial Results

Revenue: Revenue for the first quarter of 2024 was $0.6 million, compared to $6.8 million for the first quarter of 2023. The decrease was primarily due to the timing of research activities pursuant to the Company’s collaboration agreements. Revenue for the first quarter of 2024 was related solely to the collaboration agreement with Amgen which commenced in May 2023. Revenue for the first quarter of 2023 was related solely to the collaboration agreement with Novartis, which concluded in March 2023.

R&D Expenses: Research and development expenses for the first quarter of 2024 were $24.9 million, compared to $21.8 million for the first quarter of 2023. The increase of $3.1 million was primarily driven by an increase in personnel expenses due to additional headcount in support of expanded research and development activities, as well as an increase in clinical studies expense associated with the ongoing enrollment of our Phase 1 heme study and start-up activities for the Phase 1 solid tumor clinical trial. Research and development expenses included non-cash stock compensation expense of $1.1 million and $0.4 million for the first quarter of 2024 and 2023, respectively.

G&A Expenses: General and administrative expenses for the first quarter of 2024 were $7.1 million, compared to $7.8 million for the first quarter of 2023. The decrease of $0.7 million was primarily driven by lower facility-related and personnel costs. General and administrative expenses included non-cash stock compensation expense of $0.9 million and $0.7 million for the first quarter of 2024 and 2023, respectively.

Net Loss: Net loss was $30.1 million for the first quarter of 2024, compared to $22.6 million for the first quarter of 2023, and included net interest income of $1.2 million and $0.2 million, respectively.

Cash Position: Cash, cash equivalents, and marketable securities as of March 31, 2024, were $162.8 million, excluding $5.0 million of restricted cash. The Company believes that its existing cash resources along with the $161.4 million net proceeds received from its April 2024 underwritten public offering will be sufficient to fund its current operating plan into the fourth quarter of 2026.

Share Count: As of March 31, 2024, the Company had issued and outstanding shares of 47,904,737, which consists of 43,628,149 shares of voting common stock and 4,276,588 shares of non-voting common stock, and outstanding pre-funded warrants to purchase 47,010,526 shares of voting common stock at an exercise price of $0.0001 per share.

Subsequent to the end of the first quarter of 2024, in connection with its April 2024 underwritten public offering, the Company issued an additional 4,958,068 shares of voting common stock, and pre-funded warrants to purchase up to 18,577,419 shares of voting common stock with an exercise price of $0.0001 per share.

On May 13, 2024 Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a fully-integrated biopharmaceutical company with marketed products and a pipeline of development candidates, reported financial results for the first quarter ended March 31, 2024, and provided an overview of recent operational highlights (Press release, TONIX Pharmaceuticals, MAY 13, 2024, View Source [SID1234643159]).

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"Our near-term priority continues to be the submission of our New Drug Application (NDA) for Tonmya (cyclobenzaprine HCl sublingual tablets) for the management of fibromyalgia, while continuing to build out our commercial strategy for the anticipated product launch in the event of FDA approval, which we currently estimate to occur in the second half of 2025," said Seth Lederman, M.D., Chief Executive Officer of Tonix.

Dr. Lederman added, "The well-known treatment-limiting side effects of the three currently approved drugs have led to widespread patient dissatisfaction, creating what we believe is a significant opportunity for a new therapeutic. Tonmya has a differentiated mechanism of action and is generally free of common side effects associated with the currently approved products, including weight gain, fatigue, insomnia, increased blood pressure, gastrointestinal issues or sexual dysfunction. As such, we believe Tonmya, if approved, could become the treatment of choice for the approximately 10 million people in the U.S. suffering the debilitating effects of fibromyalgia."

The Company is also advancing other key pipeline programs including those for immunology, obesity, eating disorders, infectious and rare diseases, many through a capital efficient strategy involving partnerships, grants and in-kind contributions.

Recent Highlights – Key Product Candidates*

Central Nervous System (CNS) Pipeline

Tonmya (also known as TNX-102 SL; cyclobenzaprine HCl sublingual tablets): a centrally-acting, non-opioid, small molecule analgesic taken once-daily at bedtime for the management of fibromyalgia (FM).

In January 2024, Tonix presented additional safety and tolerability data from the pivotal Phase 3 RESILIENT study that showed Tonmya treatment was not associated with increases in systolic or diastolic blood pressure or body weight, nor were there any reported sexual side effects. The Company had previously announced in December 2023 that the Phase 3 RESILIENT study, a registration-quality, double-blind, placebo-controlled study evaluating Tonyma met its pre-specified primary endpoint in the second of two positive Phase 3 clinical trials, significantly reducing daily pain compared to placebo (p-value=0.00005) in participants with fibromyalgia. Statistically significant and clinically meaningful results were also seen in all pre-specified key secondary endpoints including those related to improving sleep quality, reducing fatigue, and improving patient global ratings and overall fibromyalgia symptoms and function. Tonmya was well tolerated with an adverse event profile comparable to prior studies and no new safety signals observed. In addition, Tonmya therapy showed activity on improving female sexual function relative to placebo with a nominal p-value=0.010 by the Changes in Sexual Functioning Questionnaire short-form, female version.
Tonix plans to submit an NDA to the FDA in the second half of 2024 for Tonmya for the management of fibromyalgia. In February 2024, Tonix announced the engagement of Rho, Inc. as our contract research organization (CRO) to support NDA submission.
In February 2024, Tonix announced statistically significant results from its clinical pharmacokinetic (PK) bridging study of Tonmya in healthy adult male and female ethnic Japanese and Chinese volunteers. Results indicate that key PK parameters of cyclobenzaprine are comparable in ethnic Japanese and Chinese volunteers to Caucasian volunteers from a prior PK study. Tonmya was generally well tolerated in the ethnic Japanese and Chinese healthy volunteers. The company expects these data to fulfill the requirement for a bridging study, and enables Tonix to rely on Phase 3 studies RESILIENT and RELIEF results to support regulatory filings for clinical studies in Japan and China where cyclobenzaprine is a new chemical entity (NCE). Tonix holds issued patents for market exclusivity rights of Tonmya in Japan, China, Hong Kong and Taiwan.
In March 2024, Tonix announced the selection of two contract manufacturing organizations (CMOs), including Almac Pharma Services, as dual supply sources for the potential launch and commercialization of Tonmya in the U.S.
In March 2024, Tonix selected EVERSANA, a leading provider of commercialization services to the global life sciences industry, to support the launch strategy and commercial planning of Tonmya in the U.S.
Tonix presented additional efficacy data from RESILIENT at the 6th International Congress on Controversies in Fibromyalgia in Brussels, Belgium, March 7-8, 2024. The data showed that Tonmya treatment resulted in an improvement in cognitive dysfunction, or ‘brain fog’, measured by the change in the Fibromyalgia Impact Questionnaire-Revised (FIQ-R) memory item. The FIQ-R cognitive item showed nominal improvement in Tonmya-treated patients vs placebo-treated patients with a nominal p-value=0.001 and effect size of 0.31.
TNX-102 SL for the treatment of acute stress reaction (ASR) and acute stress disorder (ASD), and prophylaxis against development of posttraumatic stress disorder (PTSD)

In February 2024, the Company announced the FDA cleared the Investigational New Drug (IND) application for the Phase 2 investigator-initiated OASIS trial to evaluate TNX-102 SL in reducing the severity of ASR and the frequency of ASD and PTSD. The trial is sponsored by the University of North Carolina Institute for Trauma Recovery and supported by a $3 million grant from the U.S. Department of Defense, which was awarded in September 2023. The proposed Phase 2, Optimizing Acute Stress Reaction Interventions with TNX-102 SL (OASIS) study will examine the safety and efficacy of TNX-102 SL to reduce adverse posttraumatic neuropsychiatric sequelae among patients presenting to the emergency department (ED) after a motor vehicle collision. The study will enroll approximately 180 trauma survivors at ED study sites in the U.S. Participants will be randomized in the ED to receive a two-week course of either TNX-102 SL 5.6 mg or placebo.
Tonix anticipates the Phase 2 OASIS trial will initiate in the second quarter of 2024.
TNX-102 SL for the treatment of Fibromyalgia-Type Long COVID, also known as Post-Acute Sequelae of COVID-19 (PASC)

In January 2024, the Company announced the online publication of a research paper in the Journal Pain. The article titled, "Chronic Overlapping Pain Conditions Increase the Risk of Long COVID Features, Regardless of Acute COVID Status," by Bergmans, et al.1, found that patients with pre-existing chronic overlapping pain conditions (COPCs) had an increased risk of being diagnosed with symptoms of Long COVID1. Faculty at the University of Michigan directed the research. Commentary on the article titled, "A step towards better understanding chronic overlapping pain conditions" by Fitzcharles, et al,2 is in the same issue of the journal. COPCs include fibromyalgia, chronic fatigue syndrome, migraine headache, irritable bowel syndrome, endometriosis and low back pain. These results contribute to a growing body of evidence that common symptoms of Long COVID in many patients are at least partly driven by central nervous system mechanisms.
TNX-1300 (recombinant double mutant cocaine esterase): biologic for life-threatening cocaine intoxication

Tonix expects to initiate a Phase 2 clinical study of TNX-1300 for the treatment of cocaine intoxication in emergency rooms in the second quarter of 2024. In 2022, Tonix was awarded a Cooperative Agreement grant from the National Institutes of Health (NIH)’s National Institute of Drug Abuse (NIDA) to support development of TNX-1300.
TNX-1300 has been granted Breakthrough Therapy designation by the FDA.
TNX-1900 (intranasal potentiated oxytocin): small peptide in development through investigator-initiated studies for adolescent obesity, binge eating disorder, bone health in autism and social anxiety disorder (SAD).

TNX-1900 continues to be studied in four ongoing investigator-initiated Phase 2 studies as follows: Massachusetts General Hospital (MGH): (1) Phase 2 study for binge-eating disorder (BED); (2) Phase 2 study for adolescent obesity; (3) Phase 2 study for improving bone health in children with autism spectrum disorder (BOX); and at University of Washington, (4) Phase 2 study for social anxiety disorder (SAD). The BED study and the adolescent obesity study will investigate whether TNX-1900 has effects on eating behaviors in specialized populations.
Rare Disease Pipeline

TNX-2900 (intranasal potentiated oxytocin): small peptide for the treatment of Prader-Willi syndrome (PWS)

In March 2024, Tonix announced that it received Rare Pediatric Disease designation from the FDA for TNX-2900 for the treatment of PWS. Tonix has an IND to support clinical development of TNX-2900 to treat PWS in children and adolescents. The planned Phase 2 study is a dose-finding study involving approximately 36 PWS patients divided into four groups with approximately nine per group. One group will receive placebo and three groups will receive different dosage regimens of TNX-2900. TNX-2900 for the treatment of PWS was granted Orphan Drug designation by the FDA in 2022. PWS is a genetic disorder that affects several body systems, with cognitive and behavioral symptoms including pathological over-eating beginning in childhood and leading to severe metabolic sequelae.
Immunology Pipeline

TNX-1500 (anti-CD40L Fc-modified humanized monoclonal antibody): third generation anti-CD40L monoclonal antibody for prophylaxis of organ transplant rejection and treatment of autoimmune disorders.

The first proposed indication for TNX-1500 is prophylaxis of organ rejection in adult patients receiving a kidney transplant; but multiple additional indications are possible, including autoimmune diseases. Two peer reviewed publications described the work with TNX-1500 at the Massachusetts General Hospital (MGH) on allogeneic transplants in animals.3,4
Preclinical studies have shown that TNX-1500 maintains the activity of first-generation monoclonal antibodies (mAbs), yet with reduced risk of thrombotic complications.3-5 Modeling studies from animal pharmacokinetic data3 predict a half-life of greater than three weeks for TNX-1500 in humans, which supports a monthly i.v. dosing regimen. This analysis together with TNX-1500’s activity and tolerability in animals, suggests that the protein engineering of TNX-1500’s Fc region has achieved its design goals.
In February 2024, Tonix announced the completion of the clinical stage of its Phase 1 single ascending dose study of TNX-1500 in healthy volunteers. The primary objectives of the study are to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of intravenous TNX-1500. This first-in-human study is intended to support dosing in a planned Phase 2 trial in kidney transplant recipients.
In March of 2024, the MGH announced the first transplant of a genetically modified pig kidney into a living patient in collaboration with eGenesis, which produced the pig donors and used an anti-CD40L mAb from another company.5 Some of the pre-clinical work that supported the living human transplant was performed in collaboration with Tonix and used TNX-1500.6 The patient was able to return home after the transplant, but died after approximately two months.7
Marketed Products – Recent Highlights

As of April 1, 2024, Tonix completed the transition to becoming a fully integrated pharmaceutical company. Tonix Pharmaceuticals has implemented personnel, systems and contracts required to support a commercial organization and has assumed responsibility for distribution, selling and marketing of Zembrace SymTouch and Tosymra, as well as supply chain, regulatory and quality control of the two products.
Facilities – Recent Highlights

In the fourth quarter of 2023, Tonix engaged CBRE, an international real estate brokerage firm, to potentially find a strategic partner for, or buyer of, its Advanced Development Center (ADC) to align with the Company’s current business objectives and priorities. At this time, the Company does not have a commitment in place to sell the building. ADC, located in the New Bedford business park in Dartmouth, Massachusetts, is an approximately 45,000 square foot BSL-2 facility intended for clinical scale manufacturing of live-virus vaccines and biologics.
*All of Tonix’s product candidates are investigational new drugs or biologics and none have been approved for any indication.

Tonmya is conditionally accepted by the U.S. Food and Drug Administration (FDA) as the tradename for TNX-102 SL for the management of fibromyalgia. Tonmya has not been approved for any indication.

1 Bergmans RS, et al. PAIN. 2023. DOI: 10.1097/j.pain.0000000000003110.

2 Fitzcharles M-A, et al. PAIN. 2023. DOI: 10.1097/j.pain.0000000000003129.

3 Lassiter G., et al. Am J Transplantation. 2023. View Source

4 Miura S., et al. Am J Transplantation. 2023. View Source

5 Massachusetts General Hospital press release. March 21, 2024. "World’s First Genetically Edited Pig Kidney Transplant into Living Recipient Performed at Massachusetts General Hospital." www.massgeneral.org/news/press-release/worlds-first-genetically-edited-pig-kidney-transplant-into-living-recipient (accessed March 29, 2024)

6 Anand, R.P., et al Nature. 622, 393–401 (2023). View Source

7 Stoico, N. Boston Globe. May 11, 2023. "Mass Man who received first kidney transplant from genetically engineered pig has died, family says".

Recent Highlights – Financial

As of March 31, 2024, Tonix had $7.0 million of cash and cash equivalents, compared to $24.9 million as of December 31, 2023. Net cash used in operations was approximately $17.6 million for first quarter 2024, compared to net cash used in operations of $32.9 million for the same period in 2023.

On April 1, 2024, the Company closed a financing with existing healthcare-focused institutional investors for upfront gross proceeds of approximately $4.4 million through a registered direct offering.

First Quarter 2024 Financial Results

Net product revenue for the first quarter 2024 was approximately $2.5 million. Net product revenue consisted of combined net sales of Zembrace SymTouch and Tosymra, which were acquired from Upsher-Smith Laboratories, LLC on June 30, 2023. Cost of Sales for the first quarter 2024 was approximately $1.7 million.

Research and development expenses for the first quarter 2024 were $12.9 million, compared to $26.5 million for the same period in 2023. This decrease is predominantly due to decreased clinical, non-clinical and manufacturing expenses.

General and administrative expenses for the first quarter 2024 were $9.3 million, compared to $7.4 million for the same period in 2023. The increase was primarily due to sales and marketing and the transition services expenses associated with the Company’s recently acquired marketed products offset by a decrease in financial reporting expenses.

Net loss was $(14.9) million, or $(0.18) per share, basic and diluted, for the first quarter 2024, compared to net loss of $(33.0) million, or $(3.21) per share, basic and diluted, for the same period in 2023. The basic and diluted weighted average common shares outstanding for the first quarter 2024 was 80,879,108 compared to 10,268,500 shares for the same period in 2023.

On May 13, 2024 Think Bioscience ("Think"), a Boulder-based synthetic biology company focused on developing small-molecule therapeutics that target "undruggable" proteins, reported a $6M Seed Expansion (Press release, Think Bioscience, MAY 13, 2024, View Source;utm_medium=rss&utm_campaign=think-bioscience-extends-seed-round-to-accelerate-pocket-finding-platform [SID1234643158]). Existing investors are joined by YK Bioventures, bringing total funding to $26M. The additional raise will accelerate the company’s pipeline and expand their unique approach for finding new functional pockets on challenging targets.

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Ultimately, the biochemical properties of any small-molecule drug are constrained by its binding site, a pocket on its protein target. Despite advances in computational chemistry and structural biology, new functional pockets remain challenging to find. Think’s pocket-finding process programs microbes to build small molecules that bind to functional pockets, and they use these pockets to find hits in drug-like space. They have extended their platform technology to a striking variety of targets such as protein tyrosine phosphatases (PTPs), kinases (PTKs), proteases, and GTPases.

"Nature has endowed living systems with an extraordinary ability to build potent bioactive compounds, usually to fulfill important ecological functions, such as defense," said Dr. Jerome Fox, co-founder and CEO of Think Bioscience. "We are using a similar selection process to discover new functional pockets on challenging drug targets. New pockets are gold."

Gary Yeung, a managing partner at YK Bioventures, added, "We invested in Think Bioscience because of its transformational platform for new discoveries. Think Bio has already identified multiple novel pockets and generated strong pre-clinical data for its lead programs. We are eager to support Think Bio’s science-driven leadership team to advance these programs and to fund its growth."

Dr. Wendy Young will join the Board of Directors, which includes fellow veteran drug developer Nick Saccomano. Dr. Young has held leadership positions in biopharma for over 30 years and most recently spent 15 years at Genentech where she served as Senior Vice President of Small Molecule Drug Discovery and co-led research. She currently serves as a SAB member to Think, as well as several other biotech companies, and is an advisor at Google Ventures. "We look forward to working even more closely with Wendy," said Dr. Jerome Fox, co-founder and CEO at Think Bioscience. "Wendy and Nick bring a wealth of drug development expertise. Their guidance will be invaluable as we expand our platform to new classes of important targets and push pipeline programs toward the clinic."

On May 13, 2024 Terns Pharmaceuticals, Inc. ("Terns" or the "Company") (Nasdaq: TERN), a clinical stage biopharmaceutical company developing a portfolio of small molecule product candidates to address serious diseases, including oncology and obesity, reported financial results for the first quarter ended March 31, 2024 and provided corporate updates (Press release, Terns Pharmaceuticals, MAY 13, 2024, View Source [SID1234643157]).

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"We continue to execute our strategy and make important progress on our pipeline of potential best-in-class small molecule therapies as we prepare for key data readouts from our two lead programs," said Amy Burroughs, chief executive officer of Terns, "We look forward to reporting both interim dose escalation data from the ongoing Phase 1 CARDINAL trial of TERN-701 in CML and top-line data from the Phase 1 trial of TERN-601 in obesity in the second half of this year."

"We are particularly pleased with the recent findings from our Phase 1 study of TERN-701 in healthy volunteers, which showed lack of food effect and supports once-daily dosing. This represents a key potential differentiator as the only approved allosteric BCR-ABL inhibitor requires three hours of fasting with each dose and twice-daily dosing in multiple clinical settings," added Ms. Burroughs.

Recent Pipeline Developments and Anticipated Milestones

TERN-701: Oral, allosteric BCR-ABL inhibitor for chronic myeloid leukemia (CML)

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Terns’ Phase 1 CARDINAL trial of TERN-701 in CML is ongoing and interim data from initial CARDINAL dose escalation cohorts are expected in the second half of 2024
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CARDINAL is a global, multicenter, open-label, two-part Phase 1 clinical trial to evaluate the safety, pharmacokinetics (PK), and efficacy of TERN-701 in patients with previously treated CML
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In April, Terns announced findings from a concurrent Phase 1 PK study of TERN-701 in U.S. healthy volunteers, which indicated TERN-701 can be administered once-daily (QD) with or without food at doses that achieve clinically efficacious exposures
•
In March 2024, the United States Food and Drug Administration (FDA) granted Orphan Drug Designation for TERN-701 for the treatment of CML
•
Terns plans to host a TERN-701-focused virtual key opinion leader (KOL) event in mid-2024

TERN-601: Oral, small molecule glucagon-like peptide-1 (GLP-1) receptor agonist for obesity

•
Phase 1 first-in-human clinical trial of Terns’ lead oral GLP-1 receptor agonist in obese and overweight participants is progressing
•
The multiple ascending dose (MAD) portion of the study is underway, testing once-daily administration of TERN-601, and is on track to report top-line 28-day weight loss data in the second half of 2024
•
Preliminary safety findings from the ongoing, blinded Phase 1 SAD/MAD study have been unremarkable to date with no observations of liver enzyme elevations, drug induced liver injury or discontinuations due to treatment-related adverse events

TERN-501: Oral, thyroid hormone receptor-beta (THR-β) agonist

•
Terns continues to evaluate opportunities for TERN-501 in metabolic diseases
o
Based on non-clinical studies, THR-β is an orthogonal mechanism to GLP-1, potentially providing broader metabolic and liver benefits in addition to increased weight loss
o
Non-clinical data suggests that TERN-501 may augment the weight loss effects of a GLP-1 receptor agonist, as demonstrated in a diet-induced obese mouse model

TERN-800 Series: Oral, small molecule glucose-dependent insulinotropic polypeptide receptor (GIPR) modulators

•
Discovery efforts are ongoing for small molecule GIPR modulators for obesity, which have the potential for combination with GLP-1 receptor agonists, such as TERN-601
•
Terns is prioritizing its discovery efforts on nominating a GIPR antagonist development candidate based on in-house discoveries and growing scientific rationale supporting the potential of GLP-1 agonist/GIPR antagonist combinations for obesity

Corporate Updates

•
In April 2024, Terns announced the appointment of Melita Sun Jung as chief business officer of Terns
•
In May 2024, Terns announced the appointment of Scott Harris as chief development officer and the upcoming departure of Erin Quirk, M.D., president, head of research and development

First Quarter 2024 Financial Results

Cash Position: As of March 31, 2024, cash, cash equivalents and marketable securities were $240.7 million, as compared with $263.4 million as of December 31, 2023. Based on its current operating plan, Terns expects these funds will be sufficient to support its planned operating expenses into 2026.

Research and Development (R&D) Expenses: R&D expenses were $18.6 million for the quarter ended March 31, 2024, as compared with $17.1 million for the quarter ended March 31, 2023.

General and Administrative (G&A) Expenses: G&A expenses were $6.9 million for the quarter ended March 31, 2024, as compared with $7.1 million for the quarter ended March 31, 2023.

Net Loss: Net loss was $22.4 million for the quarter ended March 31, 2024, as compared with $21.5 million for the quarter ended March 31, 2023.

Financial Tables

Terns Pharmaceuticals, Inc.

Condensed Consolidated Statements of Operations

(Unaudited; in thousands except share and per share amounts)

Three Months Ended March 31,

2024

2023

Operating expenses:

Research and development

$

18,587

$

17,056

General and administrative

6,859

7,101

Total operating expenses

25,446

24,157

Loss from operations

(25,446

)

(24,157

)

Interest income

3,182

2,693

Other expense, net

(12

)

(4

)

Loss before income taxes

(22,276

)

(21,468

)

Income tax expense

(97

)

(60

)

Net loss

$

(22,373

)

$

(21,528

)

Net loss per share, basic and diluted

$

(0.30

)

$

(0.31

)

Weighted average common stock outstanding, basic and diluted

74,399,378

69,778,420

Terns Pharmaceuticals, Inc.

Selected Balance Sheet Data

(Unaudited; in thousands)

March 31, 2024

December 31, 2023

Cash, cash equivalents and marketable securities

$

240,654

$

263,440

Total assets

246,766

268,517

Total liabilities

10,046

13,150

Total stockholders’ equity

236,720

255,367