On November 3, 2021 NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology platform company Drugging the Genome to address disease at the base level using a new class of precision genetic medicines, reported that Dietrich A. Stephan, Ph.D., Chief Executive Officer of NeuBase, will present in person a corporate overview at the Jefferies London Healthcare Conference being held November 16 – 18 (Press release, NeuBase Therapeutics, NOV 3, 2021, View Source [SID1234594220]).

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Jefferies London Healthcare Conference
Date: Wednesday, November 17
Time: 8:40 a.m. – 9:15 a.m. GMT
Location: Webcast Link

On November 3, 2021 Gritstone bio, Inc. (Nasdaq: GRTS), a clinical-stage biotechnology company developing next generation cancer and infectious disease immunotherapies, reported financial results for the third quarter ended September 30, 2021 and reviewed business highlights (Press release, Gritstone Oncology, NOV 3, 2021, View Source [SID1234594219]).

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"We are pleased with our strengthened cash position and the significant clinical progress achieved during this third quarter," said Andrew Allen, M.D., Ph.D., Co-Founder, President and Chief Executive Officer of Gritstone. "Our leadership in neoantigen-derived immunotherapy and vaccination strategies for cancer and infectious diseases has been validated by several key partnerships and was further supported by a $55 million dollar PIPE financing led by Frazier Life Sciences Public Fund, enabling the rapid clinical development of our second-generation mRNA and integrated vaccine manufacturing capabilities. The data generated to date using both heterologous and homologous prime-boost approaches with our two vectors further validate the vast potential of our platform to deliver broad and durable immune responses across infectious diseases and common, solid cancers. We look forward to upcoming CORAL milestones, including imminent non-human primate viral challenge data from our collaboration with NIAID and Phase 1 data from our company-sponsored trial evaluating CORAL as a boost and immunogenicity enhancer for first-generation COVID-19 vaccines in older adults early in the first quarter of 2022."

Corporate Highlights

Entered into a funding agreement of up to $20.6 million with the Coalition for Epidemic Preparedness Innovations (CEPI) to advance the clinical development of Gritstone’s CORAL second generation mRNA COVID-19 vaccine program, including a clinical trial in South Africa and manufacturing optimization (CEPI Press Release, August 2021)

Received gross proceeds of $55.0 million from a private investment in public equity (PIPE) financing led by Frazier Life Sciences Public Fund, with additional participation from Redmile Group and Gilead Sciences (PIPE Press Release, September 2021)
Clinical Highlights and Updates

Tumor-Specific Neoantigen (TSNA) Oncology Programs

GRANITE – Individualized, TSNA-directed immunotherapy using an adenoviral priming vector and self-amplifying mRNA boost vector

Updated Phase 1/2 results evaluating the safety, immunogenicity and clinical activity of GRANITE individualized neoantigen immunotherapy in combination with PD-1 checkpoint inhibitor nivolumab and subcutaneous anti-CTLA-4 antibody ipilimumab in advanced solid tumors presented at ESMO (Free ESMO Whitepaper) 2021 (GRANITE Press Release, September 2021)
GRANITE immunotherapy demonstrated a 44% molecular response rate (4/9) as measured by >50% reduction in circulating tumor DNA (ctDNA) with associated median overall survival >17 months in patients with end-stage microsatellite-stable colorectal cancer (MSS-CRC) for whom median overall survival is typically 6-7 months (as observed in the 5/9 patients without molecular response)
A Phase 2/3 randomized, controlled trial of maintenance GRANITE immunotherapy in newly diagnosed metastatic, MSS-CRC patients is expected to initiate in the first quarter of 2022
This Phase 2/3 trial has registrational intent and has been discussed with the FDA
A Phase 2 randomized trial of adjuvant GRANITE immunotherapy in MSS-CRC patients with stage II/III disease who are ctDNA+ after definitive surgery is expected to initiate in the second quarter of 2022
SLATE – "Off-the-shelf" shared neoantigen-directed immunotherapy using an adenoviral priming vector and self-amplifying mRNA boost vector

Presented Phase 1/2 data demonstrating SLATE v1 + nivolumab + ipilimumab in 26 patients with metastatic solid tumors
Observed multiple molecular responses and an unconfirmed RECIST radiologic response were observed in patients with metastatic NSCLC, all of whom had progressed on prior (chemo)immunotherapy (SLATE Press Release, September 2021)
SLATE v2, a mutant KRAS-focused version optimized for increased immune response, is currently being evaluated in a Phase 2 trial in patients with NSCLC and MSS-CRC. Initial clinical data are anticipated by mid-2022.
Infectious Disease Programs

CORAL – second-generation SARS-CoV-2 vaccine program delivering both spike and highly conserved non-spike T cell epitopes within either a self-amplifying mRNA vector or an optimized chimpanzee adenoviral vector (ChAdV). This approach offers potential for more durable clinical protection and broader immunity against SARS-CoV-2 variants than 1st generation products by inducing potent CD8+ T cells in addition to neutralizing antibody responses

Dosed first volunteer in a company-sponsored Phase 1 trial evaluating GRT-R910, a self-amplifying mRNA (SAM) second generation SARS-CoV-2 vaccine designed to boost and expand the immunogenicity of first-generation COVID-19 vaccines in subjects 60 years of age or older. (GRT-R910 Press Release, September 2021)
Initial Phase 1 data from this study are expected early in the first quarter 2022
Our collaboration with the National Institute of Allergy and Infectious Disease (NIAID) to evaluate the safety, tolerability and immunogenicity of investigational ChAdV and SAM SARS-CoV-2 vaccines continues:
Non-human primate viral challenge data are expected in November 2021
Preliminary results from a Phase 1 trial are now expected in the first half of 2022 due to protocol amendments necessitated by the changing landscape of vaccine use in the United States
Third Quarter 2021 Financial Results

Cash, cash equivalents. marketable securities and restricted cash were $216.4 million as of September 30, 2021 compared to $172.1 million as of December 31, 2020.
Research and development expenses were $24.4 million for the three months ended September 30, 2021, compared to $22.1 million for the three months ended September 30, 2020. The increase was primarily due to increases of $1.7 million in personnel-related expenses and $2.4 million in outside services, offset by $1.8 million decreases in laboratory supplies.
General and administrative expenses were $6.4 million for the three months ended September 30, 2021, compared to $5.0 million for the three months ended September 30, 2020. The increase was primarily attributable to increases in personnel-related expenses and outside services.

Collaboration revenue was $2.4 million for the three months ended September 30, 2021, compared to $0.8 million for the three months ended September 30, 2020. The increase was due to the collaboration revenue associated with the collaboration agreement with Gilead from January 2021.

On November 3, 2021 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of drugs to treat severe metabolic, oncologic and neuropsychiatric disorders by modulating the effects of the hormone cortisol, reported its results for the quarter ended September 30, 2021 (Press release, Corcept Therapeutics, NOV 3, 2021, https://ir.corcept.com/news-releases/news-release-details/corcept-therapeutics-announces-third-quarter-financial-results-0 [SID1234594218]).

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Financial Results

Revenue of $96.1 million, an 11 percent increase from third quarter 2020
GAAP diluted net income of $0.24 per share, compared to $0.17 per share in third quarter 2020
Non-GAAP diluted net income of $0.30 per share, compared to $0.24 per share in third quarter 2020
Cash and investments of $495.2 million, compared to $471.6 million at June 30, 2021
Tightening of 2021 revenue guidance to $365 – $375 million
"The strong growth of our commercial business in the third quarter reflects the continued easing of COVID-related public health restrictions," said Joseph K. Belanoff, MD, Corcept’s Chief Executive Officer. "Physicians are seeing their patients more frequently and, as a result, are better able to diagnose and treat patients with Cushing’s syndrome. As pandemic conditions recede, we expect our growth to continue. Korlym is an excellent treatment for Cushing’s syndrome and there are many eligible patients who have yet to receive it."

Corcept’s third quarter 2021 revenue was $96.1 million, compared to $86.3 million in the third quarter of 2020. Third quarter 2021 GAAP net income was $30.5 million, compared to $21.6 million in the third quarter of 2020. Excluding non-cash expenses related to stock-based compensation and the utilization of deferred tax assets, together with related income tax effects, non-GAAP net income in the third quarter was $37.0 million, compared to $30.0 million in the third quarter of 2020. A reconciliation of GAAP to non-GAAP net income is included below.

Corcept narrowed its 2021 revenue guidance to $365 – $375 million. The company’s previous guidance was $355 – $385 million.

Third quarter operating expenses were $59.9 million, compared to $61.6 million in the third quarter of 2020, primarily due to the conclusion of our studies in ovarian and pancreatic cancer, partially offset by increased employee compensation expenses, commercial spending and increased preclinical activities.

Cash and investments of $495.2 million at September 30, 2021 reflects the acquisition in the third quarter of $28.0 million of Corcept common stock – 1.2 million shares pursuant to the company’s stock repurchase program and 0.2 million shares in connection with the exercise of employee stock options. Over the term of the stock repurchase program, which began in November 2020, Corcept acquired 4.3 million shares of common stock at a cost of $98.2 million.

Clinical Development

"Cortisol modulation has the potential to help treat many serious diseases," said Dr. Belanoff. "Currently, our clinical programs are evaluating treatments for patients with solid tumors, non-alcoholic steatohepatitis (NASH), antipsychotic-induced weight gain (AIWG) and Cushing’s syndrome. We also continue to advance new cortisol modulators to the clinic. Early next year, we plan to start a Phase 2 trial in patients with amyotrophic lateral sclerosis (ALS)."

Solid Tumors

Phase 3 trial in patients with recurrent platinum-resistant ovarian cancer planned to start
in the first quarter of 2022; updated overall survival data from the Phase 2 trial expected in the first quarter of 2022
Selection of the optimum dose of exicorilant plus enzalutamide in patients with castration-resistant prostate cancer (CRPC) expected later this year
Enrollment continues in 20-patient, open-label, Phase 1b trial of relacorilant plus PD-1 checkpoint inhibitor pembrolizumab in patients with adrenal cancer with cortisol excess
"Leading gynecological oncologists have given us extremely positive feedback regarding the statistically significant and clinically meaningful results of our 178-patient, randomized, controlled Phase 2 trial in patients with recurrent platinum-resistant ovarian cancer and our plans to initiate a Phase 3 trial," said Bill Guyer, PharmD, Corcept’s Chief Development Officer. "We and our investigators are excited to advance relacorilant for the potential treatment of ovarian cancer and plan to meet with the FDA in the coming months to arrive at the best path forward."

Metabolic Diseases

Initiated Phase 1b dose-finding trial in patients with presumed NASH; results of our Phase 2 study to be presented at the American Association for the Study of Liver Diseases (AASLD) meeting, November 12 – 15
Completion of enrollment in GRATITUDE II, a 150-patient, double-blind, placebo-controlled Phase 2 trial of miricorilant to reverse long-standing AIWG, expected by year-end
Completion of enrollment in GRATITUDE, a 100-patient double-blind, placebo-controlled Phase 2 trial of miricorilant to reverse recent AIWG, expected by mid-2022
"At the AASLD meeting this month, we will present results from our Phase 2 trial, in which patients with presumed-NASH who received miricorilant experienced exceptionally large and rapid reductions in liver fat, accompanied by substantial, but transient, elevations of the liver enzymes ALT and AST," said Dr. Guyer. "Our hypothesis is that the rapidity and magnitude of miricorilant’s fat reducing effect irritated the liver. The objective of our Phase 1b study, initiated last month, is to identify a dosing regimen that can produce significant reductions in fat without causing liver irritation."

Cushing’s Syndrome

Enrollment continues in Phase 3 GRACE trial of relacorilant as a treatment for patients with all etiologies of Cushing’s syndrome; new drug application (NDA) submission expected in the second quarter of 2023
Enrollment continues in Phase 3 GRADIENT trial of relacorilant as a treatment for patients with Cushing’s syndrome caused by adrenal adenomas
"We expect our GRACE trial, which is accruing patients and generating data, to serve as the basis for relacorilant’s NDA in Cushing’s syndrome, and we are on track to make this submission in the second quarter of 2023," said Dr. Guyer. "The Phase 3 GRADIENT trial will produce valuable data about an etiology of Cushing’s syndrome that affects many patients, but has not been subject to rigorous, controlled study."

Conference Call

We will hold a conference call on November 3, 2021, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time). To participate, Click this link (listen-only mode) or dial 1-833-693-0540 from the United States or 1-661-407-1581 internationally approximately 15 minutes before the start of the call. The passcode will be 5763926. A replay will be available on the Investors / Past Events tab of our website.

Hypercortisolism

Hypercortisolism, often referred to as Cushing’s syndrome, is caused by excessive activity of the hormone cortisol. Endogenous Cushing’s syndrome is an orphan disease that most often affects adults aged 20-50. In the United States, an estimated 20,000 patients have Cushing’s syndrome, with about 3,000 new patients diagnosed each year. Symptoms vary, but most patients experience one or more of the following manifestations: high blood sugar, diabetes, high blood pressure, upper-body obesity, rounded face, increased fat around the neck, thinning arms and legs, severe fatigue and weak muscles. Irritability, anxiety, cognitive disturbances and depression are also common. Hypercortisolism can affect every organ system and can be lethal if not treated effectively. Corcept holds patents covering the composition of relacorilant and the use of cortisol modulators, including Korlym, in the treatment of patients with hypercortisolism.

On November 3, 2021 ChromaDex Corp. (NASDAQ:CDXC) reported financial results for the third quarter of 2021 (Press release, ChromaDex, NOV 3, 2021, View Source [SID1234594217]).

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Third Quarter 2021 and Recent Highlights

Total net sales were $17.3 million, up 22% from the prior year quarter.
Tru Niagen net sales were $14.8 million, a 24% increase from the prior year quarter.
Gross margin was 61.1%, a 150 basis point increase from the prior year quarter.
Net loss was $(8.9) million or $(0.13) per share, a decline of $0.06 per share from the prior year quarter.
Adjusted EBITDA excluding total legal expense, a non-GAAP measure, was a loss of $(0.6) million, a $0.5 million decline from the prior year quarter.
Announced partnership with Sinopharm Xingsha, a subsidiary of one of China’s largest pharmaceutical companies, to conduct cross-border sales of Tru Niagen in Mainland China, and collaborate to secure Health Food Registration.
Study published in September 2021 on nicotinamide riboside (NR) supplementation in children with Ataxia-Telangiectasia (AT), a prematurely aging population, showed improvement in ataxia scores and increased antibodies in immune-compromised patients.
Clinical study published in October 2021 in Molecular Systems Biology finds nutritional protocol including nicotinamide riboside (NR) significantly decreases liver fat and improves liver function.
"This quarter marks an inflection point for ChromaDex," said CEO, Rob Fried. "With the litigation largely behind us, we are focusing more resources, both human and financial, on building the Tru Niagen brand, furthering our science, and developing our global partnerships. We recently partnered with Sinopharm Xingsha, one of the most prestigious healthcare companies in China, which will enable us to capitalize on the tremendous opportunity for Tru Niagen in that market."

Results of operations for the three months ended September 30, 2021

For the three months ended September 30, 2021 ("Q3 2021"), ChromaDex reported net sales of $17.3 million, up $3.1 million or 22% compared to the third quarter of 2020 ("Q3 2020"). The increase in Q3 2021 revenues was largely driven by growth in sales of Tru Niagen paired with slight growth in Niagen and other ingredient sales.

Gross margin percentage improved by 150 basis points to 61.1% in Q3 2021 compared to 59.6% in Q3 2020. The improvement in gross margin percentage was driven by the positive impact of increased Tru Niagen consumer product sales and product cost savings initiatives.

Operating expenses increased by $6.8 million to $19.4 million in Q3 2021, compared to $12.7 million in Q3 2020. The increase in operating expenses was driven by $2.0 million of higher selling and marketing expenses and a $4.6 million increase in general and administrative expense. The increase in general and administrative expense was primarily driven by $3.7 million of higher legal expense.

The net loss for Q3 2021 was $(8.9) million or $(0.13) per share as reported compared to a net loss of $(4.2) million or $(0.07) per share for Q3 2020 as reported. Adjusted EBITDA excluding total legal expense, a non-GAAP measure, was a loss of $(0.6) million for Q3 2021, compared to a loss of $(0.1) million for Q3 2020, a $0.5 million decline. See "Reconciliation of Non-GAAP Financial Measures" for a reconciliation of non-GAAP Adjusted EBITDA excluding total legal expense to net loss, the most directly comparable GAAP measure.

For Q3 2021, the net cash outflow from operating activities was $(5.9) million, compared to $(3.8) million in Q3 2020.

2021 Full Year Outlook

Looking forward, for the full year, the Company expects continued, steady revenue growth driven by its global e-commerce business, as well as growth with existing and new strategic partners. The Company expects slightly better than 60% gross margin and slightly higher general and administrative expense, excluding severance, restructuring and legal expense, for full year 2021. The Company plans to increase investments and resources to drive brand awareness and accelerate its research and development pipeline to capitalize on growth in the nicotinamide adenine dinucleotide (NAD+) market globally. Accordingly, the Company expects higher research and development expense and higher selling and marketing expense as a percentage of net sales year-over-year.

Investor Conference Call

A live webcast will be held Wednesday, November 3, 2021 at 4:30 p.m. Eastern time (1:30 p.m. Pacific time) to discuss ChromaDex’s third-quarter financial results and provide a general business update.

To listen to the webcast, or to view the earnings press release and its accompanying financial exhibits, please visit the Investors Relations section of ChromaDex’s website at View Source The toll-free dial-in information for this call is 1-888-510-2008 with Conference ID: 4126168.

The webcast will be recorded, and will be available for replay via the website from 7:30 p.m. Eastern time on November 3, 2021 to 11:59 p.m. Eastern time on November 10, 2021. The replay of the call can also be accessed by dialing 800-770-2030, using the Replay ID: 4126168.

On November 3, 2021 TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted cancer therapeutics and utilizing a cost efficient, CRO-independent product development platform to partner with ex-U.S. companies to develop and commercialize innovative products in the U.S., reported financial results for the third quarter ended September 30, 2021 (Press release, Tracon Pharmaceuticals, NOV 3, 2021, View Source [SID1234594216]). The Company will host a conference call and webcast today at 4:30 PM Eastern Time / 1:30 PM Pacific Time.

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"We have once again utilized our CRO-independent Product Development Platform to augment our pipeline. The license of the potential best-in-class CTLA-4 antibody YH001 complements our prior license of the subcutaneously administered PD-L1 antibody envafolimab and satisfies our goal of owning complementary checkpoint inhibitors. We expect to initiate a trial combining these two drug candidates in sarcoma in the coming months," said Charles Theuer, M.D., Ph.D., President and CEO of TRACON. "In the meantime, ENVASARC continues to enroll well at 26 sites, and we look forward to releasing interim efficacy data before the end of the year."

Recent Corporate Highlights

In October, TRACON licensed the CTLA-4 antibody YH001 from Eucure Biopharma. The Company expects to file an IND and initiate a Phase 1/2 trial of YH001 combined with envafolimab in soft tissue sarcoma subtypes in early 2022, and initiate trials of YH001 in other indications in combination with approved standard of care agents in late 2022.

In September, the book Unnecessary Expense: An Antidote to the Billion Dollar Drug Problem, authored by TRACON senior management and profiling TRACON’s approach to efficient drug development, was published by ForbesBooks and is available on Amazon and other retailers.
Expected Key Upcoming Milestones

Interim ENVASARC efficacy data by end of 2021.

Request FDA breakthrough therapy designation or fast track designation for envafolimab by end of 2021, assuming positive interim efficacy data.

Decision on the envafolimab New Drug Application submitted by our partners 3D Medicines and Alphamab Oncology, in MSI-H/dMMR cancer that is under priority review by the Chinese National Medical Products Administration.
Third Quarter 2021 Financial Results

Cash, cash equivalents and short-term investments were $29.9 million at September 30, 2021, compared to $36.1 million at December 31, 2020.

Research and development expenses for the third quarter of 2021 were $2.7 million, compared to $1.8 million for the third quarter of 2020.

General and administrative expenses for the third quarter of 2021 were $4.2 million, compared to $2.1 million for the third quarter of 2020. The increase was primarily attributable to legal expenses incurred due to the ongoing arbitration with I-Mab related to the TJ4309 and bispecific antibody agreements.

Net loss for the third quarter of 2021 was $7.0 million, compared to $4.0 million for the third quarter of 2020.
Conference Call Details

Wednesday, November 3, at 4:30 PM Eastern Time / 1:30 PM Pacific Time
Domestic: 855-779-9066
International: 631-485-4859
Conference ID: 4846079
A live webcast of the conference call will be available online from the Investor/Events and Presentations 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 novel, single-domain antibody against PD-L1, is the first subcutaneously injected PD-(L)1 inhibitor to be studied in pivotal trials. Envafolimab is currently being studied in the ENVASARC Phase 2 pivotal trial in the U.S. sponsored by TRACON, as well as in a Phase 2 pivotal trial as a single agent in MSI-H/dMMR advanced solid tumor patients and a Phase 3 pivotal trial in combination with gemcitabine and oxaliplatin in advanced biliary tract cancer patients in China sponsored by TRACON’s corporate partners, Alphamab Oncology and 3D Medicines. Alphamab Oncology and 3D Medicines submitted an NDA to the NMPA in China for envafolimab in MSI-H/dMMR cancer that was accepted for review in December 2020 and granted priority review in January 2021. In the Phase 2 MSI-H/dMMR advanced solid tumor trial, the confirmed objective response rate (ORR) by blinded independent central review in MSI-H/dMMR colorectal cancer (CRC) patients treated with envafolimab who failed a fluoropyrimidine, oxaliplatin and irinotecan was 32%, which was similar to the 28% confirmed ORR reported in the Opdivo package insert in MSI-H/dMMR CRC patients who failed a fluoropyrimidine, oxaliplatin, and irinotecan and the 33% confirmed ORR reported for Keytruda in MSI-H/dMMR CRC patients who failed a fluoropyrimidine, oxaliplatin and irinotecan in cohort A of KEYNOTE-164.

About ENVASARC (NCT04480502)

The ENVASARC pivotal trial is a multicenter, open label, randomized, non-comparative, parallel cohort study at approximately 25 top cancer centers in the United States that began dosing in December 2020. TRACON expects the trial to enroll 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 cohort A of treatment with single agent envafolimab and 80 patients enrolled into cohort B of treatment with envafolimab and Yervoy. The primary endpoint is ORR by blinded independent 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 (ADCC) and complement dependent cytotoxicity (CDC) 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 dosed as a single agent in a Phase 1 trial in China (NCT04699929) and in combination with the PD-1 antibody toripalimab in a Phase 1 trial in Australia (NCT04357756).

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 U.S. FDA in malignant glioma, including glioblastoma.

About TJ004309

TJ004309 is a novel, humanized antibody against CD73, an ecto-enzyme expressed on stromal cells and tumors that converts extracellular adenosine monophosphate (AMP) to adenosine, which is highly immunosuppressive. TJ004309 is currently being studied in an ongoing Phase 1 trial to assess safety and preliminary efficacy as a single agent and when combined with the PD-L1 checkpoint inhibitor Tecentriq in patients with advanced solid tumors.