On May 13, 2020 Equillium, Inc. (Nasdaq: EQ), a clinical-stage biotechnology company leveraging deep understanding of immunobiology to develop products to treat severe autoimmune and inflammatory disorders, reported financial results for the first quarter 2020 (Press release, Equillium, MAY 13, 2020, View Source [SID1234557903]).

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"We continue to advance our EQUATE clinical trial for itolizumab in patients with acute graft-versus-host disease (aGVHD), an acute life-threatening disease that remains a medical priority in the midst of the coronavirus pandemic," said Bruce Steel, chief executive officer of Equillium. "Our EQUIP and EQUALISE trials in uncontrolled asthma and lupus nephritis, respectively, remain paused as healthcare professionals have prioritized resources around the pandemic. We continue to monitor the situation closely to assess the feasibility of recommencing these trials."

Business Highlights:

Strengthened the leadership team with the appointments of Maple Fung, M.D., as vice president of clinical development and Matthew Ritter, Ph.D., as vice president of corporate development.

Presented translational data supporting the potential of itolizumab in the treatment of graft-versus-host disease (GVHD) at the Transplantation & Cellular Therapy (TCT) Meeting.

Entered into a common stock purchase agreement for up to $15 million with Lincoln Park Capital Fund, LLC. With this potential funding source and Equillium’s cash and cash equivalents at the end of the first quarter 2020, Equillium believes it has sufficient resources to support operations into the second half of 2021.

Upcoming Catalysts:

Initial data from the Phase 1b part of the EQUATE trial in aGVHD expected in the second half of 2020

First Quarter 2020 Financial Results

Research and development (R&D) expenses. Total R&D expenses for the three months ended March 31, 2020 were $4.7 million, compared with $3.8 million for the same period in 2019. The increase in R&D expenses was primarily driven by the initiation and ramp-up of clinical development activities associated with the EQUIP, EQUATE and EQUALISE clinical trials and increased headcount expenses offset by slight decreases in expenses related to preclinical research activities and general overhead expenses.

General and administrative (G&A) expenses. Total G&A expenses for the three months ended March 31, 2020 were $2.7 million, compared with $2.6 million for the same period in 2019. The increase in G&A expenses was primarily driven by increased non-cash stock based compensation expense and consulting expenses, offset by slight decreases in legal and other professional fees.

Net loss. Net loss for the three months ended March 31, 2020 was $7.8 million, or $(0.45) per basic and diluted share, compared with a net loss of $6.0 million, or $(0.34) per basic and diluted share for the same period in 2019.

Cash, cash equivalents and short-term investments. Equillium held cash, cash equivalents and short-term investments totaling $47.7 million at March 31, 2020, compared to $53.1 million at December 31, 2019.

On May 13, 2020 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported that three abstracts highlighting its drug candidate tipifarnib have been accepted for presentation at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Virtual Scientific Program, to be held May 29-31, 2020 (Press release, Kura Oncology, MAY 13, 2020, View Source [SID1234557919]).

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"We are pleased to announce the presentation of three clinical abstracts for tipifarnib, including an oral presentation highlighting single-agent activity in multiple solid tumors with HRAS mutations," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "In addition, the oral presentation will feature compelling overall survival data from our RUN-HN Phase 2 trial in head and neck squamous cell carcinoma (HNSCC) patients with high HRAS mutant variant allele frequency, and we look forward to sharing those data at ASCO (Free ASCO Whitepaper) later this month."

The three abstracts are listed below and are now available on the ASCO (Free ASCO Whitepaper) meeting website at View Source

Preliminary activity of tipifarnib in tumors of the head and neck, salivary gland and urothelial tract with HRAS mutations
Oral Abstract Session: Head and Neck Cancer
Abstract: 6504

The AIM-HN and SEQ-HN Study: A Pivotal Study Evaluating the Efficacy of Tipifarnib in Patients with Head and Neck Squamous Cell Carcinoma (HNSCC) with HRAS Mutations (AIM-HN) and the Impact of HRAS Mutations on Response to First Line Systemic Therapies for HNSCC (SEQ-HN)
Poster Session: Head and Neck Cancer
Abstract / Poster: TPS6593 / 254

Tipifarnib, a farnesyltransferase inhibitor, for metastatic urothelial carcinoma harboring HRAS mutations
Poster Session: Genitourinary Cancer—Kidney and Bladder
Abstract / Poster: 5086 / 155

Copies of the presentations will be available on Kura’s website at www.kuraoncology.com/pipeline/publications/ following presentation at the meeting.

About Tipifarnib

Tipifarnib, is a potent, selective and orally bioavailable inhibitor of farnesyl transferase in-licensed from Janssen in December 2014. Previously, tipifarnib was studied in more than 5,000 cancer patients and showed compelling and durable anti-cancer activity in certain patient subsets; however, no molecular mechanism of action had been determined that could explain its clinical activity across a range of solid tumor and hematologic indications. Leveraging advances in next generation sequencing as well as emerging information about cancer genetics and tumor biology, the Company is seeking to identify those patients most likely to benefit from tipifarnib. Tipifarnib has been granted Fast Track designation by the U.S. Food and Drug Administration for the treatment of patients with HRAS mutant HNSCC. Kura has received multiple issued patents for tipifarnib, providing patent exclusivity in the U.S. and foreign countries.

On May 13, 2020 Tarveda Therapeutics, Inc., a clinical stage biopharmaceutical company developing a new class of potent and selective precision oncology medicines, which it refers to as Pentarin miniature drug conjugates, reported the complete data from the Phase 1 dose escalation portion of a Phase 1/2a study of PEN-866 in advanced solid tumor malignancies (Press release, Tarveda Therapeutics, MAY 13, 2020, View Source [SID1234557936]). PEN-866 was well-tolerated, demonstrated a high therapeutic index, and showed evidence of anti-tumor activity across multiple tumors and dose ranges (wide therapeutic range), including one partial response. The data also confirm the favorable pharmacokinetic profile of PEN-866 and its ability to engage with its intended target and concentrate delivery of its toxic SN-38 payload intra-tumorally.

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The full results of the Phase 1 portion of the Phase 1/2a study will be presented at the American Society for Clinical Oncology’s ASCO (Free ASCO Whitepaper)20 Virtual Scientific Program in a presentation titled, "PEN-866, a Miniature Drug Conjugate of a Heat Shock Protein 90 (HSP90) Ligand Linked to SN38 for Patients with Advanced Solid Malignancies: Phase 1 and Expansion Cohort Results". Along with the Sarah Cannon Research Institute, the National Cancer Institute (NCI) at the National Institutes of Health (NIH), Stephenson Cancer Center at the University of Oklahoma, and Tennessee Oncology are also participating as trial sites. NCI investigators, Drs. Anish Thomas, MBBS, M.D., and Yves Pommier, M.D., Ph.D., Developmental Therapeutics Branch, Center for Cancer Research, NCI, are collaborating with Tarveda through a clinical Cooperative Research and Development Agreement (CRADA).

"The full data from the Phase 1 portion of the PEN-866 Phase 1/2a study show that through binding to HSP90, which is upregulated and activated in solid tumors, PEN-866 accumulates and is retained in solid tumors where it releases its toxic payload, as designed," said Gerald Falchook, Sarah Cannon Research Institute. "PEN-866 was well tolerated and demonstrated a strong therapeutic index across multiple tumor types. We were also highly encouraged by the anti-tumor activity in this advanced patient population, including several patients experiencing prolonged stable disease and one experiencing a partial response."

"The results seen from the Phase 1 study of PEN-866 show the potential not just of PEN-866 itself but of Tarveda’s HSP90 binding miniature drug conjugate platform," said Jeffrey Bloss, M.D., Chief Medical Officer of Tarveda. "Results from this trial confirm that PEN-866 works as designed and successfully concentrated its potent SN-38 anti-tumor payload in the tumor. We will be progressing with further evaluations of PEN-866 both as a monotherapy in patients with a range of solid tumor malignancies in a Phase 2a trial, as well as in combination with cytotoxic chemotherapy and other targeted agents such as DDR, immuno-oncology drugs and key pathway inhibitors. We are also building out our HSP90 binding miniature drug conjugate platform by exploring and developing new HSP90 binding miniature drug conjugates with promising anti-cancer payloads."

PEN-866 is an HSP90 binding miniature drug conjugate that preferentially binds to activated HSP90 in solid tumors and is linked to the topoisomerase 1 inhibitor SN-38, a potent anti-cancer payload. PEN-866 is designed to accumulate and be retained in solid tumors while clearing rapidly from plasma intact over 24 to 48 hours. As the SN-38 payload is cleaved in the tumor over time, the sustained release of SN-38 in the tumor results in prolonged DNA damage and tumor regressions as demonstrated in multiple patient-derived and cancer cell line xenograft models. PEN-866 is the first miniature drug conjugate from Tarveda’s HSP90 binding drug conjugate platform.

Phase 1 Trial Design

In this dose escalating Phase 1 portion of the trial, 30 patients were enrolled in seven dose levels. Two to seven patients with advanced solid tumor malignancies were treated in each cohort. Patients received escalating doses of PEN-866 weekly for three out of four weeks in a 28-day cycle. Patients in cohorts 1-5 were treated with flat dosing and patients in cohorts 6-7 were switched to body surface area dosing based on emerging data indicating variable exposure in patients treated with flat doses.

Safety Data

Results of the study show that PEN-866 was well tolerated with no dose limiting toxicities (DLTs) in the first six cohorts (15 mg/m2 – 175 mg/m2). Three DLTs were observed above the Maximum Tolerated Dose (MTD) which was 175 mg/m2. One fatal event of dehydration occurred 11 days following the last dose of PEN-866 at the dose level above the MTD. The most frequent adverse events observed were nausea, fatigue, diarrhea, vomiting, and alopecia and the most common Grade 3 adverse event was neutropenia. The neutropenia seen at the MTD or below was uncomplicated, did not require growth factors for patients to recover, and did not result in missed or delayed doses. The recommended Phase 2 dose for PEN-866 monotherapy was determined to be 175 mg/m2.

Efficacy Data

Antitumor activity was observed across multiple tumor types and at a wide range of doses. One patient (3.8%) of 26 evaluable patients per RECIST v1.1 achieved partial response (PR) and 11 patients (42.3%) experienced stable disease (SD). Patients who experienced clinical benefit include:

One patient with anal squamous carcinoma had a PR, approximately a 50% reduction in tumor size within four cycles (duration of response > 11 months)
Three patients with pancreatic cancer had prolonged SD (8 months, 5 months and 4.5 months, respectively)
One patient with liposarcoma had prolonged SD (>12 months)
One patient with acinar cell cancer of the pancreas remained on therapy for over a year with SD
ASCO20 Virtual Scientific Program Presentation

Full results of the study will be presented at the ASCO (Free ASCO Whitepaper)20 Virtual Scientific Program. Details of the poster presentation are as follows:

Title: PEN-866, a Miniature Drug Conjugate of a Heat Shock Protein 90 (HSP90) Ligand Linked to SN-38 for Patients with Advanced Solid Malignancies: Phase 1 and Expansion Cohort Results
Date: Friday, May 29, 2020
Time: 8:00 AM ET
Location: ASCO (Free ASCO Whitepaper)20 Virtual Scientific Program accessible at View Source

Additional information on the Phase 1/2a clinical trial for PEN-866 is available at clinicaltrials.gov, through identifier number NCT03221400.

On May 13, 2020 Polynoma LLC, a U.S. immuno-oncology focused biopharmaceutical company and wholly-owned subsidiary of Hong Kong-listed CK Life Sciences Int’l., (Holdings) Inc., reported final analysis of clinical data from Part B1 of MAVIS (Melanoma Antigen Vaccine Immunotherapy Study), a Phase III study of seviprotimut-L, an investigational melanoma vaccine candidate, at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Virtual Scientific Program, to be held online May 29-31, 2020 (Press release, Polynoma, MAY 13, 2020, View Source [SID1234557952]). The study abstract is one of 12 abstracts selected for discussion in the Melanoma/Skin Cancers poster discussion session.

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MAVIS is a multicenter, double-blind, placebo-controlled adaptive Phase III trial to assess the safety and efficacy of seviprotimut-L, with primary endpoints of recurrence-free survival (RFS) and overall survival (OS) in patients with American Joint Committee on Cancer (AJCC) Stage IIB/C, IIIA, IIIB/C melanoma at high risk of recurrence after definitive surgical resection.

Highlights of the data presented include:

Improved outcomes in Stage IIB/C patients: Final analysis of subgroups confirmed the findings from the interim analysis, suggesting enhanced RFS for seviprotimut-L in patients with AJCC Stage IIB/IIC melanoma, particularly those under age 60, and those with ulceration, whose lesions are considered more serious because they have a greater risk of metastasis.1
Early evidence of survival benefit in Stage IIB/C patients: For Stage IIB/IIC melanoma patients under 60, there was a trend toward improved overall survival for those treated with seviprotimut-L.
Favorable adverse event profile: Seviprotimut-L was well-tolerated with treatment-emergent adverse events (AEs) similar to patients given placebo. There were no treatment-related serious adverse events.
Melanoma is the most diagnosed cancer among 25 to 29 year-olds in the United States, and passage from Stage II to Stage III melanoma marks a critical therapeutic intervention point to improve survival. Treatment of Stage IIB/IIC melanoma is primarily limited to surgery, coupled with a "wait and see" approach. However, recurrence of the disease can occur following definitive resection of the melanoma. Many patients progress to more advanced stages following resection and 5-year survival rates fall sharply after a patient passes from localized Stage II melanoma into regional Stage III disease (98.4% to 63.6%). Five-year survival rates are distinctly lower (22.5%) for metastatic Stage IV.2

"The final analysis of this part of the study reinforces the findings from our interim analysis last year, suggesting improved outcomes with seviprotimut-L in Stage IIB/IIC melanoma patients, particularly in those aged under 60. Furthermore, the latest findings extend the benefit to include disease with ulceration," said Melvin Toh, Chief Technology Officer at Polynoma and Vice President & Chief Scientific Officer at CK Life Sciences. "With a median patient follow-up of more than 48 months, the data show a durable RFS clinical benefit of seviprotimut-L in Stage IIB/IIC melanoma. We believe seviprotimut-L will be an important new option for the adjuvant treatment of patients with localized melanoma and look forward to advancing seviprotimut-L into the definitive part of the MAVIS Study."

"These data show promise for seviprotimut-L as a vaccine-based treatment of melanoma," said Craig L. Slingluff Jr., MD, Professor of Surgery and Director of the Human Immune Therapy Center and co-leader of the Cancer Therapeutics Program of the UVA Cancer Center. "These findings support moving forward with a pivotal trial testing seviprotimut-L as an adjuvant treatment for patients with Stage IIB/C melanoma, with stratification by age."

FURTHER DETAILS ON POSTER PRESENTATION AND DISCUSSION SESSION:

Abstract 10017: Final analysis of relapse-free survival in a multicenter, double-blind, placebo-controlled trial of seviprotimut-L polyvalent melanoma vaccine after resection of high-risk melanoma.

Poster: 366
Authors: Craig L. Slingluff, Jr., MD; Brent A. Blumenstein, PhD; Karl D. Lewis, MD; Robert H. Andtbacka, MD, CM, FACS, FRCSC; John Hyngstrom, MD; Mohammed Milhem, MBBS; Svetomir N. Markovic, MD, PhD; Omid Hamid, MD; Leonel Hernandez-Aya, MD PhD; Tawnya L. Bowles, MD; Prejesh Philips, MD; Sekwon Jang, MD; Jose Lutzky, MD, FACP; Anna Bar, MD; Peter D. Beitsch, MD
Poster Discussion Session

Session Title: Melanoma/Skin Cancers
Presentation Title: Adjuvant/Neoadjuvant Approaches
Discussant: Kenneth F. Grossmann, MD, PhD | Huntsman Cancer Institute, University of Utah
Poster and Discussion: will be available "on demand" on the ASCO (Free ASCO Whitepaper) website starting May 29 at 8:00 am EDT
The data being presented assessed the treatment effects of seviprotimut-L in patients with AJCCv7 Stage IIB-III cutaneous melanoma after surgical resection. 347 patients ages 18-75, ECOG PS 0-1, were enrolled and randomized 2:1 to seviprotimut-L 40 mcg or placebo, administered intradermally every 2 weeks x 5, then monthly x 4, then every 3 months to month 24. Patients were stratified by Stage (IIB/C, IIIA, IIIB/C).

By intent-to-treat (ITT) analysis, RFS was not significantly enhanced for seviprotimut-L in the full study population but trended toward benefit (Hazard Ratio "HR" = 0.88). Subgroup analysis based on planned stratification revealed the HR for the Stage IIB/IIC subset to be 0.65 (number of patients, "N" =111, 95% CI [0.37, 1.17]), favoring seviprotimut-L.

Age has been identified as a cause of decreased immune competence;3 thus, outcomes were assessed as a function of age as an effect modifier. Final efficacy analysis of subgroups confirmed treatment with seviprotimut-L enhanced RFS for patients less than 60 years overall (N=191, HR=0.64, 95% CI [0.38, 1.08]) and among Stage IIB/IIC melanoma patients (N=52, HR=0.32, 95% CI [0.12, 0.86]).

Furthermore, in a multivariable RFS model, for Stage IIB/IIC patients less than 60 years with ulceration, the HR was 0.209 (N=38, 95% CI [0.07,0.61]). For OS, for patients less than 60, HR = 0.41 [0.33,1.14] (n=191, 19 deaths) and for those ≥60, HR = 0.92 [0.39,2.12] (n = 156, 24 deaths).

In the study, seviprotimut-L was well-tolerated with treatment-emergent adverse events (AEs) similar to placebo patients. There were no treatment-related serious adverse events (SAEs) in the 347 patients studied, and the vast majority of events were Grade 1-2 injection site reactions that were managed by topical cream/s or an over-the-counter antihistamine.

About MAVIS
MAVIS (Melanoma Antigen Vaccine Immunotherapy Study) is a multicenter, double-blind, placebo-controlled adaptive Phase III trial to assess the safety and efficacy of seviprotimut-L, with primary endpoints of recurrence-free survival (RFS) and overall survival (OS) in patients with melanoma at high risk of recurrence after definitive surgical resection. MAVIS is being conducted under a Special Protocol Assessment (SPA) agreement with the FDA. For additional information about the trial, please visit View Source

About Seviprotimut-L
Seviprotimut-L is an allogeneic, polyvalent, partially purified shed melanoma antigen vaccine derived from three proprietary human melanoma cell lines. Seviprotimut-L stimulates humoral and cellular immune responses. Melanoma-associated antigens (MAAs) found in seviprotimut-L are taken up by antigen-presenting cells (e.g., dendritic cells) which then activate the production of antigen-specific cytotoxic T-lymphocytes (CTLs) as well as develop antibody responses against MAAs. These CTLs and antibodies then recognize and act on tumor cells expressing the MAAs on their surfaces, causing cell death. Seviprotimut-L is currently in development for the adjuvant treatment of patients with Stages IIB to IIIC melanoma, following definitive resection.

On May 13, 2020 HTG Molecular Diagnostics, Inc. (Nasdaq: HTGM) (HTG), a life science company whose mission is to advance precision medicine, reported its financial results for the first quarter ended March 31, 2020.

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"With the COVID-19 pandemic impacting businesses globally, the first quarter of 2020 was unlike anything we would have anticipated. As work from home restrictions directly impacted our customers and their normal ordering patterns, we felt the immediate impact," said John Lubniewski, President and CEO of HTG. "However, we have managed to take advantage of this time to leverage our teams, improve our processes, and accomplish detailed long-term planning all in preparation of emerging from this period as a stronger company. While we cannot be certain of the ultimate impact of the COVID-19 pandemic on us or our customers, we are optimistic that demand for our products and services will return."

Mr. Lubniewski continued, "Our product development teams have made progress towards the key milestones that we had set for ourselves during the quarter. Our California and Arizona development teams continue to implement our development strategy and we are on track to deliver development milestones. While the pandemic has created some near-term uncertainty, we are very proud of how our employees have faced the challenges – with flexibility and a commitment to ensuring our long-term success. When our customers eventually return to a more normal work environment, we will be prepared to meet demand and for growth in our business."

First Quarter 2020 Financial Highlights:

Total revenue for the first quarter ended March 31, 2020 was $2.2 million, compared with $3.2 million for the same period in 2019. The decrease in revenue is a result of the impact of the COVID-19 pandemic requiring the closure of customer facilities around the globe. These closures limited the company’s ability to deliver its products to customer facilities and created challenges for customers in preparing and shipping samples to the company for processing.

Product and product-related services revenue was $2.0 million, compared with $2.7 million for the same period in 2019. This decrease in direct revenue primarily reflects a decline in lower margin, subcontracted laboratory services revenue reflected in RUO sample processing revenue when compared with first quarter of 2019 revenue.

Collaborative development services revenue for the quarter ended March 31, 2020 was $0.2 million compared with $0.5 million for the same period in 2019. The decrease in collaborative development services revenue reflects a decrease in activity as partners approached key decision points in current active programs.

Net loss from operations for the first quarter ended March 31, 2020 was $5.4 million, compared with $5.3 million for the first quarter of 2019. Net loss per share was $(0.08) for the first quarter of 2020 compared with $(0.19) for the first quarter of 2019.

Cash, cash equivalents and short-term available-for-sale securities totaled $32.0 million as of March 31, 2020, with current liabilities of approximately $8.4 million and non-current liabilities of $11.6 million. An additional $3.3 million of restricted cash was held in connection with a convertible note that is included in current liabilities as of March 31, 2020.

Conference Call and Webcast:

HTG will host a conference call for the investment community today beginning at 4:30 p.m. Eastern Time. Conference call and webcast details are as follows: