On August 5, 2020 Harpoon Therapeutics, Inc. (Nasdaq: HARP), a clinical-stage immunotherapy company developing a novel class of T cell engagers, reported financial results for the second quarter ended June 30, 2020 and provided a corporate update (Press release, Harpoon Therapeutics, AUG 5, 2020, View Source [SID1234562924]).

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"Our TriTAC T cell engager pipeline continues to advance and we were pleased with the encouraging interim Phase 1 data for our lead program, HPN424, that we presented at the ASCO (Free ASCO Whitepaper)20 Virtual meeting," said Gerald McMahon, Ph.D., President and Chief Executive Officer of Harpoon Therapeutics. "We also announced early data from our second program, HPN536, which has continued to advance in a dose escalation trial for mesothelin malignancies. In addition, we advanced our third clinical program, HPN217, into the clinic which triggered a $50 million milestone payment from AbbVie, and submitted an IND for HPN328, our fourth TriTAC pipeline program. Looking ahead to the second half of 2020, we are preparing to advance HPN328 into the clinic for the treatment of DLL3-expressing tumors including small cell lung cancer."

Second Quarter 2020 Business Highlights and Other Recent Developments

In April, Harpoon announced the first patient was dosed with HPN217 in a Phase 1/2 clinical trial focused on relapsed/refractory multiple myeloma (RRMM). HPN217 is covered by a global development and option agreement with AbbVie Inc. (NYSE: ABBV) and dosing of the first patient in the clinical trial triggered a $50 million milestone payment, which was received in June. HPN217 targets B-cell maturation antigen (BCMA), a well-validated target expressed on multiple myeloma cells. HPN217 is Harpoon’s third product candidate to enter the clinic.

In April, Harpoon appointed Andrew R. Robbins and Joseph S. Bailes, M.D., to its Board of Directors as independent board members. Among his many achievements, Mr. Robbins is credited with leading the highly successful U.S. launch of BRAFTOVI (encorafenib) + MEKTOVI (binimetinib) in BRAF-mutant metastatic melanoma. Dr. Bailes is a medical oncologist with substantial experience in clinical practice, legislation, public policy and advocacy. For nearly two decades, he served in various executive leadership capacities with ASCO (Free ASCO Whitepaper), including as President.

In May, Harpoon presented interim data from the ongoing dose-escalation portion of a Phase 1 trial for HPN424 in patients with metastatic castration-resistant prostate cancer (mCRPC) at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 Virtual Scientific Program. At the time of the data cutoff, 44 patients with progressive mCRPC had been treated in 11 cohorts. Initial safety data showed that HPN424 is generally well tolerated, and cytokine-related adverse events were transient and manageable. Early signals of clinical activity were suggested by multiple patients remaining on study for more than 24 weeks, and several patients with serum PSA declines. Additionally, pharmacokinetic data confirmed half-life extension, which supports the weekly dosing schedule, and pharmacodynamic data supports T cell activation and target engagement, which are consistent with the expected mechanism of action. Patient enrollment continues in the dose escalation phase of the trial in the U.S. and Europe, with a goal to identify a dose for an expansion phase planned for the second half of 2020.

In May, Harpoon provided a corporate pipeline update. In addition to reiterating and expanding upon HPN424 data presented at ASCO (Free ASCO Whitepaper)20, Harpoon provided an update on HPN536, its TriTAC currently being studied for the treatment of mesothelin-expressing tumors. Harpoon highlighted that the dose escalation portion of the study was progressing and, as of May 2020, included 15 ovarian and 10 pancreatic cancer patients. Adverse events were shown to be transient and manageable, and early pharmacokinetic data showed half-life extension supporting once-weekly dosing. In addition, Harpoon also presented advancements in the company’s second platform, ProTriTAC, which builds upon the core elements of the TriTAC platform by utilizing a prodrug approach, designed to allow T cell engagers to address cancer targets that would be limited by on-target toxicities.

In July, Harpoon appointed Joanne Viney, Ph.D., to its board of directors as an independent board member. Dr. Viney is an entrepreneurial scientist and experienced biotech executive with deep autoimmune and inflammatory disease expertise and currently serves as President, CSO and Co-founder of Pandion Therapeutics.

Second Quarter 2020 Financial Results

Harpoon ended the second quarter of 2020 with $175.4 million in cash, cash equivalents, and marketable securities compared to $155.1 million as of December 31, 2019. The increase was due to a $50.0 million milestone payment received from AbbVie, partially offset by cash used in operations.

Revenue for the second quarter ended June 30, 2020 was $2.8 million compared to $1.1 million for the second quarter ended June 30, 2019. For the six months ended June 30, 2020, revenue was $6.1 million compared to $2.1 million for the six months ended June 30, 2019. During both the three and six month periods, the increase in revenue was primarily due to revenue recognized from the upfront payment under the development and option agreement with AbbVie, signed in November 2019.

Research and development (R&D) expense for the second quarter ended June 30, 2020 was $11.9 million compared to $10.0 million for the second quarter ended June 30, 2019. For the six months ended June 30, 2020, R&D expense was $24.4 million, compared to $19.4 million for the six months ended June 30, 2019. The increase for both periods primarily arose from higher clinical development and personnel-related expense, which included conducting preclinical studies and the continuation and preparation of the clinical trials for HPN424, HPN536, HPN217 and HPN328. These higher expenses were offset by a decrease in manufacturing costs due to a scale up of manufacturing activities in 2019 compared to 2020 to support our four TriTAC product candidates in various stages of development.

General and administrative (G&A) expenses for the quarter ended June 30, 2020 was $3.9 million compared to $3.7 million for the quarter ended June 30, 2019. G&A expense for the six months ended June 30, 2020 was $7.9 million compared to $9.6 million for the six months ended June 30, 2019. For the quarter ended June 30, 2020, the increase was primarily attributable to an increase in

personnel expenses due to an increase in headcount, offset by a decrease in legal fees associated with the Maverick Litigation incurred in 2019. For the six months ended June 30, 2020, the decrease was due to higher expenses incurred in 2019 primarily related to legal fees associated with Maverick litigation and consulting and accounting services, partially offset by an increase in personnel expenses related to an increase in headcount and other professional services to support our operations as a public company.

Net loss for the quarter ended June 30, 2020 was $12.7 million compared to $11.8 million for the quarter ended June 30, 2019. The net loss for the six months ended June 30, 2020 was $25.2 million compared to $25.4 million in the first six months of the prior year.

COVID-19 Update

In response to the ongoing COVID19 pandemic, Harpoon’s executive offices remain closed in compliance with county and state shelter-in-place orders, substantially all of the company’s employees continue to telecommute, with only a limited the number of staff working in the company’s laboratory. Harpoon is currently continuing its clinical trials it has underway at sites in the United States, and has not yet experienced any material delays or impacts as a result of the pandemic. In addition, Harpoon’s third-party contract manufacturers continue to operate at or near normal levels and the company does not currently anticipate material interruptions. Harpoon continues to assess the potential impact of the COVID-19 pandemic on its business and operations, including its programs, expected timelines, expenses, manufacturing and clinical trials. The full extent to which the COVID-19 pandemic may have a negative impact on Harpoon’s business, results of operations and financial condition will depend on future developments that are highly uncertain and cannot be accurately predicted.

On August 5, 2020 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 second quarter ended June 30, 2020 (Press release, Tracon Pharmaceuticals, AUG 5, 2020, View Source [SID1234562923]). 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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Recent Corporate Highlights

In July, TRACON filed the ENVASARC pivotal trial protocol with the U.S. Food and Drug Administration (FDA) as part of an Investigational New Drug (IND) application. Following the 30 day FDA review period, TRACON expects to enroll the first patient in the ENVASARC trial evaluating the PD-L1 single domain antibody envafolimab in the soft tissue sarcoma subtypes of undifferentiated pleomorphic sarcoma (UPS) and myxofibrosarcoma (MFS) in the fourth quarter of 2020. Key elements of the ENVASARC pivotal tri
Multi-center, open-label, randomized, non-comparative, parallel cohort study.

Eligible patients will have undifferentiated pleomorphic sarcoma (UPS) or myxofibrosarcoma (MFS) and will have received one or two prior lines of cancer therapy, and no prior immune checkpoint inhibitor therapy.

Planned total enrollment of 160 patients, with 80 patients enrolled into cohort A of treatment with single agent envafolimab and 80 patients enrolled in cohort B of treatment with envafolimab and Yervoy.

Primary endpoint of objective response rate (ORR) with duration of response a key secondary endpoint. Nine of 80 objective responses (11.25% ORR) are required to demonstrate an ORR that is statistically higher than the 4% ORR reported for Votrient, the only approved therapy for refractory UPS/MFS, in its package insert.

Open-label format with blinded independent central review of efficacy endpoint data.

In May, TRACON announced positive results for envafolimab at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Virtual Scientific Program from the Company’s corporate partners, 3D Medicines and Alphamab Oncology, that showed single agent envafolimab demonstrated a 30.0% confirmed ORR in 50 patients with MSI-H/dMMR colorectal cancer (CRC) who failed a fluoropyrimidine, oxaliplatin and irinotecan (n=39) or those with advanced gastric cancer who failed at least one prior systemic treatment (n=11), with at least two on-study tumor assessments. The confirmed ORR in MSI-H/dMMR CRC patients treated with envafolimab who failed a fluoropyrimidine, oxaliplatin and irinotecan was 28.2%, which was nearly identical 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 27.9% confirmed ORR reported for Keytruda in MSI-H/dMMR CRC patients who failed a fluoropyrimidine, oxaliplatin and irinotecan in cohort A of KEYNOTE-164.

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URL: www.traconpharma.com

In May, TRACON highlighted data at the 2020 ASCO (Free ASCO Whitepaper) Virtual Scientific Program from the Alliance for Clinical Trials in Oncology that showed a 29% confirmed ORR in patients (n=14) with highly refractory UPS who received dual checkpoint inhibition with Opdivo in combination with Yervoy. For reference, cohort B of the ENVASARC pivotal trial will enroll UPS/MFS patients who will receive dual checkpoint inhibition with envafolimab with Yervoy.

In May, positive data were presented from multiple TRC102 clinical trials at the 2020 ASCO (Free ASCO Whitepaper) Virtual Scientific Program. In a Phase 2 trial, two of 14 mesothelioma patients who progressed previously on Alimta had objective responses following treatment with Alimta and TRC102. In a Phase 1 trial, TRC102 in combination with chemo-radiation resulted in an objective response in all 15 patients with locally advanced non-squamous non-small cell lung cancer, including three patients who demonstrated a complete response to treatment. The 100% ORR indicates a significant improvement as compared to historical data of chemoradiation without TRC102 in advanced lung cancer.

"We were excited by the ASCO (Free ASCO Whitepaper) data presented by our corporate partners showing that envafolimab activity was comparable to the activity of the approved products Opdivo and Keytruda in separate trials of MSI-H/dMMR CRC. Moreover, we were encouraged by additional data presented at ASCO (Free ASCO Whitepaper) from the Alliance group which showed an impressive response rate for dual checkpoint inhibition with Opdivo and Yervoy in UPS. Importantly, these data provide the rationale for testing dual checkpoint inhibition using envafolimab and Yervoy in the ENVASARC trial," said Charles Theuer, M.D., Ph.D., President and CEO of TRACON. "Achieving a 29% response rate in ENVASARC would represent a marked improvement in the treatment of patients with refractory UPS/MFS, where the only approved therapy demonstrated a 4% response rate. We look forward to enrolling the first patient in ENVASARC later this year and plan to provide interim data in 2021, final data in 2022, and assuming positive clinical data and regulatory approval, potentially commercialize envafolimab in 2023."

Expected Upcoming Milestones

Completion of the 30 day FDA review period for the ENVASARC protocol, a pivotal trial in the sarcoma subtypes of UPS and MFS, that was submitted to the FDA on July 16, 2020.

Enroll the first patient in ENVASARC during the fourth quarter of 2020.

Submission of the envafolimab BLA with the National Medical Products Association in China (NMPA) by our partners, 3D Medicines and Alphamab Oncology.

Report top-line data from the Phase 1 dose escalation study of TJ4309, a CD73 antibody, as a single agent and in combination with Tecentriq (a PD-L1 antibody being supplied by Roche), in the second half of 2020.

Second Quarter 2020 Financial Results

Cash and cash equivalents were $14.5 million at June 30, 2020, compared to $16.4 million at December 31, 2019. We expect our current cash and cash equivalents to fund operations into the second quarter of 2021. If the remaining $11.7 million available under the Aspire Capital agreement as of June 30, 2020 were fully utilized, cash runway would extend late into the third quarter of 2021.

Research and development expenses for the second quarter of 2020 were $2.2 million, compared to $4.3 million for the second quarter of 2019. The decrease was primarily attributable to lower manufacturing expenses and clinical trial expenses due to the discontinuation of the Phase 3 TRC105 program.

General and administrative expenses for the second quarter of 2020 were $2.1 million, compared to $1.9 million for the second quarter of 2019.

Net loss for the second quarter of 2020 was $4.5 million, compared to $6.3 million for the second quarter of 2019.

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 registrational trials. Envafolimab is currently dosing in a Phase 2 registration trial as a single agent in MSI-H/dMMR advanced solid tumor patients and a Phase 3 registration trial in combination with gemcitabine and oxaliplatin in advanced biliary tract cancer patients in China. 3D Medicines and Alphamab Oncology, TRACON’s corporate partners for this program, plan to submit a BLA to the NMPA in China for envafolimab in 2020 based on the ORR in MSI-H/dMMR advanced solid tumor patients. The confirmed ORR in MSI-H/dMMR colorectal cancer patients treated with envafolimab who failed a fluoropyrimidine, oxaliplatin and irinotecan reported at ASCO (Free ASCO Whitepaper) 2020 was 28.2%, which was nearly identical to the 28% confirmed ORR reported in the Opdivo package insert in MSI-H/dMMR colorectal cancer patients who failed a fluoropyrimidine, oxaliplatin, and irinotecan and the 27.9% 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 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 or Case Comprehensive Cancer Center.

About TRC253

TRC253 is a Phase 3 ready novel, orally bioavailable small molecule drug that is a potent, high affinity competitive inhibitor of the androgen receptor (AR) and AR mutations, including the F877L mutation. The AR F877L mutation results in an alteration in the AR ligand binding domain that confers resistance to therapies for prostate cancer. Therapies targeting the AR have demonstrated clinical efficacy by extending time to disease progression, and in some cases, the survival of patients with metastatic castration-resistant prostate cancer. However, resistance to these agents is often observed and several molecular mechanisms of resistance have been identified, including gene amplification, overexpression, alternative splicing, and point mutation of the AR. TRC253 recently completed a Phase 1/2 clinical trial in prostate cancer conducted by TRACON. TRACON believes TRC253 can be developed and commercialized successfully in China and is actively seeking a strategic collaboration.

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 a 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.

On August 5, 2020 Heron Therapeutics, Inc. (Nasdaq: HRTX), a commercial-stage biotechnology company focused on improving the lives of patients by developing best‑in-class treatments to address some of the most important unmet patient needs, reported financial results for the three and six months ended June 30, 2020 and highlighted recent corporate updates (Press release, Heron Therapeutics, AUG 5, 2020, View Source [SID1234562922]).

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Recent Corporate Updates

Pain Management Franchise

Positive CHMP Opinion Received for ZYNRELEF for the Management of Postoperative Pain: In July 2020, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for ZYNRELEF (formerly known as HTX-011), intended for the treatment of postoperative pain. The CHMP’s positive opinion will now be reviewed by the European Commission (EC), with a final decision on the Marketing Authorisation Application expected in the coming months. An EC marketing authorisation through the centralized procedure is valid in all 27 European Union (EU) member countries as well as the European Economic Area countries. The CHMP recommended that ZYNRELEF be indicated for treatment of somatic postoperative pain from small- to medium-sized surgical wounds in adults.

Complete Response Letter Received from the FDA Regarding the NDA for HTX-011 for the Management of Postoperative Pain: In June 2020, Heron received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding the New Drug Application (NDA) for HTX-011. The CRL stated that the FDA is unable to approve the NDA in its present form based on the need for additional non-clinical information. Based on the complete review of the NDA, the FDA did not identify any clinical safety or efficacy issues or chemistry, manufacturing and controls (CMC) issues. There are four non-clinical issues in the CRL, none of which relate to any observed toxicity. Three relate to confirming exposure of excipients in preclinical reproductive toxicology studies, and the fourth relates to changing the manufacturing release specification of the allowable level of an impurity based on animal toxicology coverage. We do not believe that any of the issues are significant barriers to ultimate approval, as all of the excipients have extensive histories of use in pharmaceuticals and the specification can be revised.

Initiation of Phase 1b/2 Clinical Study of HTX-034 for the Treatment of Postoperative Pain: In May 2020, Heron initiated a Phase 1b/2 clinical study in patients undergoing bunionectomy of HTX-034, Heron’s next-generation product for the treatment of postoperative pain. The study initiation followed clearance from the FDA of Heron’s Investigational New Drug (IND) application for HTX-034 for the treatment of postoperative pain.

CINV Franchise

Initiation of Phase 2 Clinical Study of CINVANTI for the Treatment of COVID-19: In July 2020, Heron initiated the GUARDS-1 Study, a Phase 2 clinical study evaluating CINVANTI (aprepitant) injectable emulsion in early hospitalized patients with Coronavirus Disease 2019 (COVID-19). The study initiation followed clearance from the FDA of Heron’s IND application for CINVANTI for the treatment of COVID-19.

CINV Net Product Sales: For the three and six months ended June 30, 2020, chemotherapy‑induced nausea and vomiting (CINV) franchise net product sales were $22.7 million and $48.1 million, respectively, compared to $36.7 million and $68.3 million, respectively, for the same periods in 2019.

CINVANTI Net Product Sales: Net product sales of CINVANTI for the three and six months ended June 30, 2020 were $22.6 million and $47.8 million, respectively, compared to $33.2 million and $61.2 million, respectively, for the same periods in 2019. Heron expects the impact of the generic arbitrage to be resolved in 2020, with a return to growth in 2021 and beyond.

SUSTOL Net Product Sales: Net product sales of SUSTOL (granisetron) extended‑release injection for the three and six months ended June 30, 2020 were $0.1 million and $0.3 million, respectively, compared to $3.5 million and $7.1 million, respectively, for the same periods in 2019. On October 1, 2019, the Company discontinued all discounting of SUSTOL, which resulted in significantly lower SUSTOL net product sales. Heron expects SUSTOL to return to growth in 2021 and beyond.

2020 Net Product Sales Guidance: Although Heron anticipates a decrease in new diagnoses and chemotherapy patient starts because of the onging COVID-19 pandemic, the Company is maintaining its 2020 guidance for net product sales for the CINV franchise of $70 million to $80 million.

"We are pleased with the CHMP’s recent positive opinion for ZYNRELEF in the EU, and we remain committed to bringing this important non-opioid analgesic to patients in the U.S. as soon as possible. We have submitted a request for a Type A meeting with the FDA and look forward to working with the FDA to achieve this goal," said Barry Quart, Pharm.D., President and Chief Executive Officer of Heron. "In our CINV franchise, we are encouraged by the continued performance of CINVANTI during both a generic arbitrage period and the COVID-19 pandemic and are maintaining our 2020 net product sales guidance of $70 million to $80 million."

Financial Results

Net product sales for the three and six months ended June 30, 2020 were $22.7 million and $48.1 million, respectively, compared to $36.7 million and $68.3 million, respectively, for the same periods in 2019.

Heron’s net loss for the three and six months ended June 30, 2020 was $55.2 million and $106.8 million, or $0.61 per share and $1.18 per share, respectively, compared to $50.2 million and $113.2 million, or $0.63 per share and $1.43 per share, respectively, for the same periods in 2019. Net loss for the three and six months ended June 30, 2020 included non-cash, stock-based compensation expense of $11.1 million and $23.1 million, respectively, compared to $12.7 million and $30.6 million, respectively, for the same periods in 2019.

As of June 30, 2020, Heron had cash, cash equivalents and short-term investments of $300.8 million, compared to $391.0 million as of December 31, 2019. Net cash used for operating activities for the six months ended June 30, 2020 was $90.2 million, compared to $72.1 million for the same period in 2019. Heron expects that its current cash, cash equivalents and short-term investments will be sufficient to fund its operations into 2022.

About HTX-011 (ZYNRELEF in the European Union) for Postoperative Pain

HTX-011 (ZYNRELEF in the European Union), an investigational non-opioid analgesic, is a dual-acting, fixed-dose combination of the local anesthetic bupivacaine with a low dose of the nonsteroidal anti-inflammatory drug meloxicam. It is the first and only extended-release local anesthetic to demonstrate in Phase 3 studies significantly reduced pain and opioid use through 72 hours compared to bupivacaine solution, the current standard-of-care local anesthetic for postoperative pain control. HTX-011 was granted Fast Track designation from the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2017 and Breakthrough Therapy designation in the second quarter of 2018. Heron submitted a new drug application (NDA) to the FDA for HTX-011 in October 2018 and received Priority Review designation in December 2018. A complete response letter (CRL) was received from the FDA regarding the NDA for HTX-011 on June 26, 2020 relating to non‑clinical information. No clinical safety or efficacy issues and no chemistry, manufacturing and controls (CMC) issues were identified. The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for ZYNRELEF in July 2020. Heron’s New Drug Submission (NDS) for HTX-011 for the management of postoperative pain was granted Priority Review status by Health Canada in October 2019 and accepted by Health Canada in November 2019. Heron is working to respond to a list of questions received from Health Canada in July 2020.

About CINVANTI (Aprepitant) Injectable Emulsion

CINVANTI, in combination with other antiemetic agents, is indicated in adults for the prevention of acute and delayed nausea and vomiting associated with initial and repeat courses of highly emetogenic cancer chemotherapy (HEC) including high-dose cisplatin as a single-dose regimen, delayed nausea and vomiting associated with initial and repeat courses of moderately emetogenic cancer chemotherapy (MEC) as a single-dose regimen, and nausea and vomiting associated with initial and repeat courses of MEC as a 3-day regimen. CINVANTI is an IV formulation of aprepitant, a substance P/neurokinin-1 (NK1) receptor antagonist (RA). CINVANTI is the first IV formulation to directly deliver aprepitant, the active ingredient in EMEND capsules. Aprepitant (including its prodrug, fosaprepitant) is the only single-agent NK1 RA to significantly reduce nausea and vomiting in both the acute phase (0–24 hours after chemotherapy) and the delayed phase (24–120 hours after chemotherapy). The FDA-approved dosing administration included in the United States prescribing information for CINVANTI is a 30-minute IV infusion or a 2-minute IV injection.

CINVANTI is under investigation for the treatment of COVID-19 as a daily 2-minute IV injection when added to the current standard of care.

About SUSTOL (Granisetron) Extended-Release Injection

SUSTOL is indicated in combination with other antiemetics in adults for the prevention of acute and delayed nausea and vomiting associated with initial and repeat courses of moderately emetogenic chemotherapy (MEC) or anthracycline and cyclophosphamide (AC) combination chemotherapy regimens. SUSTOL is an extended-release, injectable 5-HT3 receptor antagonist that utilizes Heron’s Biochronomer drug delivery technology to maintain therapeutic levels of granisetron for ≥5 days. The SUSTOL global Phase 3 development program was comprised of two, large, guideline-based clinical studies that evaluated SUSTOL’s efficacy and safety in more than 2,000 patients with cancer. SUSTOL’s efficacy in preventing nausea and vomiting was evaluated in both the acute phase (0–24 hours after chemotherapy) and delayed phase (24–120 hours after chemotherapy).

About HTX-034 for Postoperative Pain

HTX-034, an investigational non-opioid, is a fixed-dose combination, extended‑release solution of the local anesthetic bupivacaine, the nonsteroidal anti-inflammatory drug meloxicam and an additional agent that further potentiates the activity of bupivacaine. HTX-034 is formulated in the same proprietary polymer as HTX-011. By combining two different mechanisms that each enhance the activity of the local anesthetic bupivacaine, HTX-034 is designed to provide superior and prolonged analgesia. Local administration of HTX-034 in a validated preclinical postoperative pain model resulted in sustained analgesia for 7 days.

On August 5, 2020 ORIC Pharmaceuticals, Inc. (Nasdaq: ORIC), a clinical stage oncology company focused on developing treatments that address mechanisms of therapeutic resistance, reported financial results for the quarter ended June 30, 2020 (Press release, ORIC Pharmaceuticals, AUG 5, 2020, View Source [SID1234562921]).

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"Since the beginning of the year, we have realized substantial progress advancing our wholly owned, internally developed pipeline," said Jacob Chacko, M.D., president and chief executive officer. "Looking forward, we see a number of important events as we continue to execute against our strategic plans. We expect to select the recommended Phase 2 dose and initiate the expansion cohorts in both of our ongoing ORIC-101 clinical studies in the second half of the year. Additionally, we are preparing for the development of our newly licensed, potential best-in-class PRC2 inhibitor, ORIC-944, which along with ORIC-533, we anticipate as IND candidates for 2021."

Second Quarter 2020 and Other Recent Highlights

Licensed Exclusive Worldwide Rights to PRC2 Inhibitors: In August 2020, ORIC licensed exclusive worldwide development and commercialization rights to a potential best-in-class PRC2 inhibitor, ORIC-944, from Mirati Therapeutics, Inc. Under the terms of the agreement with Mirati, ORIC paid to Mirati a one-time non-cash payment of $20 million in shares of ORIC common stock. The number of shares issued was based on a price of $34.00 per share, representing a premium of 10% to the 60-day trailing volume weighted average trading price of ORIC’s common stock. ORIC is not subject to any future milestone or royalty payment obligations to Mirati.
Preclinical Data on ORIC-101 Presented at AACR (Free AACR Whitepaper): In June 2020, ORIC presented three poster presentations at the 2020 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Virtual Meeting II. Key findings of the presentations included:
— A transcriptional signature of glucocorticoid receptor (GR) activity was identified in a panel of 32 cell lines across triple negative breast cancer, non-small cell lung cancer and pancreatic ductal adenocarcinoma, which translated from preclinical models to human tumors;
— ORIC-101 overcame GR-mediated resistance to chemotherapeutic agents including taxanes, antimetabolites and platinum agents, in both in vitro and in vivo efficacy studies spanning a variety of solid tumors; and
— Transcriptional and histological profiling showed that ORIC-101 reversed GR-activated pathways involved in drug resistance and reversed in vivo markers of epithelial-to-mesenchymal transition, antiapoptosis, and hypoxia.
Preclinical Data on CD73 Inhibitor Program Presented at AACR (Free AACR Whitepaper): In June 2020, ORIC presented two poster presentations at the 2020 AACR (Free AACR Whitepaper) Annual Virtual Meeting II. Key findings of the presentations included:
— ORIC’s CD73 inhibitors demonstrated suppression of adenosine production in vitro across multiple cell types and rescued activation of CD8+ T cells exposed to AMP with greater potency than competitor compounds;
— ORIC-533 was shown to result in sustained inhibition of adenosine production after drug washout, consistent with its slow off-rate, and differentiating from other CD73 inhibitors;
— ORIC-533 potency in high AMP environments distinguishes it from other compounds, with activity in AMP concentrations as high as 1 millimolar, which may better reflect certain tumor microenvironments; and
— Daily oral delivery of ORIC’s CD73 inhibitors significantly inhibited tumor growth, with corresponding in vivo reduction of adenosine levels in tumors, and immune modulation consistent with decreased immunosuppression.
Expanded and Strengthened its Board: In June 2020, the company appointed Lori Kunkel, M.D., to its board of directors. Dr. Kunkel brings more than twenty-five years of experience in oncology and immunology drug development and commercialization.
Completed $138 Million Initial Public Offering: On April 28, 2020, the company completed its initial public offering (IPO), selling 8,625,000 shares of common stock, which included the full exercise by the underwriters of their option to purchase up to 1,125,000 additional shares, at $16.00 per share. Gross proceeds from the IPO, excluding underwriting discounts and commissions and other estimated offering costs, were $138.0 million.
Anticipated Milestones

ORIC expects to select the recommended Phase 2 dose for its two ongoing ORIC-101 combination trials in the second half of 2020 and to report interim data from one of the trials in the first half of 2021 and from the other trial in the second half of 2021.
ORIC expects to file an Investigational New Drug (IND) Application for ORIC-533 with the Food and Drug Administration (FDA) in the first half of 2021.
ORIC expects to file an IND Application for ORIC-944 with the FDA in the second half of 2021.
Second Quarter 2020 Financial Results

Cash and Cash Equivalents: Cash and cash equivalents totaled $196.6 million as of June 30, 2020, which includes the gross proceeds of $138.0 million from the company’s IPO in April 2020. The company expects its current cash and cash equivalents will be sufficient to fund its current operating plan into the fourth quarter of 2022.
R&D Expenses: Research and development (R&D) expenses were $7.7 million for the three months ended June 30, 2020, compared to $5.0 million for the three months ended June 30, 2019, an increase of $2.7 million. For the six months ended June 30, 2020, R&D expenses were $15.0 million compared to $10.3 million for the same period of 2019, an increase of $4.7 million. The increases were primarily due to the continued advancement of the ORIC-101 and ORIC-533 programs and higher personnel and related expenses, including non-cash stock-based compensation.
G&A Expenses: General and administrative (G&A) expenses were $3.4 million for the three months ended June 30, 2020, compared to $1.3 million for the three months ended June 30, 2019, an increase of $2.1 million. For six months ended June 30, 2020, general and administrative expenses were $5.3 million compared to $2.4 million for the same period in 2019, an increase of $2.9 million. These increases were primarily due to higher professional services and related costs to operate as a public company, and higher personnel costs, including non-cash stock-based compensation.
Webcast and Conference Call
ORIC will host a webcast and conference call today, August 5th, at 4:30 p.m. ET. To participate in the conference call, please dial (866) 393-4306 (domestic) or (734) 385-2616 (international) and refer to conference ID: 5167646. Please join the conference call at least 15 minutes early to register. A live webcast will be available in the Investors section of the company’s website at www.oricpharma.com. The webcast will be archived for 60 days following the presentation.

On August 5, 2020 bluebird bio, Inc. (NASDAQ: BLUE) reported financial results and business highlights for the second quarter ended June 30, 2020 and shared recent operational progress (Press release, bluebird bio, AUG 5, 2020, View Source [SID1234562920]).

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"I am incredibly proud of the progress made at bluebird this quarter, and the way in which our employees have continued to execute on behalf of patients in the midst of an ongoing global pandemic," said Nick Leschly, chief bluebird. "It is especially gratifying that despite these challenges, we have continued to treat patients in our clinical studies at levels consistent with prior quarters. Within the quarter, we presented compelling clinical data across three of our core four programs: sickle cell disease, β-thalassemia, and multiple myeloma, and made important progress across all of our programs. In sickle cell disease, we reached an important milestone on our transition to commercial manufacturing process with the successful dosing of the first sickle cell patient using drug product manufactured with suspension-based lentiviral vector. The European launch of ZYNTEGLO continues to progress, with positive ongoing discussions with payers across Europe and we expect to treat our first commercial patients in the second half of this year. Additionally, our multiple myeloma program, partnered with BMS, continues to advance with our submission of the BLA to the FDA and BMS’ validated MAA submission in Europe. With this foundation and additional cash runway, we are confident in our ability to bring our core four programs to patients in the commercial setting by 2022 and grow our sustainable pipeline of transformative gene and cell therapies. All of this progress is made possible by our undaunted bluebirds, who have shown time and again their resourcefulness and ingenuity even under the most challenging of circumstances to bring our therapies to patients."

RECENT HIGHLIGHTS

SUSPENSION LVV MANUFACTURING FOR SCD – Today, bluebird bio announced that it has treated the first sickle cell patient with drug product manufactured with suspension-based lentiviral vector (sLVV). This process is intended to allow for larger scale and more efficient manufacturing of LVV. The company intends to submit data supporting the use of sLVV to the FDA as part of its submission for regulatory approval of LentiGlobin gene therapy for SCD in the second half of 2021.
ELI-CEL ACCELERATED ASSESSMENT – In July 2020, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) granted an accelerated assessment to elivaldogene autotemcel (eli-cel, Lenti-D gene therapy) for the treatment of cerebral adrenoleukodystrophy (CALD). The company plans to submit a Marketing Authorization Application (MAA) to the EMA for eli-cel in 2020. Accelerated assessment reduces the timeframe for the EMA to review an MAA to 150 evaluation days rather than 210. The CHMP grants review under the accelerated assessment procedure if the medicinal product is of major interest for public health, especially from the point of view of therapeutic innovation.
NEW ZYNTEGLO QTC – Today, bluebird bio announced that it has contracted with a second qualified treatment center for ZYNTEGLO. The center, in Essen, Germany, is prepared to treat patients with β-thalassemia in 2020.
IDE-CEL BIOLOGICS LICENSE APPLICATION (BLA) SUBMISSION – On July 29, 2020, bluebird bio and BMS announced the submission of their Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for idecabtagene vicleucel (ide-cel; bb2121), the companies’ investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy. This submission provides further details on the Chemistry, Manufacturing and Controls (CMC) module to address the outstanding regulatory requests from the FDA in May 2020 following the original BLA submission.
SCD DATA AT EHA (Free EHA Whitepaper) – On June 12, 2020, bluebird bio presented new data showing a near elimination of sickle cell disease-related vaso-occlusive crises and acute chest syndrome in the phase 1/2 clinical study of bluebird bio’s LentiGlobin gene therapy for sickle cell disease at 25th EHA (Free EHA Whitepaper) Congress. The company plan to submit its BLA to the FDA based on an analysis of clinical data from this study using complete resolution of severe vaso-occlusive events (VOEs) as the primary endpoint with at least 18 months of follow-up post-treatment with LentiGlobin for SCD. The company continues to plan to submit the U.S. BLA for SCD in the second half of 2021.
TDT DATA AT EHA (Free EHA Whitepaper) – On June 12, 2020, bluebird bio presented new data showing that the majority of evaluable patients across genotypes achieve transfusion independence and maintain it with near-normal hemoglobin levels in phase 3 Studies of betibeglogene autotemcel (beti-cel; formerly LentiGlobin for β-thalassemia) gene therapy presented at EHA (Free EHA Whitepaper) Congress. The company presented data from the Northstar-2 (HGB-207) clinical study of beti-cel in patients with transfusion-dependent β-thalassemia who do not have a β0/β0 genotype and the Northstar-3 (HGB-212) clinical study of beti-cel in patients with transfusion-dependent β-thalassemia who have a β0/β0 genotype or IVS-I-110 mutation.
IDE-CEL MARKETING AUTHORIZATION APPLICATION (MAA) VALIDATION – On May 22, 2020, BMS announced that the European Medicines Agency (EMA) has validated its Marketing Authorization Applications (MAA) for ide-cel. Validation of the application confirms the submission is complete and begins the EMA’s centralized review process.
KARMMA DATA AT ASCO (Free ASCO Whitepaper) – On May 13, 2020, Bristol Myers Squibb (NYSE: BMY) and bluebird announced positive updated results from the pivotal, Phase 2 KarMMa study evaluating the efficacy and safety of the companies’ investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, ide-cel, in patients with relapsed and refractory multiple myeloma. The data from this study formed the basis of recent regulatory submissions.
EXTENDED CASH RUNWAY – In June 2020, bluebird bio raised approximately $541.5 million in net proceeds through a public equity offering. bluebird bio anticipates that its cash, cash equivalents and marketable securities as of June 30, 2020, together with projected revenue generated under our collaborative arrangements and projected sales of products, will be sufficient to fund operations into 2023 based on the company’s current business plan.
UPCOMING ANTICIPATED MILESTONES

Regulatory

Submission of a Marketing Authorization Application to the European Medicines Agency for eli-cel in patients with cerebral adrenoleukodystrophy by the end of 2020.
Clinical

Updated data presentation from HGB-206 clinical study in patients with SCD by the end of 2020.
Presentation of ide-cel clinical data from the CRB-401 study in patients with multiple myeloma in 2020, in partnership with BMS.
Updated data presentation from ALD-102 clinical study in patients with CALD by the end of 2020.
Commercial and Foundation Building

ZYNTEGLO first commercial patients treated in Europe in the second half of 2020.
ZYNTEGLO access and reimbursement in additional EU countries established by the end of 2020.
SECOND QUARTER 2020 FINANCIAL RESULTS

Cash Position: Cash, cash equivalents and marketable securities as of June 30, 2020 and December 31, 2019 were $1.60 billion and $1.24 billion, respectively. The increase in cash, cash equivalents and marketable securities is primarily a result of proceeds received from the May 2020 public offering of the Company’s common stock and a one-time upfront payment received in connection with the Company’s amended collaboration with BMS, partially offset by cash used in support of ordinary course operating and commercial-readiness activities.
Revenues: Total revenues were $198.9 million for the three months ended June 30, 2020 compared to $13.3 million for the three months ended June 30, 2019. Total revenues were $220.8 million for the six months ended June 30, 2020 compared to $25.8 million for the six months ended June 30, 2019. The increase for both periods was primarily attributable to the recent amended BMS collaboration and monetization for ex-U.S. milestones and royalties from ide-cel and bb21217, with the majority of the revenue recognized relating to ide-cel license and manufacturing services. Deferred revenue under our BMS collaboration will be recognized over time as the associated obligation to provide vector manufacturing through development is satisfied.
R&D Expenses: Research and development expenses were $156.3 million for the three months ended June 30, 2020 compared to $146.5 million for the three months ended June 30, 2019. Research and development expenses were $310.4 million for the six months ended June 30, 2020 compared to $269.2 million for the six months ended June 30, 2019. The increase in both periods was primarily driven by costs incurred to advance and expand the company’s pipeline.
SG&A Expenses: Selling, general and administrative expenses were $68.6 million for both the three months ended June 30, 2020 and June 30, 2019. Selling, general and administrative expenses were $141.9 million for the six months ended June 30, 2020 compared to $128.9 million for the six months ended June 30, 2019. The increase in the six month period was largely attributable to costs incurred to support the Company’s ongoing operations and growth of its pipeline as well as commercial-readiness activities.
Net Loss: Net loss was $21.5 million for the three months ended June 30, 2020 compared to $195.8 million for the three months ended June 30, 2019. Net loss was $224.1 million for the six months ended June 30, 2020 compared to $360.2 million for the six months ended June 30, 2019.