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.

On August 5, 2020 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and reported financial results for the second quarter ending June 30, 2020 (Press release, PTC Therapeutics, AUG 5, 2020, View Source [SID1234562919]).

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"We are pleased with the progress in our pipeline and the strong commercial performance of our global Duchenne franchise," said Stuart W. Peltz, Ph.D., CEO of PTC Therapeutics. "Our growing revenue stream allows us to continue to invest in the development of treatments leveraging our novel technology platforms. We have a robust pipeline and with more than $1 billion in cash are well positioned to advance multiple differentiated therapies for patients living with rare disorders, not only in the near term, but also for many years to come."

Key Second Quarter and Other Corporate Updates:
•Our Duchenne muscular dystrophy franchise had strong performance in the second quarter. The greater than 30% year-over-year increase in second quarter sales for Emflaza was driven by ongoing improvements in our commercial business. New Duchenne patients continue to be identified in Europe, LATAM and other key markets for Translarna despite the challenges of COVID-19.
•As part of the annual renewal process for Translarna, the European Medicines Agency (EMA) confirmed its risk/benefit profile for the sixth consecutive year. In addition, PTC previously announced that the Committee for Medicinal Products for Human Use (CHMP) of the EMA recommended to remove the statement "efficacy has not been demonstrated in non-ambulatory patients" from the SmPC for Translarna. This label change enables healthcare professionals to use their clinical judgement to make treatment decisions for their patients on Translarna who have lost ambulation and should support reimbursement agencies granting continued access to Translarna.
•In the second quarter, PTC strengthened its pipeline and platforms with the acquisition of a late-stage asset for phenylketonuria (PKU). PTC923 is a clinical-stage investigational therapy for inborn errors of metabolism, including PKU and other diseases associated with defects in the tetrahydrobiopterin (BH4) biochemical pathways diagnosed at birth. PTC is currently conducting nonclinical studies to support the long-term dosing for the planned phase 3 trial in PKU in 2021.
•The U.S. Food and Drug Administration (FDA) PDUFA date for risdiplam is August 24, 2020. In addition, a submission of a marketing authorization application (MAA) to EMA for risdiplam is imminent. The filing of the MAA would trigger a $15 million milestone payment to PTC from Roche. Roche has submitted applications for approval in Brazil, Chile, China, Indonesia, Russia, South Korea and Taiwan.
•PTC recently announced an agreement to monetize a portion of the rights to the risdiplam royalty stream for a $650 million up front payment from Royalty Pharma plc, providing PTC with significant non-dilutive capital. PTC retains nearly 60% of the risdiplam royalty stream up until a $1.3 billion threshold is reached, after which PTC retains 100% of the risdiplam royalty stream. PTC also retains all economics associated with up to approximately $400 million in remaining regulatory and sales milestones.

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•Launch activities for Tegsedi continue to focus on patient finding. Pricing discussions in Brazil are ongoing and are expected to be completed by the end of the year.
•The submission of an application for marketing authorization for Waylivra in patients with familial chylomicronemia syndrome (FCS) has been filed with ANVISA and a decision is expected in 2021. Patient finding and early access programs are ongoing.
•PTC continues to develop manufacturing capabilities for its gene therapy products. It has expanded its collaboration with MassBiologics for the manufacturing of the aromatic L-amino acid decarboxylase deficiency (AADCd) commercial supply. In addition, PTC gained occupancy under its lease of ~220,000 square feet at the former BMS site in Hopewell, NJ. Manufacturing of clinical materials for its gene therapy programs at this site is expected to initiate in 2021.
•PTC recently launched a global internship program, the Talent Pipeline Program (TPP), aimed at providing recent graduates, especially those in minority communities, with real-world experience in the biopharmaceutical industry and related professions, including research, finance, commercial, compliance, quality, legal, information technology, and communications. The TPP will provide approximately 30 interns with a one-year paid program with real-world training experience.

Updates in PTC’s Diverse Product Pipeline
•In its Bio-e platform, PTC expects to initiate potential registrational trials with vatiquinone, formerly known as PTC743, in refractory mitochondrial epilepsy in the third quarter of 2020 and in Friedreich ataxia in the fourth quarter of 2020.
•The first subject has been dosed in the Phase 1 trial for PTC857, PTC’s second compound from the Bio-e platform. Data from the single ascending dose and multiple ascending dose studies is expected by the end of 2020.
•PTC recently initiated a Phase 2/3 clinical trial for PTC299 for COVID-19 called FITE-19. PTC expects Stage 1 to be completed in the second half of 2020 and anticipates reporting top-line results from both stages in the first half of 2021. Multiple sites have been opened in the U.S., Brazil, Spain and Australia with additional countries for Stage 2 expected to initiate in the coming months.
•In the novel splicing platform, the Huntington disease program remains on track for the initiation of first-in-human studies in 2020.
•PTC expects to initiate submission of the biologics license application (BLA) to the U.S. FDA in the second half of 2020 for its AADCd gene therapy program.
•Due to COVID-19 related delays, PTC now expects the final opinion from the CHMP on the MAA for the AADCd program in the first quarter of 2021.
•Given the evolving COVID-19 situation in the U.S., the final study muscle biopsies have not yet been collected from 8 remaining boys in the Translarna (ataluren) dystrophin study. PTC is continuously monitoring the situation to determine when it will be possible to safely obtain the final biopsies and is exploring all potential options in order to have a data read out by the end of 2020. All patients in the study remain on Translarna until they are able to complete the final study visit.

Financial Highlights:
•Total net product revenues were $75.2 million across our commercial portfolio for the second quarter of 2020, compared to total net product revenues of $85.5 million for the second quarter of 2019.
•Translarna net product revenues were $38.6 million for the second quarter of 2020, compared to $57.8 million for the second quarter of 2019. Revenues for the second quarter of 2020 were impacted by the timing of a group purchase order from Brazil, which is the primary driver for the year-over-year decrease, as the second quarter of 2019 included a significant group purchase order from Brazil. Due to the impact of the pandemic, there was an administrative delay by the

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Brazilian Ministry of Health in receiving the centralized Translarna group purchase order. PTC anticipates a group purchase order from Brazil later this year.
•Emflaza net product revenues were $36.2 million for the second quarter of 2020, compared to $27.6 million for the second quarter of 2019. Growth in net product revenues was driven by new patient prescriptions and continued operational improvements and efficiencies in our commercial business.
•Generally accepted accounting principles in the U.S. (GAAP) research and development (R&D) expenses were $176.5 million for the second quarter of 2020, compared to $60.0 million for the second quarter of 2019. The increase in R&D expenses includes one-time charges of $53.6 million related to the acquisition of Censa Pharmaceuticals, and $41.2 million related to the MassBiologics of the University of Massachusetts Medical School agreement for commercial manufacturing of our lead gene therapy program in AADCd.
•Non-GAAP R&D expenses were $168.0 million for the second quarter of 2020, excluding $8.6 million in non-cash, stock-based compensation expense, compared to $54.5 million for the second quarter of 2019, excluding $5.5 million in non-cash, stock-based compensation expense.
•GAAP selling, general and administrative (SG&A) expenses were $53.7 million for the second quarter of 2020, compared to $49.2 million for the second quarter of 2019. The increase reflects continued investment to support our commercial activities including our expanding commercial portfolio.
•Non-GAAP SG&A expenses were $45.3 million for the second quarter of 2020, excluding $8.3 million in non-cash, stock-based compensation expense, compared to $43.8 million for the second quarter of 2019, excluding $5.4 million in non-cash, stock-based compensation expense.
•Change in the fair value of deferred and contingent consideration was $7.7 million for the second quarter of 2020, compared to $5.3 million for the second quarter of 2019. The change in fair value of deferred and contingent consideration is related to the fair valuation of potential future consideration to be paid to former equity holders of Agilis Biotherapeutics, Inc. (Agilis) in connection with PTC’s acquisition of Agilis, which closed in August 2018.
•Settlement of deferred and contingent consideration was $10.6 million for the second quarter of 2020. The settlement of deferred and contingent consideration is related to a loss upon the settlement of the deferred and contingent consideration liabilities as a result of the rights exchange agreement with certain former shareholders of Agilis, whereby such former shareholders exchanged their pro rata share of specific future cash milestone payments in the aggregate amount of $225 million for a mixture of cash and equity of PTC. Under this agreement, which the former shareholders and PTC entered into on April 29, 2020, PTC has paid $36.9 million in cash and issued 2,821,176 shares of common stock in exchange for the cancellation and forfeiture of the participating shareholders’ rights to receive (i) $174.0 million, in the aggregate, of potential milestone payments based on the achievement of certain regulatory milestones and (ii) $37.6 million, in the aggregate, of $40.0 million in development milestone payments that would have been due upon the passing of the second anniversary of the closing of the Merger, regardless of whether the milestones are achieved.
•Net loss was $181.4 million for the second quarter of 2020, compared to net loss of $41.8 million for the second quarter of 2019.
•Cash, cash equivalents and marketable securities were $498.9 million as of June 30, 2020, compared to $686.6 million as of December 31, 2019.
•Shares issued and outstanding as of June 30, 2020 were 67,240,679.

On August 5, 2020 Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company discovering and developing breakthrough treatments for immune-mediated and oncologic disorders, reported its second quarter 2020 financial results and corporate highlights (Press release, Kezar Life Sciences, AUG 5, 2020, View Source [SID1234562918]).

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"Our excitement continues to build around the novel mechanism of KZR-616 and supports our conviction that targeting master regulators of cellular function – like the immunoproteasome – can benefit patients in need," said John Fowler, Kezar’s Chief Executive Officer. "Our recent fundraising has established a strong balance sheet to allow us to prosecute our robust pipeline."

Noreen Henig, M.D., Kezar’s Chief Medical Officer added, "We are encouraged by the striking early signs of efficacy and the favorable safety and tolerability we’ve seen with KZR-616 and believe in its potential to be a profound treatment option for a wide array of autoimmune diseases. Based on recent events, we plan to adjust our clinical development plans to optimize and expedite the development pathway for KZR-616. In addition, activities related to KZR-261, the first molecule to come forward from our protein secretion inhibition platform, continue as planned for an IND submission in the first quarter of 2021."

Clinical Highlights & Updates

KZR-616 – Selective Immunoproteasome Inhibitor

KZR-616 is currently being evaluated for the treatment of severe autoimmune diseases.

On June 3, 2020, Kezar provided a data update from the Phase 1b portion of the MISSION study. Overall, improvements were seen across seven measures of disease activity in a majority of patients, and two of two patients with lupus nephritis (LN) experienced a greater than 50% reduction in proteinuria, a biomarker of disease severity. A positive safety and tolerability profile was observed with step-up dosing of KZR-616. The Phase 1b dataset builds on the safety and tolerability evaluation performed in 100 healthy subjects from two Phase 1a studies.

Based upon the positive results from the Phase 1b portion of MISSION and acknowledging the recent slowdown in recruitment activities across clinical trials due to the COVID-19 pandemic, Kezar has reviewed and adapted its clinical plans to optimize the development pathway for KZR-616. Kezar will be focusing all development efforts on 60 mg and 45 mg once-weekly subcutaneous dosing of KZR-616. This decision is based on the positive safety and tolerability, pharmacology, and clinical activity results seen to date.

The revised clinical plan for the MISSION Phase 2 trial in patients with active, proliferative lupus nephritis include the following updates:

The Phase 2 protocol has been amended, and the primary endpoint has been changed from safety and tolerability to an efficacy endpoint of renal response measured by 50% or greater reduction in urine protein to creatinine ratio (UPCR) at six months. This study is now open for enrollment under the amended protocol.

The inclusion/exclusion criteria of the study have been expanded to include LN patients with histologic Class III or IV +/- Class V being treated with current standard-of-care. The open-label clinical trial is designed to enroll 20 patients with a single treatment arm evaluating a 60 mg dose (with first dose of 30 mg) of KZR-616 administered subcutaneously once weekly for 24 weeks.

Interim data are expected in late 2021. To allow for responding patients to continue treatment with KZR-616, a 12-month extension study is being planned.

The PRESIDIO Phase 2 trial of KZR-616 in dermatomyositis and polymyositis continues to enroll. A 12-month open-label extension study is being planned and will be available for patients completing the trial.

The MARINA study in patients with autoimmune hemolytic anemia and immune thrombocytopenia has been withdrawn. A combination of COVID-related slowdowns in screening activities and a need to substantially amend the current protocol to reduce high screen failure rates factored into this decision. No new clinical data has informed this decision, and Kezar’s scientific conviction level remains high that KZR-616 could be an important new therapy for patients with these serious diseases. Data from KZR-616 in other studies will inform the optimal strategy for the development of this product candidate in these indications.

KZR-261 – Protein Secretion Program

KZR-261, a first-in-class protein secretion inhibitor, targets the Sec61 translocon and has demonstrated broad anti-tumor activity in preclinical models of both solid and hematologic malignancies. Additional preclinical data further detailing the ability of novel Sec61 inhibitors to exhibit broad anti-cancer activity with minimal toxicity in vitro and in vivo was presented in an e-poster during the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) (ASCO20) Virtual Scientific Program at the end of May.

An Investigational New Drug application for KZR-261 is planned for submission in the first quarter of 2021. The first-in-human clinical trial will evaluate dose escalation and safety and tolerability in patients with solid tumors.

Business Update

On June 11, 2020, Kezar closed an underwritten public offering with gross proceeds of approximately $46.7 million, before deducting underwriting discounts and commissions and offering expenses. In the public offering, Kezar issued and sold 7,590,909 shares of common

stock at $5.50 per share and pre-funded warrants to purchase 909,091 shares of common stock at $5.499 per share, with an exercise price of $0.001 per share. In July, Kezar issued and sold an additional 427,707 shares of common stock at $5.50 per share, pursuant to the exercise by the underwriters of their option to purchase additional shares, with gross proceeds of approximately $2.4 million.

Financial Results

Cash, cash equivalents and marketable securities totaled $157.5 million as of June 30, 2020, compared to $78.2 million as of December 31, 2019. The increase in cash, cash equivalents and marketable securities was primarily attributable to the net proceeds from the underwritten public offerings in February and June 2020, net of cash used by the Company in operations to advance its clinical stage programs and preclinical research and development.

Research and development expenses for the second quarter of 2020 increased by $0.2 million to $7.1 million, compared to $6.9 million in the second quarter of 2019. This increase was primarily related to advancing the protein secretion preclinical program

General and administrative expenses for the second quarter of 2020 increased by $0.3 million to $2.7 million, compared to $2.4 million in the second quarter of 2019. The increase was primarily due to an increase in personnel expenses, including non-cash stock-based compensation.

Net loss for the second quarter of 2020 was $9.5 million, or $0.22 per basic and diluted common share, compared to a net loss of $8.7 million, or $0.46 per basic and diluted common share, for the second quarter of 2019.

Total shares of common stock outstanding were 45.8 million as of June 30, 2020. Additionally, there were outstanding pre-funded warrants to purchase 3.8 million shares of common stock at an exercise price of $0.001 per share and outstanding options to purchase 4.5 million shares of common stock at a weighted average exercise price of $6.09 per share as of June 30, 2020.

Conference Call and Webcast Information

Kezar will be a hosting a conference call and webcast at 4:30 EDT today. To access the live conference call via phone, dial 877-407-9711 (U.S. toll-free) or 412-902-1014 (toll). No conference ID is required. Additionally, a live webcast of the call will be available under the Events section of the Kezar’s Investor Relations (IR) site at View Source An archived replay of the call will also be available on the company’s IR site for 90 days following the live call.

About KZR-616

KZR-616 is a novel, first-in-class, selective immunoproteasome inhibitor with broad therapeutic potential across multiple autoimmune diseases. Preclinical research demonstrates that selective immunoproteasome inhibition results in a broad anti-inflammatory response in animal models of several autoimmune diseases, while avoiding immunosuppression. Data generated from Phase 1a and 1b trials provide evidence that KZR-616 exhibits a favorable safety and tolerability profile for development in severe, chronic autoimmune diseases. Phase 2 trials are underway in severe autoimmune diseases.

About KZR-261

KZR-261, a novel, first-in-class protein secretion inhibitor, is the first clinical candidate to be nominated from Kezar’s research and discovery efforts targeting protein secretion pathways. KZR-261 is a broad-spectrum anti-tumor agent that acts through direct interaction and inhibition of Sec61 activity. The compound was discovered at Kezar through a robust medicinal chemistry campaign in which several scaffolds were progressed through the company’s proprietary platform evaluating Sec61 modulation. As a result, Kezar has established a broad library of protein secretion inhibitors. KZR-261 has demonstrated several encouraging properties that lead to its potential to be an anti-cancer agent for the treatment of solid and hematologic malignancies. IND-enabling activities are currently underway, and an IND submission in solid tumors is expected to be filed in the first quarter of 2021.