On May 4, 2021 ERYTECH Pharma (Nasdaq & Euronext: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, today provided a business update and reported its financial results for the first quarter of 2021 (Press release, ERYtech Pharma, MAY 4, 2021, View Source(U.S.)%20and,its%20financial%20results%20for%20the [SID1234579076]).

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"The first quarter of 2021 has again been one of tremendous progress, and has further set the stage for a catalyst-rich second half of this year," said Gil Beyen, CEO of ERYTECH. "All four of our clinical programs are expected to report significant defining events before the end of the year, two of which potentially supporting the application for regulatory approvals within the next twelve months. Our 512-patient pivotal Phase 3 trial in second-line pancreatic cancer, TRYbeCA-1, is on track for final results in the fourth quarter. Subject to positive results, we expect to submit our applications for approval in this indication in the United States and Europe in the first half of 2022. The second potentially pivotal track of eryaspase is for the treatment of ALL patients who developed hypersensitivities to pegylated asparaginase. We recently requested a pre-BLA meeting with the FDA to discuss the path to approval in this indication and we are targeting the potential submission of a Biologics License Applicaton (BLA) in the second half of this year. We are also continuing work to further broaden the indication scope of eryaspase. The safety profile and encouraging clinical activity observed in the first dose cohort of the ongoing Phase 1 IST in first line pancreatic cancer add to our conviction that eryaspase can be a meaningful contributor to helping cancer patients live longer, better. "

Business Highlights

TRYbeCA-1, pivotal Phase 3 clinical trial in second-line advanced pancreatic cancer

TRYbeCA-1 is a randomized, controlled Phase 3 pivotal trial, evaluating eryaspase in second-line advanced pancreatic cancer at approximately 90 sites in the United States and in Europe. Eryaspase, in combination with standard chemotherapy (gemcitabine/nab paclitaxel or an irinotecan-based regimen), is compared with standard chemotherapy alone in a 1 to 1 randomization. The primary endpoint is overall survival (OS).

Enrollment was completed in January 2021. A total of 512 patients were randomized in the trial, above the target enrollment of 482 patients.

In February 2021, an interim efficacy and safety analysis was performed by an independent data monitoring committee (IDMC), which recommended the trial continue without modification to its final analysis. As with the three previous IDMC reviews, no safety issues have been identified and the Company remains blinded to the primary and secondary endpoint efficacy data.

Reporting of the final results from this trial is expected in the fourth quarter of 2021.

Phase 2 trial in acute lymphoblastic leukemia (ALL), sponsored by the Nordic Society of Pediatric Hematology and Oncology (NOPHO)
The NOPHO trial evaluated the safety and pharmacological profile of eryaspase in ALL patients who had previously experienced hypersensitivity reactions to pegylated asparaginase therapy. The primary objective of the trial was to assess asparaginase enzymatic activity. In December 2020, positive trial results were presented at the 2020 American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting. Eryaspase in combination with chemotherapy, administered every two weeks, provided a sustained asparaginase enzyme activity level, and was generally well tolerated with few hypersentivity reactions.

The Company continued its interactions with the FDA regarding a potential regulatory approval in this indication based on the NOPHO-sponsored trial. In April 2021, ERYTECH requested a pre-BLA meeting to discuss the submission of a Biologics License Application.

Subject to the feedback from this meeting, the Company plans to potentially submit the BLA in the second half of 2021.

TRYbeCA-2, randomized Phase 2 clinical trial in triple-negative breast cancer (TNBC)

The TRYbeCA-2 trial is evaluating eryaspase in combination with gemcitabine and carboplatin chemotherapy, compared to chemotherapy alone, in metastatic TNBC. Target enrollment is approximately 64 patients. The primary end point of the trial is objective response rate.

Following a recommendation of the trial’s investigators, the trial’s inclusion criteria were modified in January 2021 to include second-line patients.

The TRYbeCA-2 Steering Committee met in April 2021 and recommended the trial continue without modification after review of the safety data of the first 19 patients.

Initial data from the TRYbeCA-2 trial are expected to be reported in the fourth quarter of 2021.

rESPECT, Phase 1 investigator-sponsored trial in first-line pancreatic cancer

rESPECT is a Phase 1 trial, sponsored by the Georgetown Lombardi Comprehensive Cancer Center, evaluating the safety of eryaspase in combination with mFOLFIRINOX as a first-line treatment for advanced and locally advanced pancreatic cancer in approximately 18 patients.

Patient enrollment started in January 2021, and the first dose cohort of three patients was enrolled by the end of February.

After review of the safety data, the Dose Escalation Committee concluded that no dose-limiting toxicity (DLT) had been observed in the first cohort treated at a therapeutic dose of 75 U/kg eryaspase, and that the treatment was well tolerated in the cohort thus far.

After the first dose cycle of treatment, two of the three patients had a partial response and significantly decreased levels of CA19-9, a pancreatic cancer tumor marker; the third patient had stable disease after the first cycle of treatment.
The trial has been escalated to the next dosing cohort, where the dose level will be increased to 100 U/kg (the same dose as in TRYbeCA-1) eryaspase. This will be the highest dose level cohort in the trial and the presumed maximum tolerable dose (MTD) assuming no dose limiting toxicity is observed.

Determination of the MTD is expected in the second half of 2021.

First Quarter 2021 Financial Results and Cash Guidance

Key financial figures for the first quarter of 2021 compared with the same period of the previous year are summarized below:

Net loss for the first quarter of 2021 was €11.9 million, down €5.6 million (-32%) year-over-year, with a €5.8 million decrease (-31%) in operating loss and a €0.2 million decrease in financial income. The €5.8 million decrease in operating loss was attributable to the €4.8 million decrease in preclinical and clinical development expenses, concurrent with the completion of patient enrollment in the Company’s Phase 3 clinical trial in pancreatic cancer, a €0.3 million decrease in general and administrative expenses, and a €0.7 million increase in other income, mostly related to R&D tax credits. The €0.2 million decrease in financial result was mostly related to the IFRS accounting of the convertible notes.

As of March 31, 2021, ERYTECH had cash and cash equivalents totaling €37.4 million (approximately $43.9 million), compared with €44.4 million on December 31, 2020. The €7.0 million decrease in cash position during the first quarter of 2021 was the result of a 3-month €7.6 million net cash utilization, which was mostly comprised of a €16.3 million net cash utilization in operating activities, €0.1 million used for investing activities and €8.8 million generated in financing activities, while the variation of the U.S. dollar against the euro led to a €0.6 million positive currency exchange impact.

Financing activities in the first quarter of 2021 included the placement of 744,186 newly issued ordinary shares underlying American Depositary Shares (ADSs) in the United States through the Company’s at-the-market (ATM) equity financing program for net proceeds of €6.4 million, and the draw down of one tranche under the convertible notes (OCABSA) financing agreement signed with Alpha Blue Ocean, for net proceeds of €2.9 million.

As of the date of this press release, all notes of the six OCABSA tranches called to date have been converted and, together with the shares issued under the ATM program, have resulted in the issuance of 3,677,676 new shares and 202,020 warrants, representing 18.3% of the Company’s outstanding share capital to date.

Financing Round of $30 million on April 29, 2021

On April 29, 2021, the Company announced that it has entered into definitive agreements with several health-care focused institutional and accredited investors for the purchase and sale of 1,034,483 units ("Units"), each Unit consisting of four ordinary shares in the form of ADSs and three warrants, each to purchase one ordinary share, in a registered direct offering to specified categories of investors.

The subscription price for one Unit is corresponding to $7.25 (€6.01) per ADS and associated 0.75 warrant. The warrants have an exercise price of €7.50 ($9.05) per share, will be immediately exercisable upon issuance and will expire two years from the issuance date.

The aggregate gross proceeds to the Company from the sale of the Units, excluding any exercise of the warrants, is expected to be approximately $30.0 million. The Company intends to use the net proceeds from this offering to fund further development of its products candidates, including through the completion of the Phase 3 TRYbeCA-1 trial and the costs to prepare the associated regulatory filings, and for associated working capital and other general corporate purposes.

The Company believes that its current cash position and the net proceeds from the April 29, 2021 offering can fund its planned operating expenses and current programs into the first quarter of 2022. Further, the Company believes that it would be able to fund operations into the third quarter of 2022 if it utilizes the OCABSA agreement, subject to the regulatory limit of 20% dilution.

Key News Flow and Milestones Expected Over the Next 12 Months

Final results from TRYbeCA-1 Phase-3 trial of eryaspase in 2L PAC (Q4 2021)

Potential eryaspase BLA filing for ALL (2H 2021)

Initial data from the randomized Phase 2 TRYbeCA-2 trial of eryaspase in TNBC (Q4 2021)

Determination of the maximum tolerated dose in rESPECT, Phase 1 IST in 1L PAC (2H 2021)

First Quarter 2021 Conference Call Details

ERYTECH management will hold a conference call and webcast on Wednesday, May 5, 2021 at 8:30am ET / 2:30 pm CET on the business highlights and financial results for the quarter ended March 31, 2021. Gil Beyen, CEO, Eric Soyer, CFO/COO, and Iman El-Hariry, CMO, will deliver a brief presentation, followed by a Q&A session.

The call is accessible via the below teleconferencing numbers, followed by the Conference ID#: 3887923#

USA/Canada: +1 (833) 818-6807 France: +33 1 70 80 71 53
International Dial-In Number: +1 (409) 350-3501 United-Kingdom: +44 2031070289

The webcast can be followed live online via the link: View Source

An archived replay of the call will be available for 7 days by dialing + 1 855 859 2056, Conference ID: 3887923#.

An archive of the webcast will be available on ERYTECH’s website, under the "Investors" section at investors.erytech.com

Financial Calendar 2021

Business Update and Financial Highlights for the Second Quarter of 2021: September 20, 2021 (after U.S. market close), followed by a conference call & webcast on September 21, 2021 (2:30pm CET/8:30am ET)

Business Update and Financial Highlights for the Third Quarter of 2021: November 15, 2021 (after U.S. market close), followed by a conference call & webcast on November 16, 2021 (2:30pm CET/8:30am ET)

ERYTECH plans on attending the following upcoming investor conferences:

Jefferies 2021 Global Healthcare Conference, June 1-4, New-York (Virtual)

JMP Securities Life Science Conference, June 16-17, New York (Virtual)

European Midcap Event – June 24, Paris (Virtual)

BTIG Biotechnology Conference – August 9-10, New York (Virtual)

On May 4, 2021 DURECT Corporation (Nasdaq: DRRX) reported financial results for the three months ended March 31, 2021 and provided a corporate update (Press release, DURECT, MAY 4, 2021, View Source [SID1234579075]).

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Q1 2021 Accomplishments:

Initiated patient dosing in the Phase 2b AHFIRM clinical study of DUR-928 in severe Alcohol-associated Hepatitis (AH)
FDA approval of POSIMIR in adults to produce post-surgical analgesia for up to 72 hours following Arthroscopic Subacromial Decompression
Publication of DUR-928’s mechanism of action
Appointment of two highly experienced biopharmaceutical board members
Further strengthened our financial position by raising $47.8 million in equity and ending Q1 with $97.2 million in cash and investments
"We are pleased with progress made in opening clinical trial sites in the U.S. and early enrollment in the AHFIRM trial, our Phase 2b clinical trial designed to evaluate the potential life-saving capability of DUR-928 in patients with severe Alcohol-associated Hepatitis," stated James E. Brown, D.V.M, President and CEO of DURECT. "We are also in discussions with multiple potential POSIMIR partners with a goal of enabling the partner to launch the product this year."

Financial highlights for Q1 2021:

Total revenues were $2.2 million and net loss was $10.1 million for the three months ended March 31, 2021 as compared to total revenues of $1.6 million and net loss of $9.9 million for the three months ended March 31, 2020.
At March 31, 2021, cash and investments were $97.2 million, compared to cash, cash held in escrow and investments of $56.9 million at December 31, 2020. Debt at March 31, 2021 was $21.0 million, compared to $20.8 million at December 31, 2020.
Update on Selected Programs:

Epigenetic Regulator Program. DUR-928, the lead product candidate in the Company’s Epigenetic Regulator Program, is an endogenous, orally bioavailable, first-in-class small molecule, which may have broad applicability in acute organ injuries such as alcohol-associated hepatitis (AH) as well as in chronic liver diseases such as non-alcoholic steatohepatitis (NASH).

Clinical Development

Alcohol-associated Hepatitis (AH)

Enrollment is ongoing in our Phase 2b study in subjects with severe acute AH to evaluate saFety and effIcacy of DUR-928 treatMent (AHFIRM). AHFIRM is a randomized, double-blind, placebo-controlled, international, multi-center Phase 2b study to evaluate the safety and efficacy of DUR-928 in approximately 300 patients with severe AH. The study is comprised of three arms targeting approximately 100 patients each: (1) Placebo plus standard of care (SOC, which may include the use of methylprednisolone, a corticosteroid, at the discretion of the treating physician); (2) DUR-928 (30 mg); and (3) DUR-928 (90 mg). All patients in the trial receive supportive care. Patients receive an intravenous (IV) dose of DUR-928 or placebo (sterile water) on day 1 and a second IV dose on day 4 if they are still hospitalized. The primary outcome measure is 90-day survival rate for patients treated with DUR-928 compared to those treated with placebo plus SOC. Secondary endpoints include 28-day survival, the rate of adverse events, Lille and MELD (prognostic scores) and time in the intensive care unit. The Company is targeting approximately 50-60 clinical trial sites in the U.S., U.K., Europe and Australia.
Reflecting the life-threatening nature of AH and the lack of therapeutic options for this devastating condition, the FDA granted DUR-928 Fast Track Designation for the treatment of AH in December 2020. The FDA grants Fast Track Designation to facilitate development and expedite the review of therapies with the potential to treat a serious condition where there is an unmet medical need. A therapeutic that receives Fast Track Designation may benefit from early and frequent communication with the agency in addition to a rolling submission of the marketing application, with the objective of getting important new therapies to patients more quickly.
Given the high mortality rate in severe AH patients and the absence of an approved therapeutic, we believe demonstration of a robust survival benefit in the AHFIRM trial may support an NDA filing.
In March 2021, a peer-reviewed research paper describing the binding sites and proposed mechanism of action of DUR-928 was published in The Journal of Lipid Research. The publication shows that DUR-928 (referred to in the paper as 25HC3S) binds to and inhibits the activity of DNA methyltransferases (DNMTs) DNMT-1, 3a and 3b, epigenetic regulating enzymes that add methyl groups to DNA (a process called DNA methylation). As such, by inhibiting DNMT activity, DUR-928 inhibits DNA methylation, thereby regulating the expression of genes that modulate crucial cellular activities, including those associated with cell death, stress response, and lipid biosynthesis. These modulations may lead to improved cell survival, and reduced lipid accumulation and inflammation, as has been observed in various in vivo animal models and in results from DURECT’s completed clinical trials in alcohol-associated hepatitis (AH) and non-alcoholic steatohepatitis (NASH).
During 2019, we completed a Phase 2a clinical trial of DUR-928 in patients with AH. All 19 patients treated with DUR-928 survived the 28-day follow-up period, 74% of patients (14/19) were discharged in ≤ 4 days after receiving a single dose of DUR-928, and there were no drug-related serious adverse events.
Alcohol-associated hepatitis (also called alcoholic hepatitis or AH) is an acute form of alcoholic liver disease (ALD) associated with long-term heavy intake of alcohol, and often occurs after a recent period of increased alcohol consumption. AH is typically characterized by a recent onset of jaundice and hepatic failure. According to the most recent data provided by the Agency for Healthcare Research and Quality (AHRQ), a part of the US Department of Health and Human Services (HHS), there were approximately 132,000 hospitalizations for patients with AH in 2018. From a 2018 publication analyzing the mortality and costs associated with AH, the cost per patient is estimated at over $50,000 in the first year. ALD is one of the leading causes of liver transplants in the U.S., costing over $875,000 per patient. An analysis of 77 studies published between 1971 and 2016, which included data from a total of 8,184 patients, showed the overall mortality from AH was 26% at 28 days and 29% at 90 days after admission.
Non-Alcoholic Steatohepatitis (NASH)

In May 2020, we reported positive topline results from a Phase 1b randomized and open-label clinical study conducted in the U.S. to evaluate safety, pharmacokinetics and signals of biological activity (including clinical chemistry and biomarkers as well as liver fat content and liver stiffness by imaging) of DUR-928 in NASH patients with stage 1-3 fibrosis. In this 65-patient study, reductions from baseline (pre-treatment) levels were seen in liver enzymes, liver stiffness as measured by imaging, serum lipids and biomarkers. Many of these reductions were statistically significant and DUR-928 was well tolerated at all three doses evaluated.
We will present additional data from this trial at an upcoming medical conference and we are working with a number of disease experts to determine next steps for DUR-928 in NASH.
Non-alcoholic fatty liver disease (NAFLD) is the most common form of chronic liver disease in both children and adults. It is estimated that NAFLD affects approximately 30% to 40% of adults and 10% of children in the United States. NASH, a more severe and progressive form of NAFLD, is one of the most common chronic liver diseases worldwide, with an estimated prevalence of 3-5% globally. No drug is currently approved for NAFLD or NASH.
POSIMIR (bupivacaine solution) Post-Operative Pain Relief Depot. POSIMIR is DURECT’s post-operative pain relief depot that uses the Company’s patented SABER technology that delivers bupivacaine to provide up to 3 days of post-surgical analgesia.

In February 2021, POSIMIR was granted U.S. FDA approval in adults for administration into the subacromial space under direct arthroscopic visualization to produce post-surgical analgesia for up to 72 hours following arthroscopic subacromial decompression. POSIMIR is the only approved sustained-release bupivacaine product indicated for up to 72 hours of post-surgical analgesia from a single application.
The approval was based on positive data from a randomized, multicenter, assessor-blinded, placebo–controlled clinical trial in patients undergoing arthroscopic subacromial decompression surgery with an intact rotator cuff. The primary outcome measures were mean pain intensity and total opioid rescue analgesia administered, both evaluated over the first 72 hours after surgery versus placebo. POSIMIR demonstrated a statistically significant improvement in both primary outcome measures: a 1.3 point, or 20%, reduction in mean pain intensity on a 0-10 point pain scale (p=0.01), and a 67% reduction in I.V. morphine-equivalent rescue opioid use, from a median of 12 mg in the placebo group to 4 mg in the POSIMIR group (p=0.01). In connection with this approval, the Company or its licensee, will be required to conduct two postmarketing non-clinical studies. Full Prescribing Information, including the Boxed Warning, is available at www.POSIMIR.com.
DURECT is in discussions with potential licensees of commercialization rights to POSIMIR, for which DURECT currently holds worldwide rights. Our goal is to put a commercial license in place in time for the licensee to launch the product in Q4 2021.
Subacromial decompression is a type of shoulder surgery used to treat impingement syndrome, a common repetitive-use injury that causes pain when the arm is raised over the head. The procedure is typically performed arthroscopically, meaning that several small incisions are made in the skin and muscle of the shoulder through which a camera lens (arthroscope) and surgical instruments are inserted during surgery. Arthroscopic subacromial decompression is generally considered outpatient surgery, and most patients go home within a few hours of surgery. The recovery period may extend from weeks to months, but the most intense pain typically occurs during the first 3 days after surgery and is often managed with oral opioids. There are over 600,000 surgeries involving arthroscopic subacromial decompression performed each year in the U.S.
Conference Call

We will host a conference call today at 4:30 p.m. Eastern Time / 1:30 p.m. Pacific Time to discuss first quarter 2021 results and provide a corporate update:

The conference call will also be available by webcast on DURECT’s homepage at www.durect.com under the "Investors" tab. If you are unable to participate during the webcast, the call will be archived on DURECT’s website under "Event Calendar" in the "Investors" section.

On May 4, 2021 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH) reported financial results for the first quarter ended March 31, 2021, and provided a corporate update (Press release, Deciphera Pharmaceuticals, MAY 4, 2021, View Source [SID1234579074]).

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"We are excited by the continuing successful commercial launch of QINLOCK in the U.S. as we solidify its position among GIST prescribers and patients and expand access to this important medicine globally. We also remain focused on realizing QINLOCK’s potential in earlier lines of therapy," said Steve Hoerter, President and Chief Executive Officer of Deciphera. "We expect the INTRIGUE Phase 3 top-line results in the fourth quarter of this year and believe QINLOCK has the potential to transform the treatment of GIST for this larger, second-line patient population. Building on our commitment to fully explore the potential of QINLOCK to benefit patients with GIST, we are excited to announce today our plans to initiate a Phase 1b/2 study combining QINLOCK with binimetinib, an approved MEK inhibitor. Our enthusiasm for this combination is based on compelling pre-clinical data showing that this combination can induce apoptosis and has the potential to deepen and prolong responses."

Mr. Hoerter continued, "We remain very pleased with the progress and growth for the balance of our pipeline, including the upcoming initiation of the Phase 1 study for our potential first-in-class ULK kinase inhibitor, DCC-3116, in patients with cancers driven by mutant RAS or RAF genes. We look forward to presenting updated data from both the vimseltinib and rebastinib programs in the coming months and plan to finalize registration-enabling studies for both programs before the end of the year."

First Quarter 2021 Highlights and Upcoming Milestones

QINLOCK(ripretinib)
Recorded $20.0 million in QINLOCK net product revenue in the first quarter of 2021, including $19.3 million in U.S. net product revenue.
Received approval in China from the China National Medical Products Administration (NMPA) and from the Hong Kong Department of Health, via our collaboration with Zai Lab, for the treatment of adult patients with fourth-line gastrointestinal stromal tumors (GIST).
Expects potential approval from the European Medicines Agency (EMA) for QINLOCK in the fourth quarter of 2021.
Expects to announce top-line results from the INTRIGUE Phase 3 study in the fourth quarter of 2021.
Expects to present data for QINLOCK patients undergoing intra-patient dose escalation after disease progression in the INVICTUS Phase 3 study at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June.
Today announced that the Company plans to initiate a Phase 1b/2 study of QINLOCK in combination with binimetinib, an approved MEK inhibitor, to address one of the potential mechanisms of resistance to kinase inhibition, reactivation of the MAPK pathway, in post-imatinib GIST patients.
A publication supporting this approach was recently published online by Molecular Cancer Therapeutics. The results showed that treatment with QINLOCK in combination with MEK inhibitors effectively induced and enhanced apoptotic responses and prevented growth of resistant colonies in both imatinib-sensitive and -resistant GIST cell lines.
Vimseltinib
Expects to present updated data from the ongoing Phase 1/2 study in patients with tenosynovial giant cell tumor (TGCT) in the third quarter of 2021.
Plans to finalize the pivotal development plan for vimseltinib in TGCT in the second half of 2021.
Rebastinib
Expects to present updated data from the ongoing Phase 1b/2 study of rebastinib in combination with paclitaxel in the endometrial cancer cohort at the ASCO (Free ASCO Whitepaper) Annual Meeting in June.
Expects to present updated data from the ongoing Phase 1b/2 study of rebastinib in combination with paclitaxel in the platinum-resistant ovarian cancer cohort in the third quarter of 2021.
Plans to finalize the pivotal development plan for rebastinib in combination with paclitaxel in the second half of 2021.
DCC-3116
Expects to initiate the Phase 1, multicenter, open-label, first-in-human study of DCC-3116 in the second quarter of 2021. The study will evaluate DCC-3116 as a single agent and in combination with trametinib in patients with advanced or metastatic tumors with a mutant RAS or RAF gene. Currently, expansion cohorts are planned in patients with advanced or metastatic pancreatic ductal adenocarcinoma with KRAS or BRAF mutations, non-small cell lung cancer with KRAS, NRAS, or BRAF mutations, colorectal cancer with KRAS, NRAS, or BRAF mutations, and melanoma with NRAS or BRAF mutations.
Upcoming Scientific Congress Presentations

2021 ASCO (Free ASCO Whitepaper) Annual Meeting, June 4-8. E-poster presentations will be available on-demand via the ASCO (Free ASCO Whitepaper) Meeting Library beginning on Friday, June 4 at 9:00 AM ET.
QINLOCK
E-poster presentation: Intra-patient dose escalation (IPDE) of ripretinib after disease progression in patients with advanced gastrointestinal stromal tumor (GIST): Analyses from the phase 3 INVICTUS study.
Rebastinib
E-poster presentation: Open-label, multicenter, phase 1b/2 study of rebastinib in combination with paclitaxel to assess safety and efficacy in patients with advanced or metastatic endometrial cancer.
First Quarter Financial Results

Revenue: Total revenue for the first quarter of 2021 was $25.2 million, which includes $20.0 million of net product revenue from sales of QINLOCK and $5.2 million of collaboration revenue. Net product revenues for the first quarter of 2021 included U.S. sales of QINLOCK of $19.3 million and ex-U.S. sales of QINLOCK of $0.7 million. The Company also recognized $5.0 million in collaboration revenue under its license agreement with Zai Lab based on the approval of QINLOCK in China. In the first quarter of 2020, the Company did not generate product revenue.
Cost of Sales: Cost of sales were $0.2 million in the first quarter of 2021. There were no cost of sales in the first quarter of 2020 as no product sales were generated during that period. Cost of sales will not be significant until the initial pre-launch inventory is depleted, and additional inventory is manufactured and sold.
R&D Expenses: Research and development expenses for the first quarter were $55.7 million, compared to $51.4 million for the same period in 2020. The increase was primarily due to personnel and preclinical costs, partially offset by a decrease in clinical trial expenses related to the INTRIGUE Phase 3 study in second-line GIST and the INVICTUS Phase 3 study in fourth-line and fourth-line plus GIST. Non-cash, stock-based compensation was $5.0 million and $3.3 million for the first quarters of 2021 and 2020, respectively.
SG&A Expenses: Selling, general and administrative expenses for the first quarter of 2021 were $30.7 million, compared to $23.9 million for the same period in 2020. The increase was primarily due to personnel costs as well as external spend related to professional fees, including those associated with establishing a targeted commercial infrastructure in key European markets to support a potential launch of QINLOCK in Europe, if approved. Non-cash, stock-based compensation was $6.2 million and $3.7 million for the first quarters of 2021 and 2020, respectively.
Net Loss: For the first quarter of 2021, Deciphera reported a net loss of $61.3 million, or $1.06 per share, compared with a net loss of $72.8 million, or $1.36 per share, for the same period in 2020. The decrease in net loss was primarily a result of product sales during the first quarter of 2021, partially offset by an increase in R&D and SG&A expenses as described above.
Cash Position: As of March 31, 2021, cash, cash equivalents and marketable securities were $502.2 million, compared to $561.3 million as of December 31, 2020. Based on its current operating plans, Deciphera expects its current cash, cash equivalents, and marketable securities together with anticipated product and royalty revenues, but excluding any potential future milestone payments or other payments under its collaboration or license agreements, will enable the Company to fund its operating and capital expenditures into the first half of 2023.
Conference Call and Webcast

Deciphera will host a conference call and webcast to discuss this announcement today, May 4, 2021 at 4:30 PM ET. To access the live call by phone please dial (866) 930-5479 (domestic) or (409) 216-0603 (international); the conference ID is 5470938. A live audio webcast of the event may also be accessed through the "Investors" section of Deciphera’s website at www.deciphera.com. A replay of the webcast will be available for 30 days following the event.

On May 4, 2021 Plus Therapeutics, Inc. (Nasdaq: PSTV) (the "Company"), a clinical-stage pharmaceutical company developing novel, targeted therapies for rare and difficult to treat cancers, reported two collaboration agreements to support its process development and analytical chemistry activities for the cGMP manufacturing of Rhenium NanoLiposome (RNL), the Company’s lead investigational asset in clinical development for recurrent glioblastoma (Press release, Cytori Therapeutics, MAY 4, 2021, View Source [SID1234579073]).

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Plus Therapeutics signed a pre-clinical, clinical, and process development agreement with Invicro LLC (Invicro), a Konica Minolta company, a global provider of imaging biomarkers, core lab services, advanced analytics and software solutions for drug discovery and development with best-in-class expertise in radiochemistry. Under this agreement, Invicro will characterize the current manufacturing process and develop in-process manufacturing controls for the RNL active pharmaceutical ingredient (API) and final drug product and provide future clinical trial imaging support and drug development consulting.

In addition, Plus Therapeutics entered into an agreement with Eurofins BioPharma Inc. ("Eurofins"), a market leader in analytical chemistry for discovery pharmacology and advanced materials sciences. Eurofins will develop and validate test methods for purity, composition, and identity of Re-BMEDA, the API in RNL. These test methods will support release testing and compliance with cGMP requirements for new drug substances.

"Process optimization and appropriate quality controls of investigational compounds are very critical aspects in bringing novel drugs to markets," said Marc H. Hedrick M.D., President and Chief Executive Officer of Plus Therapeutics. "We have identified two best-in-class partners in Invicro and Eurofins to help us get one step further in bringing RNL to a registrational clinical trial and ultimately commercial supply."

As previously disclosed, Plus Therapeutics entered into a master services agreement with Piramal Pharma Solutions for the development, manufacture, and supply of RNL intermediate of the drug product.

On May 4, 2021 Charles River Laboratories International, Inc. (NYSE: CRL) reported its results for the first quarter of 2021 (Press release, Charles River Laboratories, MAY 4, 2021, View Source [SID1234579072]). For the quarter, revenue was $824.6 million, an increase of 16.6% from $707.1 million in the first quarter of 2020.

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Acquisitions contributed 0.7% to consolidated first-quarter revenue growth. The impact of foreign currency translation benefited reported revenue growth by 2.9%. Excluding the effect of these items, organic revenue growth of 13.0% was driven by contributions from all three business segments. The year-over-year comparison to last year’s COVID-19-related revenue impact contributed approximately 140 basis points to the reported and organic revenue growth rates in the first quarter, principally in the Research Models and Services segment.

On a GAAP basis, first-quarter net income attributable to common shareholders was $61.5 million, an increase of 21.2% from net income of $50.8 million for the same period in 2020. First-quarter diluted earnings per share on a GAAP basis were $1.20, an increase of 17.6% from $1.02 for the first quarter of 2020. The increases in the GAAP net income and earnings per share were driven primarily by higher revenue and operating margin improvement, partially offset by debt extinguishment costs and the write-off of deferred financing costs related to debt refinancing activities in the first quarter of 2021.

On a non-GAAP basis, net income from continuing operations was $129.2 million for the first quarter of 2021, an increase of 40.7% from $91.8 million for the same period in 2020. First‑quarter diluted earnings per share on a non-GAAP basis were $2.53, an increase of 37.5% from $1.84 per share for the first quarter of 2020. The non-GAAP net income and earnings per share increases were driven primarily by higher revenue and operating margin improvement.

James C. Foster, Chairman, President and Chief Executive Officer, said, "Our first-quarter performance demonstrates the power of our unique, non-clinical portfolio and the strength of the biopharmaceutical market environment. A global focus on scientific innovation is driving record levels of investment in the biopharmaceutical industry, which is generating biomedical breakthroughs across multiple therapeutic areas at a rapid pace. We believe these factors are resulting in unprecedented client demand across most of our businesses."

"To maintain and enhance our position as the leading, non-clinical CRO, we are strategically expanding our portfolio and enhancing our scientific capabilities, especially in the use of more complex research techniques and advanced drug modalities such as cell and gene therapies. These investments are enabling us to offer greater value to our clients and capitalize on the significant growth opportunities," Mr. Foster concluded.

First-Quarter Segment Results

Research Models and Services (RMS)

Revenue for the RMS segment was $176.9 million in the first quarter of 2021, an increase of 21.2% from $146.0 million in the first quarter of 2020. The impact of foreign currency translation contributed 4.2%, and acquisitions, principally Cellero which was completed in August 2020, contributed 2.2% to first-quarter RMS revenue. Organic revenue growth of 14.8% was driven by robust demand for research models in China, as well as higher revenue for research models services, particularly Genetically Engineered Models and Services (GEMS). The year-over-year comparison to last year’s COVID-19-related revenue impact contributed approximately 620 basis points to the RMS revenue growth rate in the first quarter.

In the first quarter of 2021, the RMS segment’s GAAP operating margin increased to 25.4% from 18.7% in the first quarter of 2020. On a non-GAAP basis, the operating margin increased to 28.7% from 23.0% in the first quarter of 2020. The GAAP and non-GAAP operating margin increases were driven primarily by operating leverage from higher sales volume for research models.

Discovery and Safety Assessment (DSA)

Revenue for the DSA segment was $501.2 million in the first quarter of 2021, an increase of 14.2% from $438.7 million in the first quarter of 2020. The impact of foreign currency translation contributed 2.3% to DSA revenue growth. Organic revenue growth of 11.6% was primarily driven by robust demand from global biopharmaceutical and biotechnology clients in both the Discovery Services and Safety Assessment businesses.

In the first quarter of 2021, the DSA segment’s GAAP operating margin increased to 18.1% from 16.5% in the first quarter of 2020. On a non-GAAP basis, the operating margin increased to 23.8% from 22.0% in the first quarter of 2020. The GAAP and non-GAAP operating margin increases were driven primarily by operating leverage from higher revenue in both the Discovery Services and Safety Assessment businesses.

Manufacturing Support (Manufacturing)

Revenue for the Manufacturing segment was $146.5 million in the first quarter of 2021, an increase of 19.7% from $122.4 million in the first quarter of 2020. The impact of foreign currency translation contributed 4.1% to Manufacturing revenue growth. Organic revenue growth of 15.6% was driven by strong demand in the Biologics Testing Solutions (Biologics) and Microbial Solutions businesses.

In the first quarter of 2021, the Manufacturing segment’s GAAP operating margin increased slightly to 33.8% from 33.6% in the first quarter of 2020. On a non-GAAP basis, the operating margin decreased slightly to 35.5% from 35.6% in the first quarter of 2020.

2021 Guidance

On February 17, 2021, the Company provided 2021 financial guidance, both excluding and including the impact of the Cognate BioServices acquisition. The acquisition of Cognate was subsequently completed on March 29, 2021.

The Company is increasing its revenue growth and non-GAAP earnings per share guidance for 2021, as a result of the stronger-than-expected first quarter financial performance and an expectation that robust client demand trends will continue for the remainder of the year. This updated guidance includes the acquisitions that have already been completed in 2021, including Cognate.

Footnotes to Guidance Table:
(1) The contribution from acquisitions reflects only those acquisitions that have been completed.

(2) Organic revenue growth is defined as reported revenue growth adjusted for acquisitions and foreign currency translation.

(3) Acquisition-related amortization includes an estimate of $0.45-$0.65 for the impact of the Cognate acquisition and $0.05-$0.10 for other acquisitions completed in 2021 because the preliminary purchase price allocation has not been completed.

(4) These adjustments are related to the evaluation and integration of acquisitions, and primarily include transaction, advisory, and certain third-party integration costs, as well as certain costs associated with acquisition-related efficiency initiatives.

(5) These items primarily relate to charges of a) approximately $0.15 associated with U.S. and international tax legislation, and b) approximately $0.40 associated with debt extinguishment costs and the write-off of deferred financing costs related to debt refinancing.

(6) Venture capital and other strategic investment performance only includes recognized gains or losses. The Company does not forecast the future performance of these investments.

(7) Reconciliation of the current 2021 free cash flow guidance is as follows: Cash flow from operating activities of approximately $655 million, less capital expenditures of approximately $220 million, equates to free cash flow of approximately $435 million.

Webcast

Charles River has scheduled a live webcast on Tuesday, May 4th, at 9:00 a.m. ET to discuss matters relating to this press release. To participate, please go to ir.criver.com and select the webcast link. You can also find the associated slide presentation and reconciliations of GAAP financial measures to non-GAAP financial measures on the website.

Bank of America Health Care Conference Presentation

Charles River will virtually present at the Bank of America 2021 Health Care Conference, on Wednesday, May 12th, at 10:15 a.m. ET. Management will provide an overview of Charles River’s strategic focus and business developments.

A live webcast of the presentation will be available through a link that will be posted on ir.criver.com. A webcast replay will be accessible through the same website shortly after the presentation and will remain available for approximately two weeks.

Investor Day

Charles River will host a virtual Meeting with Management on Thursday, May 27th, beginning at 8:30 a.m. ET. Investors will have the opportunity to listen to a webcast of the virtual event through the Investor Relations section of the Company’s website at ir.criver.com. A replay will be accessible through the same website.

Non-GAAP Reconciliations

The Company reports non-GAAP results in this press release, which exclude often-one-time charges and other items that are outside of normal operations. A reconciliation of GAAP to non-GAAP results is provided in the schedules at the end of this press release.