On May 4, 2022 DiaMedica Therapeutics Inc. (Nasdaq: DMAC), a clinical-stage biopharmaceutical company focused on developing novel treatments for neurological disorders and kidney diseases, reported financial results for the quarter ended March 31, 2022 (Press release, DiaMedica, MAY 4, 2022, View Source [SID1234613526]). DiaMedica will host a conference call on Thursday, May 5, 2022, at 8:00AM Eastern Time / 7:00AM Central Time, to discuss its business update and first quarter financial results.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Clinical Developments

DM199 for the Treatment of Acute Ischemic Stroke

DiaMedica reported that it has increased the number of activated clinical trial sites to 9, more than doubling the number of active sites reported in March 2022 and is currently on track with its site activation goals for its ReMEDy2 trial, which is an adaptive design, randomized, double-blind, placebo-controlled trial studying the use of the Company’s product candidate, DM199, to treat acute ischemic stroke (AIS). DiaMedica believes that it has experienced slower than expected site activations and enrollment in the ReMEDy2 trial due primarily to staffing shortages and overall resourcing at study sites due to the COVID-19 pandemic and challenges of the study sites managing logistics and compliance for patients discharged from the hospital to an intermediate care facility. The Company is putting resources and processes in place to address both of these issues and expects continued improvement in site activations and patient enrollment over the course of 2022, subject to new pandemic related challenges which may arise for study sites.

DM199 for the Treatment of Chronic Kidney Disease

As previously reported, patient enrollment is completed in DiaMedica’s Phase 2 REDUX trial, which is a multi-center, open-label, investigation to assess the safety and efficacy of multiple doses of DM199, administered over 90 days, in participants with chronic kidney disease (CKD) (Stage 2 or 3).

In total, 79 patients were enrolled and initiated treatment, including 21 African American patients in Cohort 1, 25 patients with IgAN in Cohort 2 and 33 patients with Type 2 diabetes in Cohort 3. The last patient visit was completed in March 2022 and DiaMedica is working to complete the study close-out and the final data analysis while evaluating next steps for the CKD program.

Balance Sheet and Cash Flow

DiaMedica reported total cash and investments of $41.0 million, consisting of cash and cash equivalents of $3.0 million and marketable securities of $38.0 million, current liabilities of $1.4 million and working capital of $40.7 million as of March 31, 2022, compared to total cash and investments of $45.1 million, $1.5 million in current liabilities and $43.9 million in working capital as of December 31, 2021. The decreases in cash and investments and in working capital were due primarily to cash used to fund operating activities during the quarter ended March 31, 2022.

Net cash used in operating activities for the three months ended March 31, 2022, was $3.9 million compared to $4.3 million for the three months ended March 31, 2021. This decrease relates primarily to reduced effects of changes in operating assets and liabilities on the net cash used in operating activities in the current year period.

Financial Results

Research and development (R&D) expenses were $2.0 million for the three months ended March 31, 2022, compared with $2.4 million for the three months ended March 31, 2021, a decrease of $0.4 million. This decrease was driven mainly by a reduction in costs related to the REDUX CKD trial and a lower level of DM199 manufacturing process development work in the current year quarter as compared to the prior year quarter. These decreases were partially offset by increased costs incurred in the ReMEDy2 AIS trial and higher personnel costs related to the expansion of the clinical team in the current year period.

General and administrative (G&A) expenses were $1.6 million for the three months ended March 31, 2022, up from $1.2 million for the three months ended March 31, 2021. This $0.4 million increase resulted from a combination of increased professional services costs, directors’ and officers’ liability insurance and personnel costs incurred in support of expanding the operations and clinical programs.

Conference Call and Webcast Information

DiaMedica Management will host a conference call and webcast to discuss its business update and first quarter 2022 financial results on Thursday, May 5, 2022, at 8:00 AM Eastern Time / 7:00 AM Central Time:

Interested parties may access the conference call by dialing in or listening to the simultaneous webcast. Listeners should log on to the website or dial in 15 minutes prior to the call. The webcast will remain available for play back on DiaMedica’s website, under investor relations – events and presentations, following the earnings call and for 12 months thereafter. A telephonic replay of the conference call will be available until May 12, 2022, by dialing (800) 770-2030 (US Toll Free) and entering the replay passcode: 4814247.

About ReMEDy2 Trial

The ReMEDy2 trial is an adaptive design, randomized, double-blind, placebo-controlled trial studying the use of the Company’s product candidate, DM199, to treat AIS patients. The trial is intended to enroll approximately 350 patients at 75 sites in the United States. Patients enrolled in the trial will be treated for three weeks with either DM199 or placebo beginning within 24 hours of the onset of AIS symptoms, with the final follow-up at 90 days. The trial excludes patients treated with tissue plasminogen activator (tPA) and/or mechanical thrombectomy. The study population is representative of the approximately 80% of AIS patients who do not have treatment options today, primarily due to the limitations on treatment with tPA or mechanical thrombectomy. DiaMedica believes that the proposed trial has the potential to serve as a pivotal registration study of DM199 in this patient population.

The ReMEDy2 trial has two separate, independent, primary endpoints based upon both the results observed in the first ReMEDy1 phase 2 trial and published results from the urine-derived form of KLK1 used to successfully treat AIS in China. ReMEDy2 is powered for success with either endpoint: 1) physical recovery from stroke as measured by the well-established modified Rankin Scale (mRS) at day 90, and 2) the rate of ischemic stroke recurrence through day 90. Recurrent strokes represent 25% of all ischemic strokes, often occurring in the first few weeks after an initial stroke and are typically more disabling, costly, and fatal than initial strokes.

About DM199

DM199 is a recombinant (synthetic) form of human tissue kallikrein-1 (KLK1). KLK1 is a serine protease (protein) that plays an important role in the regulation of diverse physiological processes including blood flow, inflammation, fibrosis, oxidative stress and neurogenesis via a molecular mechanism that increases production of nitric oxide and prostaglandin. KLK1 deficiency may play a role in multiple vascular and fibrotic diseases such as stroke, chronic kidney disease, retinopathy, vascular dementia, and resistant hypertension where current treatment options are limited or ineffective. DiaMedica is the first company to have developed a recombinant form of the KLK1 protein. The KLK1 protein, produced from porcine pancreas and human urine, has been used to treat patients in Japan, China and South Korea for decades. DM199 is currently being studied in patients with acute ischemic stroke and patients with chronic kidney disease. In September 2021, the U.S. Food and Drug Administration granted Fast Track Designation to DM199 for the treatment of acute ischemic stroke.

On May 4, 2022 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company developing first-in-class antibodies focused on emerging immune control mechanisms applicable to inflammation and immuno-oncology indications, reported operating results for the first quarter ended March 31, 2022 and provided pipeline updates (Press release, AnaptysBio, MAY 4, 2022, View Source [SID1234613525]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are excited with our recently released Phase 1 trial results of ANB032, our anti-BTLA agonist, and the potential to broadly treat T and B-cell driven inflammatory diseases. Our recently initiated strategic portfolio review will define the path forward across a breadth of clinical development options of potential inflammation-focused indications that could be pursued for each of our clinical and preclinical therapeutic antibody programs," said Daniel Faga, interim president and chief executive officer of AnaptysBio. "We look forward to continuing to execute on our ongoing clinical trials with multiple readouts throughout the next 18 months."
Imsidolimab (Anti-IL-36 receptor) Program
•Imsidolimab, our investigational wholly owned anti-IL-36R therapeutic antibody, is in Phase 3 trials in generalized pustular psoriasis (GPP), and we anticipate top-line data from the GEMINI-1 Phase 3 clinical trial in Q4 2023.
•We anticipate top-line data from the HARP Phase 2 trial in moderate-to-severe hidradenitis suppurativa in Q3 2022.
Rosnilimab (Anti-PD-1 agonist) Program
•Rosnilimab, our investigational wholly owned anti-PD-1 agonist therapeutic antibody, previously known as ANB030, is in the AZURE Phase 2 clinical trial in moderate-to-severe alopecia areata, and we anticipate top-line data in H1 2023.
ANB032 (Anti-BTLA agonist) Program
•Announced positive top-line phase 1 data in April 2022 of ANB032, our investigational wholly owned anti-BTLA agonist antibody, demonstrating favorable safety and tolerability with no dose limiting toxicities were observed and no serious adverse events (SAEs) reported.
•ANB032 pharmacokinetic analyses demonstrated a favorable profile including an approximate two-week half-life and Full BTLA receptor occupancy rapidly within hours and was maintained for greater than 30 days.
•ANB032 also demonstrated rapid and sustained target engagement on both T cells and B cells.
•We are advancing ANB032 with an IND filing for a Phase 2 clinical trial anticipated in H2 2022.

First Quarter Financial Results
•Cash, cash equivalents and investments totaled $596.8 million as of March 31, 2022, compared to $615.2 million as of December 31, 2021, for a decrease of $18.4 million. The decrease relates primarily to cash used for operating activities offset by cash received from stock option exercises.
•Collaboration revenue was $1.0 million for the three months ended March 31, 2022, compared to $11.2 million for the three months ended March 31, 2021. The decrease relates primarily to one milestone achieved for JEMPERLI in the first quarter of 2021 for $10.0 million, and no milestones achieved in the first quarter of 2022.
•Research and development expenses were $22.5 million for the three months ended March 31, 2022, compared to $24.2 million for the three months ended March 31, 2021. The decrease was due primarily to reduced imsidolimab manufacturing costs offset by increased costs for the Company’s clinical programs. The R&D non-cash, stock-based compensation expense was $1.7 million for the three months ended March 31, 2022 as compared to $1.2 million in the same period in 2021.
•General and administrative expenses were $10.2 million for the three months ended March 31, 2022, compared to $5.4 million for the three months ended March 31, 2021. The increase was due primarily to $3.8 million of costs incurred for our former President and CEO’s resignation in the first quarter of 2022. The G&A non-cash, stock-based compensation expense was $6.1 million for the three months ended March 31, 2022 which includes $3.2 million of the $3.8 million one-time costs described earlier as compared to $2.1 million in the same period in 2021.
•Net loss was $36.3 million for the three months ended March 31, 2022, or a net loss per share of $1.31, compared to a net loss of $18.2 million for the three months ended March 31, 2021, or a net loss per share of $0.66.

On May 4, 2022 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) and Sumitomo Pharma Co., Ltd. (Head Office: Osaka, Japan; Representative Director, President, and CEO: Hiroshi Nomura; Securities Code: 4506, Prime Market of TSE) reported that the companies have entered into an exclusive licensing agreement under which Jazz has acquired development and commercialization rights in the United States, Europe and other territories for Sumitomo Pharma’s investigational DSP-0187, a potent, highly selective oral orexin-2 receptor agonist with potential application for the treatment of narcolepsy, idiopathic hypersomnia and other sleep disorders. Jazz has designated this molecule JZP441 (Press release, Jazz Pharmaceuticals, MAY 4, 2022, View Source [SID1234613524]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Sumitomo Pharma initiated a Phase 1 trial in Japan of DSP-0187 in November 2021 to evaluate safety, tolerability and pharmacokinetics in healthy volunteers. Jazz expects to rapidly advance development of DSP-0187 based on these clinical findings.

"Orexin agonism is an exciting area of sleep disorder research and an approach that may be complementary to oxybate therapy. We believe that DSP-0187 has the potential to advance the treatment of narcolepsy and other sleep disorders based on its profile," said Rob Iannone, M.D., M.S.C.E., executive vice president, global head of research and development of Jazz Pharmaceuticals. "As a leader in sleep medicine, we are committed to delivering innovative therapies to people who are living with debilitating sleep disorders. We look forward to leveraging our expertise in sleep disorders to rapidly advance this promising therapy into clinical trials in the U.S. and Europe. This agreement exemplifies both our ability to identify promising compounds that have the potential to improve patient care and our corporate objective of making strategic, value-creating investments in our pipeline."

"DSP-0187 is a potent, highly selective oral orexin-2 receptor agonist, which is a novel small molecule compound originally created by Sumitomo Pharma by utilizing our advanced expertise in drug discovery for central nervous system disorders. We are pleased to partner with Jazz on DSP-0187 given their strong track record of development success and commercial execution in sleep disorders and long-term commitment to advancing therapies to improve the lives of people with sleep disorders," said Toru Kimura, Representative Director, Executive Vice President of Sumitomo Pharma. "Jazz’s accomplishments in sleep medicine make the company an ideal partner for us as DSP-0187 enters its next phase of development."

Orexins are neuropeptides that play an important role in the regulation of sleep and wakefulness. DSP-0187 is a potent, highly selective orexin-2 receptor agonist designed to activate orexin signaling. DSP-0187 is initially planned to be evaluated in patients with narcolepsy, a debilitating sleep disorder which can manifest clinically with cataplexy, excessive daytime sleepiness (EDS), disrupted nighttime sleep (DNS), sleep paralysis and other symptoms. There is also potential for DSP-0187 to treat other sleep disorders.

Transaction Terms

Under the terms of the agreement, Jazz will receive an exclusive license to develop and commercialize DSP-0187 throughout the world except for Japan, China and certain other Asia/Pacific countries and regions, where Sumitomo Pharma will retain all development and commercialization rights. Sumitomo Pharma will receive an upfront payment of $50 million, and is eligible to receive development, regulatory and commercial milestone payments of up to $1.09 billion. Pending approval, Sumitomo Pharma is eligible to receive a tiered, low double-digit royalty on Jazz’s net sales of DSP-0187.

On May 4, 2022 DURECT Corporation (Nasdaq: DRRX) reported financial results for the three months ended March 31, 2022 and provided a corporate update (Press release, DURECT, MAY 4, 2022, View Source [SID1234613523]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Progress continues in the larsucosterol (DUR-928) AHFIRM trial, with more patients dosed in the first quarter of 2022 than in any prior quarter, reflecting the opening of additional clinical trial sites and our ongoing efforts to work with existing sites to recruit more patients with severe alcohol-associated hepatitis (AH) that meet our enrollment criteria," stated James E. Brown, D.V.M., President and CEO of DURECT.

First Quarter and Recent Business Highlights:

Continued progress in AHFIRM enrollment – DURECT has now dosed the first patients in Australia, France and Belgium, with 57 AHFIRM study sites now open at leading hospitals in the U.S., Australia, E.U. and U.K., a net increase of 6 sites in the two months since our last earnings call. At the pace of enrollment achieved in the first quarter of 2022, we would complete dosing the last patient in the AHFIRM trial in mid-2023; we expect the pace of enrollment should improve through our clinical site expansion, clinical trial engagement activities and the potential lessening of the impact of COVID on the hospitals participating in AHFIRM.
Engaging thought leaders and increasing awareness of AH – DURECT’s medical affairs team has been substantially increasing our AH market outreach and education efforts to amplify larsucosterol awareness and facilitate AHFIRM enrollment efforts. We have recently hosted the first of multiple regional AHFIRM study update meetings with our investigators and study site coordinators, sponsored and exhibited at three liver-focused congresses in person, and retained a medical communications agency to broaden awareness of AH, the AHFIRM trial and larsucosterol.
Approaching U.S. launch of POSIMIR by our licensee – DURECT signed an exclusive U.S. licensing agreement for POSIMIR with Innocoll Pharmaceuticals in December 2021. Under the agreement, DURECT will earn low to mid double-digit royalties from net sales of POSIMIR and is eligible to receive up to $136 million in upfront and milestone payments, including the $4 million upfront license fee received in January 2022, and a $2 million milestone payment upon the first commercial sale of POSIMIR, which is anticipated in Q2 2022.
Financial highlights for Q1 2022:

Total revenues were $1.9 million and net loss was $10.8 million for the three months ended March 31, 2022 compared to total revenues of $2.2 million and net loss of $10.1 million for the three months ended March 31, 2021.
At March 31, 2022, cash and investments were $64.4 million, compared to cash and investments of $70.0 million at December 31, 2021. Debt at March 31, 2022 was $20.8 million, compared to $20.6 million at December 31, 2021.
Earnings 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 2022 results and provide a corporate update:

A live audio webcast of the presentation will be also available by accessing DURECT’s homepage at www.durect.com and clicking "Investors." If you are unable to participate during the live webcast, the call will be archived on DURECT’s website under "Event Calendar" in the "Investors" section.

About the AHFIRM Trial
Enrollment is ongoing in our Phase 2b randomized, double-blind, placebo-controlled, international, multi-center study in subjects with severe acute alcohol-associated hepatitis (AH) to evaluate saFety and effIcacy of laRsucosterol (DUR-928) treatMent (AHFIRM). The study is comprised of three arms targeting enrollment of 300 total patients, with approximately 100 patients in each arm: (1) Placebo plus standard of care (SOC) which may include the use of methylprednisolone, a corticosteroid, at the discretion of the treating physician; (2) larsucosterol (30 mg); and (3) larsucosterol (90 mg). All patients in the trial receive supportive care. The primary outcome measure is 90-Day survival rate for patients treated with larsucosterol compared to those treated with placebo plus SOC. The Company is targeting more than 60 clinical trial sites across the U.S., EU, U.K., and Australia. Reflecting the life-threatening nature of AH and the lack of therapeutic options, the U.S. Food and Drug Administration (FDA) has granted larsucosterol Fast Track Designation for the treatment of AH. We believe demonstration of a robust survival benefit in the AHFIRM trial would support an NDA filing. For more information, refer to ClinicalTrials.gov Identifier: NCT04563026.

About Alcohol-associated Hepatitis (AH)
AH is a life-threatening acute alcohol-associated liver disease (ALD) often caused by chronic heavy alcohol use and a recent period of increased alcohol consumption (i.e., a binge). It is characterized by severe inflammation and destruction of liver tissue (i.e., necrosis), potentially leading to life-threatening complications including liver failure, acute renal injury and multi-organ failure. There are no FDA approved therapies for AH and 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, 29% at 90 days and 44% at 180 days. A subsequent global study published in December 2021, which included 85 tertiary centers in 11 countries across 3 continents, prospectively enrolled 2,581 AH patients with a median MELD score of 23.5, reported mortality at 28 and 90 days of 20% and 31% respectively. Stopping alcohol consumption is not sufficient for recovery in many moderate and severe patients and the use of treatments to reduce liver inflammation, such as corticosteroids, are limited by contraindications and have been shown to provide no survival benefit at 90 days or 1 year. While liver transplantation is becoming more common for alcoholic liver disease patients, including AH patients, the procedure involves a long waiting period, a burdensome selection process, and costs more than $875,000 on average.

About Larsucosterol (DUR-928)
Larsucosterol is an endogenous sulfated oxysterol and an epigenetic regulator. Epigenetic regulators are compounds that regulate patterns of gene expression without modifying the DNA sequence. DNA hypermethylation, an example of epigenetic dysregulation, results in transcriptomic reprogramming and cellular dysfunction, and has been found to be associated with many acute (e.g., AH) or chronic diseases (e.g., NASH). As an inhibitor of DNA methyltransferases (DNMT1, DNMT3a and 3b), larsucosterol inhibits DNA methylation, which subsequently regulates expression of genes that are involved in cell signaling pathways associated with stress responses, cell death and survival, and lipid biosynthesis. This may ultimately lead to improved cell survival, reduced inflammation, and decreased lipotoxicity. As an epigenetic regulator, the proposed mechanism of action provides further scientific rationale for developing larsucosterol for the treatment of acute organ injury and certain chronic diseases.

On May 4, 2022 Adaptive Biotechnologies Corporation ("Adaptive Biotechnologies") (Nasdaq: ADPT), a commercial stage biotechnology company that aims to translate the genetics of the adaptive immune system into clinical products to diagnose and treat disease, reported financial results for the quarter ended March 31, 2022 (Press release, Adaptive Biotechnologies, MAY 4, 2022, View Source [SID1234613522]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We started the year strong and completed a strategic reorganization of our company around two key business areas: MRD and Immune Medicine," said Chad Robins, chief executive officer and co-founder of Adaptive Biotechnologies. "I am encouraged by the solid momentum across both business areas as we optimize our resource allocation to capitalize on the multiple opportunities ahead."

Recent Highlights

Revenue of $38.6 million for the first quarter 2022, representing a 0.5% increase from the first quarter 2021.

clonoSEQ test volume in the first quarter 2022 grew 45% versus the first quarter of prior year and 12% over the fourth quarter of 2021.

Signed expanded MRD pan-portfolio agreement with major partner in MM and CLL for the use of MRD status as a clinical endpoint.

Recognized $3.0 million in MRD regulatory milestone revenue resulting from a biopharmaceutical partner who used data from our MRD assay to support its drug approval.

Completed analysis from immuneSense Lyme blinded data from 990 participants, confirming T-Detect Lyme is nearly twice as sensitive as the current standard of care (54% T-Detect Lyme vs 30% STTT sensitivity, both at 99% specificity). T-Detect Lyme offering to be made available during 2022 Lyme season.

Entered a new T-MAP collaboration with the Janssen Pharmaceutical Companies of Johnson & Johnson to map T cell responses to RSV to support Janssen’s RSV vaccine program.

Announced the appointment of Tycho Peterson as chief financial officer, who brings several decades of financial leadership and experience within the life science and diagnostic industries.

First Quarter 2022 Financial Results

Revenue was $38.6 million for the quarter ended March 31, 2022, representing a 0.5% increase from the first quarter in the prior year. Immune Medicine revenue was $20.8 million for the quarter, representing a 4% increase from the first quarter in the prior year. MRD revenue was $17.8 million for the quarter, representing a 3% decrease from the first quarter in the prior year.

Operating expenses were $101.7 million for the first quarter of 2022, compared to $79.7 million in the first quarter of the prior year, representing an increase of 28%.

Net loss was $62.8 million for the first quarter of 2022, compared to $40.6 million for the same period in 2021.

Cash, cash equivalents and marketable securities was $500.7 million as of March 31, 2022.

2022 Financial Guidance

Adaptive Biotechnologies reiterates full year 2022 revenue to be in the range of $185 million to $195 million.

Webcast and Conference Call Information

Adaptive Biotechnologies will host a conference call to discuss its first quarter 2022 financial results after market close on Wednesday, May 4, 2022 at 4:30 PM Eastern Time. The conference call can be accessed at View Source The webcast will be archived and available for replay at least 90 days after the event.