On February 24, 2022 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-inflammatory diseases, reported its financial results for the fourth quarter and full year of 2021 and provided a corporate update (Press release, Aclaris Therapeutics, FEB 24, 2022, View Source [SID1234608927]).

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!

"2021 was a tremendous year for the progression of our drug development pipeline, and I’m very proud of what our team has accomplished," said Dr. Neal Walker, President & CEO of Aclaris. "We reported positive data for our Phase 2a trials of zunsemetinib in subjects with moderate to severe rheumatoid arthritis (RA) and ATI-1777 in subjects with moderate to severe atopic dermatitis (AD), and strengthened our balance sheet to continue this momentum in 2022. Moving forward, we are progressing zunsemetinib in three immuno-inflammatory indications, moving ATI-1777 forward in moderate to severe AD, and progressing ATI-2138 in SAD/MAD studies. Our KINect drug discovery platform continues to be productive and we now have three clinical-stage compounds as well as an early-stage immuno-inflammatory and oncology pipeline. We have the privilege of working toward the goal of helping address the needs of patients with immuno-inflammatory diseases as well as cancer and look forward to progressing our assets to achieve this goal."

Research and Development Highlights:

The global COVID-19 pandemic continues to rapidly evolve and has caused and may continue to cause Aclaris to experience disruptions that could impact the timing of its research and development and regulatory activities listed below.

Clinical Programs

Zunsemetinib, an investigational oral small molecule MK2 inhibitor:
Currently being developed as a potential treatment for immuno-inflammatory diseases
ATI-450-RA-202: This Phase 2b dose ranging trial to investigate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of multiple doses (20 mg and 50 mg twice daily) of zunsemetinib in combination with methotrexate in subjects with moderate to severe RA is ongoing.
Aclaris anticipates increasing the size of the patient population from approximately 195 to approximately 240 subjects and expects topline data in 2023.
ATI-450-HS-201: This Phase 2a trial to investigate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of zunsemetinib (50 mg twice daily) in subjects with moderate to severe HS is ongoing.
Aclaris expects topline data in the first half of 2023.
ATI-450-PsA-201: Aclaris plans to progress zunsemetinib (50 mg twice daily) into a Phase 2a trial in subjects with moderate to severe psoriatic arthritis in the first half of 2022.
ATI-1777, an investigational topical "soft" Janus kinase (JAK) 1/3 inhibitor:
Currently being developing as a potential treatment for moderate to severe AD
Aclaris plans to progress ATI-1777 into a Phase 2b trial in subjects with moderate to severe AD in the first half of 2022. In this trial, Aclaris plans to explore multiple concentrations of twice daily treatment with ATI-1777 and a single concentration of once daily treatment with ATI-1777, in patients 12 years and older.
ATI-2138, an investigational oral ITK/TXK/JAK3 (ITJ) inhibitor:
Currently being developed as a potential treatment for T cell-mediated autoimmune diseases
ATI-2138-PKPD-101: This Phase 1 single ascending dose (SAD) trial to investigate the safety, tolerability, pharmacokinetics and pharmacodynamics of ATI-2138 in healthy subjects is ongoing.
Aclaris expects topline data in 2022.
If the Phase 1 SAD trial is successful, Aclaris currently plans to initiate a two-week Phase 1 multiple ascending dose trial of ATI-2138 in subjects with psoriasis in 2022. Aclaris is also currently exploring alternative indications to the planned indication that are relevant to the mechanism of action.
Preclinical Programs

ATI-2231, an investigational oral MK2 inhibitor compound:
Currently being explored as a potential treatment for pancreatic cancer and metastatic breast cancer as well as in preventing bone loss in patients with metastatic breast cancer
Second MK2 inhibitor generated from Aclaris’ proprietary KINect drug discovery platform and designed to have a long half-life.
IND-enabling studies are underway, and Aclaris expects to submit an IND by the end of 2022.
Discovery Programs

Currently developing oral gut-biased JAK inhibitors with limited systemic exposure as potential treatments for inflammatory bowel disease.
Central nervous system (CNS) kinase inhibitor targets:
Currently engaged in research to identify brain penetrant kinase inhibitor candidates and assess their impact on neuronal pro-inflammatory cytokine production, microglia growth and survival, and neurodegeneration.
Other Highlights:

James Loerop appointed as Chief Business Officer in January 2022.
Aclaris plans to hire additional key leadership positions over the coming months to support its operational plans and strategic direction.
Financial Highlights:

Liquidity and Capital Resources

As of December 31, 2021, Aclaris had aggregate cash, cash equivalents and marketable securities of $225.7 million compared to $54.1 million as of December 31, 2020. The primary factors for the change in cash, cash equivalents and marketable securities during the year ended December 31, 2021 included:

Net cash used in operating activities of $52.1 million. This amount was comprised of the following:
$90.9 million net loss
$1.3 million cash used from changes in operating assets and liabilities
$24.3 million of non-cash charges for the revaluation of contingent consideration
$14.1 million of non-cash stock-based compensation expense
$1.7 million of other non-cash charges
Net cash used to repay outstanding debt and fees of $11.5 million in July 2021.
Aggregate net proceeds of $238.2 million from public offerings in January 2021 and June 2021 in which Aclaris sold a total of 14.4 million shares of common stock.
Aclaris anticipates that its cash, cash equivalents and marketable securities as of December 31, 2021 will be sufficient to fund its operations through the end of 2024, without giving effect to any potential business development transactions or financing activities.

Financial Results

Fourth Quarter 2021

Net loss was $22.8 million for the fourth quarter of 2021 compared to $13.2 million for the fourth quarter of 2020.
Total revenue was $1.5 million for the fourth quarter of 2021 compared to $1.6 million for the fourth quarter of 2020.
Research and development (R&D) expenses were $14.1 million for the quarter ended December 31, 2021 compared to $9.0 million for the prior year period.
The $5.1 million increase was the result of additional zunsemetinib expenses, including costs associated with clinical development activities for a Phase 2b trial for moderate to severe RA and a Phase 2 trial for moderate to severe HS and preclinical development activities related to ATI-2231.
General and administrative (G&A) expenses were $6.9 million for the quarter ended December 31, 2021 compared to $4.9 million for the prior year period.
The $2.0 million increase was primarily the result of higher compensation-related costs, including stock-based compensation, as well as higher accounting, compliance and professional fees. Severance payments relating to the retirement of our former Chief Legal Officer also contributed to the increase.
Revaluation of contingent consideration charges related to the Confluence acquisition was $2.2 million for the quarter ended December 31, 2021 compared to $0 for the prior year period.
Full Year 2021

Net loss was $90.9 million for the year ended December 31, 2021 compared to $51.0 million for the year ended December 31, 2020.
Total revenue was $6.8 million for the year ended December 31, 2021 compared to $6.5 million for the year ended December 31, 2020.
R&D expenses were $43.8 million for the year ended December 31, 2021 compared to $29.3 million for the prior year period.
The $14.5 million increase was primarily the result of additional zunsemetinib expenses, including costs associated with drug candidate development and clinical development activities for a Phase 2b trial for moderate to severe RA and a Phase 2a trial for moderate to severe HS. Continued investment in the further development of Aclaris’ immuno-inflammatory drug development pipeline also contributed to the increase as Aclaris progressed toward the October 2021 IND submission for ATI-2138 and began pre-clinical development activities on ATI-2231.
G&A expenses were $23.6 million for the year ended December 31, 2021 compared to $20.5 million for the prior year period.
The $3.1 million increase was primarily the result of higher stock-based compensation costs as well as higher accounting, compliance and professional fees. Severance payments relating to the retirement of our former Chief Legal Officer also contributed to the increase.
Revaluation of contingent consideration charges related to the Confluence acquisition was $24.3 million for the year ended December 31, 2021 compared to $2.4 million for the prior year period.

On February 24, 2022 Calliditas Therapeutics reported that Year-End Report, 2021 (Press release, Calliditas Therapeutics, FEB 24, 2022, View Source [SID1234608923])

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!

"The accelerated approval of TARPEYO was for the indication of reduction of proteinuria, which was the endpoint of Part A of the trial, in adult patients with IgA nephropathy with risk of rapid progression, which is characterized as generally UPCR ≥ 1.5 g/g. It is well established that patients with higher levels of proteinuria have a worse outlook and prognosis as they tend to experience a more rapid decline of their kidney function, as measured by eGFR. These patients are therefore at considerable risk of progressing to ESRD, resulting in the need for dialysis or transplantation. We are obviously thrilled to be able to bring a medication to all these patients, who before now did not have any approved treatment options that addressed their illness.

This undertaking has taken well over a decade to reach patients and is the result of the incredibly hard work and dedication of a diverse and extraordinary group of people, working as a team towards a common goal. It has been a privilege to help guide and participate in this amazing journey over the last 4 years, and I am confident that this is just the beginning of our evolution into a broad-based biopharma business with the requisite talent, resources and science to continue to deliver enduring value to all of our stakeholders."

Renée Aguiar-Lucander, CEO

Summary of Q4 2021
October 1 – December 31, 2021

Net sales amounted to SEK 31.2 million and SEK 0.4 million for the three months ended December 31, 2021 and 2020, respectively.
Operating loss amounted to SEK 222.1 million and SEK 135.9 million for the three months ended December 31, 2021 and 2020, respectively.
Loss per share before and after dilution amounted to SEK 4.19 and SEK 3.41 for the three months ended December 31, 2021 and 2020, respectively.
Cash amounted to SEK 955.5 million and SEK 996.3 million as of December 31, 2021 and 2020, respectively.
Significant events during Q4 2021, in summary
In December 2021, Calliditas announced that the US Food and Drug Administration (FDA) had granted accelerated approval for TARPEYO (budesonide) delayed release capsules indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a urine protein-to-creatinine ratio (UPCR) ≥1.5g/g. TARPEYO is the first and only FDA-approved treatment for this disease and was designed specifically to target the origin of IgA nephropathy. This approval marked the successful transition for Calliditas to a commercial-stage biopharmaceutical company.

On February 23, 2022 Blue Water Vaccines Inc. ("BWV" or "Blue Water Vaccines" or the "Company"), a biopharmaceutical company developing vaccines, reported the closing of its initial public offering of 2,222,222 shares of its common stock at a public offering price of $9.00 per share (Press release, Onconetix, FEB 23, 2022, View Source [SID1234641105]). The shares began trading on the Nasdaq Capital Market under the ticker symbol "BWV" on February 18, 2022.

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!

The aggregate gross proceeds to BWV from the initial public offering were approximately $20 million, prior to deducting underwriting discounts, commissions, and other estimated offering expenses.

Boustead Securities, LLC acted as the sole book-running manager for the offering.

The Securities and Exchange Commission ("SEC") declared effective a registration statement on Form S-1 relating to these securities on February 11, 2022. A final prospectus relating to this offering was filed with the Securities and Exchange Commission on February 22, 2022. The offering was made only by means of a prospectus, copies of which may be obtained from; Boustead Securities, LLC, Attention: Prospectus Department, 6 Venture, Suite 325, Irvine, CA 92618, or by telephone at 949-502-4408 or by email at [email protected]. Investors may also obtain these documents at no cost by visiting the SEC’s website at View Source

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

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!

On February 23, 2022 Photocure ASA reported Hexvix/Cysview revenues of NOK 94.2 million in the fourth quarter of 2021 (Q4 2020: NOK 97.7 million) (Press release, , FEB 23, 2022, View Source,million%20(NOK%2015.2%20million). [SID1234611427]). Unit sales in the U.S. and Europe rose 11% and 12%, respectively, while the revenue comparison was impacted by a one-time reversal of an accrual in the prior-year period and foreign exchange. Photocure has a solid pipeline of anticipated Blue Light Cystoscopy (BLC) tower installations in the U.S. and expects placements to accelerate during the second half of 2022.

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!

"Photocure showed strong execution and resilience in 2021 as we continued to grow our U.S. business and successfully completed our first full year of operations in continental Europe despite several surges of Covid-19. While product revenue declined 4% in the fourth quarter of 2021 due to a one-time accrual reversal in Q4 2020 and the impact of foreign exchange, fourth quarter unit sales in the U.S. and Europe rose 11% and 12%, respectively year-over-year. We are particularly pleased with the progress that we made during the 2-year pandemic period, given the challenging conditions. Procedure volumes for bladder cancer resection surgeries in the U.S. declined 11%, while we grew Cysview unit volume by 24% from 2019 to 2021, resulting in an increase in our market penetration," says Daniel Schneider, President & Chief Executive Officer of Photocure.

Photocure reported total group revenues of NOK 94.5 million in the fourth quarter of 2021 (NOK 97.9 million), and an EBITDA* of NOK -5.5 million (NOK 15.2 million). Adjusted for the one-time reversal of an accrual in the fourth quarter of 2020, Hexvix/Cysview revenues increased 5% in the fourth quarter 2021 and were up 10% in constant currencies. The EBIT ended at NOK -11.5 million (-6.2) and the cash balance at the end of the fourth quarter 2021 was NOK 322.9 million. The installed base of rigid cystoscopes in the U.S. was 311 at the end of the fourth quarter, an increase of 43 units or 16% since the same period in 2020, including a total base of 48 flexible cystoscopes.

"Demand for BLC equipment remains very strong, and while some customers are awaiting the launch of the new Karl Storz system, we placed 9 towers in the fourth quarter. Our backlog for new capital equipment continues to grow, and we are extremely pleased that Karl Storz’s new blue light system was approved by the FDA earlier this month. As a result, both Photocure and Karl Storz are energized and preparing to launch this new system in the coming months," Schneider adds.

The Company’s pipeline of anticipated BLC tower installations in the U.S. remains robust given the demand by physicians and facilities to offer the benefits of BLC with Cysview in their practices. With the new Karl Storz blue light system anticipated to launch throughout the U.S. in the second quarter of 2022, Photocure expects that the placements of new blue light rigid towers will accelerate in the back half of the year. Meanwhile, the Company will continue to focus on increasing the use of Cysview in existing accounts as well as placing flexible BLC equipment, for which installation momentum is increasing.

"While the worldwide pandemic required us to take measures to manage through a period of great uncertainty, we used that time and deployed the necessary resources to position our global commercial organization to drive significant Hexvix/Cysview growth once healthcare access returns to normal. With our European operations fully integrated, a new high-definition BLC system launching in the U.S. market, and Covid-19 becoming better managed, we believe that 2022 will be an exciting year for Photocure as we help more bladder cancer patients realize the benefits of Hexvix/Cysview," Schneider concludes.

Please find the full financial report and presentation enclosed.

EBITDA* and other alternative performance measures (APMs) are defined and reconciled to the IFRS financial statements as a part of the APM section of the fourth quarter 2021 financial report on page 24.

Photocure will present its fourth quarter 2021 report on Wednesday 23 February 2022 at 14:00 CET. The investor presentation will be streamed live and be hosted by Daniel Schneider, CEO and Erik Dahl, CFO.

The presentation will be held in English and questions can be submitted throughout the event. The streaming event is available through https://channel.royalcast.com/landingpage/hegnarmedia/20220223_10/