On March 10, 2022 AcelRx Pharmaceuticals, Inc. (Nasdaq: ACRX), (AcelRx), a specialty pharmaceutical company focused on the development and commercialization of innovative therapies for use in medically supervised settings, reported its fourth quarter and full year 2021 financial results (Press release, AcelRx Pharmaceuticals, MAR 10, 2022, View Source [SID1234609937]).

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"We are executing on our strategy to expand and diversify our product portfolio by acquiring commercial-ready, or late-stage development-ready assets that address what we consider as unmet market needs for medically supervised settings. In particular, we are currently focused on the development and ultimate approval of our newly secured pre-filled syringes and Niyad (nafamostat) product candidates which could potentially provide multiple value creating catalysts for our shareholders in the near-term," said Vince Angotti, Chief Executive Officer of AcelRx. Mr. Angotti continued, "In addition, we have taken deliberate actions to adapt to the evolving healthcare environment as many medical procedures resulting in moderate-to-severe pain that were previously performed in surgical centers or hospitals have now been shifted to procedural suites, allowing us the opportunity to introduce DSUVIA into these settings. We have thus shifted our commercial resources, beginning in the third quarter, to focus on this new customer base, resulting in solid growth in DSUVIA unit sales in the fourth quarter 2021."

FY 2021 and Recent Highlights

AcelRx entered into a license agreement with Laboratoire Aguettant (Aguettant) providing AcelRx with two innovative pre-filled syringe product candidates for the U.S. The expected market opportunity for these two product candidates exceeds $100 million, and AcelRx currently plans to file New Drug Applications for both in 2022.

A second license transaction with Aguettant was completed establishing Aguettant as the commercial partner for DZUVEO in Europe with an expected launch in the third quarter of 2022. AcelRx is entitled to receive up to approximately $55 million in combined up-front and sales-based milestone payments.

AcelRx announced the closing of its acquisition of Lowell Therapeutics, Inc. (Lowell) in January 2022 in a transaction for consideration of approximately $32.5 million plus net cash acquired and certain other adjustments, and which includes up to approximately $26.0 million of contingent consideration payable in cash or stock at AcelRx’s option, upon the achievement of regulatory and sales-based milestones. Niyad (nafamostat) is the lead product, with a targeted indication of anticoagulation of the extracorporeal circuit, and which has received Breakthrough Device Designation from the FDA, as well as an ICD-10 procedural code from CMS which allows for reimbursement. Annual peak sales potential for Niyad is expected to exceed $200 million.

Since January 2021, six articles on DSUVIA were published reporting the benefits of administering DSUVIA in place of IV opioids, including reducing perioperative opioid use, rapid recovery times, efficacy and safety among a wide range of demographics and the overall advantages of sublingual delivery. Of note, one of these articles was a commentary published in Military Medicine, identifying DSUVIA as the next evolution in battlefield pain management. An additional study of DSUVIA for painful cosmetic procedures has been accepted for publication by the American Journal of Cosmetic Surgery and another study on the use of DSUVIA during general anesthesia for lengthy plastic surgery procedures has been submitted for publication.

As of December 31, 2021, AcelRx has achieved 725 approvals compared to our initial target of 615. As of February 28, 2022, AcelRx has achieved 813 formulary approvals for DSUVIA.

In February 2022, AcelRx was notified that it had met all requirements set by the U.S. Food and Drug Administration (FDA) with regards to the FDA Warning Letter regarding certain DSUVIA promotional materials, dated February 11, 2021, and a Closeout Letter is expected in Q1 2022.
Financial Information

The cash, cash equivalents and short-term investments balance was $51.6 million as of December 31, 2021.

8,960 units of DSUVIA were sold in the fourth quarter of 2021, compared to 3,710 units in the third quarter of 2021; however, the Company has recognized only $2 thousand in net revenues in the fourth quarter 2021 as a result of a $0.3 million reserve for potential returns related to a certain wholesale customer that purchased product for the Department of Defense (DoD) in 2020. The DoD has purchased exclusively from a secondary wholesale customer instead of their primary wholesaler, and therefore the Company has recorded a reserve in the event this product is not ultimately sold to the DoD.

Unit sales growth in the first two months of Q1 2022, compared to the first two months of Q4 2021 is 63%.

Combined R&D and SG&A expenses for the fourth quarter of 2021 totaled $6.9 million compared to $8.7 million for the fourth quarter of 2020. Excluding non-cash depreciation and stock-based compensation expense, these amounts were $5.6 million for the fourth quarter of 2021, compared to $7.5 million for the fourth quarter of 2020. R&D and SG&A expenses for the year ended December 31, 2021 totaled $35.0 million compared $40.3 million for the year ended December 31, 2020. Excluding non-cash depreciation and stock-based compensation expense, these figures were $29.7 million for the year ended December 31, 2021, compared to $35.4 million for the year ended December 31, 2020. The decrease in combined R&D and SG&A expenses in the fourth quarter and year ended 2021 was primarily due to reductions in personnel-related costs, including travel expense, partially offset by increased Catalent manufacturing-related DSUVIA development expenses.

Net loss for the fourth quarter of 2021 was $7.9 million, or $0.06 per basic and diluted share, compared to $8.9 million, or $0.10 per basic and diluted share, for the fourth quarter of 2020. Net loss for the year ended December 31, 2021 was $35.1 million, or $0.29 per basic and diluted share, compared to $40.4 million, or $0.47 per basic and diluted share, for the year ended December 31, 2020.
2022 Guidance

The Company’s 2022 year-end goals include the submission of two NDAs for its pre-filled syringe product candidates, pending outcome of FDA feedback that is expected in the second quarter, and the manufacturing of initial lots of nafamostat. Quarterly combined R&D and SG&A expense is expected to be approximately $9-$10 million (and $8-$9 million excluding stock compensation and depreciation). Annual debt service is expected to approximate $10 million as the Company continues to pay down amounts outstanding under its senior debt facility that matures in June 2023. Annual capital expenditures are expected to approximate $2 million attributed mainly to the final validation of the automated packaging line at AcelRx’s contract manufacturer.

2022 financial guidance is based on the Company’s current expectations and are forward-looking statements. Actual results could differ materially depending on market conditions and the factors set forth under Forward-Looking Statements below.

Webcast and Conference Call Information
As previously announced, AcelRx will host a live webcast Thursday, March 10th at 4:30 p.m. Eastern Time (1:30 p.m. Pacific Time) to discuss these financial results and provide other corporate updates. The webcast is accessible by visiting the Investors page of AcelRx’s website at View Source and clicking on the webcast link. The webcast will be accompanied by a slide presentation. Investors who wish to participate in the conference call may do so by dialing (866) 361-2335 for domestic callers, (855) 669-9657 for Canadian callers or (412) 902-4204 for international callers. A webcast replay will be available on the AcelRx website for 90 days following the call by visiting the Investor page of AcelRx’s website at View Source.

About DSUVIA (sufentanil sublingual tablet), 30 mcg
DSUVIA, to be marketed as DZUVEO in Europe, is indicated for use in adults in certified medically supervised healthcare settings, such as hospitals, surgical centers, and emergency departments, for the management of acute pain severe enough to require an opioid analgesic, and for which alternative treatments are inadequate. DSUVIA was designed to provide rapid analgesia via a non-invasive route of administration and to eliminate dosing errors associated with intravenous (IV) administration of opioid analgesics. DSUVIA is a single-strength solid dosage form administered sublingually via a single-dose applicator (SDA) by healthcare professionals. Sufentanil is an opioid analgesic previously only marketed for IV and epidural anesthesia and analgesia. The sufentanil pharmacokinetic profile when delivered sublingually avoids the high peak plasma levels and short duration of action observed with IV administration. The European Commission approved DZUVEO for marketing in Europe and it will be commercialized by AcelRx’s European partner, Aguettant.

For more information, including important safety information and black box warning for DSUVIA, please visit www.DSUVIA.com.

About nafamostat Nafamostat is a broad spectrum, synthetic serine protease inhibitor with anticoagulant, anti-inflammatory and potential anti-viral activities. Niyad is a lyophilized formulation of nafamostat and is currently being studied under an investigational device exemption, or IDE, as an anticoagulant for the extracorporeal circuit. Niyad has received Breakthrough Device Designation Status from the FDA. LTX-608 is a proprietary nafamostat formulation for direct IV infusion that we plan to potentially develop as a COVID anti-viral treatment, as well as for the treatment of disseminated intravascular coagulation (DIC), acute respiratory distress syndrome (ARDS), and acute pancreatitis.

On March 10, 2022 Myeloid Therapeutics, Inc. ("Myeloid"), a clinical stage mRNA-immunotherapy company developing novel therapies for cancer and autoimmune diseases, reported that company management will present a corporate overview at Oppenheimer’s 32nd Annual Healthcare Conference and will participate in one-on-one meetings with investors (Press release, Myeloid Therapeutics, MAR 10, 2022, View Source [SID1234609936]). The conference is being held in a virtual format, March 15-17, 2022.

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On March 10, 2022 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary genetic engineering platform technologies to create cell and gene therapeutics with the capacity to cure, reported updates and financial results for the fourth quarter and full year ended December 31, 2021 (Press release, Poseida Therapeutics, MAR 10, 2022, View Source [SID1234609935]).

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"We continued to demonstrate strong progress on our key priorities in the fourth quarter even as the pandemic impacted all parts of our industry. In the fourth quarter, we announced our first strategic collaboration with Takeda in gene therapy as well as the FDA IND clearance for P-MUC1C-ALLO1, our allogeneic CAR-T program in solid tumor indications," said Mark Gergen, Chief Executive Officer of Poseida. "We are highly focused on our key priorities for 2022, including initial clinical data for both the P-BCMA-ALLO1 and P-MUC1C-ALLO1 programs expected in the second half of the year, additional data on P-PSMA-101 in prostate cancer, and continuing progress in our gene therapy programs as we work toward applying our technologies to redefining cell and gene therapy."

Program Highlights

BCMA Program
P-BCMA-ALLO1 is the Company’s first allogeneic CAR-T program, currently being evaluated in patients with relapsed/refractory multiple myeloma (R/R MM) with initial clinical data expected in the second half of 2022. The planned shift in operational focus to allogeneic CAR-T was announced in the fourth quarter, as the Company began winding down the autologous BCMA program, P-BCMA-101.

The Company reported interim results from its Phase 1 clinical trial of P-BCMA-101 for the treatment of R/R MM at the 2021 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December. The results shown highlight that P-BCMA-101, a non-viral transposon-based autologous CAR-T, was well tolerated and demonstrated strong anti-tumor activity in advanced, late line R/R MM patients. The Company also highlighted findings from some of the novel dosing regimens explored during the trial, including the combination with rituxan, which demonstrated an ability to improve response rates, eliminate antibodies, and show an increase in both progression free survival and overall survival in that cohort. The full results of the ASH (Free ASH Whitepaper) update can be found on the Company’s website.

PSMA Program
P-PSMA-101 is an autologous CAR-T product candidate being developed to treat patients with metastatic castrate-resistant prostate cancer (mCRPC) currently in an ongoing Phase 1 dose escalation trial.

In February, the Company presented interim data at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Symposium (ASCO GU). The results presented showed notable responses even at the lowest doses in heavily pre-treated patients with mCRPC, including one patient who demonstrated evidence of near complete tumor elimination as evidenced by PSMA PET and other measures. The full ASCO (Free ASCO Whitepaper) GU presentation can be found on the Company’s website.

MUC1-C Program
P-MUC1C-ALLO1 is an allogeneic CAR-T product candidate with the potential to treat a wide range of solid tumors derived from epithelial cells, including breast and ovarian cancers. The Phase 1 clinical trial of P-MUC1C-ALLO1 is proceeding following IND clearance in the fourth quarter of 2021, and an update on the program is expected in the second half of 2022.

Liver-Directed Gene Therapy Programs
P-OTC-101 is the Company’s first liver-directed gene therapy program for the in vivo treatment of urea cycle disease caused by congenital mutations in the ornithine transcarbamylase (OTC) gene, a condition characterized by high unmet medical need.

The Company shared an update on the program during the R&D Day held in February 2022, including the presentation of data showing that use of a non-viral nanoparticle delivery system utilizing piggyBac AAV plus lipid nanoparticle potentially enables curative outcomes in a mouse model of severe OTC. The Company is currently evaluating the P-OTC-101 program to determine the best path forward and will update expected timing on program advancement once that evaluation is complete.

P-FVIII-101 is a liver-directed gene therapy currently partnered with Takeda Pharmaceuticals USA, Inc. (Takeda) and in development for the in vivo treatment of Hemophilia A. P-FVIII-101 utilizes piggyBac gene modification delivered via lipid nanoparticle and has demonstrated stable and sustained Factor VIII expression in animal models. Data from preclinical studies were shared during the R&D Day and showed that the biodegradable nanoparticle in combination with super piggyBac may overcome the limitations of AAV-based systems, with the following potential benefits: larger cargo capacity than AAV delivery alone, the ability to re-dose patients, the ability to treat pediatric patients, and fewer safety concerns.

Platforms and Emerging Technologies
The Company also reviewed its core platform technologies and introduced emerging technology programs at the R&D Day. These emerging programs highlight the Company’s continuing focus on innovation and include: (i) emerging preclinical work demonstrating the Company’s ability to include T cell receptors (TCRs), in addition to CARs in cell therapies; (ii) early preclinical data on a version of Site-Specific piggyBac gene insertion for site-directed DNA integration; and (iii) a CAR 3.0, an approach that utilizes genetically engineered hematopoietic stem cells to treat certain cancer indications. The full presentation is currently available for a limited period of time on the Company’s website.

For these and other discovery programs, the Company may seek partnerships or collaborations to move those applications forward.

Organizational Updates
The Company announced today that Kerry Ingalls, the Company’s Chief Operating Officer (COO), intends to retire effective May 13, 2022 and will continue in a strategic advisory role with the Company through September 2022. As previously announced, effective February 1, 2022, Mark Gergen, the Company’s President and former Chief Business Officer (CBO), assumed the role of Chief Executive Officer (CEO) and Eric Ostertag, the Company’s founder and former CEO, assumed the role of Executive Chairman of the board of directors. In February 2022, the Company announced the appointment of Brent Warner as President, Gene Therapy, to lead the Company’s gene therapy efforts including management of the collaboration with Takeda.

Financial Results for the Fourth Quarter and Full Year 2021

Revenues
Revenues were $31.2 million for both the fourth quarter and the full year ended December 31, 2021 consisting of revenue earned from the collaboration and license agreement with Takeda entered into in the fourth quarter of 2021, compared to no revenue for the same periods in 2020.

Research and Development Expenses
Research and development expenses were $39.1 million for the fourth quarter ended December 31, 2021, compared to $27.9 million for the same period in 2020. For the full year ended December 31, 2021, research and development expenses were $136.7 million, compared to $103.5 million for the same period in 2020. The increase was primarily related to an increase in personnel expenses due to an increase in headcount and an increase in stock-based compensation expense, an increase in external costs related to preclinical programs due to an increased number of early stage programs, an increase in external costs related to clinical stage programs including the enrollment and manufacturing for the P-BCMA-101 clinical trial and increased enrollment of the Phase 1 P-PSMA-101 trial, and an increase in internal costs related to facilities and other expenses primarily due to the increased activities in the pilot plant.

General and Administrative Expenses
General and administrative expenses were $9.6 million for the fourth quarter ended December 31, 2021, compared to $7.5 million for the same period in 2020. General and administrative expenses were $35.9 million for the full year ended December 31, 2021, compared to $23.0 million for the same period in 2020. The increase was primarily related to an increase in personnel expenses due to an increase in headcount combined with an increase in stock-based compensation expense, and increases in insurance costs and professional fees.

Net Income (Loss)
Net income was $1.5 million for the fourth quarter ended December 31, 2021 compared to net loss of $36.1 million for the same period in 2020. Net income for the fourth quarter was primarily driven by the revenue from the Company’s Takeda collaboration and the write-off of CIRM liability related to the P-BCMA-101 grant award. For the full year ended December 31, 2021, net loss was and $125.0 million compared to net loss of $129.8 million in 2020.

Cash Position
As of December 31, 2021, cash and cash equivalents balance was $206.3 million, which does not include net proceeds of $28.3 million from the restructuring of the Company’s debt arrangement, pursuant to that certain loan and security agreement with Oxford Finance LLC, that closed in the first quarter of 2022.

On March 10, 2022 ESSA Pharma Inc. ("ESSA", or the "Company") (NASDAQ: EPIX), a clinical-stage pharmaceutical company focused on developing novel therapies for the treatment of prostate cancer, reported that the Company will be presenting at the virtual Oppenheimer 32nd Annual Healthcare Conference on Thursday, March 17, 2022 at 2:40 p.m. Eastern Time (Press release, ESSA, MAR 10, 2022, View Source [SID1234609934]).

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David. R. Parkinson, President and Chief Executive Officer of ESSA Pharma, will participate in and host one-on-one meetings. Peter Virsik, ESSA’s Chief Operating Officer, and David S. Wood, ESSA’s Chief Financial Officer, will also be participating in the one-on-one meetings.

On March 10, 2022 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported its financial results for the fourth quarter and year ended December 31, 2021 and provided a corporate update (Press release, Regulus, MAR 10, 2022, View Source [SID1234609932]).

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"Last year was a transformational year for Regulus with the accomplishments in our ADPKD program including clinical validation of the target and the prioritization and advancement of our next generation compound RGLS8429," commented Jay Hagan, CEO of Regulus Therapeutics. "We are on track with an IND filing and initiation of our Phase 1 clinical study and with the recent equity financing we believe the Company is poised and capitalized to deliver on upcoming milestones."

Program Updates

RGLS8429 for ADPKD: In January 2022, the Company announced the successful completion of a pre-investigational new drug (Pre-IND) meeting with the U.S. Food and Drug Administration (FDA). The FDA provided overall agreement with the trial design and length of the Phase 1 study. Based on this feedback, the company is on track to submit an IND application in the second quarter of 2022 to obtain clearance for the initiation of the Phase 1 clinical trial. The Company anticipates reporting top-line data from the healthy volunteer portion of the study in the second half of 2022, and top-line biomarker data in the first cohort of RGLS8429-treated patients with ADPKD in the first half of 2023.

Lademirsen (RG-012) for Alport syndrome: In February 2022, the Company announced completion of enrollment in the Phase 2 HERA clinical study evaluating lademirsen for the treatment of adult patients with Alport Syndrome under the Company’s Collaboration and License Agreement with Sanofi. Final data is expected in the first half of 2023 and if successful could provide further validation of the Company’s platform technology designed to address genetic kidney diseases and earn the Company a $25 million milestone, furthering the cash runway into 2024.

Corporate Highlights

Closed $34.6 Million Private Placement: In November 2021, the Company announced the closing of a private placement of equity. The financing was led by the Federated Hermes Kaufmann Funds and New Enterprise Associates (NEA), with participation from other new and existing investors. The Company received gross proceeds of approximately $34.6 million from the sale of 58,923,352 shares of the Company’s common stock ("Common Stock") at a purchase price of $0.36 per share. In addition, the Company sold 3,725,720 shares of non-voting Class A-4 convertible preferred stock, in lieu of shares of Common Stock, at a price of $3.60 per share. Each share of non-voting Class A-4 convertible preferred stock is convertible into 10 shares of Common Stock, subject to certain beneficial ownership conversion limitations. Net proceeds from the transaction will be used for non-clinical and clinical development activities for the Company’s product candidates and general corporate purposes. SVB Leerink acted as the lead placement agent for the financing and H.C. Wainwright and Co. acted as co-placement agent.

Expanded Team: In December 2021, the Company announced the appointment of Mohammad Ahmadian, Ph.D., as Vice President, Chemistry and Pharmaceutical Development. Dr. Ahmadian has worked for various life sciences companies in research and managerial capacities. Most recently, he was Vice President & Resident Director of Kinovate Life Sciences, Inc.

Financial Results

Cash Position: As of December 31, 2021, Regulus had $60.4 million in cash and cash equivalents.

Research and Development (R&D) Expenses: Research and development expenses were $4.4 million and $17.8 million for the fourth quarter and year ended December 31, 2021, respectively, compared to $4.0 million and $15.3 million for the same periods in 2020, respectively. These amounts reflect internal and external costs associated with advancing our clinical and preclinical pipeline.

General and Administrative (G&A) Expenses: General and administrative expenses were $2.6 million and $10.0 million for the fourth quarter and year ended December 31, 2021, respectively, compared to $2.1 million and $8.8 million for the same periods in 2020, respectively. These amounts reflect personnel-related and ongoing general business operating costs.

Net Loss: Net loss was $7.1 million, or $0.07 per share (basic and diluted), and $27.8 million, or $0.32 per share (basic and diluted), for the fourth quarter and year ended December 31, 2021, respectively, compared to $1.3 million, or $0.03 per share (basic and diluted), and $15.7 million, or $0.45 per share (basic and diluted), respectively, for the same periods in 2020.

Conference Call and Webcast Information:
The Company will host a conference call and live audio webcast today at 5:00 p.m. ET to discuss its fourth quarter and year end 2021 financial results and corporate update. To access the call, please dial (877) 257-8599 (domestic) or (970) 315-0459 (international) and refer to conference ID 3786683. To access the telephone replay of the call, dial (855) 859-2056 (domestic) or (404) 537-3406 (international), passcode ID 3786683. The webcast and telephone replay will be archived on the Company’s website at www.regulusrx.com following the call.

About ADPKD

ADPKD, caused by the mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs. Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60.

About RGLS8429

RGLS8429 is a novel, next generation oligonucleotide designed to inhibit miR-17 and to preferentially target the kidney. Administration of RGLS8429 has shown robust data in preclinical models, where clear improvements in kidney function, size, and other measures of disease severity, as well as a superior pharmacologic profile have been demonstrated. Regulus has nominated RGLS8429 as a clinical candidate for the treatment of ADPKD.