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Janux Therapeutics Reports Second Quarter 2021 Financial Results

On August 10, 2021 Janux Therapeutics, Inc. (Nasdaq: JANX) (Janux), a biopharmaceutical company developing novel T cell engager immunotherapies based on its TRACTr platform technology, reported financial results for the quarter ended June 30, 2021 (Press release, Janux Therapeutics, AUG 10, 2021, View Source [SID1234586226]).

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"With the successful completions of a Series B financing and IPO this past quarter, we are well-capitalized to advance our novel TRACTr platform technology and next-generation T cell engager immunotherapies, with the potential to improve the treatment of cancer," said David Campbell, Ph.D., President and CEO of Janux. "Janux has also significantly strengthened the organization with key hires to our senior management team. These team members bring invaluable expertise and decades of drug development experience, specifically in T cell therapeutics, to propel our broad preclinical pipeline toward the clinic."

Recent Business Highlights:

TRACTr product candidates advancing as planned. Janux’s lead TRACTr programs of next-generation T cell engagers for PSMA, EGFR, TROP2, and its costimulatory bispecific TRACIr program remain on track as they move through preclinical development. Janux continues to expect to submit at least two Investigational New Drug (IND) applications by the end of 2022.
Strengthened management team with appointment of Shahram Salek-Ardakani, Ph.D., as Chief Scientific Officer, and Wayne Godfrey, M.D., as Chief Medical Officer. Dr. Salek-Ardakani brings more than 20 years of experience as a scientific leader and drug developer in multiple disease areas, most recently serving as the Senior Director of Cancer Immunology at Pfizer, Inc., where he was responsible for formulating first-in-class discovery programs and platform initiatives to implement Pfizer’s T cell targeted therapeutics strategy. Dr. Godfrey brings more than 25 years of drug development, clinical strategy, and research experience in cancer immunology and immunotherapy at leading pharmaceutical and biotechnology companies, most recently serving as the Vice President of Clinical Development at IGM Biosciences, Inc., where he led the global clinical development of IGM’s emerging pipeline of proprietary bispecific T cell engaging IgM antibodies.
Completed Series B financing and initial public offering (IPO). In April 2021, Janux completed a Series B financing, raising gross proceeds of $125.0 million. In June 2021, Janux completed an initial public offering, selling 13,110,000 shares of its common stock at a price to the public of $17.00 per share, for aggregate gross proceeds of $222.9 million (before deducting underwriting discounts and commissions and offering costs).
Second Quarter 2021 Financial Highlights:

Cash and cash equivalents and short-term investments: As of June 30, 2021, Janux reported cash and cash equivalents and short-term investments of $394.2 million, compared to $7.8 million at December 31, 2020.
Research and development expenses: Research and development expenses for the quarter ended June 30, 2021 were $4.7 million, compared to $0.7 million for the comparable period in 2020. The increase in research and development expenses in 2021 was primarily attributable to the development of the TRACTr platform technology and programs. Janux also incurred additional personnel-related expenses, including stock-based compensation, as operations grew in support of program advances.
General and administrative expenses: General and administrative expenses for the quarter ended June 30, 2021 were $2.0 million, compared to $0.4 million for the same period in 2020. The increase in general and administrative expenses was primarily attributable to an increase in personnel-related expenses including stock-based compensation, due to increased headcount in 2021. The increase in general and administrative expenses were also due to an increase in legal fees, professional fees, and other various general and administrative expenses, as Janux now operates as a public company.
Net loss: For the quarter ended June 30, 2021, Janux reported a net loss of $6.2 million, compared to a net loss of $2.5 million for the comparable period in 2020.

Crinetics Pharmaceuticals Reports Second Quarter 2021 Financial Results and Provides Corporate Update

On August 10, 2021 Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors, reported financial results for the second quarter ended June 30, 2021 and provided a corporate update (Press release, Crinetics Pharmaceuticals, AUG 10, 2021, View Source [SID1234586225]).

"We’ve seen advancements across our pipeline over the past months, with the commencement of dosing in the Phase 3 PATHFNDR-1 trial of paltusotine in acromegaly and the announcement of data from CRN04894’s Phase 1 program," said Scott Struthers, Ph.D., Founder and Chief Executive Officer of Crinetics. "This program continues to follow the blueprint of paltusotine’s success, as early clinical data have provided proof-of-concept by demonstrating the dose-dependent and clinically-significant pharmacodynamic effects of CRN04894 on a well validated hormonal biomarker. We are also mirroring this approach with our somatostatin receptor type 5 agonist, CRN04777, and remain on track to report data from the single-ascending dose cohorts of the ongoing Phase 1 trial in September."

Dr. Struthers continued, "Looking forward, our strong financial foundation and talented team of in-house endocrinology experts leaves us well positioned to execute on our corporate and clinical objectives. Through the continued advancement of our pipeline, we aim to solidify our position as a leader in the design and development of novel small molecule drugs for endocrine diseases."

SECOND QUARTER AND SUBSEQUENT HIGHLIGHTS
Reported positive data from single-ascending dose (SAD) cohorts of first-in-human study of CRN04894. In August 2021, Crinetics announced positive data from the SAD cohorts of an ongoing Phase 1 study of its ACTH antagonist, CRN04894. Preliminary data provided evidence of clinically relevant cortisol suppression. CRN04894 demonstrated dose-dependent reductions in basal cortisol levels as well as suppression of cortisol following ACTH challenge. In addition, the data suggests that CRN04894 was orally bioavailable and demonstrated dose-proportional pharmacokinetics. CRN04894 was well-tolerated, and all adverse events were considered mild. The data is supportive of proceeding to the multiple-ascending dose cohorts of the Phase 1 study and additional data is expected in the fourth quarter of 2021.
Commenced dosing in Phase 3 PATHFNDR-1 study. In June 2021, Crinetics announced that the first patient had been dosed in PATHFNDR-1, one of two planned Phase 3 trials assessing the safety and efficacy of once-daily oral paltusotine that together will evaluate paltusotine in a wide cross section of acromegaly patients. Topline data from PATHFNDR-1 is expected to be available in 2023.
Strengthened balance sheet with successful common stock offerings. In April 2021, Crinetics completed an underwritten follow-on offering and raised gross proceeds of approximately $75.0 million and in August 2021, Crinetics entered into a securities purchase agreement with Frazier Healthcare Partners for the private placement of 851,306 shares at $17.62 per share, raising gross proceeds of $15.0 million.
Appointed Garlan Adams as General Counsel. In June 2021, Crinetics strengthened its leadership team with the appointment of Ms. Adams to the newly created role of General Counsel. Ms. Adams brings more than two decades of experience managing legal and compliance matters associated with the development and commercialization of innovative pharmaceutical and biotechnology products.

SECOND QUARTER 2021 FINANCIAL RESULTS
Research and development expenses were $20.5 million for the three months ended June 30, 2021, compared to $12.6 million for the same period in 2020. The increase was primarily due to an increase in personnel costs of $3.0 million, of which stock-based compensation was $1.1 million, and increased spending on manufacturing and development activities of $4.1 million associated with our clinical and nonclinical activities for paltusotine and our other clinical and preclinical programs.
General and administrative expenses were $5.6 million for the three months ended June 30, 2021, compared to $4.3 million for the same period in 2020. The increase was primarily due to additional personnel costs of $0.8 million, of which stock-based compensation was $0.6 million.
Net loss for the three months ended June 30, 2021 was $26.1 million, compared to a net loss of $16.5 million for the three months ended June 30, 2020.
Unrestricted cash, cash equivalents and investments totaled $203.8 million as of June 30, 2021, compared to $170.9 million as of December 31, 2020. The increase was attributable to the $72.6 million net proceeds from the common stock offering completed in April.
As of July 31, 2021, the company had 38,563,660 common shares outstanding.

ORIC Pharmaceuticals Reports Second Quarter 2021 Financial Results and Operational Update

On August 10, 2021 ORIC Pharmaceuticals, Inc. (Nasdaq: ORIC), a clinical stage oncology company focused on developing treatments that address mechanisms of therapeutic resistance, reported financial results and operational updates for the quarter ended June 30, 2021 (Press release, ORIC Pharmaceuticals, AUG 10, 2021, View Source [SID1234586224]).

"We executed well in the first half of the year, achieving multiple milestones, including the initial data readout for ORIC-101 in combination with nab-paclitaxel in solid tumors and the clearance of the IND application for ORIC-533, our small molecule CD73 inhibitor," said Jacob Chacko, M.D., president and chief executive officer. "In the second half of the year, we expect to present preliminary safety, pharmacokinetic, and translational data, as well as preliminary antitumor activity from our ongoing Phase 1 trial of ORIC-101 in combination with enzalutamide in prostate cancer. In addition, we look forward to dosing our first patient with ORIC-533 and to filing an IND for ORIC-944, our allosteric PRC2 inhibitor, and a CTA for ORIC-114, our brain penetrant EGFR/HER2 exon 20 inhibitor. Furthermore, the proceeds from our recent financing have bolstered our balance sheet with funding for the continued development of ORIC-101 and our other three product candidates through multiple data readouts expected in the 2022 and 2023 timeframe."

Second Quarter 2021 and Other Recent Highlights

Initial ORIC-101 Clinical Data Presented at ASCO (Free ASCO Whitepaper): In June 2021, ORIC presented initial clinical data from the Phase 1b trial of ORIC-101 in combination with nab-paclitaxel. The trial is a single arm, multicenter, open-label study conducted in two parts, intended to establish the recommended Phase 2 dose (RP2D), safety, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity when administered to patients with advanced or metastatic solid tumors. Following the completion of the Part I dose escalation portion of the study, the RP2D was determined to be 160 mg of ORIC-101 continuous once daily dosing in combination with 75 mg/m2 of nab-paclitaxel, without requirement for prophylactic granulocyte-colony stimulating factor (G-CSF). For the Part II dose expansion portion of the study, up to 132 patients are expected to be enrolled across four cohorts, including pancreatic ductal adenocarcinoma (PDAC), ovarian cancer, triple negative breast cancer, and other advanced solid tumors. Enrollment continues in the Part II dose expansion cohorts at 12 clinical sites across the United States. Patients in the dose expansion portion of the study are required to have previously progressed on a taxane-based therapy, with retrospective analysis of GR expression and other potentially predictive biomarkers. Key findings of the initial data presented included:

Safety Analyses:
As of March 31, 2021, a total of 31 patients were enrolled across Parts I and II of the study, which included 12 patients treated at non-RP2D doses and 19 patients treated at the RP2D. Patients treated at the RP2D were heavily pretreated, with a median of four prior therapies, and all had previously received a taxane-based therapy.
As of the database cutoff date of April 21, 2021, the RP2D was well tolerated; treatment-related adverse events were primarily Grade 1 or 2, with only three Grade 3 events, which all resolved with dose interruption.
There were no treatment-related discontinuations and no requirement for prophylactic G-CSF at the RP2D.

Preliminary Antitumor Activity (as of the database cutoff date of April 21, 2021):
The efficacy evaluable population included a total of 23 patients who had an opportunity for at least one on-treatment tumor assessment.
Five partial responses were observed, one confirmed and four unconfirmed, including in heavily pretreated patients with PDAC, endometrial and breast cancers, who previously progressed on or after a taxane-based therapy.
Further evidence of antitumor activity was demonstrated by prolonged disease stabilization across multiple solid tumors, including PDAC, breast, gastric, esophageal, and testicular cancers.
Notably, three of the four efficacy evaluable patients with late-line relapsed PDAC treated at the RP2D demonstrated extended progression free survival ranging from 3.6 months to 5.3+ months in the third-line or later setting, despite having already previously progressed on nab-paclitaxel.
ORIC-533 IND Cleared by FDA: In June 2021, ORIC announced that the U.S. Food and Drug Administration (FDA) cleared its Investigational New Drug Application (IND) for ORIC-533 to proceed into a first-in-human clinical trial. ORIC-533 is a highly potent, orally bioavailable small molecule inhibitor of CD73, a key node in the adenosine pathway believed to play a central role in resistance to chemotherapy and immunotherapy-based treatment regimens. Based on a preclinical collaboration with an academic key opinion leader that generated compelling single agent activity in patient derived model systems in an undisclosed tumor type, the company plans to pursue a single agent clinical development plan in this indication. ORIC plans to initiate the Phase 1 clinical trial with ORIC-533 in the second half of 2021 to evaluate safety, PK and preliminary efficacy in cancer patients.

Preclinical Data Presented at AACR (Free AACR Whitepaper): In April 2021, ORIC presented posters on four programs at the 2021 American Association for Cancer Research (AACR) (Free AACR Whitepaper) virtual annual meeting.

Corporate: In July 2021, ORIC raised gross proceeds of $50.0 million through the sale of approximately 2.6 million shares under its ATM offering, with participation based on unsolicited interest received from a healthcare specialist fund. The company sold the shares at a purchase price per share of $19.25, a premium to the market price at the time of the sale.
Anticipated Milestones

ORIC anticipates the following milestones in the second half of 2021:
ORIC-101: Report interim safety, pharmacokinetics, translational data, and preliminary antitumor activity from the ongoing combination trial with enzalutamide
ORIC-533: Initiate Phase 1 study
ORIC-944: File IND
ORIC-114: File CTA
Present additional preclinical data at scientific conferences
Second Quarter 2021 Financial Results

Cash, Cash Equivalents and Short-term Investments: Cash, cash equivalents, and short-term investments totaled $261.1 million as of June 30, 2021. Along with the July 2021 ATM offering gross proceeds of $50.0 million, the company expects its cash, cash equivalents, and short-term investments of $305.9 million as of July 31, 2021, will be sufficient to fund its current operating plan into 2024.

R&D Expenses: Research and development expenses were $15.5 million for the three months ended June 30, 2021, compared to $7.7 million for the three months ended June 30, 2020, an increase of $7.8 million. For the six months ended June 30, 2021, R&D expenses were $27.2 million compared to $15.0 million for the same period of 2020, an increase of $12.2 million. The increases for the 2021 periods were primarily driven by an increase in external expenses related to the advancement of ORIC-101 and our other product candidates of $6.4 million and $10.0 million for the three and six months ended June 30, 2021, respectively, as well as higher personnel costs, including additional non-cash stock-based compensation of $0.7 million and $1.5 million for the three and six months ended June 30, 2021, as compared to the same periods in 2020, respectively.

G&A Expenses: General and administrative expenses were $5.5 million for the three months ended June 30, 2021, compared to $3.4 million for the three months ended June 30, 2020, an increase of $2.1 million. For the six months ended June 30, 2021, G&A expenses were $10.4 million compared to $5.3 million for the same period of 2020, an increase of $5.1 million. The increases were primarily due to higher personnel costs, including additional non-cash stock-based compensation of $1.1 million and $2.5 million for the three months and six months ended June 30, 2021, as compared to the same periods in 2020, respectively, as well as higher professional services and related costs to operate as a public company.

Alpine Immune Sciences Provides Corporate Update and Reports Second Quarter 2021 Financial Results

On August 10, 2021 Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for cancer and autoimmune/inflammatory diseases, reported financial results for the second quarter ended June 30, 2021 (Press release, Alpine Immune Sciences, AUG 10, 2021, View Source [SID1234586223]).

"We have had a highly productive second quarter with substantial progress made across our portfolio highlighted by the initiation of Synergy, our international Phase 2 trial of ALPN-101, or acazicolcept, in lupus patients, the achievement of $45 million in development milestones as part of our AbbVie agreement, and the first presentation of clinical data from ALPN-202 highlighting the potential differentiation of safely agonizing CD28. Alpine has evolved into a global development company with clinical programs in both Immunology and Oncology and two strong pharmaceutical partners in AbbVie and Merck," said Mitchell H. Gold, M.D., Executive Chairman and Chief Executive Officer of Alpine. "Looking forward, we plan to initiate our Phase 1 study for ALPN-303 by the fourth quarter and look forward to providing other updates as we progress on all our clinical studies."

Second Quarter 2021 and Recent Corporate and Clinical Updates

Acazicolcept: Dual CD28/ICOS inhibitor
Initiated Synergy, an international, double-blind placebo-controlled Phase 2 study of acazicolcept in patients with Systemic Lupus Erythematosus (SLE).
Achieved $45 million in pre-option exercise development milestones as part of the 2020 Option and License Agreement with AbbVie, with the potential of an additional $30 million in pre-option exercise development milestones.
ALPN-202: Conditional CD28 costimulator and dual checkpoint inhibitor
Presented initial dose escalation experience with ALPN-202 monotherapy in the NEON-1 clinical trial at the 2021 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. ALPN-202 was well-tolerated to date, with evidence of peripheral T cell modulation consistent with CD28 agonism, and clinical benefit observed in the majority of participants, despite the population consisting of heavily pre-treated, advanced tumor types traditionally considered unresponsive to immunotherapies.
Entered a clinical trial collaboration and supply agreement with Merck and initiated enrollment in NEON-2, a dose escalation and expansion study to evaluate the safety and efficacy of ALPN-202 in combination with KEYTRUDA (pembrolizumab).
ALPN-303: Dual APRIL/BAFF inhibitor
Presented an oral abstract at the 2021 European Alliance of Associations for Rheumatology (EULAR) virtual meeting demonstrating superior preclinical activity of ALPN-303 compared to multiple comparators, including wild-type TACI-Fc fusion proteins.
Targeting completion of activities to support initiation of a Phase 1 healthy volunteer study with ALPN-303 in the fourth quarter of this year.
General Corporate
Appointed Zelanna Goldberg, M.D., M.A.S. as Chief Medical Officer: Dr. Goldberg brings over 20 years of industry and clinical practice experience, including strategic and/or operational responsibility for multiple therapeutic products. Most recently Dr. Goldberg was Senior Vice President of Clinical Science at Iovance Biotherapeutics.
Added to the Russel 3000 Index.
Second Quarter 2021 Financial Results

As of June 30, 2021, we had cash, cash equivalents, restricted cash, and investments totaling $100.4 million, which does not include the $45.0 million in achieved milestones from the AbbVie collaboration expected to be received in the third quarter. We recorded net losses of $11.0 million and $9.9 million for the quarters ended June 30, 2021 and 2020, respectively.

Collaboration revenue for the quarter ended June 30, 2021 was $7.2 million compared to $0.7 million for the quarter ended June 30, 2020. The increase was primarily attributable to the revenue recognized under our AbbVie Agreement.

Research and development expenses for the quarter ended June 30, 2021 were $14.6 million compared to $7.1 million for the quarter ended June 30, 2020. The increase was primarily attributable to contract manufacturing and process development of our product candidates, personnel-related expenses, clinical trial activities, and direct research activities.

General and administrative expenses for the quarter ended June 30, 2021 were $3.3 million compared to $3.3 million for the quarter ended June 30, 2020. While the costs remained relatively constant, decreases in professional and legal services were partially offset by increases in personnel-related expenses to support the growth and expansion of our business.

Alpine expects that its current cash resources, combined with the $45 million in achieved milestones and the potential additional $30 million in pre-option exercise milestones payable under its option and license agreement with AbbVie, for the development and commercialization of acazicolcept, are sufficient to fund Alpine’s planned operations through 2023.

Second Quarter 2021 Conference Call and Webcast Details

Alpine will host a conference call and live webcast to discuss the second quarter performance today, August 10, 2021 at 4:30 p.m. ET/1:30 p.m. PT.

To access the live call by phone, dial (800) 816-3005 (domestic) or (857) 770-0069 (international) and reference conference ID: 8145346. A live webcast of the presentation will be available online in the investor relations section of the company’s website at View Source A replay of the presentation will be available on the company website for 90 days following the webcast.

Regulus Therapeutics Reports Second Quarter 2021 Financial Results and Recent Updates

On August 10, 2021 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported financial results for the second quarter ended June 30, 2021 and provided a corporate update (Press release, Regulus, AUG 10, 2021, View Source [SID1234586222]).

"The team at Regulus accomplished a lot over the last several months, including the achievement of several major milestones in our ADPKD program, the completion of meeting preparations with FDA and extending our cash runway into Q4 of next year," commented Jay Hagan, CEO of Regulus. "In addition, we are pleased to have completed enrollment of the second cohort of our Phase 1b trial of RGLS4326 for ADPKD, for which we expect to report topline data in the next several months. Additionally, we look forward to obtaining FDA feedback on our approach for addressing the remaining partial clinical hold requirements through our meeting request."

Program Updates

RGLS4326 for ADPKD: In June 2021, the Company presented additional data from the first cohort of patients in its Phase 1b clinical trial of RGLS4326 for the treatment of ADPKD, as well as new preclinical data from relevant animal models of the disease, at PKD Connect 2021. The additional data presented at PKD Connect demonstrated clinical proof of mechanism by showing statistically significant increases in polycystin levels in ADPKD patients by targeting miR-17 in the kidneys. Levels of PC1 and PC2 have previously been shown to be inversely correlated with disease severity. RGLS4326 was well-tolerated with no serious adverse events.

The Phase 1b study is an adaptive design, open-label, multiple dose study in up to three cohorts of patients with ADPKD evaluating the safety, pharmacokinetics, and effects on pharmacodynamic biomarkers of multiple doses of RGLS4326. In the first cohort of the study, nine patients were enrolled and received four doses of 1mg/kg of RGLS4326 administered every other week.

The Company also presented new data from relevant preclinical models showing treatment with RGLS4326 resulted in increased gene and polycystin levels in vitro. Improvements in key disease markers including serum creatinine and BUN were demonstrated in the Pkd1(F/RC) mouse model that harbors a mutation in the Pkd1 gene equivalent to human ADPKD. Mutation to the Pkd1 gene is reported for 85% of diagnosed patients with ADPKD.

In late July 2021, the Company completed enrollment of the second cohort of patients in its Phase 1b clinical study of RGLS4326. The patients in the second cohort are being administered 0.3mg/kg of RGLS4326 every other week for four doses. The dose of RGLS4326 for the third and final cohort will be chosen based on the results of this second cohort and enrollment is expected to commence thereafter. The Company expects to report topline data from the second cohort in the fourth quarter.

The Company requested a Type A meeting with FDA to discuss the data supporting its approach to addressing the remaining partial clinical hold requirements. FDA typically responds to a sponsor’s request with the meeting being scheduled within 30 days from the receipt of the meeting request.

Corporate Highlights

Raised $15.4 Million in Gross Proceeds Through its ATM Facility: A total of 12,007,546 shares were sold and settled for gross proceeds of $15.4 million at a weighted average price per share of $1.28 under the ATM facility during the three months ended June 30, 2021. The Company expects its existing cash will provide cash resources to fund planned activities into Q4 2022.

Financial Results

Cash Position: As of June 30, 2021, Regulus had $41.4 million in cash and cash equivalents.

Research and Development (R&D) Expenses: Research and development expenses were $4.2 million and $7.5 million for the three and six months ended June 30, 2021, respectively, compared to $4.2 million and $7.4 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 $2.5 million and $5.0 million for the three and six months ended June 30, 2021, respectively, compared to $2.3 million and $4.7 million for the same periods in 2020, respectively. These amounts reflect personnel-related and ongoing general business operating costs.

Net Loss: Net loss was $6.0 million, or $0.08 per share (basic and diluted), and $12.0 million, or $0.16 per share (basic and diluted), for the three and six months ended June 30, 2021, compared to $6.9 million, or $0.23 per share (basic and diluted), and $12.9 million, or $0.48 per share (basic and diluted), for the same periods in 2020.

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 RGLS4326

RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 and preferentially target the kidney. Preclinical studies with RGLS4326 have demonstrated direct regulation of Pkd1 and Pkd2, reduction of cyst growth in human in vitro ADPKD models, and attenuation of cyst proliferation and improvement of kidney function in mouse models of ADPKD. The RGLS4326 IND is currently on a partial clinical hold for treatment of extended duration by FDA until the second set of requirements outlined by the agency have been satisfactorily addressed. The Company will use information from the Phase 1 clinical studies, including the first cohort of the Phase 1b together with information from the recently completed additional nonclinical studies generated in 2020, in its plan to address the second set of requirements outlined in the Partial Clinical Hold letter to support studies of extended duration. Regulus plans to discuss its approach to addressing the remaining Partial Clinical Hold requirements with FDA in the third quarter 2021. RGLS4326 received orphan drug designation from FDA in July 2020.