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]).

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"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.

On August 10, 2021 Biodesix, Inc. (Nasdaq: BDSX), a leading data-driven diagnostic solutions company with a focus in lung disease, reported its financial and operating results for the second quarter ended June 30, 2021 and provided a corporate update (Press release, Biodesix, AUG 10, 2021, View Source [SID1234586221]).

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"It was a productive quarter with 180% year over year growth in total revenue. Our first four blood-based tests which make up our core lung diagnostic testing, grew 109% year over year and 20% over the prior quarter," said Scott Hutton, CEO of Biodesix. "We expect growth in core lung diagnostic testing to continue through 2021 driven by the U.S.’s emergence from the pandemic, productivity from our growing salesforce, and our efforts to build on the body of evidence supporting the use of our tests. Beyond our currently marketed products, we also continue to develop our Diagnostic Cortex Artificial Intelligence (AI) platform and expand our pipeline of products capable of providing broad lung diagnostics to address patients’ needs over the course of their disease. The launch of our 72-hour liquid NGS test is now projected for the first quarter of 2022 and will supplement our 36-hour VeriStrat and GeneStrat tests. With this accelerated launch and multiple studies underway for our Risk of Recurrence (ROR) and Primary Immune Response (PIR) tests as well as our ALTITUDE, BEACON, INSIGHT, and ORACLE prospective studies actively enrolling or following patients, we look forward to a productive and exciting second half of the year."

Second Quarter 2021 Financial Results

For the three-month period ended June 30, 2021, as compared to the same period of 2020 (where applicable):

Total revenue of $11.9 million, an increase of 180%;

Core lung diagnostic revenue of $4.8 million, an increase of 109%;
Continued sequential recovery from initial rapid uptake of COVID-19 immunizations.
Growth driven primarily by Nodify XL2 and Nodify CDT.
Nodify XL2 growth triggered the previously disclosed milestone resulting in a $37 million obligation payable by us over six quarterly installments of approximately $4.6 million beginning January 2022 and a final payment of approximately $9.3 million in July 2023.
COVID-19 testing revenue of $6.1 million, an increase of 345%;
Sequential quarter over quarter decline commensurate with our prior commentary as US immunizations accelerated in second quarter.
Services revenue of $1.0 million, an increase of 76%;
Clinical trial services showed improvement and we expect further recovery in clinical trial enrollments in second half of 2021.

Gross profit of $4.8 million, an increase of 103%, and gross margin percentage of 40% as compared to 56% representing a decline primarily attributable to the lower gross margin COVID-19 testing;

Operating expenses (excluding direct costs and expenses) of $15.4 million, which includes an investment in the planned expansion of our sales force, increased 93% over second quarter 2020;
On track to double size of lung focused direct and dedicated sales force in 2021.
Non-cash stock compensation expense of $0.5 million as compared to $0.1 million.
Non-cash expense for change in fair value of contingent consideration of $0.6 million as compared to a gain of $1.0 million.

Net loss of $11.4 million, an increase of 38% over second quarter of 2020; and

Maintained fiscal discipline and strong liquidity with cash and cash equivalents of $56.3 million as of June 30, 2021 and expect to qualify for PPP loan forgiveness of $3.1 million later in 2021.
For a full list of Biodesix’s press releases and webinars, please visit Biodesix.com.

Conference call and webcast information

Management will host an investor conference call and webcast today, August 10, 2021 at 4:30 p.m. Eastern Time.

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 [SID1234586220]).

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"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.

On August 10, 2021 Almac Sciences, global provider of integrated drug development services and member of the Almac Group, reported that the company has signed a manufacturing agreement with Swedish biotech company, PILA PHARMA, for the production of XEN-D0501 active pharmaceutical ingredient (API) (Press release, Almac, AUG 10, 2021, View Source [SID1234586219]).

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From its global headquarter facilities in Northern Ireland, Almac Sciences will manufacture XEN-D0501, a highly selective and very potent small molecule TRPV1 antagonist, previously in development by Bayer Healthcare and Xention/Ario Pharma. The TRPV1 target (also called the "chili-receptor") has demonstrated applications across pain and inflammatory diseases and possibly in diabetes as well. XEN-D0501 was acquired by PILA PHARMA in March 2016, and subsequently, PILA PHARMA has demonstrated good safety results as well as efficacy, i.e. a statistically improved endogenous insulin response to oral glucose versus placebo in patients with type 2 diabetes, thus demonstrating proof of principle of XEN-D0501.

Commented by Professor Tom Moody, VP Technology Development and Commercialisation, Almac Sciences and Arran Chemical Company, "Almac Sciences is delighted to be working with PILA PHARMA and supporting their API manufacture. The project showcases deployment of our technology platforms including integration of green chemistries and we look forward to further opportunities in this space."

Dorte X. Gram, CEO, PILA PHARMA states, "The availability of new API is key for us to be able to conduct planned 3-month toxicology studies, that are in turn a prerequisite for initiating our main goal, conducting a clinical phase 2b study with XEN-D0501 in type-2 diabetics. I am, therefore, very pleased to have signed off this agreement and we look forward to the collaboration with Almac on timely delivery of new API."

The global company has proven expertise in small and large molecule analytics, API supply, stable & 14C radiolabelling, formulation development & solid-state services and earlier this year announced the completion of its £5 million two-storey centre for biocatalysis, flow chemistry technologies and peptide research and development.

On August 10, 2021 Pulmatrix, Inc. (NASDAQ: PULM), a clinical stage biopharmaceutical company developing innovative inhaled therapies to address serious pulmonary and non-pulmonary disease using its patented iSPERSE technology, reported its second quarter 2021 financial results and provides a business update (Press release, Pulmatrix, AUG 10, 2021, View Source [SID1234586218]).

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"We have made steady progress across our pipeline in the second quarter," said Ted Raad, Chief Executive Officer of Pulmatrix. "Recent toxicology data from PUR1800 suggests the potential to expand into indications that require chronic dosing. We look forward to presenting topline data from the fully enrolled, ongoing Phase 1b study of PUR1800 in Q1 2022. We are also rapidly advancing towards the clinic with PUR3100 in acute migraine. We believe that our strong cash position allows us to advance our pipeline through major data milestones into 2023."

Second Quarter and Recent Highlights:

PUR1800

Completed enrollment in ongoing Phase 1b clinical study of PUR1800 in acute exacerbations in COPD (AECOPD). Study endpoints include safety, tolerability, and exploratory biomarkers to demonstrate target engagement and anti-inflammatory effect.
PUR1800 Phase 1b top-line data is expected in Q1 2022.
Results from 6-month rat and 9-month dog toxicology results demonstrate no progression of 28-day findings, suggesting potential for chronic dosing of PUR1800 in indications beyond AECOPD including, but not limited to, steroid resistant asthma, chronic obstructive pulmonary disease (COPD) and idiopathic pulmonary fibrosis (IPF).
The Company estimates an approximate peak net revenue opportunity of $2.4B in AECOPD1, due to the significant unmet need beyond standard of care oral steroids and/or antibiotic therapy. Expansion to chronic indications should further broaden the market potential of PUR1800.
PUR3100

14-day toxicology results for PUR3100, Pulmatrix’s dry powder iSPERSE formulation of dihydroergotamine (DHE) for pulmonary delivery to treat acute migraine are expected in Q3 2021.
IND filing planned in Q4 2021, with a Phase 1/ Phase 2 clinical study start date anticipated in Q1 2022. Data from this proof-of-concept study are expected in Q4 2022.
Pulmazole

Pulmatrix notified Cipla Technologies LLC (Cipla) that it is in material breach of the Development and Commercialization Agreement (the Cipla Agreement) in May 2021. The Company continues to seek Cipla’s reaffirmation of all of its obligations under the Cipla Agreement and, in the absence of such reaffirmation, to pursue all available remedies.
1 Market research and analysis from ClearView Healthcare Partners

Financials

As of June 30, 2021, Pulmatrix had $56.9 million in cash and cash equivalents, compared to $31.7 million for the year ended December 31, 2020.

Revenue for the second quarter of 2021 was $2.2 million, compared to $3.5 million for the same period in 2020. The revenue for the second quarter of 2021 was the result of the collaboration and licensing agreements with Cipla and JJEI.

Research and development expense was $4.5 million in the second quarter of 2021 compared to $3.2 million for the same period in 2020. The increase year–over-year was primarily attributable to increased preclinical and manufacturing costs related to the PUR3100 project partially offset by decreased spend on the Pulmazole clinical trial.

General and administrative expense was $1.6 million for the second quarter of 2021 compared to $1.5 million for the same period in 2020. The increase year–over-year was primarily attributable to increase legal, patent, ad public company costs partially offset by decreased employment costs.

Net loss was $3.9 million for the second quarter of 2021 compared to a net loss of $1.2 million for the same period of 2020. The $2.7 million increase in net loss year-over-year was due to increased spend for preclinical and manufacturing expenses on the PUR3100 program and reduced revenue recognized that related to the Pulmazole program.