On August 4, 2022 Cytokinetics, Incorporated (Nasdaq: CYTK) reported financial results for the second quarter of 2022. Net loss for the second quarter was $19.8 million, or $0.23 per share, compared to net loss for the second quarter of 2021 of $61.6 million, or $0.86 per share (Press release, Cytokinetics, AUG 4, 2022, View Source,million%20at%20June%2030%2C%202022. [SID1234617702]). Cash, cash equivalents and investments totaled $596.7 million at June 30, 2022.

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In July, Cytokinetics raised $540 million netting approximately $383 million in proceeds through the offering of convertible senior notes, after deducting the purchasers’ discounts and commissions and Cytokinetics’ estimated offering expenses, as well as deducting $140.3 million to repurchase previously outstanding convertible senior notes. Cytokinetics expects to end 2022 with more than $800 million in cash.

"During the second quarter we made progress across regulatory, clinical and financial objectives. We engaged extensively with FDA in planning for its review of our NDA for omecamtiv mecarbil and advanced the enrollment and conduct of SEQUOIA-HCM and COURAGE-ALS while also planning for a second Phase 3 trial of aficamten," said Robert I Blum, Cytokinetics’ President and Chief Executive Officer. "We also bolstered our balance sheet by executing on a fundraising of over $500 million while reducing our planned nearer-term spending in 2022. We believe that we are in a strong position to bring our potential therapies to patients while also executing well on late-stage clinical development programs."

Q2 and Recent Highlights

Cardiac Muscle Programs

omecamtiv mecarbil (cardiac myosin activator)

Participated in a mid-cycle review meeting with the U.S. Food and Drug Administration (FDA) related to our New Drug Application (NDA) for omecamtiv mecarbil. Engaged extensively with FDA in other interactions related to its review of our NDA.

Received notice that the FDA plans to convene an Advisory Committee meeting to review the NDA for omecamtiv mecarbil, and that the meeting is currently scheduled for December 13, 2022.

Received notice that the FDA extended the Prescription Drug User Fee Act (PDUFA) date for omecamtiv mecarbil to February 28, 2023 due to additional pharmacokinetic analyses of omecamtiv mecarbil provided to the FDA upon request and that are deemed to constitute a major amendment to the NDA.

Hired and deployed Managed Healthcare Medical Scientists and initiated medical support of our global value, access and distribution services. Continued building headquarters-based commercial infrastructure and engaging in launch readiness activities, including pre-approval information exchange discussions with key payers. Conducted Business Process inventory for enterprise resource planning implementation and strategic sourcing activities to mitigate supply chain risks.

Presented additional data from GALACTIC-HF (Global Approach to Lowering Adverse Cardiac Outcomes Through Improving Contractility in Heart Failure) at Heart Failure 2022, an International Congress of the European Society of Cardiology, including:

An analysis showing that in patients with low blood pressure, there was a greater treatment effect from omecamtiv mecarbil on the primary composite endpoint of cardiovascular death or first heart failure event than in patients without low blood pressure such that there was an absolute risk reduction of 9.8 events per 100 patient-years (hazard ratio, 0.81; 95% confidence interval [CI] 0.70, 0.94; interaction p=0.051).

An analysis showing that the treatment effect of omecamtiv mecarbil on the primary composite endpoint was consistent across patients with no tricuspid regurgitation (TR), mild TR and moderate/severe TR such that baseline TR did not modify the treatment effect (interaction p=0.91).
Presented additional data from GALACTIC-HF at the American College of Cardiology 71st Annual Scientific Session (ACC.22) including:

An analysis showing that treatment with omecamtiv mecarbil in a subgroup of patients in GALACTIC-HF led to a reduction in resource intensity, with an estimated cost offset of $3,085, or a 19% reduction in costs per patient. The majority of cost reductions were due to heart failure hospitalizations avoided by patients treated with omecamtiv mecarbil.

An analysis showing that the effect of treatment with omecamtiv mecarbil was associated with similar risk reduction in the primary composite endpoint in both hospitalized patients and in outpatients, indicating that initiation of omecamtiv mecarbil was safe and well tolerated in both hospitalized patients and outpatients.
Announced results of METEORIC-HF (Multicenter Exercise Tolerance Evaluation of Omecamtiv Mecarbil Related to Increased Contractility in Heart Failure), a Phase 3 clinical trial of omecamtiv mecarbil in patients with HFrEF that evaluated the effect of treatment with omecamtiv mecarbil compared to placebo on exercise capacity as determined by cardiopulmonary exercise testing (CPET). After 20 weeks of treatment, there was no change in peak oxygen uptake (pVO2) in patients treated with omecamtiv mecarbil versus placebo. Adverse events, including major cardiac events, were similar between the treatment arms, and the safety profile of omecamtiv mecarbil was consistent with prior clinical trials, including GALACTIC-HF.

Published a manuscript entitled "Effects of Omecamtiv Mecarbil in Heart Failure with Reduced Ejection Fraction According to Blood Pressure: The GALACTIC-HF Trial" in the European Heart Journal.

Published a manuscript entitled "The Effect of Omecamtiv Mecarbil on Exercise Capacity in Chronic Heart Failure with Reduced Ejection Fraction: The METEORIC-HF Randomized Trial" in the Journal of the American Medical Association.

aficamten (cardiac myosin inhibitor)

Presented the first long-term data from REDWOOD-HCM OLE (Randomized Evaluation of Dosing With CK-274 in Obstructive Outflow Disease in HCM Open Label Extension) at Heart Failure 2022 from 38 patients treated for up to 24 weeks showing that treatment with aficamten was associated with substantial reductions in the average resting left ventricular outflow tract gradient (LVOT-G) and Valsalva LVOT-G. The reductions started to occur within two weeks of treatment, were sustained through 24 weeks of treatment, and were achieved with only modest decreases in the average LVEF. Improvements were also observed in New York Heart Association (NYHA) Functional Class and cardiac biomarkers including NTpro-BNP and cardiac troponin. Treatment with aficamten was well-tolerated with one temporary discontinuation due to LVEF <50% and one temporary down-titration, neither related to drug. Both patients remain on treatment with aficamten.

Presented additional data from REDWOOD-HCM at the American Society of Echocardiography (ASE) 33rd Annual Scientific Sessions showing that measures of cardiac structure, diastolic and mitral valve function improved in patients treated with aficamten after 10 weeks, including a significant reduction in left atrial volume index (p<0.01) and a trend towards a reduction in left ventricular hypertrophy (left ventricular mass index; p=0.06). Treatment with aficamten also resulted in improved ventricular relaxation and filling, as indicated by a reduction in lateral E/e’ (p<0.01) and an increase in lateral e’ (p<0.05).

Announced positive data from Cohort 3 of REDWOOD-HCM, which enrolled patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM) and a resting left LVOT-G ≥50, or resting LVOT-G ≥30 mmHg and post-Valsalva LVOT-G ≥50 mmHg, whose background therapy included disopyramide and in the majority a beta-adrenergic blocker. Results showed that substantial reductions in the average resting LVOT-G as well as the post-Valsalva LVOT-G (defined as resting gradient <30 mmHg and post-Valsalva gradient <50 mmHg) were achieved. The safety and tolerability of aficamten were consistent with prior experience in REDWOOD-HCM with no treatment interruptions and no serious adverse events attributed to treatment reported by the investigators.

Began developing the go-to-market strategy for aficamten to support its potential future commercialization.

Published a manuscript entitled "Characteristics of Patients with Obstructive Hypertrophic Cardiomyopathy in Real-World Community-Based Cardiovascular Practices" in The American Journal of Cardiology.

Skeletal Muscle Program

reldesemtiv (fast skeletal muscle troponin activator (FSTA))

Continued conduct and enrollment of COURAGE-ALS (Clinical Outcomes Using Reldesemtiv on ALSFRS-R in a Global Evaluation in ALS), the Phase 3 clinical trial of reldesemtiv in patients with amyotrophic lateral sclerosis (ALS).

Started COURAGE-ALS OLE (Clinical Outcomes Using Reldesemtiv on ALSFRS-R in a Global Evaluation in ALS Open Label Extension), an open-label extension clinical study designed to assess the long-term safety and tolerability of reldesemtiv in people with ALS who have previously participated in COURAGE-ALS.

Pre-Clinical Development and Ongoing Research

Continued to advance new muscle directed compounds and conduct IND-enabling studies with the expectation of our potentially moving 1-2 drug candidates into clinical development in the next year.

Continued research activities directed to our other muscle biology research programs.

Corporate

Raised $523.3 million in net proceeds, after deducting underwriters’ discounts and transaction fees, and before repurchasing previously outstanding 2026 convertible senior notes.

Announced changes to the Board of Directors including the retirement of L. Patrick Gage, Ph.D., former Chairman of the Board, the appointment of John T. Henderson, M.B., Ch.B. as the company’s new Chairman, and the appointment of Robert A. Harrington, M.D., Arthur L. Bloomfield Professor and Chair, Department of Medicine, Stanford University, to the Board.

Announced the continuation of our partnership with The ALS Association in the fight against ALS.

Upcoming Corporate Milestones

Cardiac Muscle Programs

omecamtiv mecarbil (cardiac myosin activator)

Participate in Advisory Committee meeting to review the NDA for omecamtiv mecarbil on December 13, 2022.

Launch omecamtiv mecarbil in the U.S. subject to FDA approval in Q1 2023.

aficamten (cardiac myosin inhibitor)

Continue enrolling patients with obstructive HCM in SEQUOIA-HCM through 2022 with results expected in 2H 2023.

Continue enrolling patients with non-obstructive HCM in Cohort 4 of REDWOOD-HCM with results expected in 1H 2023.

Begin second Phase 3 clinical trial of aficamten in obstructive HCM in Q4 2022.

Expect to share additional data from the open label extension study of aficamten, REDWOOD-HCM OLE in 2H 2022.

CK-3828136 (CK-136) (cardiac troponin activator)

Reactivate development program for CK-136 in 2H 2022.

Skeletal Muscle Program

reldesemtiv (fast skeletal muscle troponin activator (FSTA))

Expect the Data Monitoring committee to conduct the first interim analysis (assessing for futility) from COURAGE-ALS in Q4 2022.

Financials

Revenues for the three and six months ended June 30, 2022 were $89.0 million and $90.1 million, respectively, compared to $2.8 million and $9.4 million for the corresponding period in 2021. The increase in revenues was primarily due to the recognition of $87.0 million of deferred revenue for royalties on the net sales of products containing mavacamten as a result of the extinguishment of royalty obligations.

Research and development expenses for the three and six months ended June 30, 2022 increased to $57.1 million and $103.1 million, respectively, compared to $36.4 million and $68.0 million for the same period in 2021. The changes were primarily due to increases in spending for clinical development activities for COURAGE-ALS, for our cardiac muscle inhibitor programs, and early research programs.

General and administrative expenses for the three and six months ended June 30, 2022 increased to $42.7 million and $75.8 million, respectively, from $21.2 million and $36.8 for the same period in 2021 due primarily to higher outside services spending in anticipation of the potential commercial launch of omecamtiv mecarbil, and an increase in personnel related costs including stock-based compensation.

Revised 2022 Financial Guidance

The company today revised its financial guidance related to a reduction in expected operating expenses as a result of the three-month extension of the PDUFA date for omecamtiv mecarbil to February 28, 2023, which shifted certain hiring and activities previously planned to occur in 2022 to 2023. The company anticipates operating expenses for 2022 will be in the range of $375 to $385 million, and net cash utilization will be approximately $360 to $365 million. The company expects to end 2022 with more than $800 million, representing between two and three years of forward cash.

Conference Call and Webcast Information

The conference call will be simultaneously webcast and can be accessed from the homepage and in the Investors & Media section of Cytokinetics’ website at www.cytokinetics.com. The live audio of the conference call can also be accessed by telephone by registering in advance at the following link: Cytokinetics Q2 2022 Earnings Conference Call. Upon registration, participants will receive a dial-in number and a unique passcode to access the call. An archived replay of the webcast will be available via Cytokinetics’ website for twelve months.

On August 4, 2022 Galapagos NV (Euronext & NASDAQ: GLPG) reported its first half-year 2022 financial results, a year-to-date business update and its outlook for the remainder of 2022 (Press release, FierceBiotech, AUG 4, 2022, https://www.fiercebiotech.com/biotech/galapagos-sends-another-4-programs-extinction-pivot-car-t-continues [SID1234617693]). The results are further detailed in the H1 2022 financial report available on the financial reports section of the website.

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"This quarter, we took a first key step in our strategic transformation by entering the field of oncology with the acquisitions of CellPoint and AboundBio. The combined transactions offer the potential for a paradigm shift in CAR-T1 therapy through CellPoint’s breakthrough, decentralized point-of-care supply model, developed in a global strategic collaboration with Lonza, and AboundBio’s cutting-edge fully human antibody-based capabilities to design next-generation CAR-Ts. Patient enrolment in the ongoing Phase 1/2a trials in rrNHL and rrCLL2 is progressing well, and we expect topline results in the first half of next year. Our near-term goal is to bring three additional differentiated, next-generation CAR-T candidates in the clinic over the next three years," said Dr. Paul Stoffels3, CEO and chairman of the board of directors of Galapagos. "We strongly believe that we are taking the right steps in our transformation to accelerate value creation, and we look forward to presenting an in-depth update on our strategy later this year."

"Our Jyseleca franchise is performing very well with robust sales momentum, supported by the regulatory approvals in ulcerative colitis (UC) in Great Britain and Japan earlier this year. The adoption of Jyseleca is strong across Europe with reimbursement for rheumatoid arthritis (RA) in 15 and for UC in 6 countries," added Bart Filius, President, COO and CFO of Galapagos. "Following the acquisitions of CellPoint and AboundBio, we expect that second half operating expenses will increase by approximately €30 million. Therefore, we revised our cash burni guidance of €450-€490 million for the full year 2022 to €480-€520 million. As a result of the strong Jyseleca performance, we increase our full-year net sales guidance of €65-€75 million to €75-€85 million."

Year-to-date operational overview
Commercial & regulatory progress:

Strong adoption across Europe with reimbursement for RA in 15 countries and for UC in 6 countries
Sobi, our distribution and commercialization partner in Eastern and Central Europe, Portugal, Greece, and the Baltic countries, launched Jyseleca in RA in the Czech Republic and Portugal, resulting in €2 million milestone payments to Galapagos in H1
Filed a type II variation for the label update for Jyseleca based on data from the MANTA and MANTA-RAy studies
At the EULAR4 2022 European Congress of Rheumatology, Galapagos hosted several expert sessions and presented 11 abstracts, further establishing us as a key player in RA
Article 20 pharmacovigilance procedure ongoing by the European Medicines Agency’s (EMA) Pharmacovigilance Risk Assessment Committee (PRAC), investigating the safety data of all JAK inhibitors for the treatment of certain chronic inflammatory disorders
Pipeline update:

Decided to move forward with GLPG3667 (TYK2 inhibitor) in dermatomyositis with the aim to start a Phase 2 study before year-end
Discontinued development of 4 early-stage programs as part of ongoing scientific and strategic exercise: GLPG3121, a local release formulation JAK1/TYK2 inhibitor with potential in inflammatory diseases; GLPG0555, a JAK1 inhibitor evaluated in osteoarthritis; GLPG4586, a compound with undisclosed mode of action directed toward fibrosis; and GLPG4716, a chitinase inhibitor directed toward idiopathic pulmonary fibrosis
Corporate update:

Entered the field of oncology through the combined acquisitions of CellPoint and AboundBio in all-cash transactions
Received a transparency notification from FMR LLC in Q2 indicating that its shareholding in Galapagos increased and crossed the 5% threshold, to 5.04% of the current outstanding Galapagos shares
Raised €3.6 million through the exercise of subscription rights
Created new subscription rights plans, offering all Galapagos employees the opportunity to participate
All proposed resolutions regarding the extraordinary and annual shareholders’ meetings were adopted by Galapagos’ shareholders on 26 April 2022

H1 2022 financial results
We reported product net sales of Jyseleca in Europe for the first six months of 2022 amounting to €35.4 million (€0.5 million in the first six months of 2021). Our counterparties for the sales of Jyseleca were mainly hospitals and wholesalers located in Belgium, the Netherlands, France, Italy, Spain, Germany, Great Britain, Ireland, Austria, Norway, Sweden and Finland.

Cost of sales related to Jyseleca net sales in the first six months of 2022 amounted to €5.5 million.

Collaboration revenues amounted to €238.6 million for the first six months of 2022, compared to €253.2 million for the first six months of 2021.

Revenues recognized related to the collaboration agreement with Gilead for the filgotinib development were €115.3 million in the first six months of 2022 compared to €136.1 million for the same period last year. This decrease was due to a lower increase in the percentage of completion, partly offset by a higher revenue recognition of milestone payments, strongly influenced by the milestone achieved related to the regulatory approval in Japan for UC in the first half-year of 2022. The revenue recognition related to the exclusive access rights for Gilead to our drug discovery platform amounted to €114.9 million for the first six months of 2022 (€115.7 million for the same period last year).

We have recognized royalty income from Gilead for Jyseleca for €6.3 million in the first six months of 2022 (compared to €1.4 million in the same period last year) of which €3.6 million royalties on milestone income for UC approval in Japan.

Additionally, we recorded milestones of €2.0 million triggered by the first sale of Jyseleca in the Czech Republic and Portugal by our distribution and commercialization partner Sobi, in the first half-year of 2022.

Our deferred income balance on 30 June 2022 includes €1.6 billion allocated to our drug discovery platform that is recognized linearly over the remaining period of our 10-year collaboration, and €0.5 billion allocated to the filgotinib development that is recognized over time until the end of the development period.

Our R&D expenditure in the first six months of 2022 amounted to €249.5 million, compared to €268.8 million for the first six months of 2021. This decrease was primarily explained by a decrease in subcontracting costs from €139.2 million in the first six months of 2021 to €104.1 million in the first six months of 2022, primarily due to the winding down of the ziritaxestat (IPF) program and reduced spend on our Toledo (SIKi) and TYK2 programs. This was partly offset by cost increases for our filgotinib program, on a six month basis compared to the same period in 2021. Personnel costs decreased from €94.2 million in the first half of 2021 to €86.0 million for the same period this year mainly due to a lower number of FTEs as well as lower costs for our subscription right plans. Depreciation and impairment amounted to €32.6 million for the first six months of 2022 (€8.1 million for the same period last year). This increase was primarily due to an impairment of €26.7 million of previously capitalized upfront fees related to our collaboration with Molecure on the dual chitinase inhibitor OATD-01 (GLPG4716). As part of an ongoing strategic exercise to renew and accelerate our portfolio, we decided to return all rights to OATD-01 to Molecure.

Our G&A and S&M expenses amounted to €134.0 million in the first six months of 2022, compared to €105.8 million in the first six months of 2021. This increase was primarily due to the termination of our 50/50 filgotinib co-commercialization cost sharing agreement with Gilead for filgotinib in 2022. The cost increase was also explained by an increase in personnel costs for the first six months of 2022 compared to the same period last year explained by an increase in the commercial work force driven by the commercial launch of filgotinib in Europe.

Other operating income (€17.6 million vs €23.6 million for the same period last year) decreased, mainly driven by lower grant and R&D incentives income.

Net financial income in the first six months of 2022 amounted to €67.7 million, compared to net financial income of €19.9 million for the first six months of 2021. Net financial income in the first six months of 2022 was primarily attributable to €57.4 million of unrealized currency exchange gains on our cash and cash equivalents and current financial investments at amortized cost in U.S. dollars, and to €11.8 million of positive changes in (fair) value of current financial investments. The financial expenses also contained the effect of discounting our long term deferred income of €3.8 million.

We realized a net loss from continuing operations of €32.3 million for the first six months of 2022, compared to a net loss of €77.2 million for the first six months of 2021.

The net profit from discontinued operations for the six months ended 30 June 2021 consisted of the gain on the sale of Fidelta, our fee-for-services business, for €22.2 million.

We reported a group net loss for the first six months of 2022 of €32.3 million, compared to a group net loss of €55.0 million for the first six months of 2021.

Cash position
Current financial investments and cash and cash equivalents totaled €4,429.0 million on 30 June 2022, as compared to €4,703.2 million on 31 December 2021.

Total net decrease in cash and cash equivalents and current financial investments amounted to €274.2 million during the first six months of 2022, compared to a net decrease of €162.7 million during the first six months of 2021. This net decrease was composed of (i) €217.1 million of operational cash burn, (ii) offset by €3.6 million of cash proceeds from capital and share premium increase from exercise of subscription rights in the first six months of 2022, (iii) €11.8 million positive changes in (fair) value of current financial investments and €60.4 million of mainly positive exchange rate differences, and (iv) the cash out from the acquisitions of CellPoint and AboundBio, net of cash acquired, of €132.9 million.

Acquisitions of CellPoint and AboundBio
The preliminary accounting of the acquisitions of CellPoint and AboundBio are included in our H1 2022 condensed consolidated financial statements. To date, we have performed a preliminary fair value analysis of the business combinations. We expect the provisional amount of goodwill to change significantly upon the completion of the purchase price allocation, resulting from the valuation of the different assets and liabilities acquired.

Outlook 2022
Financial guidance:
Following the acquisitions of CellPoint and AboundBio, we revised our cash burn guidance for full year 2022 from €450-€490 million to €480-€520 million. Additionally, we increased our anticipated net sales guidance for Jyseleca from €65-€75 million to between €75 and €85 million.

Expected regulatory events:
We anticipate a Committee for Medicinal Products for Human Use (CHMP) opinion on the type II variation for the Jyseleca label, based on the data from the MANTA and MANTA-RAy studies around year-end. We also expect reimbursement decisions in most key European markets in UC and anticipate that Sobi will further progress with reimbursement discussions in RA and UC in Eastern and Central Europe, Greece, and the Baltic countries. As part of the ongoing article 20 pharmacovigilance procedure on all JAK inhibitors approved in Europe, we expect a CHMP opinion by the end of the year, followed by an adoption by the European Commission shortly afterwards.

Anticipated R&D milestones:
Patient enrolment in the Phase 1/2a trials in rrNHL and rrCLL is progressing well and we anticipate that additional clinical sites will be active by year-end. We are on track to report topline results of both trials in the first half of next year.
We plan to progress TYK2 inhibitor GLPG3667 into a Phase 2 program in dermatomyositis with first patients potentially recruited around year-end.

We continue to explore additional business development opportunities to further leverage our internal capabilities and renew our portfolio, and we look forward to presenting an in-depth update on our corporate strategy later this year.

First half-year 2022 financial report

Galapagos’ financial report for the first six months ended 30 June 2022, including details of the unaudited consolidated results, is accessible on the financial reports section of our website.

Conference call and webcast presentation

Management will host a conference call and webcast presentation followed by Q&A tomorrow 5 August 2022, at 14:00 CET / 8 AM ET. To participate in the conference call, please register in advance using this link. Upon registration, the dial-in numbers will be provided. The conference call can be accessed 10 minutes prior to the start time by using the conference access information provided in the e-mail received at the point of registering, or by selecting the call me feature.

The live webcast can be accessed on the investors section of the Galapagos website, and a replay will be made available shortly after the close of the call.

On August 4, 2022 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, reported recent business highlights and second quarter 2022 financial results (Press release, Sangamo Therapeutics, AUG 4, 2022, View Source [SID1234617647]).

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"We made meaningful progress advancing our clinical-stage programs in the second quarter," said Sandy Macrae, Chief Executive Officer of Sangamo. "We are engaging in pivotal study-enabling activities in two of our clinical stage programs and are preparing to complete dosing of the first cohort in our TX200 program, which recently received Orphan Medicinal Product Designation from the European Commission. Coupled with strong advances in our preclinical pipeline, we believe this progress positions us well to advance the development of potentially transformational genomic medicines for patients in need and to generate long-term value for our shareholders."

Recent Business Highlights

Fabry disease – Received endorsement to progress into the Ph1/2 study’s expansion phase; continued to recruit patients and activate sites; Phase 3 planning progresses.

In June, the Safety Monitoring Committee endorsed progressing the Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, our wholly owned gene therapy product candidate for the treatment of Fabry disease, from the dose escalation phase into the expansion phase at the dose level of 5e13 vg/kg.
We expect to dose two additional patients imminently, and have multiple patients in screening, including both male and female candidates.
Enzyme replacement therapy (ERT) withdrawal was completed for an additional two patients previously dosed in the STAAR study, achieving a total of four patients to date who have successfully been withdrawn.
A total of 16 study sites are now open and recruiting, including the first sites in Canada, Italy and Australia.
We plan to provide updated results from the STAAR study in the second half of 2022, including at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium, taking place August 30-September 2, 2022.
We continue to actively prepare for a potential pivotal Phase 3 trial.
Sickle cell disease – Completed transition of program back to Sangamo; advanced manufacturing activities in anticipation of dosing in Q3; Phase 3 planning progresses.

We completed the transition of Sanofi’s rights and obligations under the collaboration developing BIVV003, formerly known as SAR445136, our zinc finger nuclease gene-edited cell therapy candidate for the treatment of sickle cell disease, back to Sangamo on June 28, 2022.
Manufacturing of product candidates using improved methods progressed in the Phase 1/2 study. These improved manufacturing methods have been shown in internal experiments to increase the number of long-term progenitor cells in the final product.
Dosing of the next patient is anticipated in the third quarter of 2022.
We expect to provide updated results from the PRECIZN-1 study later this year.
Phase 3 enabling activities and manufacturing readiness are in progress.
Hemophilia A – Pfizer advised us that it continues to expect resumption of dosing in Q3 2022; pivotal data read-out expected in late 2023 or early 2024.

Pfizer advised us that it continues to anticipate resuming the dosing of additional patients in the Phase 3 AFFINE trial of giroctocogene fitelparvovec, an investigational gene therapy we are developing with Pfizer for patients with moderately severe to severe hemophilia A, in the third quarter of 2022.
A pivotal data readout is estimated in late 2023 or early 2024.
Over 50% of the patients have been enrolled in the Phase 3 AFFINE trial.
Renal Transplant Rejection – Received Orphan Medicinal Product Designation from the European Commission; progressed manufacturing and clinical activities ahead of anticipated Q3 dosing.

Since we dosed the first patient in the Phase 1/2 STEADFAST study evaluating TX200, our wholly owned autologous CAR-Treg cell therapy treating patients receiving an HLA-A2 mismatched kidney from a living donor, the product candidate continues to be generally well tolerated, with no treatment related adverse events.
We completed manufacturing of the dose for the second patient, who recently received a kidney transplant. Dosing of this second patient is expected later in the third quarter of 2022.
We plan to complete dosing of the first cohort, comprised of three patients, by the end of 2022.
The European Commission granted Orphan Medicinal Product Designation to TX200, for treatment in solid organ transplantation, following a positive opinion from the European Medicines Agency’s Committee for Orphan Medicinal Products.
American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) – Profiled significant pre-clinical progress across Sangamo’s innovative pipeline and platform.

Presented seven posters and one oral presentation at ASGCT (Free ASGCT Whitepaper) on May 16-19, 2022, including pre-clinical updates across our CAR-Treg autoimmune cell therapy platform, innovations in our genome engineering platform and advances in our AAV capsid engineering program.
Second Quarter 2022 Financial Results

Consolidated net loss for the second quarter ended June 30, 2022, was $43.2 million, or $0.29 per share, compared to a net loss of $47.2 million, or $0.33 per share, for the same period in 2021.

Revenues

Revenues for the second quarter ended June 30, 2022, were $29.4 million, compared to $27.9 million for the same period in 2021.

The increase of $1.5 million in revenues was primarily attributed to an increase of $1.3 million in revenue related to our collaboration agreement with Novartis and an increase of $0.8 million in revenue related to our collaboration agreement with Sanofi. These increases were partially offset by a decrease of $0.7 million in revenue related to our collaboration agreement with Biogen.

Total operating expenses on a GAAP basis for the second quarter ended June 30, 2022, were $75.1 million, compared to $76.6 million for the same period in 2021. Non-GAAP operating expenses, which exclude stock-based compensation expense, for the second quarter ended June 30, 2022, were $67.2 million, compared to $67.1 million for the same period in 2021.

The decrease in total operating expenses on a GAAP basis was primarily due to the timing of certain research and development activities.

Cash, cash equivalents and marketable securities

Cash, cash equivalents and marketable securities as of June 30, 2022, were $363.7 million, compared to $464.7 million as of December 31, 2021. Since the beginning of the second quarter, we have raised approximately $43.1 million in net proceeds under our previously announced at the market offering program.

Financial Guidance for 2022 Reiterated (initial guidance provided on February 24, 2022)

On a GAAP basis, we continue to expect total operating expenses in the range of approximately $320 million to $350 million in 2022, which includes non-cash stock-based compensation expense.

We continue to expect non-GAAP total operating expenses, excluding estimated non-cash stock-based compensation expense of approximately $40 million, in the range of approximately $280 million to $310 million in 2022.

Upcoming Events

Sangamo plans to participate in the following events in the third quarter:

Scientific / Medical Conferences

Society for the Study of Inborn Errors of Metabolism (SSIEM), August 30-September 2, 2022, Freiburg, Germany
Prion 2022, September 13-16, 2022, Gottingen, Germany
Investor Conferences

Wedbush PacGrow Healthcare Conference, August 9-10, 2022 [9:10-9:40am EDT]
H.C. Wainwright 24th Annual Global Investment Conference, September 12-14, 2022
Jefferies Cell and Genetic Medicine Summit, September 29-30, 2022
Access links for these investor conferences will be available on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. Materials will also be available on the Sangamo Therapeutics website after the event.

Conference Call to Discuss Second Quarter 2022 Results

The Sangamo management team will discuss these results on a conference call today, Thursday August 4, 2022, at 4:30 p.m. Eastern Time.

Participants should register for, and access, the call using this link. While not required, it is recommended you join 10 minutes prior to the event start. Once registered, participants will be given the option to either dial into the call with the number and unique passcode provided, or to use the dial-out option to connect their phone instantly.

The link to access the live webcast can also be found on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations.

A replay will be available following the conference call, accessible under Events and Presentations.

On August 4, 2022 Moberg Pharma AB (OMX: MOB) reported that has signed a distribution agreement with Padagis Israel Agencies Ltd. for MOB-015 in Israel and the Palestinian territories (Press release, Moberg Pharma, AUG 4, 2022, View Source [SID1234617620]). Under the agreement Padagis is granted exclusive rights to market and sell MOB-015 in Israel and the Palestinian territories. Moberg Pharma assumes production and supply responsibility.

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Padagis will conduct registration activities in Israel, and will be marketing, distributing and selling MOB-015 in Israel and the Palestinian territories upon completion of registration.

"This is the sixth commercial agreement for MOB-015, this time with a leading provider of extended topical and other specialty pharmaceuticals in Israel. We look forward to work with Padagis and making MOB-015 available in Israel, contributing to our vision of making MOB-015 the leading nail fungus treatment worldwide", says Anna Ljung, CEO of Moberg Pharma.

"As a leading pharmaceutical company in Israel, Padagis Israel welcomes the signing of this agreement with Moberg Pharma and look forward to a fruitful cooperation and collaboration between the two companies", says Shlomi Leibovich, SVP & CEO of Padagis Israel.

According to Moberg Pharma’s market intelligence, the Israelian market for topical drugs for onychomycosis amounts to approximately €6.5 million.

About this information
This information is information that Moberg Pharma AB is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out above, on August 4th, 2022, at 8.00 am CEST.

On August 4, 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 financial results for the fiscal third quarter ended June 30, 2022 (Press release, ESSA, AUG 4, 2022, View Source [SID1234617619]). All references to "$" in this release refer to United States dollars, unless otherwise indicated.

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"It is a busy and important time for ESSA as we advance our lead candidate for the treatment of prostate cancer, EPI-7386, into the dose expansion phase of the single agent trial, dose the second cohort of patients in the Company-sponsored combination trial with enzalutamide, and plan to initiate additional trials of EPI-7386 in earlier-line patients," stated David Parkinson, M.D., President and CEO of ESSA. "During this past quarter, we were pleased to report clinical results from the Phase 1a dose escalation study demonstrating that EPI-7386 was safe and well-tolerated at all dose levels tested and that tumor volume decreases were observed in a subgroup of patients with measurable disease who were on therapy for more than 12 weeks. We also shared preliminary results from the first cohort of patients in our Phase 1/2 combination trial with enzalutamide."

Clinical Highlights

EPI-7386 Monotherapy

In June 2022, ESSA reported clinical results from the Phase 1a dose escalation study of EPI-7386 in patients with metastatic castration-resistant prostate cancer ("mCRPC") resistant to current standard-of-care therapies. The initial data demonstrate that EPI-7386 was well-tolerated, exhibited a favorable pharmacokinetic profile, and demonstrated initial anti-tumor activity in heavily pretreated patients. EPI-7386 was safe and well-tolerated at all dose levels and schedules tested, with no dose-limiting toxicities.

The Phase 1b expansion study is expected to begin in the third quarter of calendar 2022; the trial is expected to enroll two dose cohorts as well as an additional cohort of patients with non-metastatic castration-resistant prostate cancer ("nmCRPC") who have not yet been treated with a second-generation antiandrogen in a 12-week window of opportunity study.
EPI-7386 Clinical Collaborations

The Company has completed dosing of the first cohort of patients and is currently enrolling the second cohort of patients in the Company-sponsored Phase 1/2 study of EPI-7386 in combination with Astellas Pharma Inc.’s and Pfizer Inc.’s enzalutamide in patients with mCRPC who have not been treated with second-generation antiandrogens. In June 2022, the Company reported preliminary results from the first cohort suggesting that the drugs can be combined safely and result in active drug levels of both EPI-7386 and enzalutamide.

Janssen Research and Development LLC continues to enroll patients in the Phase 1/2 trial of EPI-7386 in combination with apalutamide or abiraterone acetate plus prednisone in earlier line mCRPC patients.

The Bayer-led Phase 1/2 trial will evaluate EPI-7386 in combination with darolutamide in earlier line mCRPC patients.

The Company expects to initiate a Phase 2 investigator-sponsored neoadjuvant study to evaluate darolutamide compared to EPI-7386 + darolutamide in patients undergoing prostatectomy for high-risk localized prostate cancer by year-end.
Summary Financial Results

Net Loss. ESSA recorded a net loss of $8.8 million ($0.20 loss per common share based on 44,059,700 weighted average common shares outstanding) for the quarter ended June 30, 2022, compared to a net loss of $8.8 million ($0.21 loss per common share based on 41,018,024 weighted average common shares outstanding) for the quarter ended June 30, 2021. For the quarter ended June 30, 2022, this included non-cash share-based payments of $1.6 million compared to $2.8 million for the comparable period in 2021, recognized for stock options granted and vesting.

Research and Development ("R&D") expenditures. R&D expenditures for the quarter ended June 30, 2022 were $6.4 million compared to $6.2 million for the quarter ended June 30, 2021 and included non-cash costs related to share-based payments ($872,531 for the quarter ended June 30, 2022 compared to $1.2 million for the quarter ended June 30, 2021). The increase in R&D expenditures for the fiscal quarter ended June 30, 2022 was primarily related to preclinical and clinical data analysis associated with the Phase 1a clinical study.

General and administration ("G&A") expenditures. G&A expenditures for the quarter ended June 30, 2022 were $2.9 million compared to $3.1 million for the quarter ended June 30, 2021 and included non-cash costs related to share-based payments of $718,469 for the quarter ended June 30, 2022 compared to $1.5 million for the comparable period in 2021. The decreased expenditure is the result of decreased professional fees from collaboration contracts in the prior period and decreased non-cash share-based payments.
Liquidity and Outstanding Share Capital

At June 30, 2022, the Company had available cash reserves and short-term investments of $174.6 million reflecting the gross proceeds of the February 2021 financing of approximately $150.0 million and July 2020 financing of $48.9 million, less operating expenses in the intervening period. The Company’s cash position is expected to be sufficient to fund current and planned operations through 2024.

As of June 30, 2022, the Company had 44,073,076 common shares issued and outstanding.

In addition, as of June 30, 2022 there were 3,234,750 common shares issuable upon the exercise of warrants and broker warrants. This includes 2,920,000 prefunded warrants at an exercise price of $0.0001, and 314,750 warrants at a weighted average exercise price of $49.69. There were 7,852,061 common shares issuable upon the exercise of outstanding stock options at a weighted-average exercise price of $5.15 per common share.

About EPI-7386

EPI-7386 is an investigational, highly-selective, oral, small molecule inhibitor of the N-terminal domain of the androgen receptor. EPI-7386 is currently being studied in a Phase 1 clinical trial (NCT04421222) in men with CRPC and mCRPC whose tumors have progressed on current standard-of-care therapies. The Phase 1 clinical trial of EPI-7386 began in calendar Q3 of 2020 following FDA allowance of ESSA’s Investigational New Drug application and Health Canada acceptance. The Phase 1b component of the study comprises two cohorts enrolling in parallel. Cohort A – a dose expansion study of EPI-7386 to evaluate the safety, tolerability, pharmacokinetic, and preliminary anti-tumor activity and Cohort B – a window of opportunity study with clinical endpoints to assess the anti-tumor activity in nmCRPC patients unperturbed by previous second generation anti-androgen therapies or chemotherapy. EPI-7386 is also being studied in earlier line mCRPC patients in Phase 1/2 trials in combination with enzalutamide, apalutamide and abiraterone acetate with prednisone. The U.S. FDA has granted Fast Track designation to EPI-7386 for the treatment of adult male patients with mCRPC resistant to standard-of-care treatment. ESSA retains all rights to EPI-7386 worldwide.