On August 12, 2021 F-star Therapeutics, Inc. (NASDAQ: FSTX), a clinical-stage biopharmaceutical company dedicated to developing next generation bispecific immunotherapies to transform the lives of patients with cancer, reported second quarter 2021 financial results and provides a corporate update (Press release, F-star, AUG 12, 2021, View Source [SID1234586419]).

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The Company today announces an expanded clinical development strategy for FS118, F-star’s first-in-class bispecific antibody targeting LAG-3 and PD-L1, into checkpoint inhibitor naïve patients. This new study with FS118 is in biomarker enriched subsets of patients with non-small cell lung cancer (NSCLC) and diffuse large B-cell lymphoma (DLBCL). By moving into earlier lines of therapy, FS118 has the potential not only to delay, but also to prevent checkpoint resistance, which could provide an alternative treatment for patients who do not see a durable benefit from currently approved immunotherapies.

In addition to the expanded FS118 clinical strategy, F-star has three other ongoing clinical stage programs. These programs are combined with a strong balance sheet, having closed an underwritten public offering of stock and its "at the market" facility during Q2, which will enable the company to deliver multiple clinical data milestones. At the end of the second quarter the company had over $80 million in cash and cash equivalents. In the past quarter, F-star has also delivered on clinical and business development objectives, specifically the recent interim Phase 1 update on SB 11285 and the announcement of two major collaborations, a licensing agreement with AstraZeneca Plc for STING inhibitors and a supply agreement with MSD (Merck & Co., Inc., Kenilworth, NJ, USA) to evaluate the combination of FS120, F-star’s first-in-class dual-agonist, tetravalent, bispecific antibody targeting CD137 and OX40, with KEYTRUDA (pembrolizumab), MSD’s anti-PD-1 therapy.

Eliot Forster, CEO of F-star Therapeutics, Inc., said, "I’m happy to share today the expansion of the FS118 clinical development program into checkpoint naïve patients. By targeting LAG-3, which was recently externally validated in the clinic as an important target, and PD-L1 simultaneously, we believe FS118 has the opportunity to improve patient outcomes over those seen with first generation immunotherapies. We also continue to focus on the checkpoint inhibitor refractory patient population in our ongoing FS118 proof-of-concept study in head and neck cancers. We are also pleased to have concluded two new collaborations, with AstraZeneca and MSD for STING antagonists and combining Keytruda with FS120, respectively.

"We believe that our strong balance sheet, expanded strategy for FS118, continuing and beneficial partnerships, along with our dedicated staff and investors, position F-star for a promising 2021 and beyond. We look forward to sharing data with you at upcoming medical conferences, including at ESMO (Free ESMO Whitepaper) next month."

SECOND QUARTER 2021 AND RECENT HIGHLIGHTS

FS118 development expansion plans following recent external clinical validation of LAG-3: Phase 3 data with LAG-3 at ASCO (Free ASCO Whitepaper) 2021, further supported F-star’s expansion of the FS118 clinical development plan into checkpoint naïve, biomarker enriched NSCLC and DLBCL patients. This study is anticipated to begin in the second half of 2021.

Strong financial position: In Q2 2021, the Company received $82.6 million in gross proceeds through the combination of an underwritten public offering of stock and use of its "at the market" facility.

FS222 Poster presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in 2021: In April, F-star presented a poster at AACR (Free AACR Whitepaper) 2021 entitled ‘FS222, a Tetravalent Bispecific Antibody Targeting CD137 and PD-L1, is Designed for Optimal CD137 Interactions Resulting in Potent T cell Activation Without Toxicity’. This poster and the associated data showcased the differentiation of FS222 from competitor molecules and highlighted the importance of ‘tuning’ for both the affinity and avidity of bispecific antibodies.

Combination of FS120 with KEYTRUDA: Last week, F-star announced a clinical trial collaboration and supply agreement with MSD (Merck & Co., Inc., Kenilworth, NJ, USA), to evaluate the combination of FS120, F-star’s first-in-class dual-agonist tetravalent bispecific antibody targeting CD137 and OX40, with KEYTRUDA (pembrolizumab), MSD’s anti-PD-1 therapy.

SB 11285 Phase 1 interim update: In July, F-star provided an interim update on the safety, tolerability and pharmacokinetics of its intravenously administered novel STING agonist, alone and in combination with atezolizumab. SB 11285 appeared to be well tolerated both alone and in combination across all dose levels tested to-date, including five dose levels as monotherapy and three dose levels as a combination. The Part 1a/1b study database lock, as defined in the contingent value rights (CVR) agreement entered into in connection with the transaction with Spring Bank Pharmaceuticals, Inc., has been completed. Based on the positive emerging clinical data, further dose escalations are ongoing and a further clinical update is planned for 2022.

SB 11285 in Nature publication and composition of matter patent granted in the US: F-star published on its second-generation STING agonist, SB 11285, in the April 2021 issue of Nature Communications. The study, entitled ‘STING enhances cell death through regulation of reactive oxygen species and DNA damage’ demonstrated that systemic administration of a STING agonist in combination with radiation in a preclinical model enhances local control in Head and Neck Squamous Cell Carcinoma (HNSCC) and suggests that STING expression in the tumor is required for maximal therapeutic benefit. The US Patent Office granted a patent during the quarter with claims protecting the composition of matter of F-star’s SB 11285 molecule giving protection until 2037.

AstraZeneca licenses STING inhibitors: In July, F-star entered into an exclusive licensing agreement with AstraZeneca plc under which AstraZeneca received global rights to research, develop and commercialize next generation Stimulator of Interferon Genes (STING) inhibitor compounds. AstraZeneca was granted exclusive access to F-star’s novel preclinical STING inhibitors and will be responsible for all future research, development and commercialization of the STING inhibitor compounds. F-star retains rights to all STING agonists, currently in clinical development for patients with cancer. This agreement is subject to the second CVR with the former shareholders of Spring Bank Pharmaceuticals.

SECOND QUARTER 2021 FINANCIAL SUMMARY

Cash and cash equivalents as of June 30, 2021, were $81.6 million, compared to $18.5 million at December 31, 2020.

Research & Development (R&D) expenses were $8.4 million for the quarter ended June 30, 2021, compared to $2.1 million for the corresponding quarter in 2020. The $6.3 million increase was primarily related to manufacturing costs and clinical costs with four programs now in the clinic versus one clinical program in Q2 2020.

General & Administrative (G&A) expenses were $6.5 million for the quarter ended June 30, 2021, compared to $3.2 million for the second quarter of 2020. This $3.3 million increase in G&A expense was primarily due to increased non-cash stock-based compensation expense, legal and professional fees and costs associated with operating as a public company.

Net loss was $15.6 million or a loss per share of $0.92 (basic and diluted), for the quarter ended June 30, 2021, compared to a net loss of $6.5 million or a loss per share of $3.53 (basic and diluted) for the quarter ended June 30, 2020.

CONFERENCE CALL AND WEBCAST

F-star will host a conference call today, August 12, 2021, beginning at 9:00 AM EDT. To join the webcast, go to our website. To join by phone, participants may dial 1-833-471-0868 in the US/Canada or 1-914-987-7751 for International calls or 0800 0288438 or 0203 1070289 for the United Kingdom, at least 10 minutes prior to the start of the call.

A recording of the conference call will be available on the ‘Events & Presentations’ section of the Company’s website at www.f-star.com from August 13, 2021.

On August 12, 2021 Veru Inc. (NASDAQ: VERU), an oncology biopharmaceutical company with a focus on developing novel medicines for the management of prostate and breast cancer, reported that net revenues increased 71% and gross profit rose 113% for its fiscal 2021 third quarter ended June 30, 2021, setting new quarterly records for both net revenues and gross profit (Press release, Veru, AUG 12, 2021, View Source [SID1234586418]).

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Third Quarter Financial Highlights: Fiscal 2021 vs Fiscal 2020

Net revenues increased 71% to $17.7 million from $10.3 million
FC2 prescription net revenues climbed 150% to $13.5 million from $5.4 million
Gross profit rose 113% to $13.9 million from $6.5 million
Gross margin increased to 79% of net revenues from 63% of net revenues
Operating loss was $2.9 million versus $1.4 million
Net loss was $2.7 million, or $0.03 per share, compared with $3.0 million, or $0.05 per share.
Year-to-Date Financial Highlights: Fiscal 2021 vs Fiscal 2020

Net revenues increased 48% to $45.6 million from $30.8 million, a record high not only for the nine-month period ended June 30, 2021, but also a record high when compared to any prior full fiscal year
FC2 prescription net revenues climbed 79% to $32.9 million from $18.4 million
Gross profit rose 68% to $35.6 million from $21.2 million
Gross margin increased to 78% of net revenues from 69% of net revenues
Operating income was $14.8 million, which includes an $18.4 million gain on the December 2020 sale of the PREBOOST business. Adjusted operating loss, which excludes the gain on the sale of the PREBOOST business, was $3.6 million versus $3.5 million
Net income, which includes the gain on the sale of the PREBOOST business, was $11.7 million and diluted EPS was $0.14. Adjusted net loss, which excludes the gain on the sale of the PREBOOST business, was $6.7 million compared with $7.1 million and adjusted diluted loss per share was $0.09, compared with $0.11 per share
Balance Sheet Information

Cash and cash equivalents were $123.2 million as of June 30, 2021, versus $13.6 million as of September 30, 2020
Net accounts receivable were $8.3 million as of June 30, 2021, versus $5.2 million as of September 30, 2020
"We reported another record quarter based on all-time high quarterly and year to date FC2 net revenues from the U.S. business," said Mitchell Steiner, M.D., Chairman, President and Chief Executive Officer of Veru Inc. "We have enjoyed having this significant revenue stream to invest in our pharmaceutical programs. In fact, we have made great progress advancing our late-stage drug pipeline this past quarter, as we have initiated and are actively enrolling our: (i) Phase 3 VERACITY clinical trial of sabizabulin for metastatic castration and androgen receptor targeting agent resistant prostate cancer; (ii) Phase 2 clinical trial of VERU-100, a novel long-acting GnRH antagonist injection formulation for androgen deprivation therapy for advanced prostate cancer, and (iii) Phase 3 clinical trial of sabizabulin in hospitalized patients with COVID-19 at high risk for ARDS. Unfortunately, yet another COVID-19 viral infection wave has hit the United States and globally, making our COVID-19 drug development program highly relevant and timely as COVID-19 remains a severe threat with few effective therapies to complement the COVID-19 vaccination strategy."

Dr. Steiner further noted: "We are also making significant progress on our novel oral drug candidates for the treatment of advanced breast cancer. In the second half of the calendar year, we anticipate starting our: (i) Phase 3 ARTEST clinical trial of enobosarm monotherapy in a 3rd line metastatic setting in AR+ER+HER2- metastatic breast cancer; (ii) Phase 2b clinical trial of enobosarm in combination with abemaciclib, CDK 4/6 inhibitor, in a 2nd line metastatic setting in AR+ER+HER2- metastatic breast cancer; and (iii) Phase 2b clinical trial evaluating sabizabulin monotherapy and sabizabulin + Trodelvy (sacituzumab govitecan-hziy) combination therapy versus Trodelvy monotherapy in metastatic triple negative breast cancer. We have solidly transformed Veru into a premium oncology biopharmaceutical company seeking large market opportunities."

Pharmaceutical Pipeline Highlights:

Prostate Cancer Program

Sabizabulin, a Novel Oral Androgen Receptor Transport Disruptor, for the Treatment of Metastatic Castration and Androgen Receptor Targeting Agent Resistant Prostate Cancer – Phase 3 VERACITY Clinical Study – Enrolling.

Sabizabulin is a novel oral new chemical entity that targets microtubules in the cytoskeleton to disrupt androgen receptor transport. In June, the Company initiated the open label, randomized (2:1), multicenter Phase 3 VERACITY clinical trial evaluating sabizabulin 32mg versus an alternative androgen receptor targeting agent for the treatment of chemotherapy naïve men with metastatic castration resistant prostate cancer who have failed at least one androgen receptor targeting agent. The primary endpoint is median radiographic progression free survival. The Phase 3 VERACITY clinical trial is expected to enroll approximately 245 patients from 45 clinical centers.

VERU-100, a Novel Proprietary Long-Acting Gonadotropin-Releasing Hormone (GnRH) Antagonist Peptide 3-Month Subcutaneous Depot Formulation, for Androgen Deprivation Therapy of Advanced Prostate Cancer – Phase 2 Clinical Study – Enrolling.

In June, the Company initiated the Phase 2 clinical trial of VERU-100 androgen deprivation therapy for hormone sensitive advanced prostate cancer. The Phase 2 VERU-100 clinical trial is expected to enroll approximately 35 patients. VERU-100 formulation is designed to address the current limitations of commercially available androgen deprivation therapy. Androgen deprivation therapy is currently the mainstay of advanced prostate cancer treatment and is used as a foundation of treatment throughout the course of the disease even as other endocrine, chemotherapy, or radiation treatments are added or stopped. Specifically, VERU-100 is a chronic, long-acting GnRH antagonist peptide administered as a small volume, three-month depot subcutaneous injection without a loading dose. VERU-100 immediately suppresses testosterone with no testosterone surge upon initial or repeated administration, a problem that occurs with currently approved luteinizing hormone-releasing hormone agonists used for androgen deprivation therapy. There are no GnRH antagonist depot injectable formulations commercially approved beyond a one-month injection. A Phase 3 registration clinical trial has been agreed upon with FDA and will enroll approximately 100 men. The Phase 3 clinical study is anticipated to begin in Q4 calendar year 2021.

Breast Cancer Program

Enobosarm, a Novel Oral Selective Androgen Receptor Targeted Agonist, for the 3rd Line Treatment of Androgen Receptor Positive (AR+), Estrogen Receptor Positive (ER+) and Human Epidermal Growth Factor Receptor 2 Negative (HER2-) Metastatic Breast Cancer – Phase 3 ARTEST Clinical Study.

Enobosarm is the first new class of targeted endocrine therapy in advanced breast cancer in decades. Enobosarm is an oral, new chemical entity, selective androgen receptor agonist that targets and activates the androgen receptor, a tumor suppressor, in AR+ER+HER2- metastatic breast cancer without the unwanted masculinizing side effects. Enobosarm has extensive nonclinical and clinical experience having been evaluated in 25 separate clinical studies in over 2,000 patients including three Phase 2 clinical studies in advanced breast cancer involving more than 250 patients. In the two Phase 2 clinical studies conducted in women with AR+ER+HER2- metastatic breast cancer, enobosarm demonstrated significant antitumor efficacy in heavily pretreated cohorts that failed estrogen receptor targeting agents, chemotherapy, and/or CDK 4/6 inhibitors and was well tolerated with a favorable safety profile. The Phase 3 multicenter, international, open label, and randomized (1:1) ARTEST registration clinical trial is designed to evaluate the efficacy and safety of enobosarm monotherapy versus physician’s choice of either exemestane everolimus or a SERM as an active comparator for the 3rd line treatment of metastatic AR+ER+HER2- breast cancer in approximately 210 patients who have failed a nonsteroidal aromatase inhibitor, fulvestrant and a CDK4/6 inhibitor. The primary endpoint is median radiographic progression-free survival. We expect to commence our pivotal enobosarm Phase 3 ARTEST clinical study in the second half of calendar year 2021.

Enobosarm in Combination with Abemaciclib, CDK 4/6 Inhibitor, for the 2nd Line Treatment of AR+ER+HER2- Metastatic Breast Cancer- Phase 2b Clinical Study.

CDK 4/6 inhibitor and estrogen blocking agent combination has become first line therapy for patients with ER+HER2- advanced breast cancer. Unfortunately, almost all patients will develop resistance to CDK 4/6 inhibitors, and will eventually, have breast cancer progression. Based on positive Phase 2 clinical data and the preclinical data supporting the use of enobosarm in combination with a CDK 4/6 inhibitor in patients that are CDK 4/6 inhibitor and estrogen blocking agent resistant, we plan to evaluate as 2nd line treatment in the metastatic setting the addition of enobosarm in combination with a CDK 4/6 inhibitor (abemaciclib) in a Phase 2b clinical study in subjects with AR+ER+HER2 metastatic breast cancer that have experienced disease progression on 1st line palbociclib + estrogen blocking agent in approximately 186 patients. The Phase 2b clinical study is expected to commence in calendar 2H 2021.

Sabizabulin, Novel Oral Cytoskeleton Disruptor Agent, Versus Trodelvy for the Treatment of Metastatic Triple Negative Breast Cancer- Phase 2b Clinical Study.

Sabizabulin is an oral, first-in-class, new chemical entity that targets and inhibits microtubules to disrupt the cytoskeleton. Sabizabulin is not a substrate for P-glycoprotein drug resistance protein. Over expression of P-glycoprotein is a common mechanism that results in taxane and chemotherapy resistance in metastatic triple negative breast cancer. Preclinical studies in human triple negative breast cancer grown in animal models demonstrate that sabizabulin significantly inhibits cancer proliferation, migration, metastases, and invasion of triple negative breast cancer tumors that have become resistant to paclitaxel (taxane). A three cohort Phase 2b clinical study is planned to start in calendar Q3 2021 to evaluate sabizabulin monotherapy and sabizabulin + Trodelvy combination therapy versus Trodelvy monotherapy in approximately 216 women with metastatic triple negative breast cancer that have become resistant to at least 2 systemic chemotherapies.

COVID-19 Program

Sabizabulin for the Treatment of Hospitalized COVID-19 Patients at High Risk for Acute Respiratory Distress Syndrome (ARDS) Phase 3 Clinical Study- Enrolling.

In May, we initiated our Phase 3 clinical trial of sabizabulin, which has both broad anti-viral and anti-inflammatory activities and which may serve a two-pronged approach to the treatment of COVID-19 virus infection and the subsequent debilitating inflammatory effects that lead to ARDS and death, in high risk hospitalized COVID-19 patients. The Phase 3 clinical trial is a double-blind, multicenter, multinational, randomized (2:1), placebo-controlled trial evaluating daily oral doses of 9mg sabizabulin for up to 21 days versus placebo in 300 hospitalized COVID-19 patients (200 subjects will be treated with sabizabulin and 100 subjects will receive placebo/standard of care) who are at high risk for ARDS. The primary efficacy endpoint will be proportion of patients that die on study up to Day 60. Secondary endpoints will include the proportion of patients without respiratory failure, days in ICU, WHO Ordinal Scale for Clinical Improvement change from baseline, days on mechanical ventilations, days in the hospital, and viral load. The study is being conducted in the United States, Brazil, Argentina, Mexico, and Colombia.

Benign Prostatic Hyperplasia Program

TADFIN (Tadalafil 5mg and Finasteride 5mg Combination Capsule) for the Treatment of Benign Prostatic Hyperplasia (BPH) – PDUFA Date December 2021.

TADFIN (tadalafil 5mg and finasteride 5mg combination capsule) was developed to treat urinary tract symptoms caused by BPH. Tadalafil (CIALIS) is currently approved for treatment of BPH and erectile dysfunction and finasteride is currently approved for treatment of BPH (finasteride 5mg PROSCAR) and male pattern hair loss (finasteride 1mg PROPECIA). The co-administration of tadalafil and finasteride has been shown to be more effective for the treatment of BPH than finasteride alone with the additional benefit of ameliorating erectile dysfunction. The PDUFA date is scheduled for December 2021. If approved, we expect to market and distribute TADFIN by telemedicine and telepharmacy sales channels.

Non-GAAP Financial Information
Certain financial results for fiscal years 2021 and 2020 are presented on both a reported and a non-GAAP, adjusted basis. Reported results were prepared in accordance with U.S. GAAP and include all revenue and expenses recognized during the period. The non-GAAP results are adjusted to exclude the one-time gain on sale of PREBOOST in the first quarter of fiscal year 2021. Management believes non-GAAP financial measures provide useful information to investors regarding the Company’s results of operations and assist management, analysts, and investors in evaluating the performance of the Company’s business. Non-GAAP financial measures should be considered in addition to, and not as a substitute for, measures of financial performance prepared in accordance with GAAP. The Company has reconciled these non-GAAP financial measures to the nearest reported GAAP measures in the reconciliation table below.

Event Details
Interested parties may access the call by dialing 800-341-1602 from the U.S. or 412-902-6706 from outside the U.S. and asking to be joined into the Veru Inc. call. The call will also be available through a live, listen-only audio broadcast via the Internet at www.verupharma.com. Listeners are encouraged to visit the website at least 10 minutes prior to the start of the scheduled presentation to register, download and install any necessary software. A playback of the call will be archived and accessible on the same website for at least three months. A telephonic replay of the conference call will be available, beginning the same day at approximately 12 p.m. (noon) ET by dialing 877-344-7529 for U.S. callers, or 412-317-0088 from outside the U.S., passcode 10157539, for one week.

On August 12, 2021 Biofrontera AG (NASDAQ: BFRA; Frankfurt Stock Exchange: B8F) (the "Company"), an international biopharmaceutical company, reported that it will be releasing its financial results for the first six months ended June 30, 2021 on Thursday, August 19, 2021 (Press release, Biofrontera, AUG 12, 2021, View Source [SID1234586417]).

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Conference calls for shareholders and interested investors will be held on Friday, August 20, 2021, at the following times:

On August 12, 2021 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a late clinical-stage biopharmaceutical company focused on oncology, reported it will release its unaudited financial results for the quarter ended June 30, 2021 on Wednesday, August 18, 2021, before the US markets open (Press release, BioLineRx, AUG 12, 2021, View Source [SID1234586416]).

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The Company will host a conference call on Wednesday, August 18, 2021 at 10:00 a.m. EDT featuring remarks by Philip Serlin, Chief Executive Officer. The conference call will be available via webcast and can be accessed through the Investor Relations page of BioLineRx’s website. Please allow extra time prior to the call to visit the site and download any necessary software to listen to the live broadcast.

To dial into the conference call, please dial +1-866-744-5399 from the U.S. or +972-3-918-0644 internationally. A replay of the conference call will be available approximately two hours after completion of the live conference call on the Investor Relations page of BioLineRx’s website. A dial-in replay of the call will be available until August 20, 2021; please dial +1-888-295-2634 from the U.S. or +972-3-925-5904 internationally.

On August 12, 2021 Exicure, Inc. (NASDAQ:XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) technology, reported financial results for the quarter ended June 30, 2021 and provided an update on corporate progress (Press release, Exicure, AUG 12, 2021, View Source [SID1234586415]).

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"We continue to drive towards a number of key value inflection points across our platforms and programs," commented Dr. David Giljohann, Exicure’s Chief Executive Officer. "Notably, we believe our momentum in neurology is particularly strong on the heels of our recently announced collaboration with Ipsen for Huntington’s disease and Angelman syndrome. Our team has also made impressive progress in our preclinical neurology pipeline, with a planned IND filing in Friedreich’s ataxia and work in pain and Batten disease using our SNA technology."

Pipeline Highlights & Updates

Neurology

Ipsen Collaboration

On August 2, 2021, IPSEN BIOPHARM LIMITED (Ipsen) and Exicure announced an exclusive collaboration agreement to research, develop, and commercialize novel SNAs as potential investigational treatments for Huntington’s disease (HD) and Angelman syndrome (AS). Under the terms of the collaboration:
Ipsen obtains two exclusive options to SNAs currently under discovery evaluation for HD and AS;
Exicure will be responsible for discovery and certain preclinical development activities. In the event Ipsen exercises an option with respect to the two collaboration programs, Ipsen will be responsible for further development and worldwide commercialization for the corresponding licensed product;
Exicure received a $20 million upfront payment and is eligible to receive up to $1 billion in option exercise fees and milestone payments should Ipsen opt into both programs, as well as tiered royalties.
XCUR-FXN – Friedreich’s Ataxia

The Company hosted a virtual R&D Day on July 15, 2021 to present new and previously unreleased preclinical data and discuss progress with XCUR-FXN:
Observed 2-3x fold change in measurable Frataxin protein in the cerebellum and dorsal root ganglia (amongst other important brain and spinal regions) in Pook800J mouse model indicating potential for disease resolution;
Showed no adverse, test-related histopathological findings in repeat dose range finding rat study.
The Company continues to expect to file for an IND for XCUR-FXN in FA by the end of 2021 and to dose the first human patient in the first half of 2022.
SCN9A – Neuropathic Pain

Presented supporting data for targeting the SCN9A gene which encodes for the NaV1.7 sodium channel, a sought after, but difficult to drug target with highly selective SNA approach.
CLN3 – Batten Disease

Generated in vivo Proof of Concept data of an SNA splice switching mechanisms to correct mutations in the CLN3 gene and upregulate CLN3 mRNA in the retinae of Batten disease mice.
Immuno-Oncology

Cavrotolimod (AST-008)

The Phase 1b/2 clinical trial of intra-tumoral cavrotolimod in combination with approved checkpoint inhibitors pembrolizumab or cemiplimab, for the treatment of patients with advanced or metastatic Merkel cell carcinoma (MCC) or cutaneous squamous cell carcinoma (CSCC), is open and actively enrolling patients:
As of August 4, 2021, total trial enrollment for the Phase 1b/2 trial including primary and exploratory cohorts was 51 patients.
Interim results from the Phase 2 portion of the clinical trial were reported on August 5, 2021 on 26 patients, 17 of whom were evaluable.
A complete response (CR) in one MCC patient met the pre-specified Phase 2 stage threshold to continue advancing patient enrollment in the MCC cohort;
Injected and non-injected tumor lesions completely resolved in the MCC patient with a CR, supporting systemic (abscopal) effects;
Eight evaluable patients were enrolled across either the CSCC dose-expansion cohort, in which enrollment and data accrual is continuing, or exploratory cohort;
The confirmed overall response rate (ORR) in all evaluable MCC patients enrolled in total in the Phase 1b/2 study was 21% (3 of 14) as of the July 1, 2021 data cutoff date, comprised of two CRs and one partial response (PR);
The Company expects to provide ORR results from the Phase 2 portion of the trial in the first half of 2022.
Corporate Updates

Expanded the executive management team with the appointments of Brian C. Bock as Chief Financial Officer and Elias D. Papadimas as Chief Accounting Officer.
First Quarter Financial Results and Financial Guidance

Cash Position: Cash, cash equivalents, short-term investments, and restricted cash were $57.3 million as of June 30, 2021 compared to $68.6 million as of March 31, 2021.

Research and Development (R&D) Expenses: R&D expenses were $10.8 million for the quarter ended June 30, 2021, compared to $7.0 million for the quarter ended June 30, 2020. The Company has increased full-time headcount in R&D from 41 at June 30, 2020 to 66 at June 30, 2021. The increase in R&D expense reflects this increased headcount and the related increase in R&D activities, in addition to increased clinical trial activities.

General and Administrative Expenses: General and administrative expenses were $3.1 million for the quarter ended June 30, 2021, compared to $2.2 million for the quarter ended June 30, 2020. This increase is primarily due to costs related to new hires needed to grow the Company as it evolves.

Net Loss: Exicure had a net loss of $14.3 million for the quarter ended June 30, 2021 compared to net loss of $4.3 million for the quarter ended June 30, 2020. The increase in net loss was primarily driven by lower revenue associated with Exicure’s collaboration with AbbVie as well as higher R&D costs to advance our pipeline and higher G&A costs associated with an increase in headcount.

Cash Runway Guidance: The Company believes that, based on its current operating plans and estimates of future expenses, as of the date of this press release, its existing cash, cash equivalents and short-term investments, including the $20 million upfront payment received relating to the Company’s announced partnership with Ipsen subsequent to June 30, 2021, will be sufficient to fund its operations for at least the next 12 months.