Mustang Bio Announces Exclusive Worldwide License Agreement with Leiden University Medical Centre for Clinical-Stage Lentiviral Gene Therapy with Curative Potential for RAG1 Severe Combined Immunodeficiency

On November 10, 2021 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported that the company has executed an exclusive license agreement with Leiden University Medical Centre ("LUMC") for a first-in-class ex vivo lentiviral gene therapy for the treatment of RAG1 severe combined immunodeficiency ("RAG1-SCID") (Press release, Mustang Bio, NOV 10, 2021, View Source [SID1234595269]).

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The therapy, which includes low-dose conditioning prior to reinfusion of the patients’ own gene-modified blood stem cells, is currently being evaluated in a Phase 1/2 multicenter clinical trial in Europe. The ongoing clinical trial recently enrolled its first patient, and additional clinical sites are expected to be added in the near future. The RAG1-SCID program has been granted Orphan Drug Designation by the European Medicines Agency.

Mustang also established an ongoing partnership with Frank J. Staal, Ph.D., professor of Molecular Stem Cell Biology and molecular immunologist, whose laboratory developed the therapy. Dr. Staal will continue the development of additional lentiviral gene therapies in his lab, to which Mustang Bio has rights under the agreement.

The RAG1-SCID therapy expands the pipeline of ex vivo lentiviral gene therapies currently in development at Mustang. The Company’s lead programs, MB-107 and MB-207, are being investigated for the treatment of X-linked severe combined immunodeficiency ("XSCID"). A pivotal multicenter trial studying MB-107 is expected to enroll its first patient in the first quarter of 2022. XSCID and RAG1-SCID make up almost 60% of all SCID cases1 combined.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang said, "We are excited to add RAG1-SCID to the Mustang portfolio as it enables us to leverage our lentiviral gene therapy expertise and experience and our state-of-the-art cell processing facility. Mustang is establishing itself as the leader in developing treatments for patients with severe combined immunodeficiency, an area of high unmet need. We have made great progress in moving our XSCID therapy into a registrational trial and look forward to similarly advancing this RAG1-SCID therapy to make it available for patients in need of life-saving treatment."

About RAG1-SCID
Severe combined immunodeficiency (SCID) due to complete recombinase-activating gene-1 (RAG1) deficiency is a rare, genetic severe combined immunodeficiency disorder due to null mutations in the RAG1 gene resulting in less than 1% of wild type V(D)J recombination activity. Patients present with neonatal onset of life-threatening, severe, recurrent infections by opportunistic fungal, viral and bacterial micro-organisms, as well as skin rashes, chronic diarrhea, failure to thrive and fever. Immunologic observations include profound T- and B-cell lymphopenia, low or absent serum immunoglobulins, and normal natural killer cell counts. As is the case with other types of SCID, RAG1-SCID is fatal in infancy unless immune reconstitution is achieved with hematopoietic stem cell transplantation (HSCT).

EDAP TMS SA to Announce Third Quarter 2021 Financial Results on Wednesday, November 17, 2021

On November 10, 2021 EDAP TMS SA (Nasdaq: EDAP), the global leader in therapeutic ultrasound, reported that it will release its financial results for the third quarter ended September 30, 2021 after the markets close on Wednesday, November 17, 2021 (Press release, EDAP TMS, NOV 10, 2021, View Source [SID1234595268]).

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An accompanying conference call and webcast will be conducted by Marc Oczachowski, Chairman of the Board and Chief Executive Officer, Ryan Rhodes, Chief Executive Officer of EDAP U.S.A., and François Dietsch, Chief Financial Officer. The call will be held at 8:30am ET on Thursday, November 18, 2021. Please refer to the information below for conference call dial-in information and webcast registration.

DiaMedica Therapeutics Provides a Business Update and Announces Third Quarter 2021 Financial Results

On November 10, 2021 DiaMedica Therapeutics Inc. (Nasdaq: DMAC), a clinical-stage biopharmaceutical company focused on developing novel treatments for neurological disorders and kidney diseases, reported a business update and financial results for the quarter ended September 30, 2021 (Press release, DiaMedica, NOV 10, 2021, View Source [SID1234595239]). Management will host a conference call Thursday, November 11, 2021, at 7:00AM Central Time/8:00AM Eastern Time to discuss its business update and third quarter financial results.

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Pivotal Phase 2/3 ReMEDy Trial of DM199 for Acute Ischemic Stroke Initiated & First Patient Dosed

The Company previously announced the initiation of the first site and reported dosing of the first patient for its pivotal ReMEDy2 trial. The ReMEDy2 trial is a randomized, double-blind, placebo-controlled Phase 2/3 adaptive trial intended to enroll approximately 350 patients. Patients enrolled in the study will be treated with either DM199 or placebo within 24 hours of the onset of acute ischemic stroke (AIS) symptoms. The trial is studying AIS in a patient population for whom thrombolysis and/or a catheter-based procedure, mechanical thrombectomy, are not medically appropriate or available due to constraints of clot location, comorbidity risks or time from estimated onset of stroke, which represents approximately 80% of all AIS patients.

Also as previously announced, the U.S. Food and Drug Administration (FDA) has accepted the Company’s protocol amendment to evaluate the effects of DM119 on the rate of recurrent AIS as a second, independent, primary endpoint. The FDA’s acceptance of the amendment allows the Company to evaluate the effects of DM199 on both stroke recoveries post AIS, as measured by the well-established modified Rankin Scale (mRS), and the incidence of AIS recurrence at day 90 as two separate, independent, primary endpoints, with each statistically powered for success. Recurrent strokes represent 25% of all ischemic strokes, often occur in the first few weeks after an initial stroke, and are typically more disabling, costly, and fatal than initial strokes. The Company further notes that there are no changes in treatment, duration, or study population of the trial as part of this protocol amendment.

Secondary endpoints for the study will evaluate participant deaths, mRS shift (which shows the treatment effect on participants across the full spectrum of stroke severity) and additional standard stroke scores (NIHSS and Barthel Index scores).

Fast Track Designation Granted to DM199 for Treatment of Acute Ischemic Stroke

The FDA granted Fast Track Designation to the Company’s lead candidate DM199 for the treatment of AIS. Fast Track is a process intended to facilitate the development and expedite the review of investigational drugs for the treatment of serious or life-threatening conditions where there is a significant unmet medical need. Drugs that receive Fast Track Designation may be eligible for more frequent communications and meetings with the FDA to review the drug’s development plan, including the design of the proposed clinical trials, use of biomarkers, and the extent of data needed for approval. Drugs with Fast Track Designation may also qualify for accelerated and priority review of new drug applications if relevant criteria are met.

Additional Interim Data From REDUX Phase 2 CKD Study for IgAN Presented at the ASN Kidney Week 2021 Virtual Conference

Additional data from the Company’s Phase 2 REDUX trial of DM199 in chronic kidney disease (CKD) was presented at the American Society of Nephrology’s (ASN) annual Kidney Week meeting taking place during the first week of November 2021. In the IgA Nephropathy (IgAN) cohort, in addition to continuing to show statistically significant reductions (over 30% decrease) in albuminuria in participants with moderate to severe baseline albuminuria, also demonstrated early signals of potential disease modification with the APRIL and IgA1 biomarkers decreasing 35% and 22%, respectively.

Balance Sheet Strengthened with $30 Million Private Placement

In September 2021, the Company issued and sold in a private placement an aggregate 7,653,060 common shares at a purchase price of $3.92 per share to ten accredited investors, resulting in gross proceeds of $30.0 million and net proceeds of $29.9 million, after deducting offering expenses.

"We are pleased that our regulatory interactions have led to acceptance of the protocol with the FDA and the Fast Track Designation status which will give the continued focus that this trial deserves given the unmet medical need," said Rick Pauls, DiaMedica’s President and Chief Executive Officer. " We believe that we are well positioned to advance toward our goal of offering a treatment option for the millions of patients around the world who suffer from a stroke and need a better chance to recover and avoid recurrent strokes."

Financial Results

Research and development (R&D) expenses increased slightly to $2.3 million for the three months ended September 30, 2021, up from $2.2 million for the three months ended September 30, 2020. R&D expenses increased to $6.9 million for the nine months ended September 30, 2021, compared to $5.1 million for the nine months ended September 30, 2020, an increase of $1.8 million. The increase for the nine month comparison was primarily due to a number of factors including costs incurred for the Company’s pivotal ReMEDy2 clinical study, increased year-over-year costs related to manufacturing process development and increased personnel costs associated with adding staff to support R&D operations. These increases were partially offset by decreased costs incurred for the earlier ReMEDy Phase 2 stroke study, which completed in the prior year, and the REDUX study as the number of enrollments declined in the later stages of the study.

General and administrative (G&A) expenses were $1.1 million for the three months ended September 30, 2021, down from $1.2 million for the three months ended September 30, 2020. G&A expenses increased to $3.5 million for the nine months ended September 30, 2021, up $0.2 million from $3.3 million for the nine months ended September 30, 2020. The increase for the nine month comparison was primarily due to increased professional services costs and increased personnel costs to support the Company’s expanding clinical programs. These increases were partially offset by reduced non-cash, share based compensation costs.

Balance Sheet and Cash Flow

As of September 30, 2021, DiaMedica had cash, cash equivalents and marketable securities of $48.1 million, working capital of $46.9 million and shareholders’ equity of $47.0 million, compared to $27.5 million in cash, cash equivalents and marketable securities, $25.9 million in working capital and shareholders’ equity of $26.0 million as of December 31, 2020. The increases in combined cash, cash equivalents and marketable securities and in working capital are due primarily to the net proceeds from the Company’s September 2021 private placement offering, partially offset by operating costs incurred during the quarter.

Net cash used in operating activities for the nine months ended September 30, 2021, was $9.4 million compared to $6.2 million for the nine months ended September 30, 2020. This increase relates primarily to the increase in the net loss, partially offset by non-cash share-based compensation and the effects of the changes in operating assets and liabilities.

Conference Call and Webcast Information

DiaMedica Management will host a conference call and webcast to discuss its business update and third quarter 2021 financial results on Thursday, November 11, 2021, at 7:00AM Central Time:

Interested parties may access the conference call by dialing in or listening to the simultaneous webcast. Listeners should log on to the website or dial in 15 minutes prior to the call. The webcast will remain available for play back on DiaMedica’s website, under investor relations – events and presentations, following the earnings call and for 12 months thereafter. A telephonic replay of the conference call will be available until November 18, 2021, by dialing (800) 770-2030 (US Toll Free) and entering the replay passcode: 4814247.

About DM199

DM199 is a recombinant (synthetic) form of human tissue kallikrein-1 (KLK1). KLK1 is a serine protease (protein) that plays an important role in the regulation of diverse physiological processes including blood flow, inflammation, fibrosis, oxidative stress and neurogenesis via a molecular mechanism that increases production of nitric oxide and prostaglandin. KLK1 deficiency may play a role in multiple vascular and fibrotic diseases such as stroke, chronic kidney disease, retinopathy, vascular dementia, and resistant hypertension where current treatment options are limited or ineffective. DiaMedica is the first company to have developed a recombinant form of the KLK1 protein. The KLK1 protein, produced from porcine pancreas and human urine, has been used to treat patients in Japan, China and South Korea for decades. DM199 is currently being studied in patients with acute ischemic stroke and patients with chronic kidney disease. In September 2021, the FDA granted Fast Track Designation to DM199 for the treatment of AIS.

2seventy bio to Present at the 5th Annual Cowen IO Next Summit

On November 10, 2021 2seventy bio, Inc. (Nasdaq: TSVT) reported that it will participate in a fireside chat at the 5th Annual Cowen IO Next Summit on November 15, at 1:15pm ET (Press release, 2seventy bio, NOV 10, 2021, View Source [SID1234595237]).

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A webcast of the presentation will be available via the investor relations section of the 2seventy bio website for 30 days following the event.

Erasca Reports Third Quarter 2021 Financial Results and Business Updates

On November 10, 2021 Erasca, Inc. (Nasdaq: ERAS), a clinical-stage precision oncology company singularly focused on discovering, developing, and commercializing therapies for patients with RAS/MAPK pathway-driven cancers, reported financial results for the fiscal quarter ended September 30, 2021, and provided business updates (Press release, Erasca, NOV 10, 2021, View Source [SID1234595236]).

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"Erasca continued our strong execution this quarter, delivering on key milestones, advancing new clinical trials, and generating exciting preclinical data," said Jonathan E. Lim, M.D., Erasca’s chairman, CEO, and co-founder. "With the initiation in September of our HERKULES-2 and -3 trials in non-small cell lung cancer and gastrointestinal malignancies, respectively, we currently have four ongoing clinical trials with multiple clinical data readouts anticipated in 2022. We are excited about recent preclinical data that further support the planned IND filings for ERAS-801 (CNS-penetrant EGFR inhibitor for refractory glioblastoma multiforme) in the first quarter of 2022 and ERAS-3490 (CNS-penetrant KRAS G12C inhibitor) in the second half of 2022."

Dr. Lim continued, "At the corporate level, we are honored to be named one of Fierce Biotech’s ‘Fierce 15’ most promising and innovative biotechnology companies of 2021. We remain focused on our mission to erase cancer and believe this mission goes beyond therapeutic development to include a broader social contribution. We were pleased to donate 1% of our pre-IPO capital stock to the Erasca Foundation in conjunction with the successful completion of our $345 million initial public offering in July. The Erasca Foundation was established to fund charitable activities designed to have a positive impact on society, particularly in underserved populations. With our strong cash position supported by top-tier institutional investors, we remain well-positioned to advance the industry’s deepest portfolio singularly focused on shutting down the RAS/MAPK pathway and to deliver on our upcoming milestones."

Research and Development (R&D) Highlights

Dosed First Patient in HERKULES-2 Trial: In September 2021, Erasca dosed the first patient in HERKULES-2, a Phase 1b/2 trial evaluating ERAS-007 (ERK1/2 inhibitor) in multiple combinations as part of Erasca’s lung cancer master protocol, with a focus on patients with advanced non-small cell lung cancer (NSCLC)
Dosed First Patient in HERKULES-3 Trial: In September 2021, Erasca dosed the first patient in HERKULES-3, a Phase 1b/2 trial evaluating ERAS-007 (ERK1/2 inhibitor) in multiple combinations as part of Erasca’s gastrointestinal cancer master protocol, with an initial focus on patients with advanced colorectal cancer (CRC)
Presented Preclinical Data for ERAS-801: In October 2021, Erasca announced the presentation of preclinical data for ERAS-801, a central nervous system (CNS)-penetrant epidermal growth factor receptor (EGFR) inhibitor for the treatment of refractory glioblastoma multiforme (GBM), at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Special Virtual Conference on Brain Cancer
Corporate Highlights

Completed $345 Million Initial Public Offering: In July 2021, Erasca sold 21,562,500 shares of common stock, which included the exercise in full by the underwriters of their option to purchase 2,812,500 shares of common stock, at a public offering price of $16 per share. The gross proceeds from the offering were $345 million
Named a 2021 "Fierce 15" Winner: Named one of Fierce Biotech’s "Fierce 15" most promising biotechnology companies of 2021
Entered into a Clinical Trial Collaboration and Supply Agreement with Pfizer: Pfizer will supply its BRAF inhibitor encorafenib (BRAFTOVI) at no cost in connection with a clinical proof-of-concept study evaluating ERAS-007 in combination with encorafenib and the EGFR inhibitor cetuximab for the treatment of patients with BRAF V600E-mutant metastatic CRC as part of the ongoing Phase 1b/2 HERKULES-3 trial
Contributed to the Erasca Foundation: Issued $17.5 million of common shares to the Erasca Foundation to fund charitable activities related to Erasca’s mission
Key Upcoming Milestones

ERAS-801: CNS-penetrant EGFR inhibitor
IND filing expected in first quarter of 2022
HERKULES-4: Phase 1b/2 clinical trial for ERAS-007/MAPKlamp in combination with various agents in patients with hematological malignancies
Dosing of the first patient expected in first quarter of 2022
Third Quarter 2021 Financial Results

Cash Position: Cash, cash equivalents, and investments were $486.6 million as of September 30, 2021, as compared to $118.7 million as of December 31, 2020. During the third quarter, Erasca completed an IPO raising net proceeds of $317.0 million, after deducting underwriting discounts, commissions, and other offering expenses. Erasca expects its current cash, cash equivalents, and investments balance to fund operations for at least the next 24 months.

Research and Development (R&D) Expenses: R&D expenses were $20.0 million for the quarter ended September 30, 2021, compared to $9.1 million for the quarter ended September 30, 2020. The increase was primarily driven by expenses incurred in connection with clinical trials and preclinical studies, personnel costs due to increased headcount to support increased development activities, and outsourced services and consulting fees. Erasca also recorded $1.7 million of in-process research and development expense during the quarter ended September 30, 2021, for a milestone payment made to the University of California, San Francisco.

General and Administrative (G&A) Expenses: G&A expenses were $6.9 million for the quarter ended September 30, 2021, compared to $2.0 million for the quarter ended September 30, 2020. The increase was primarily driven by personnel costs, insurance costs, and legal fees. For the quarter ended September 30, 2021, $17.5 million was recorded as additional G&A expense for the common shares issued to the Erasca Foundation.

Net Loss: For the quarter ended September 30, 2021, Erasca reported a net loss of $46.1 million, inclusive of the $17.5 million in expense recorded for the common shares issued to the Erasca Foundation, or $(0.46) per basic and diluted share, compared to a net loss of $10.6 million, or $(0.50) per basic and diluted share, for the quarter ended September 30, 2020.