Panbela Announces 1st Patient Enrolled in its Aspire Trial Studying SBP-101 in Combination with Gemcitabine and Nab-Paclitaxel in Patients with Metastatic Pancreatic Ductal Adenocarcinoma

On August 11, 2022 Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs, reported it has enrolled the first patient in its global clinical trial to study SBP-101 in combination with Gemcitabine and Nab-Paclitaxel in patients with metastatic pancreatic ductal adenocarcinoma, which is referred to as the ASPIRE trial, a randomized double-blind placebo-controlled trial, with a primary endpoint of overall survival (Press release, Panbela Therapeutics, AUG 11, 2022, View Source [SID1234618116]). Detailed information on the trial can be located at View Source

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"While site initiation was gradual, we are pleased with the current momentum of the ASPIRE trial. We expect that a significant number of global sites will be open by year-end with the full complement of sites open by the first quarter 2023. Australia opened last week with the first site activated within the country, and now they’ve enrolled the first patient into the study," commented, Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer of Panbela. "Australian study centers have been wonderful to work with and were important contributors to our phase 1 trial, enrolling a heavy preponderance of the 50 patients. We’re excited to reach this milestone of first patient enrolled, as we move forward towards interim analysis which is expected to complete in early 2024."

Australia marked the second country activated for the ASPIRE trial, and first to enroll, with approximately 90 additional sites expected to be activated across 10 countries by early 2023.

Additionally, in response to European and FDA regulatory feedback, the study was amended to include the total trial sample size (600 subjects) and the design modified to utilize overall survival as the primary endpoint to be examined at an interim analysis. While the trial is expected to take approximately 36 months to fully enroll, the interim analysis is still expected to occur in early 2024.

About our Pipeline

The pipeline consists of assets currently in clinical trials with an initial focus on familial adenomatous polyposis (FAP), first-line metastatic pancreatic cancer, neoadjuvant pancreatic cancer, colorectal cancer prevention and ovarian cancer. The combined development programs have a steady cadence of catalysts with programs ranging from pre-clinical to registration studies.

SBP-101

SBP-101 is a proprietary polyamine analogue designed to induce polyamine metabolic inhibition (PMI) by exploiting an observed high affinity of the compound for pancreatic ductal adenocarcinoma and other tumors. The molecule has shown signals of tumor growth inhibition in clinical studies of US and Australian metastatic pancreatic cancer patients, demonstrating a median overall survival (OS) of 14.6 months which is final, and an objective response rate (ORR) of 48%, both exceeding what is seen typically with the standard of care of gemcitabine + nab-paclitaxel suggesting potential complementary activity with the existing FDA-approved standard chemotherapy regimen. In data evaluated from clinical studies to date, SBP-101 has not shown exacerbation of bone marrow suppression and peripheral neuropathy, which can be chemotherapy-related adverse events. Serious visual adverse events have been evaluated and patients with a history of retinopathy or at risk of retinal detachment will be excluded from future SBP-101 studies. The safety data and PMI profile observed in the current Panbela sponsored clinical trial provides support for continued evaluation of SBP-101 in a randomized clinical trial. For more information, please visit View Source .

Flynpovi

Flynpovi is a combination of CPP-1X (eflornithine) and sulindac with a dual mechanism inhibiting polyamine synthesis and increase polyamine export and catabolism. In a Phase 3 clinical trial in patients with sporadic large bowel polyps, the combination prevented > 90% subsequent pre-cancerous sporadic adenomas versus placebo. Focusing on FAP patients with lower gastrointestinal tract anatomy in the recent Phase 3 trial comparing Flynpovi to single agent eflornithine and single agent sulindac, FAP patients with lower GI anatomy (patients with an intact colon, retained rectum or surgical pouch), Flynpovi showed statistically significant benefit compared to both single agents (p≤0.02) in delaying surgical events in the lower GI for up to four years. The safety profile for Flynpovi did not significantly differ from the single agents and supports the continued evaluation of Flynpovi for FAP.

CPP-1X

CPP-1X (eflornithine) is being developed as a single agent tablet or high dose power sachet for several indications including prevention of gastric cancer, treatment of neuroblastoma and recent onset Type 1 diabetes. Preclinical studies as well as Phase 1 or Phase 2 investigator-initiated trials suggest that CPP-1X treatment is well tolerated and has potential activity.

Veru Reports Third Quarter Fiscal 2022 Financial Results; Progresses Sabizabulin Treatment for COVID-19 Towards Regulatory Decisions in Key Territories

On August 11, 2022 Veru Inc. (NASDAQ: VERU), a biopharmaceutical company focused on developing novel medicines for COVID-19 and other viral and Acute Respiratory Distress Syndrome (ARDS)-related diseases and for the management of breast and prostate cancers, reported financial results for its fiscal 2022 third quarter ended June 30, 2022, and sabizabulin for COVID progress towards regulatory decisions in key territories (Press release, Veru, AUG 11, 2022, View Source [SID1234618094]).

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Third Quarter Financial Summary: Fiscal 2022 vs Fiscal 2021

Total net revenues decreased 46% to $9.6 million from $17.7 million

US FC2 prescription net revenues decreased 50% to $6.7 million from $13.5 million

Gross profit decreased 49% to $7.1 million from $13.9 million

Gross margin decreased to 74% of net revenues from 79% of net revenues

Research and development expenses increased to $18.1 million from $11.2 million

Operating loss was $21.8 million versus $2.9 million

Net loss was $22.2 million, or $0.28 per share, compared to $2.7 million, or $0.03 per share

Year-to-Date Financial Summary: Fiscal 2022 vs Fiscal 2021

Total net revenues decreased 19% to $36.8 million from $45.6 million

US FC2 prescription net revenues decreased 9% to $29.9 million from $32.9 million

Gross profit decreased 16% to $30.1 million from $35.6 million

Gross margin increased to 82% of net revenues from 78% of net revenues

Research and development expenses increased to $43.8 million from $24.4 million

Operating loss was $38.6 million compared with operating income of $14.8 million, which included an $18.4 million gain on the December 2020 sale of the PREBOOST business

Net loss was $42.8 million, or $0.53 per diluted share, compared with net income, which included the gain on sale of the PREBOOST business, of $11.7 million, or $0.14 per diluted share

Balance Sheet Information

Cash and cash equivalents were $100.6 million as of June 30, 2022 versus $122.4 million as of September 30, 2021

Net accounts receivable were $8.3 million as of June 30, 2022 versus $8.8 million as of September 30, 2021

"COVID-19 new cases and hospitalizations are on the rise again with both summer and fall-winter surges expected. Unfortunately, the death rate in hospitalized patients with moderate to severe COVID-19 who are at risk for ARDS remains unacceptably high with current standard of care," said Mitchell Steiner, M.D., Chairman, President, and Chief Executive Officer of Veru. "By reducing deaths in hospitalized COVID-19 patients, sabizabulin has great potential to play a critical role in the battle against COVID-19."

Dr. Steiner added: "I am proud of how expeditiously the Veru team moved to get the Emergency Use Authorization (EUA) application submitted to FDA in June. I was also very pleased to see the UK’s Medicines and Healthcare Products Regulatory Agency’s decision to expedite review of a marketing authorization application for sabizabulin as well as the European Medicines Agency’s Emergency Task Force’s decision to initiate data review, for the first time ever under their article 18, for potential emergency use of sabizabulin in European Union member countries. Veru has scaled up manufacturing of sabizabulin 9mg capsules to meet the needs of patients in the U.S. and ex-US, if authorizations are received, and we are building our U.S. and ex-U.S. infectious disease commercial franchises."

Finally, Dr. Steiner noted: "We expect to have significant near-term revenue from sabizabulin for the treatment of hospitalized COVID-19 patients at high risk for ARDS, if the EUA is granted by the U.S. FDA. The decrease in the third quarter FC2 net revenues in the U.S. prescription channel is primarily due to lower volume from telemedicine customers because of some business challenges experienced by our customers which resulted in a slow-down in orders during the current quarter. We expect their historical ordering patterns to resume in future quarters, although there is uncertainty as to timing of the resumption, and we also anticipate new revenues from the launch of ENTADFI which is now commercially available."

Pharmaceutical Pipeline Highlights:

Infectious Disease Franchise:

The Company has Completed a Positive Phase 3 COVID-19 Study in Hospitalized Moderate to Severe COVID-19 Patients at High Risk for ARDS.

A double-blind, randomized, placebo-controlled Phase 3 COVID-19 clinical trial was conducted in approximately 210 hospitalized COVID-19 patients with moderate to severe COVID (≥ WHO 4-supplemental oxygen) at high risk for ARDS and death. The primary endpoint was the proportion of deaths by Day 60. Based on a planned interim analysis of the first 150 patients randomized, the Independent Data Monitoring Committee unanimously halted the study for overwhelming efficacy and safety. Treatment with sabizabulin 9mg once daily, an oral, first-in-class, new chemical entity, microtubule disruptor that has dual anti-inflammatory and antiviral properties, resulted in a clinically meaningful and statistically significant 55.2% relative reduction in deaths.

On June 6, 2022, the Company submitted a request for emergency use authorization to FDA. On July 6, 2022, the Company announced the publication of the Phase 3 COVID-19 trial results evaluating the efficacy and safety of oral sabizabulin in The New England Journal of Medicine Evidence. On July 25, 2022, the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) informed the Company that the sabizabulin marketing authorization application will receive expedited review. On July 27, 2022, the European Medicines Agency’s Emergency Task Force initiated the review of sabizabulin treatment for hospitalized COVID-19 patients for emergency use in European Union countries.

The Company has scaled up manufacturing processes and will be able to produce commercial drug supply to address anticipated drug needs following potential FDA authorization and subsequent authorizations in the U.S. as well as other countries and regions.

The Company has initiated discussions with government agencies to discuss potential government purchases of sabizabulin in the U.S. and other countries around the world.

Breast Cancer Program

Enobosarm, a Novel Oral Selective Androgen Receptor Targeting Agonist, for the 3rd Line Treatment of AR+ ER+ HER2- Metastatic Breast Cancer with AR ≥ 40% Expression—Phase 3 ARTEST Clinical Study- Enrolling.

Enobosarm is an oral, new chemical entity, selective androgen receptor targeting agonist that activates the androgen receptor (AR), a tumor suppressor, in AR+ER+HER2- metastatic breast cancer without causing unwanted masculinizing side effects. Enobosarm has extensive nonclinical and clinical experience having been evaluated in 25 separate clinical studies in approximately 1,450 subjects dosed, including three Phase 2 clinical studies in advanced metastatic 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 previously failed estrogen receptor blocking agents, chemotherapy, and/or CDK 4/6 inhibitors and enobosarm was well tolerated with a favorable safety profile.

We are enrolling the Phase 3 multicenter, international, open label, and randomized (1:1) ARTEST registration clinical trial design to evaluate enobosarm monotherapy versus physician’s choice of either exemestane ☐ everolimus or a selective estrogen receptor modulator (SERM) as the active comparator for the treatment of AR+ ER+ HER2- metastatic breast cancer in approximately 210 patients with AR expression ≥40% in their breast cancer tissue who had previously received a nonsteroidal aromatase inhibitor, fulvestrant, and a CDK4/6 inhibitor. In January 2022, the FDA granted Fast Track designation to the ARTEST Phase 3 registration program, a distinction that underscores the urgent need for novel, targeted therapies for this important unmet medical need.

Enobosarm and Abemaciclib, CDK 4/6 Inhibitor, Combination Therapy for the 2nd Line Treatment of AR+ER+HER2- Metastatic Breast Cancer with AR ≥ 40% Expression – Phase 3 ENABLAR-2 Clinical Study-Enrolling.

We are enrolling the Phase 3 multicenter, open label, randomized (1:1), active control clinical study, named ENABLAR-2 to evaluate the treatment of the enobosarm and abemaciclib combination versus an alternative estrogen blocking agent (fulvestrant or an aromatase inhibitor) in subjects with AR+ ER+ HER2- metastatic breast cancer who have failed first line palbociclib (a CDK 4/6 inhibitor) plus an estrogen blocking agent (non-steroidal aromatase inhibitor or fulvestrant) and who have an AR ≥ 40% expression in their breast cancer tissue in approximately 186 subjects. We have a clinical trial collaboration and supply agreement with Lilly for our Phase 3 ENABLAR-2 trial.

Sabizabulin, Novel Oral Cytoskeleton Disruptor Agent, for the 3rd Line Treatment of AR+ER+HER2- Metastatic Breast Cancer with AR< 40% Expression—Phase 2b Clinical Study.

We intend to conduct a Phase 2b clinical study which will be an open label, multicenter, and randomized (1:1) study evaluating sabizabulin 32mg monotherapy versus active comparator (exemestane ± everolimus or a SERM, physician’s choice) for the treatment of AR+ ER+ HER2- metastatic breast cancer in approximately 200 patients with AR <40% expression in their breast cancer tissue who have previously received a nonsteroidal aromatase inhibitor, fulvestrant, and a CDK4/6 inhibitor.

Prostate Cancer Program

Sabizabulin for the Treatment of Metastatic Castration and Androgen Receptor Targeting Agent Resistant Prostate Cancer – Phase 3 VERACITY Clinical Study—Enrolling.

The Company is enrolling the open label, randomized (2:1), multicenter Phase 3 VERACITY clinical study 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 tumor progression after previously receiving at least one androgen receptor targeting agent. The primary endpoint is radiographic progression free survival in 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.

VERU-100 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. In June 2021, the Company initiated the Phase 2 dose finding clinical study of VERU-100 androgen deprivation therapy for hormone sensitive advanced prostate cancer. The Phase 2 VERU-100 clinical study is expected to enroll approximately 45 patients. A Phase 3 registration clinical study has been agreed upon with FDA and will enroll approximately 100 men.

Urev – Sexual Health Division

ENTADFI (tadalafil and finasteride) capsule, a new Treatment for Benign Prostatic Hyperplasia (BPH) – commercially launched

Today the Company initiated the U.S. commercial launch and availability of ENTADFI– a new oral treatment for benign prostatic hyperplasia. ENTADFI has been shown to be faster and more effective to treat urinary tract symptoms caused by BPH with less potential for adverse sexual side effects compared to finasteride monotherapy. ENTADFI dosing is one capsule orally once a day, and the FDA approved indication is to initiate treatment of the signs and symptoms of BPH in men with an enlarged prostate for up to 26 weeks. ENTADFI will be featured on GoodRx, a leading consumer-focused digital healthcare platform, to drive awareness among consumers and providers.

FC2 Female Condom/Internal Condom

The Company markets and sells the FC2, an FDA-approved product for dual protection against unplanned pregnancy and the transmission of sexually transmitted infections.

Event Details

Interested parties may access the call by dialing 1-800-341-1602 from the U.S. or 1-412-902-6706 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 1-877-344-7529 for U.S. callers, or 1-412-317-0088 from outside the U.S., passcode 1902173, for one week.

Synlogic Reports Second Quarter 2022 Financial Results and Provides Business Update

On August 11, 2022 Synlogic, Inc. (Nasdaq: SYBX), a clinical-stage biotechnology company developing medicines for metabolic and immunological diseases through its proprietary approach to synthetic biology, reported financial results for the second quarter ended June 30, 2022 and provided an update on its pipeline programs (Press release, Synlogic, AUG 11, 2022, View Source [SID1234618093]).

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"We look forward to three clinical data readouts in the remainder of this year for our programs in phenylketonuria (PKU), homocystinuria (HCU) and enteric hyperoxaluria (EH), reflecting the progress of our Synthetic Biotic platform," said Aoife Brennan, M.B. Ch.B, Synlogic President and Chief Executive Officer. "Our research productivity continues with the naming of our latest metabolic disease drug candidate and pipeline addition."

Anticipated Upcoming Milestones

Report additional Phase 2 data and final candidate selection for PKU program in H2 2022
Initiate the Phase 3 trial for the PKU program in H1 2023 with Phase 3 readiness activities currently underway
Share data from the Phase 1 trial in healthy volunteers for SYNB1353 for HCU in H2 2022
Share proof of concept data for SYNB8802 for EH in H2 2022
Recent Business Highlights

Received Positive Opinion on Orphan Designation from the European Medicines Agency for SYNB1618 for the treatment of PKU
Initiated the Phase 1 study of SYNB1353 in healthy volunteers for the treatment of HCU
Named SYNB2081, a Synthetic Biotic designed to lower uric acid, for the treatment of gout, our second drug candidate through our partnership with Ginkgo Bioworks
Brendan St. Amant appointed to General Counsel and Corporate Secretary
Participated and presented data at the following scientific congresses and patient conferences:
EH program data were presented at the American Urological Association’s Annual Meeting held May 13-16, 2022; IBD program data were presented at Digestive Disease Week held May 21-24, 2022; and HCU program data were presented at the Boston Bacterial Meeting on June 14, 2022.
PKU and HCU program data were presented at the 2022 HCU Network America, Organic Acidemia Association and Propionic Acidemia Foundation 2022 Conference held June 25-26, 2022, and at the National PKU Alliance’s (NPKUA) biennial conference held July 7-10, 2022.
Second Quarter 2022 Financial Results

As of June 30, 2022, Synlogic had cash, cash equivalents and short-term investments of $106.8 million.

Revenue for the three months ended June 30, 2022 and for the corresponding period in 2021 was $0.2 million. Revenue in both periods was associated with the ongoing research collaboration with Roche for the discovery of a novel Synthetic Biotic for the treatment of IBD.

For the three months ended June 30, 2022, Synlogic reported a consolidated net loss of $15.8 million, or $0.22 per share, compared to a consolidated net loss of $14.5 million, or $0.28 per share, for the corresponding period in 2021.

Research and development expenses were $12.1 million for the three months ended June 30, 2022 compared to $10.7 million for the corresponding period in 2021.

General and administrative expenses were $4.1 million for the three months ended June 30, 2022 and for the corresponding period in 2021.

Financial Outlook

Based upon its current operating plan and balance sheet as of June 30, 2022, Synlogic expects to have sufficient cash to be able to fund operations into 2024.

Investor and Industry Conference Participation

Synlogic will participate in the following upcoming investor and industry conferences:

The H.C. Wainwright Global Investment Conference. Michael Jensen, Chief Financial Officer, and Molly Harper, Chief Business Officer, will provide a company presentation on Monday, September 12, 2022 during the H.C. Wainwright 24th Annual Global Investment Conference being held September 12-14, 2022 virtually and in-person in New York City.
Live webcasts of the presentations, if available, will be accessible under the "Event Calendar" in the Investors & Media section of the Synlogic website. Archived versions will be available afterwards at the same location.

Conference Call & Webcast Information

Synlogic will host a conference call and live webcast at 8:30 a.m. ET today, August 11, 2022. To access the webcast, please register here. To access the call by phone, please register here and you will be provided with dial-in details. You can also access these links on the "Events Calendar" section of the Investors & Media webpage. For those unable to participate in the conference call or webcast, a replay will be available for 30 days on the Synlogic website.

Spectrum Pharmaceuticals Reports Second Quarter 2022 Financial Results and Provides Corporate Update

On August 11, 2022 Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, reported financial results for the three-month period ended June 30, 2022 and provided a corporate update (Press release, Spectrum Pharmaceuticals, AUG 11, 2022, View Source [SID1234618092]).

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"The completion of the FDA re-inspection of the drug substance facility for eflapegrastim is a critical step in the regulatory review process. With this hurdle behind us, we have turned our focus to our potential approval and commercialization," said Tom Riga, President and Chief Executive Officer of Spectrum Pharmaceuticals. "Additionally, our team is engaged in extensive preparations ahead of poziotinib’s ODAC review in September, and we look forward to moving a step closer to bringing this therapy to patients."

Pipeline Updates

Eflapegrastim, a novel long-acting G-CSF

The Biologics License Application (BLA) for eflapegrastim is under active review at the U.S. Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) date of September 9, 2022. The pre-approval inspection of the drug substance manufacturing facility has been completed by the FDA. The company anticipates an FDA decision by the PDUFA date and is actively preparing for the potential commercial launch.
Poziotinib, a Pan ErbB inhibitor targeting HER2 exon 20 mutations

The New Drug Application (NDA) for poziotinib is under active review at the FDA with Fast Track designation and a PDUFA date of November 24, 2022. The NDA is based on the positive results of Cohort 2 from the ZENITH20 clinical trial in patients with previously treated locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring HER2 exon 20 insertion mutations. There is currently no FDA approved therapy for patients with NSCLC harboring HER2 exon 20 insertion mutations.
An abstract showing a high level of activity for poziotinib in patients with a G778_P780dup mutation, the second most prevalent mutation in HER2 exon 20 NSCLC, has been accepted for presentation at ESMO (Free ESMO Whitepaper) 2022 being held September 9-13 in Paris. The data comes from Cohorts 2 and 4 of the ZENITH20 clinical trial.
A study for poziotinib is in progress to confirm the clinical benefit seen in Cohort 2, as required for an accelerated approval. The trial, Study SPI-POZ-301 (PINNACLE), is designed to enroll 268 patients with previously treated NSCLC harboring HER2 exon 20 mutations. Patients are being randomized 2-to-1 into one of two treatment arms using 8mg of poziotinib orally administered BID (twice daily) versus 75mg/m2 of docetaxel administered intravenously every three weeks. The primary endpoint is progression free survival.
The FDA’s Oncologic Drugs Advisory Committee (ODAC) is scheduled to review poziotinib for the treatment of patients with previously treated locally advanced or metastatic NSCLC harboring HER2 exon 20 insertion mutations. The poziotinib ODAC review is scheduled for September 22, 2022 at 9 a.m. ET. ODAC is an independent panel of experts that evaluates data concerning the efficacy and safety of marketed and investigational products for use in the treatment of cancer and makes non-binding recommendations to the FDA. The final decision regarding the approval of the product is made solely by the FDA.
The company presented data on the predictive ability of circulating tumor DNA (ctDNA) in poziotinib treated patients with NSCLC harboring HER2 exon 20 insertion mutations at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June. Preliminary results suggest that decreases in plasma ctDNA during poziotinib therapy correlate with clinical response in patients with advanced NSCLC with HER2 exon 20 insertion mutations.
Three-Month Period Ended June 30, 2022 (All numbers are from Continuing Operations)

Spectrum recorded a net loss of $29.0 million, or a $0.17 loss per basic and diluted share, in the three-month period ended June 30, 2022, compared to a net loss of $49.9 million, or a $0.32 loss per basic and diluted share, in the comparable period in 2021. Total research and development expenses were $16.0 million in the quarter, as compared to $29.1 million in the same period in 2021. Selling, general and administrative expenses were $9.4 million in the quarter, compared to $15.0 million in the same period in 2021.

Cash Position and Guidance

The company’s cash, cash equivalents and marketable securities balance was approximately $68 million at June 30, 2022, which provides for a cash runway into 2023.

Conference Call

Thursday, August 11, 2022 @ 8:30 a.m. Eastern/5:30 a.m. Pacific

To access the live call by phone, please go to this link (registration link), and you will be provided with dial in details. To avoid delays, participants are encouraged to dial into the conference call fifteen minutes ahead of the scheduled start time.

This conference call will also be webcast. Listeners may access the webcast, which will be available on the investor relations page of Spectrum Pharmaceuticals’ website: View Source on August 11, 2022 at 8:30 a.m. Eastern/5:30 a.m. Pacific.

NeuBase Therapeutics Reports Business Update and Financial Results for the Third Quarter of Fiscal Year 2022

On August 11, 2022 NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology platform company Drugging the Genome to address disease at the base level using a new class of precision genetic medicines, reported its financial results for the three-month period ended June 30, 2022, and other recent developments (Press release, NeuBase Therapeutics, AUG 11, 2022, View Source [SID1234618091]).

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"Thus far in 2022, we have generated new data that strengthens application of the PATrOLTM platform and its potential to output novel genetic medicines that are broadly biodistributed after systemic routes of administration and engage double-stranded nucleic acid targets in the nucleus to resolve gene dysfunction. This includes new PK and biodistribution data for our DM1 development candidate, which were presented at the ASGCT (Free ASGCT Whitepaper) 25th Annual Meeting, that support a differentiated whole-body solution for DM1 patients," said Dietrich A. Stephan, Ph.D., Founder, Chief Executive Officer, and Chairman of NeuBase. "The data in each tissue we evaluated also displayed an extended elimination phase with tissue concentrations measurable for at least four weeks following a single intravenous (IV) dose administration and consistent with the durable pharmacology in the transgenic animal models. With these data in hand, we decided to perform additional IND-enabling studies to further characterize our development candidate and confirm our expectations of safety and efficacy. While conducting these additional studies will require us to push our guidance on the timing for our DM1 IND filing to mid-CY2023, patient safety and benefit are of utmost importance to us. We also believe the information that will be generated by these studies will benefit not only our DM1 program but all of our programs and better prepare us to enter the clinic and fulfill our mission of delivering new medicines to patients who currently have no therapeutic options."

Dr. Stephan continued, "We continue to generate positive data from other programs in our pipeline, including in HD, that support the ability of the PATrOLTM platform to develop differentiated genetic medicines. We are confident in the ability of our platform and team to advance these drug candidates to clinical trials, and we look forward to providing further updates on our progress."

Third Quarter of Fiscal Year 2022 and Recent Operating Highlights

Myotonic Dystrophy Type 1 (DM1) Program: NeuBase is making steady progress advancing IND-enabling studies for its development candidate in the DM1 program, which includes PK, absorption, distribution, metabolism, and excretion (ADME), and bioavailability via IV and subcutaneous routes of administration, exploratory and IND-enabling Good Laboratory Practice (GLP) toxicology, and mechanism of action studies. In addition, Good Manufacturing Practice (GMP) of NeuBase’s development candidate to support Phase 1/2 clinical trials has been successfully implemented via contract manufacturing organizations.
In May 2022, the Company presented preclinical PK and biodistribution data at the ASGCT (Free ASGCT Whitepaper) 25th Annual Meeting for its lead development candidate, NT-0231.F, supporting a differentiated whole body treatment solution for DM1. Following a single IV injection of 30 mg/kg in wild-type BALB/c mice, NT-0231.F was cleared rapidly from the systemic compartment and demonstrated rapid and wide distribution into tibialis anterior muscle, heart muscle, and brain tissues. NT-0231.F rapidly cleared the plasma, and each tissue evaluated displayed an extended elimination phase with tissue concentrations measurable for at least four weeks following a single IV dose administration.
Completed exploratory toxicology work in non-human primates and rats.
Initiated a collaboration with Inserm to further study NT-0231.F using the DMSXL mouse model to measure central nervous system and heart pharmacology and to refine the human equivalent dose.
Committed to support the Myotonic Dystrophy Clinical Research Network, the world’s largest patient organization focused solely on myotonic dystrophy.
The Company expects to submit an IND application to the FDA for NT-0231.F in mid-CY2023.
Huntington’s Disease (HD) Program: Preclinical development activities in the Company’s HD program progressed during the second quarter of CY2022. During the second half of CY2022, NeuBase expects to present new preclinical data at upcoming scientific conferences describing the pharmacology of a candidate compound in the brain after systemic administration, nominate a development candidate, and initiate scale-up and toxicology activities.
KRAS Oncology Program: The Company continued in vitro mechanistic studies and in vivo pharmacology studies for the KRAS program (KRAS G12V and G12D mutations) to support the development of an allele-selective approach to engaging mutant KRAS at the DNA and RNA levels.
Management Update: William (Bill) Mann, Ph.D. was promoted to President of the Company. Dr. Mann continues to serve as NeuBase’s Chief Operating Officer.
Financial Results for the Third Fiscal Quarter Ended June 30, 2022

As of June 30, 2022, the Company had cash and cash equivalents of approximately $29.8 million, compared with approximately $52.9 million as of September 30, 2021.
For the fiscal quarter ended June 30, 2022, the Company reported a net loss of approximately $8.5 million, or a net loss of $0.26 per share, compared with a net loss of approximately $8.7 million, or a net loss of $0.29 per share, for the same period last year.
For the fiscal quarter ended June 30, 2022, total operating expenses were approximately $8.4 million, consisting of approximately $3.6 million in general and administrative expenses and $4.8 million of research and development expenses. This compares with total operating expenses of approximately $8.8 million for the same period last year, consisting of approximately $3.5 million in general and administrative expenses and $5.3 million in research and development expenses.
Financial Results for the Nine-Month Period Ended June 30, 2022

For the nine-month period ended June 30, 2022, the Company reported a net loss of approximately $26.1 million, or a net loss of $0.80 per share, compared with a net loss of approximately $18.3 million, or a net loss of $0.72 per share, for the same period last year.
For the nine-month period ended June 30, 2022, total operating expenses were approximately $25.6 million, consisting of approximately $9.6 million in general and administrative expenses and $16.0 million of research and development expenses. This compares with total operating expenses of approximately $19.4 million for the same period last year, consisting of approximately $8.8 million in general and administrative expenses and $10.6 million in research and development expenses.