On November 3, 2021 OS Therapies, a research and clinical-stage biopharmaceutical company whose lead program uses OST-HER2 (Listeria monocytogenes) is being developed for therapies to treat and cure Osteosarcoma (OS), reported the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RDD) for OST-HER2 (OST31-164) for the treatment of Osteosarcoma (Press release, OS Therapies, NOV 3, 2021, View Source [SID1234594282]).

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"The timing and determination by the FDA that OST-HER2 is a potentially much-needed new treatment in this underserved disease underscores the importance of our recently initiated PhIIb clinical trial in recurred, resected Osteosarcoma" said Paul Romness, CEO of OS Therapies. "The RDD ensures a well-deserved expedited review by the FDA, as well as a Priority Review Voucher (PRV) if our current trial is successful."

The FDA grants RDD (Rare Disease Designation) status for serious and life-threatening diseases that primarily affect children ages 18 years or younger and involves fewer than 200,000 people in the U.S. In addition to expedited review, if a PRV (Priority Review Voucher) is issued it can – at the election of OS Therapies – be transferred to larger Pharmaceutical and Biotechnology companies for a cash or other benefit-in-kind.

"The entire team at OS Therapies has been working diligently through a global pandemic to address the necessary regulatory hurdles in order to get this technology to pediatric patients as soon as possible," said Dr. Colin Goddard, Executive Chair of OS Therapies. "Not only are we addressing an unmet medical need, but the PRV and expedited review will have significant financing advantages that will enable us to support our ever-expanding pipeline for patients with other solid tumors."

About Osteosarcoma
Osteosarcoma is a solid tumor of the bone that predominantly occurs in adolescent and young adults (AYA). Standard treatment includes surgery and chemotherapy. For patients with initially metastatic or recurrence after chemotherapy, there is a significantly poorer prognosis.

On November 3, 2021 TransThera Sciences (Nanjing) Inc., a clinical-stage biopharmaceutical company focusing on discovering and developing innovative small molecule drug therapies for oncology, inflammatory and cardiovascular diseases, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its phase II stage product TT-00420 for the treatment of patients with Cholangiocarcinoma (CCA) who have no standard treatment options (Press release, TransThera Biosciences, NOV 3, 2021, View Source [SID1234594281]).

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TT-00420 is a spectrum selective kinase inhibitor that is found to show high potency to a variety of clinically identified FGFR2 mutations in preclinical experiments. The unique combination of TT-00420’s target profile and mechanism of action also allow it to effectively treat patients with heterogeneous tumors, such as CCA patients without clear biomarkers.

The grant of Fast Track Designation is primarily based upon the clinical results of TT-00420 in its completed phase I study, in which nine CCA patients were enrolled and treated with TT-00420 at different dose levels. Seven patients had at least one post treatment efficacy assessment. Two achieved PR and five achieved SD. Among these evaluable patients, (i) Five harbored FGFR2 fusion or rearrangement and developed acquired resistance to prior treatment of FGFR inhibitors. One patient achieved PR with PFS about ten months. The other four patients achieved SD. (ii) One patient with primary resistance achieved PR with PFS about eight months. (iii) One patient without FGFR alterations achieved SD with shrinkage of target lesion. The clinical evidence supports the continuing exploration of TT-00420 in treating CCA patients with no standard treatment options.

"Receiving Fast Track Designation is an important milestone for the development of TT-00420." commented excitedly by Dr. Frank Wu, CEO of TransThera, "We have been and will continue to actively work with FDA, expediting the clinical development of TT-00420 in CCA field."

In November 2019, TT-00420 was already granted Orphan Drug Designation by FDA to treat Cholangiocarcinoma.

About TT-00420

TT-00420 is a highly innovative clinical-stage spectrum-selective kinase inhibitor that exerts antitumor effects by targeting tumor cells and improving the tumor microenvironment. A large number of preclinical studies have found that TT-00420 has promising inhibitory effect on triple-negative breast cancer, cholangiocarcinoma and other malignant tumors. In September 2018, TT-00420 was approved by the US FDA for the first human clinical trial; in February 2019, it was approved by China’s NMPA for human clinical trials; and in November of the same year, TT-00420 was granted the "Orphan Drug Designation" status (ODD) by FDA for the treatment of cholangiocarcinoma. In November 2020, it was again approved by the US FDA targeting the clinical trial of new indications for cholangiocarcinoma.

On November 3, 2021 Heron Therapeutics, Inc. (Nasdaq: HRTX), a commercial-stage biotechnology company focused on improving the lives of patients by developing best-in-class treatments to address some of the most important unmet patient needs, reported financial results for the three and nine months ended September 30, 2021 and highlighted recent corporate updates (Press release, Heron Therapeutics, NOV 3, 2021, https://www.prnewswire.com/news-releases/heron-therapeutics-announces-financial-results-for-the-three-and-nine-months-ended-september-30-2021-and-highlights-recent-corporate-updates-301415716.html [SID1234594280]).

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Recent Corporate Updates

Acute Care Franchise

ZYNRELEF Now Available:
The ZYNRELEF (bupivacaine and meloxicam) extended-release solution New Drug Application (NDA) was approved by the U.S. Food and Drug Administration (FDA) in May 2021. ZYNRELEF became commercially available in the U.S. on July 1, 2021, and net product sales for the three and nine months ended September 30, 2021 were $2.1 million.
During the first quarter of commercial launch, 160 unique accounts purchased ZYNRELEF with 50% of those accounts reordering the product.
As of October 31, 2021, ZYNRELEF has received 126 formulary approvals, representing over a 91% hospital approval rate, and an additional 150 formulary review meetings are scheduled before the end of 2021.
Multiple commercial and Medicaid payers covering over 88 million lives have agreed to reimburse ZYNRELEF outside of the surgical bundle payment for surgeries performed in ambulatory surgical centers (ASC), with many of these covered lives also having their hospital outpatient procedures reimbursed outside the surgical bundle payment. Commercial and Medicaid payers represent >80% of our target patients in the outpatient setting. On November 2, 2021, we were issued a specific C-code (C9088) for separate reimbursement in the ASC setting of care effective January 1, 2022. Medicare is currently reimbursing ZYNRELEF in outpatient settings of care under a miscellaneous C-code at 95% of the Average Wholesale Price until December 31, 2021.
Heron announced on October 4, 2021, the filing of a supplemental NDA (sNDA) for expansion of the ZYNRELEF indication statement based on the outcome of a Type C meeting with the FDA. At this meeting, Heron aligned with the FDA on the content of this sNDA to support the proposed revised indication statement to include foot and ankle, small–to–medium open abdominal, and lower extremity total joint arthroplasty surgical procedures.
At the Type C meeting, Heron also aligned with the FDA on the data needed to support a future sNDA to further expand the ZYNRELEF indication statement to broadly include soft tissue and orthopedic surgical procedures with pharmacodynamic, pharmacokinetic and safety data from a limited number of additional procedures. The studies in these additional surgeries are already in progress with the plan to submit the next sNDA in the second half of 2022.
Heron recently received FDA approval in less than 4 months of a manufacturing supplement to the NDA for ZYNRELEF to add a large-scale secondary supplier of our proprietary polymer, which will allow for the manufacturing of millions of doses of ZYNRELEF annually at a significantly reduced cost of products sales.
NDA for HTX-019 for Prevention of PONV in Adults Planned in Late 2021: A 505(b)(2) NDA for HTX-019 is planned for the prevention of postoperative nausea and vomiting (PONV) in adults. A Pre-NDA meeting with the FDA was held in August 2021 and the NDA is on track for filing in the fourth quarter of this year.
Oncology Care Franchise

2021 Oncology Care Franchise Net Product Sales: For the three and nine months ended September 30, 2021, oncology care franchise net product sales were $21.1 million and $63.6 million, respectively, compared to $20.0 million and $68.0 million, respectively, for the same periods in 2020. During 2021, Heron’s oncology care franchise net product sales have stabilized. Key factors influencing our results are the lower rate of new cancer patient treatment starts due to the continued impact of the COVID-19 pandemic, resulting in fewer clinic anti-emetic administrations during the first nine months of 2021 compared to the prior year, and the impact of value-based payer reimbursement.
CINVANTI Net Product Sales: Net product sales of CINVANTI (aprepitant) injectable emulsion for the three and nine months ended September 30, 2021 were $18.0 million and $56.2 million, respectively, compared to $19.8 million and $67.6 million, respectively, for the same periods in 2020.
SUSTOL Net Product Sales: Net product sales of SUSTOL (granisetron) extended-release injection for the three and nine months ended September 30, 2021 were $3.1 million and $7.4 million, respectively, compared to $0.2 million and $0.4 million, respectively, for the same periods in 2020.
2021 Oncology Care Franchise Net Product Sales Guidance: Heron currently expects fourth quarter of 2021 net product sales for the oncology care franchise in the range of $20 million to $22 million.
"The approval and commercial launch of ZYNRELEF, the first and only FDA-approved extended-release dual-acting local anesthetic, is an important advancement in the field of pain management. Laying the groundwork for long-term growth, we have had excellent success with the pharmacy and therapeutics committee approvals in our first quarter of launch and the number of accounts already ordering the product. With agreement from the FDA, we have submitted the first of our planned sNDAs designed to expand the ZYNRELEF label, which we believe will substantially expand the commercial opportunity for ZYNRELEF," said Barry Quart, Pharm.D., Chairman and Chief Executive Officer of Heron. "For our oncology care franchise, we have stabilized sales in a market dominated by generics and expect to see moderate growth in net product sales in 2022. Overall, we have reduced the net cash used for operating activities by 15% compared to the prior quarter and we expect to continue to further reduce it by more than 10% next quarter."

Financial Results

Net product sales for the three and nine months ended September 30, 2021 were $23.2 million and $65.7 million, respectively, compared to $20.0 million and $68.0 million, respectively, for the same periods in 2020.

Heron’s net loss for the three and nine months ended September 30, 2021 was $52.4 million and $166.0 million, or $0.51 per share and $1.71 per share, respectively, compared to $58.2 million and $165.0 million, or $0.64 per share and $1.82 per share, respectively, for the same periods in 2020. Net loss for the three and nine months ended September 30, 2021 included non-cash, stock-based compensation expense of $11.2 million and $34.0 million, respectively, compared to $11.1 million and $34.2 million, respectively, for the same periods in 2020.

As of September 30, 2021, Heron had cash, cash equivalents and short-term investments of $202.8 million, compared to $208.5 million as of December 31, 2020. Net cash used for operating activities for the nine months ended September 30, 2021 was $158.1 million, compared to $132.3 million for the same period in 2020. The increase in our net cash used for operating activities was primarily due to changes in working capital related to the launch of ZYNRELEF in July 2021, including manufacturing of commercial inventory. We expect net cash used for operating activities of $45 million to $48 million in the fourth quarter of 2021, and we anticipate that our net cash usage will continue to moderate lower in 2022 as net product sales increase and we realize cost savings from anticipated larger-scale manufacturing.

Conference Call and Webcast

Heron will host a conference call and webcast on November 3, 2021 at 4:15 p.m. ET. The conference call can be accessed by dialing 877-311-5906 for domestic callers and 281-241-6150 for international callers. Please provide the operator with the passcode 7242566 to join the conference call. The conference call will also be available via webcast under the Investor Relations section of Heron’s website at www.herontx.com. An archive of the teleconference and webcast will also be made available on Heron’s website for 60 days following the call.

About ZYNRELEF for Postoperative Pain

ZYNRELEF is the first and only dual-acting local anesthetic that delivers a fixed-dose combination of the local anesthetic bupivacaine and a low dose of nonsteroidal anti-inflammatory drug meloxicam. ZYNRELEF is the first modified-release local anesthetic to be classified by the FDA as an "extended-release" product because ZYNRELEF is also the first and only extended-release local anesthetic to demonstrate in Phase 3 studies significantly reduced pain and significantly increased proportion of patients requiring no opioids through the first 72 hours following surgery compared to bupivacaine solution, the current standard-of-care local anesthetic for postoperative pain control. ZYNRELEF was approved by the FDA in May 2021 for use in adults for soft tissue or periarticular instillation to produce postsurgical analgesia for up to 72 hours after bunionectomy, open inguinal herniorrhaphy and total knee arthroplasty. Safety and efficacy have not been established in highly vascular surgeries, such as intrathoracic, large multilevel spinal, and head and neck procedures. At a Type C meeting with the FDA, following the ZYNRELEF NDA approval, we aligned with the FDA on the content of an sNDA for proposed expansion of the ZYNRELEF indication statement to include foot and ankle, small–to–medium open abdominal, and lower extremity total joint arthroplasty surgical procedures without the need for additional clinical studies. The sNDA is based on the consistent safety, efficacy and pharmacokinetic data from previously completed clinical trials and was submitted to the FDA in late September 2021. In September 2020, the European Commission granted a marketing authorization for ZYNRELEF for the treatment of somatic postoperative pain from small- to medium-sized surgical wounds in adults. As of January 1, 2021, ZYNRELEF is approved in 31 European countries including the countries of the European Union and European Economic Area and the United Kingdom.

Please see full prescribing information, including Boxed Warning, at www.ZYNRELEF.com.

About HTX-019 for PONV

HTX-019 is an IV injectable emulsion formulation designed to directly deliver aprepitant, the active ingredient in EMEND (aprepitant) capsules, which is the only substance P/neurokinin-1 (NK1) receptor antagonist (RA) to be approved in the U.S. for the prevention of PONV in adults. The FDA-approved dose of oral EMEND is 40 mg for PONV, which is given within 3 hours prior to induction of anesthesia for surgery. In a Phase 1 clinical trial, 32 mg of HTX-019 as a 30-second IV injection was demonstrated to be bioequivalent to oral aprepitant 40 mg. A successful Pre-NDA meeting with the FDA was held in August 2021 and the NDA is on track for filing in the fourth quarter of this year.

About CINVANTI for Chemotherapy Induced Nausea and Vomiting (CINV) Prevention

CINVANTI, in combination with other antiemetic agents, is indicated in adults for the prevention of acute and delayed nausea and vomiting associated with initial and repeat courses of highly emetogenic cancer chemotherapy (HEC) including high-dose cisplatin as a single-dose regimen, delayed nausea and vomiting associated with initial and repeat courses of moderately emetogenic cancer chemotherapy (MEC) as a single-dose regimen, and nausea and vomiting associated with initial and repeat courses of MEC as a 3-day regimen. CINVANTI is an IV formulation of aprepitant, an NK1 RA. CINVANTI is the first IV formulation to directly deliver aprepitant, the active ingredient in EMEND capsules. Aprepitant (including its prodrug, fosaprepitant) is the only single-agent NK1 RA to significantly reduce nausea and vomiting in both the acute phase (0–24 hours after chemotherapy) and the delayed phase (24–120 hours after chemotherapy). The FDA-approved dosing administration included in the U.S. prescribing information for CINVANTI is a 30-minute IV infusion or a 2-minute IV injection.

Please see full prescribing information at www.CINVANTI.com.

About SUSTOL for CINV Prevention

SUSTOL is indicated in combination with other antiemetics in adults for the prevention of acute and delayed nausea and vomiting associated with initial and repeat courses of moderately emetogenic chemotherapy (MEC) or anthracycline and cyclophosphamide (AC) combination chemotherapy regimens. SUSTOL is an extended-release, injectable 5-hydroxytryptamine type 3 RA that utilizes Heron’s Biochronomer drug delivery technology to maintain therapeutic levels of granisetron for ≥5 days. The SUSTOL global Phase 3 development program was comprised of two, large, guideline-based clinical studies that evaluated SUSTOL’s efficacy and safety in more than 2,000 patients with cancer. SUSTOL’s efficacy in preventing nausea and vomiting was evaluated in both the acute phase (0–24 hours after chemotherapy) and delayed phase (24–120 hours after chemotherapy).

Please see full prescribing information at www.SUSTOL.com.

On November 3, 2021 Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) reported financial and operational results for the third quarter ended September 30, 2021 (Press release, Vanda Pharmaceuticals, NOV 3, 2021, View Source [SID1234594279]).

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"Vanda is approaching another pivotal moment in its 18-year history with the upcoming completion of the Phase III study of tradipitant in gastroparesis and anticipated results by the end of this year. We believe that tradipitant has the potential to become the first new product in 40 years for the treatment of gastroparesis, a serious and debilitating disorder that severely impacts the lives of many people. Additionally, the launch of HETLIOZ and HETLIOZ LQ for nighttime sleep disturbances in people with Smith-Magenis Syndrome is progressing well as we are beginning our broad awareness campaign. Across all of our programs, patient access to innovative treatments remains a key goal of Vanda. We are currently working to address the escalating reimbursement delays for HETLIOZ, which are especially impacting Non-24 patients with light perception, and we are confident in our ability to drive positive outcomes for patients. I am proud of the Vanda team for continuing to advance Vanda’s innovation objectives and creating value for patients and all of the company’s stakeholders," said Mihael H. Polymeropoulos, M.D., Vanda’s President, CEO and Chairman of the Board.

Financial Highlights

Third Quarter of 2021

Total net product sales from HETLIOZ and Fanapt were $70.1 million in the third quarter of 2021, a 16% increase compared to $60.3 million in the third quarter of 2020.
HETLIOZ net product sales were $45.6 million in the third quarter of 2021, a 15% increase compared to $39.6 million in the third quarter of 2020.
Fanapt net product sales were $24.5 million in the third quarter of 2021, an 18% increase compared to $20.7 million in the third quarter of 2020.
Income before taxes was $10.7 million in the third quarter of 2021 compared to $8.4 million in the third quarter of 2020.
First Nine Months of 2021

Total net product sales from HETLIOZ and Fanapt were $200.7 million in the first nine months of 2021, an 11% increase compared to $180.5 million in the first nine months of 2020.
HETLIOZ net product sales were $129.5 million in the first nine months of 2021, an 11% increase compared to $116.5 million in the first nine months of 2020.
Fanapt net product sales were $71.2 million in the first nine months of 2021, an 11% increase compared to $64.0 million in the first nine months of 2020.
Income before taxes was $33.8 million in the first nine months of 2021 compared to $20.7 million in the first nine months of 2020.
Cash, cash equivalents and marketable securities (Cash) was $406.0 million as of September 30, 2021, representing an increase to Cash of $57.4 million compared to September 30, 2020.

Key Operational Highlights

Tradipitant

Enrollment of the randomized portion of the Phase III clinical study of tradipitant in gastroparesis is complete. The randomized portion of the study is a 12-week study of approximately 200 patients with idiopathic or diabetic gastroparesis. Results are expected by the end of 2021. Enrollment of the open label portion of the study is ongoing with more than 250 patients enrolled.
HETLIOZ (tasimelteon)

Reimbursement challenges with HETLIOZ prescriptions for patients with Non-24 have increased significantly. While Vanda is working to address these challenges and is confident in its ability to successfully navigate the current environment, Vanda is revising its HETLIOZ net product sales guidance to $170 to $190 million from the prior HETLIOZ net product sales guidance of $180 to $200 million.
In December 2020, the FDA approved HETLIOZ capsule and liquid formulations for the treatment of adults and children, respectively, with nighttime sleep disturbances in Smith-Magenis Syndrome (SMS).1 To date, more than 90 patients with SMS have been prescribed HETLIOZ or HETLIOZ LQ and a broad awareness campaign is expected to begin during the fourth quarter of 2021.
A Phase III clinical study of HETLIOZ in delayed sleep phase disorder (DSPD) is currently enrolling patients. The study has a 28-day randomized evaluation period and plans to enroll approximately 300 patients. DSPD is likely the most prevalent circadian-rhythm sleep disorder, affecting approximately 1% of the population, and there is no FDA approved treatment at this time.2
Fanapt (iloperidone)

A Phase III clinical study of Fanapt in acute bipolar mania disorder is currently enrolling. The study is a placebo controlled four-week evaluation of approximately 400 patients at sites in the U.S. and Europe.
A repeat-dose clinical pharmacology study of the long acting injectable (LAI) formulation of Fanapt is ongoing. A Phase III study of the LAI formulation for the treatment of acute schizophrenia is planned to follow the pharmacology study.
Evaluation of P88, the active metabolite of iloperidone, has been initiated. P88 has the potential to improve the clinical profile of Fanapt and create sustained, long-term value in the treatment of psychiatric disorders.
GAAP Financial Results

Income before taxes was $10.7 million in the third quarter of 2021 compared to $8.4 million in the third quarter of 2020. Net income was $7.8 million in the third quarter of 2021 compared to net income of $5.9 million in the third quarter of 2020. Diluted net income per share was $0.14 in the third quarter of 2021 compared to diluted net income per share of $0.11 in the third quarter of 2020.

Income before taxes was $33.8 million in the first nine months of 2021 compared to $20.7 million in the first nine months of 2020. Net income was $26.1 million in the first nine months of 2021 compared to net income of $15.1 million in the first nine months of 2020. Diluted net income per share was $0.46 in the first nine months of 2021 compared to diluted net income per share of $0.28 in the first nine months of 2020.

2021 Financial Guidance

Vanda is updating its 2021 financial guidance and expects to achieve the following financial objectives in 2021:

Conference Call

Vanda has scheduled a conference call for today, Wednesday, November 3, 2021, at 4:30 PM ET. During the call, Vanda’s management will discuss the third quarter 2021 financial results and other corporate activities. Investors can call 1-866-688-9426 (domestic) or 1-409-216-0816 (international) and use passcode 5961858. A replay of the call will be available on Wednesday, November 3, 2021, beginning at 7:30 PM ET and will be accessible until Wednesday, November 10, 2021 at 7:30 PM ET. The replay call-in number is 1-855-859-2056 for domestic callers and 1-404-537-3406 for international callers. The passcode number is 5961858.

The conference call will be broadcast simultaneously on Vanda’s website, www.vandapharma.com. Investors should click on the Investors tab and are advised to go to the website at least 15 minutes early to register, download, and install any necessary software or presentations. The call will also be archived on Vanda’s website for a period of 30 days.

References

Refer to Company press release titled "FDA Approves HETLIOZ (tasimelteon) for the Treatment of Nighttime Sleep Disturbances in Smith-Magenis Syndrome" issued on December 1, 2020. View Source
P.J. Murphy, Delayed Sleep-Phase Type, Encyclopedia of Sleep, Academic Press, 2013, Pages 22-25, ISBN 9780123786111, View Source

On November 3, 2021 Synthetic Biologics, Inc. (NYSE American: SYN), a diversified clinical-stage company leveraging the microbiome to develop therapeutics designed to prevent and treat gastrointestinal ("GI") diseases in areas of high unmet need, reported financial results for the third quarter ended September 30, 2021 (Press release, Synthetic Biologics, NOV 3, 2021, View Source [SID1234594278]).

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Recent Developments:

Initiated a Phase 1, placebo-controlled, multiple ascending dose clinical study of SYN-020 intestinal alkaline phosphatase ("IAP")
Continuing enrollment in the Phase 1b/2a clinical trial of SYN-004 (ribaxamase) in allogeneic hematopoietic cell transplant ("HCT") recipients
Current cash position of approximately $72.1 million
Current cash runway expected to provide funding to complete Phase 1b/2a clinical trial of SYN-004, clinical trials of SYN-020 through proof-of-concept, and other key milestones into 2023
Anticipated Milestones:

Topline data readout from the first antibiotic cohort of the SYN-004 Phase 1b/2a clinical trial is expected during Q1 2022
Expect to announce topline data readout from the Company’s Phase 1 MAD clinical trial of SYN-020 during Q2 2022
"During the third quarter, we significantly advanced our portfolio of GI and microbiome-focused clinical programs," said Steven A. Shallcross, Chief Executive Officer of Synthetic Biologics. "We were pleased to initiate a Phase 1 multiple ascending dose clinical trial of our SYN-020 IAP program during the third quarter of 2021. At this time, the first cohort of 8 study participants is nearing completion with dosing of the second cohort of 8 study participants expected to begin shortly thereafter, pending a safety review. We anticipate reporting topline data from this clinical trial during the second quarter of 2022. This study follows our recent Phase 1 single ascending dose ("SAD") clinical trial of SYN-020, in which SYN-020 was well tolerated and demonstrated a favorable safety profile at all doses. Both clinical trials are designed to support the advancement of SYN-020 in multiple potential therapeutic indications, including radiation enteropathy, celiac disease, non-alcoholic fatty liver disease (NAFLD), and age-related metabolic and inflammatory diseases. We continue to view SYN-020 as a multi-indication platform program, which has the potential to help address a considerable need for innovative new therapies targeting disorders that stem from immune and inflammatory damage to the intestine."

Mr. Shallcross continued, "We also continue to progress the SYN-004 Phase 1b/2a clinical trial in allogeneic hematopoietic cell transplant ("HCT") recipients. Patient screening and enrollment is ongoing at the Washington University School of Medicine in St. Louis ("Washington University"). As a result, we currently anticipate announcing topline results from the first of three antibiotic cohorts during the first quarter of 2022. We believe SYN-004 has the potential to address a significant unmet medical need by improving outcomes for allogeneic HCT recipients. Overall, we remain very excited about the potential for each of our clinical programs and near-term clinical milestones that we believe could drive significant value for shareholders."

Clinical Development and Operational Update

Announced commencement of the Company’s Phase 1, placebo-controlled, multiple ascending dose clinical trial of SYN-020
The ongoing Phase 1 MAD clinical trial is intended to evaluate the safety, tolerability and biodistribution of SYN-020 upon repeated dosing in up to 32 healthy adult volunteers.
The study is divided into 4 sequential cohorts of 8 participants, with SYN-020 (5 mg, 15, mg, 45 mg or 75 mg) given orally twice daily for 14 days.
At this time, the first cohort of 8 study participants is nearing completion and dosing of the second cohort of 8 study participants is expected to begin shortly thereafter, pending a safety review.
A safety review will be conducted at the end of each cohort to determine whether progression to the next higher dose cohort is permissible.
A topline data readout from this clinical trial is anticipated during the second quarter of 2022, pandemic conditions permitting.
A previously completed Phase 1 single ascending dose clinical study of SYN-020 enrolled 24 healthy adult volunteers into four cohorts with SYN-020 given orally as a single dose ranging from 5 mg to 150 mg. Analyses of preliminary data demonstrated that SYN-020 maintained a favorable safety profile, was well tolerated at all dose levels, and no adverse events were attributed to study drug. No serious adverse events were reported.
Both studies are intended to support the development of SYN-020 in multiple potential clinical indications including radiation enteropathy, celiac disease, NAFLD, as well as indications supported by the Company’s collaboration with Massachusetts General Hospital.
Enrollment in the Company’s Phase 1b/2a clinical trial of SYN-004 in allogeneic HCT recipients for the prevention of acute graft-versus-host-disease ("aGVHD") remains ongoing
The Phase 1b/2a clinical trial comprises a single center, randomized, double-blind, placebo-controlled clinical trial of oral SYN-004 in up to 36 evaluable adult allogeneic HCT recipients.
The goal of this clinical trial is to evaluate the safety, tolerability, and potential absorption into the systemic circulation (if any) of oral SYN-004 administered to allogeneic HCT recipients who receive an IV beta-lactam antibiotic to treat fever.
Study participants will be enrolled into three sequential cohorts and administered a different study-assigned IV beta-lactam antibiotic. Eight participants in each cohort will receive SYN-004 and 4 will receive placebo.
Safety and pharmacokinetic data for each cohort will be reviewed by an independent Data and Safety Monitoring Committee ("DSMC"), which will make a recommendation on whether to proceed to the next IV beta-lactam antibiotic.
A topline data readout for the first of three antibiotic cohorts is anticipated during the first quarter of 2022, pandemic conditions permitting.
Quarter Ended September 30, 2021 Financial Results

General and administrative expenses increased by 9% to approximately $1.3 million for the three months ended September 30, 2021, from approximately $1.2 million for the three months ended September 30, 2020. This increase is primarily due to higher insurance costs, audit fees and registration fees offset by lower legal costs and vacation expense. The charge related to stock-based compensation expense was $83,000 for the three months ended September 30, 2021, compared to $67,000 for the three months ended September 30, 2020.

Research and development expenses increased by 116% to approximately $2.0 million for the three months ended September 30, 2021, from approximately $900,000 for the three months ended September 30, 2020. This increase is primarily the result of increased clinical trial expenses as we continued dosing patients in the Phase 1b/2a clinical trial of SYN-004 and by higher indirect program costs for the three months ended September 30, 2021, including an increase in manufacturing costs for SYN-020. We anticipate research and development expense to increase as our ongoing clinical trials continue to enroll patients. The charge related to stock-based compensation expense was $19,000 for the three months ended September 30, 2021, compared to $15,000 related to stock-based compensation expense for the three months ended September 30, 2020.

Other income was $2,000 for the three months ended September 30, 2021, compared to other income of $134 for the three months ended September 30, 2020. Other income for the three months ended September 30, 2021 and 2020 is primarily comprised of interest income.

Cash and cash equivalents as of September 30, 2021 totaled $72.1 million, an increase of $65.9 million from December 31, 2020.

Conference Call

Synthetic Biologics will hold a conference call today, Wednesday, November 3, 2021, at 4:30 p.m. (EST). The dial-in information for the call is as follows, U.S. toll free: 1-888-347-5280 or International: +1 412-902-4280. Participants are asked to dial in 15 minutes before the start of the call to register. The call will also be webcast over the Internet at View Source." target="_blank" title="View Source." rel="nofollow">View Source An archive of the call will be available for replay at the same URL, View Source, for 90 days after the call.