On May 13, 2024 Verrica Pharmaceuticals Inc. ("Verrica") (Nasdaq: VRCA), a dermatology therapeutics company developing medications for skin diseases requiring medical interventions, reported financial results for the first quarter ended March 31, 2024 (Press release, Verrica Pharmaceuticals, MAY 13, 2024, View Source [SID1234643163]).

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"The first quarter of 2024 marked a period of significant accomplishments across our business, as we continued to expand utilization of YCANTH, received a permanent J-Code from CMS, and secured new chemical entity status from the FDA for YCANTH," said Ted White, Verrica’s President and Chief Executive Officer. "I am also pleased to report that we have seen a meaningful uptick in prescription growth and onboarding of buy and bill accounts following the listing of the permanent J-Code for YCANTH, which went into effect on April 1.

"Looking ahead, this quarter we expect to announce Phase 2 results from our lead pipeline candidate, VP-315, which is being evaluated for the treatment of basal cell carcinoma. As a potential first-in-class oncolytic peptide, VP-315 is designed to have a direct killing activity of the cancer cells, and also to stimulate the immune system to recognize, infiltrate, and attack the cancer. We expect to share data from the Phase 2 study later this quarter, and we are excited about VP-315’s potential to provide an important treatment alternative for the thousands of patients who are diagnosed each year with BCC."

Conference Call and Webcast Information

The Company will host a conference call today, Monday, May 13, 2024, at 8:30 AM, Eastern Time, to discuss its first quarter 2024 financial results and provide a business update. To participate in the conference call, please utilize the following information:

Domestic Dial-In Number: Toll-Free: 1-877-407-4018

International Dial-In Number: 1-201-689-8471

Conference ID: 13746100

Call me:

•
View Source;passcode=13741589&h=true&info=company-email&r=true&B=6

•
Participants can use Guest dial-in #s above and be answered by an operator OR click the Call me link for instant telephone access to the event.

•
Call me link will be made active 15 minutes prior to scheduled start time.

The call will also be broadcast live over the Web and can be accessed on Verrica Pharmaceuticals’ website: www.verrica.com or directly at View Source;tp_key=caf7d1fe6b

The conference call will also be available for replay for one month on the Company’s website in the Events Calendar of the Investors section.

Business Highlights and Recent Developments

YCANTH (VP-102)

•
On March 26, 2024, the Company announced that YCANTH received New Chemical Entity ("NCE") Status and a listing in the Orange Book from the U.S. Food and Drug Administration ("FDA"), providing a minimum five years of regulatory exclusivity. The Company’s U.S. patents and pending patent applications related to YCANTH are projected to expire between 2034 and 2041, excluding any patent term adjustment or patent term extension.

•
On January 29, 2024, the Company announced that the Centers for Medicare & Medicaid Services (CMS) issued a permanent J-Code (J7354) for YCANTH. Under the Healthcare Common Procedure Coding System (HCPCS) process, the J-Code for YCANTH will become fully published April 1, 2024. The Company believes that securing a permanent J-Code will accelerate utilization of YCANTH among the U.S. Medicaid and Medicare patient populations and will streamline billing and the reimbursement process.

•
On January 4, 2024, the Company announced that it received the minutes from the Company’s recent Type C meeting with the FDA, which was held on November 6, 2023, to discuss the Phase 3 clinical development plan for YCANTH for the treatment of common warts. Verrica believes that the Type C meeting satisfied its objective of gaining the FDA’s advice and agreement on the overall design of a pivotal Phase 3 study of YCANTH that would support an efficacy supplement for the proposed indication of common warts.

•
On January 3, 2024, the Company announced that it expanded its distribution network by entering into an agreement with Walgreen Co. to distribute YCANTH through its specialty pharmacy.

VP-315

•
On January 5, 2024, the Company announced that the last patient had been dosed in Part 2 of its ongoing Phase 2 trial of VP-315, a potential first-in-class oncolytic peptide, for the treatment of basal cell carcinoma. The Phase 2 trial is a two-part, open-label, multicenter, dose-escalation, proof-of-concept study with a safety run-in designed to assess the safety, pharmacokinetics, and efficacy of VP-315 when administered intratumorally to adults with biopsy-proven basal cell carcinoma. The study is expected to enroll approximately 80 adult subjects with a histological diagnosis of basal cell carcinoma in at least one eligible target lesion. For additional information about this clinical trial, please visit clinicaltrials.gov, identifier NCT05188729.

First Quarter 2024 Financial Results

•
Verrica recognized product revenue of $3.2 million in the first quarter of 2024. As commercial sales of YCANTH began in the third quarter of 2023, Verrica did not recognize any product revenue prior to that point.

•
Verrica recognized collaboration revenues of $0.6 million for the three months ended March 31, 2024 related to the Collaboration and License Agreement with Torii Pharmaceutical Co., Ltd ("Torii") for supplies and development activity with Torii.

•
Selling, general and administrative expenses were $16.3 million in the first quarter of 2024, compared to $4.3 million for the same period in 2023. The increase of $12.0 million was primarily due to higher expenses related to commercial activities for YCANTH, including increased compensation, recruiting fees, benefits and travel due to ramp-up of sales force of $6.2 million, increased marketing and sponsorship costs of $2.3 million, other commercial activity of $1.9 million, and increased legal costs of $0.6 million.

•
Research and development expenses were $4.9 million in the first quarter of 2024, compared to $2.7 million for the same period in 2023. The increase of $2.2 million was primarily related to increased clinical costs for VP-315 of $1.5 million and increased headcount related costs of $0.6 million.

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Costs of product revenue were $0.5 million for the quarter ended March 31, 2024 including product costs of $0.2 million and obsolete inventory write-off of $0.3 million. Product costs were $0.1 million lower as some materials were expensed as research and development costs prior to FDA approval.

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Costs of collaboration revenue were $0.6 million for the quarter ended March 31, 2024, compared to $0.1 million for the quarter ended March 31, 2023. These costs of collaboration revenue consisted of payments for manufacturing supply to support development and testing services pursuant to the Torii Clinical Supply Agreement.

•
Interest income was $0.6 million for the three months ended March 31, 2024, compared to $0.5 million for the same period in 2023. The increase of $0.1 million was primarily due to higher interest rates.

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Interest expense of $2.3 million for the three months ended March 31, 2024 consisted of interest expense related to the OrbiMed Credit Agreement that commenced in July 2023.

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For the quarter ended March 31, 2024, net loss was $20.3 million, or $0.44 per share, compared to a net loss of $6.6 million, or $0.15 per share, for the same period in 2023.

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For the quarter ended March 31, 2024, non-GAAP net loss was $17.8 million, or $0.38 per share, compared to a non-GAAP net loss of $5.5 million, or $0.13 per share, for the same period in 2023.

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As of March 31, 2024, Verrica had cash and cash equivalents of $48.9 million. Verrica believes that its existing cash and cash equivalents as of March 31, 2024 will be sufficient to support planned operations into the first quarter of 2025.

On May 13, 2024 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported financial results for the first quarter ended March 31, 2024, and provided an overview of recent developments (Press release, UroGen Pharma, MAY 13, 2024, View Source [SID1234643162]).

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"In 2024 to date, UroGen has made excellent progress in both our commercial business and pipeline of innovative products designed to treat urothelial and specialty cancers," said Liz Barrett, President, and Chief Executive Officer of UroGen. "The upcoming announcement of 12-month duration of response results from ENVISION will be a key clinical milestone and we expect the data to support completion of our NDA for UGN-102. Pre-launch activities are underway and we estimate that UGN-102 will address a more than $3 billion market opportunity. If approved, we believe this product will become the major growth driver for UroGen and could establish a new standard of care in LG-IR-NMIBC."

Q1 2024 and Recent Business Highlights:

UGN-102 (mitomycin) for intravesical solution:

In January 2024, UroGen initiated submission of a rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for UGN-102 as a treatment of low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). The company plans to complete the submission in Q3 2024 with a potential FDA decision as early as the first quarter of 2025.
In May 2024, a subgroup analysis from the UGN-102 ATLAS trial was featured in a podium presentation at the American Urological Association (AUA) 2024 Annual Meeting in San Antonio, Texas. The analysis showed that patients with new and recurrent LG-IR-NMIBC treated with UGN-102 ± TURBT (trans urethral resection of bladder tumor) had high probabilities of remaining event-free for disease free survival (DFS) and high probabilities of remaining in complete response.
JELMYTO (mitomycin) for pyelocalyceal solution in low-grade upper tract urothelial cancer (LG-UTUC):

A soon-to-be-published post-hoc analysis of the OLYMPUS trial assessed the long-term effects in treating LG-UTUC with JELMYTO. Of the 71 patients who enrolled in OLYMPUS, 41 achieved a complete-response and their health outcomes were tracked for up to 12 months. 20 of these patients remaining in CR enrolled in a 5-year rollover study. All 41 patients with an initial CR indicated a promising median duration of response of 47.8 months, based on a median follow-up of 28.1 months. In the 5-year rollover trial, 75% (N=15) showed no disease recurrence, indicating potential for extended disease-free periods.
JELMYTO was featured in three presentations at the AUA 2024 Annual Meeting. Independent long-term real-world analyses explored use of the product in broad patient types and with different methods of administration. The results show that JELMYTO treatment demonstrated favorable recurrence free survival rates for patients with LG-UTUC who respond to initial induction. The results were consistent regardless of JELMYTO administration, original tumor size, multifocality or tumor location.
Continued strong demand for Jelmyto with record patient enrollments forms. Generated net product revenue of $18.8 million in the first quarter of 2024, compared with $17.2 million in the first quarter of 2023, representing ~10% annual growth.
Next-generation novel mitomycin-based formulation for urothelial cancers

In January 2024, UroGen announced a license and supply agreement with medac GmbH to develop a next-generation novel mitomycin-based formulation for urothelial cancers. UGN-103 and UGN-104 combine UroGen’s RTGel technology with medac’s licensed mitomycin formulation. The agreement and development program potentially offer both manufacturing efficiencies and IP protection for the Company’s next-generation urothelial cancer franchise.
In April 2024, the FDA accepted the Company’s Investigational New Drug (IND) application for UGN-103. If approved, UGN-103 is expected to provide several advantages related to production, cost, supply, and product convenience.
UroGen plans to initiate Phase 3 studies to explore the safety and efficacy of UGN-103 in LG-IR-NMIBC in 2024 and UGN-104 in LG-UTUC shortly thereafter.
First Quarter 2024 Financial Results

JELMYTO Revenue: JELMYTO net product revenues were $18.8 million and $17.2 million for the three months ended March 31, 2024 and 2023, respectively.

R&D Expense: Research and development expenses for the first quarter of 2024 were $15.5 million, including non-cash share-based compensation expense of $0.5 million as compared to $12.5 million, including non-cash share-based compensation expense of $0.5 million, for the same period in 2023.

SG&A Expense: Selling, general and administrative expenses for the first quarter of 2024 were $27.3 million, including non-cash share-based compensation expense of $2.2 million. This compares to $24.5 million, including non-cash share-based compensation expense of $1.8 million, for the same period in 2023.

Financing on Prepaid Forward Obligation: UroGen reported non-cash financing expense related to the prepaid forward obligation to RTW Investments of $5.7 million in the first quarter of 2024, compared to $5.2 million in the same period in 2023.

Interest Expense on Long-Term Debt: Interest expense related to the $100 million term loan facility with funds managed by Pharmakon Advisors was $2.4 million in the first quarter of 2024, compared to $3.6 million in the same period in 2023.

Net Loss: UroGen reported a net loss of $32.3 million or ($0.97) per basic and diluted share in the first quarter of 2024 compared with a net loss of $30.2 million or ($1.30) per basic and diluted share in the same period in 2023.

Cash & Cash Equivalents: As of March 31, 2024, cash, cash equivalents and marketable securities totaled $164.5 million.

2024 Revenue, Operating Expense and RTW Expense Guidance: The Company is reiterating full year 2024 net product revenues guidance from JELMYTO in the range of $95 to $102 million. Increased discounts related to Medicare refunds for discarded drugs and 340B purchases will further impact net revenues in 2024. The Company also expects full year 2024 operating expenses in the range of $175 to $185 million, including non-cash share-based compensation expense of $6 to $11 million, subject to market conditions. The Company also reiterates the anticipated full year 2024 non-cash financing expense related to the prepaid obligation to RTW Investments in the range of $21 to $26 million. Of this amount approximately $12.4 to $13.3 million is expected to be in cash.

Conference Call & Webcast Information: Members of UroGen’s management team will host a live conference call and webcast today at 10:00 AM Eastern Time to review UroGen’s financial results and provide a general business update.

The live webcast can be accessed by visiting the Investors section of the Company’s website at View Source Please connect at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast.

UROGEN PHARMA LTD.

SELECTED CONSOLIDATED BALANCE SHEETS

(U.S. dollars in thousands)

(Unaudited)

March 31, 2024

December 31, 2023

Cash and cash equivalents and marketable securities

$

164,525

$

141,470

Total assets

$

200,574

$

178,311

Total liabilities

$

240,708

$

243,523

Total shareholders’ deficit

$

(40,134

)

$

(65,212

)

UROGEN PHARMA LTD.

CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS

(U.S. dollars in thousands, except share and per share data)

(Unaudited)

Three months ended March 31,

2024

2023

Revenue

$

18,781

$

17,192

Cost of revenue

1,728

2,265

Gross profit

17,053

14,927

Operating expenses:

Research and development expenses

15,494

12,498

Selling, general and administrative expenses

27,299

24,474

Total operating expenses

42,793

36,972

Operating loss

(25,740

)

(22,045

)

Financing on prepaid forward obligation

(5,660

)

(5,224

)

Interest expense on long-term debt

(2,447

)

(3,553

)

Interest and other income, net

1,615

630

Loss before income taxes

$

(32,232

)

$

(30,192

)

Income tax expense

(54

)

(21

)

Net loss

$

(32,286

)

$

(30,213

)

Net loss per ordinary share, basic and diluted

$

(0.97

)

$

(1.30

)

Weighted average shares outstanding, basic and diluted

33,379,786

23,279,951

About JELMYTO

JELMYTO (mitomycin) for pyelocalyceal solution is a mitomycin-containing reverse thermal gel containing 4 mg mitomycin per mL gel indicated for the treatment of adult patients with LG-UTUC. JELMYTO is a viscous liquid when cooled and becomes a semi-solid gel at body temperature. The drug slowly dissolves over four to six hours after instillation and is removed from the urinary tract by normal urine flow and voiding. It is approved for administration in a retrograde manner via ureteral catheter or antegrade through nephrostomy tube. The delivery system allows the initial liquid to coat and conform to the upper urinary tract anatomy. The eventual semisolid gel allows for chemoablative therapy to remain in the collecting system for four to six hours without immediately being diluted or washed away by urine flow.

APPROVED USE FOR JELMYTO

JELMYTO is a prescription medicine used to treat adults with a type of cancer of the lining of the upper urinary tract including the kidney called low-grade Upper Tract Urothelial Cancer (LG-UTUC).

IMPORTANT SAFETY INFORMATION

You should not receive JELMYTO if you have a hole or tear (perforation) of your bladder or upper urinary tract.

Before receiving JELMYTO, tell your healthcare provider about all your medical conditions, including if you:

are pregnant or plan to become pregnant. JELMYTO can harm your unborn baby. You should not become pregnant during treatment with JELMYTO. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with JELMYTO. Females who are able to become pregnant: You should use effective birth control (contraception) during treatment with JELMYTO and for 6 months after the last dose. Males being treated with JELMYTO: If you have a female partner who is able to become pregnant, you should use effective birth control (contraception) during treatment with JELMYTO and for 3 months after the last dose.
are breastfeeding or plan to breastfeed. It is not known if JELMYTO passes into your breast milk. Do not breastfeed during treatment with JELMYTO and for 1 week after the last dose.
Tell your healthcare provider if you take water pills (diuretic).
How will I receive JELMYTO?

Your healthcare provider will tell you to take a medicine called sodium bicarbonate before each JELMYTO treatment.
You will receive your JELMYTO dose from your healthcare provider 1 time a week for 6 weeks. It is important that you receive all 6 doses of JELMYTO according to your healthcare provider’s instructions. If you miss any appointments, call your healthcare provider as soon as possible to reschedule your appointment. Your healthcare provider may recommend up to an additional 11 monthly doses.
JELMYTO is given to your kidney through a tube called a catheter.
During treatment with JELMYTO, your healthcare provider may tell you to take additional medicines or change how you take your current medicines.
After receiving JELMYTO:

JELMYTO may cause your urine color to change to a violet to blue color. Avoid contact between your skin and urine for at least 6 hours.
To urinate, males and females should sit on a toilet and flush the toilet several times after you use it. After going to the bathroom, wash your hands, your inner thighs, and genital area well with soap and water.
Clothing that comes in contact with urine should be washed right away and washed separately from other clothing.
JELMYTO may cause serious side effects, including:

Swelling and narrowing of the tube that carries urine from the kidney to the bladder (ureteric obstruction). If you develop swelling and narrowing, and to protect your kidney from damage, your healthcare provider may recommend the placement of a small plastic tube (stent) in the ureter to help the kidney drain. Tell your healthcare provider right away if you develop side pain or fever during treatment with JELMYTO.
Bone marrow problems. JELMYTO can affect your bone marrow and can cause a decrease in your white blood cell, red blood cell, and platelet counts. Your healthcare provider will do blood tests prior to each treatment to check your blood cell counts during treatment with JELMYTO. Your healthcare provider may need to temporarily or permanently stop JELMYTO if you develop bone marrow problems during treatment with JELMYTO.
The most common side effects of JELMYTO include: urinary tract infection, blood in your urine, side pain, nausea, trouble with urination, kidney problems, vomiting, tiredness, stomach (abdomen) pain.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1‑800‑FDA‑1088. You may also report side effects to UroGen Pharma at 1-855-987-6436.

Please see JELMYTO Full Prescribing Information, including the Patient Information, for additional information.

About Upper Tract Urothelial Cancer (UTUC)

Urothelial cancer is the ninth most common cancer globally and the eighth most lethal neoplasm in men in the U.S. Between five percent and ten percent of primary urothelial cancers originate in the ureter or renal pelvis and are collectively referred to as upper tract urothelial cancers (UTUC). In the U.S., there are approximately 6,000 – 7,000 new or recurrent low-grade UTUC patients annually. Most cases are diagnosed in patients over 70 years old, and these older patients often face comorbidities. There are limited treatment options for UTUC, with the most common being endoscopic surgery or nephroureterectomy (removal of the entire kidney and ureter). These treatments can lead to a high rate of recurrence and relapse.

About UGN-102

UGN-102 (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin, currently in Phase 3 development for the treatment of low-grade, intermediate-risk, non-muscle invasive bladder cancer (LG-IR-NMIBC). Utilizing UroGen’s proprietary RTGel technology, a sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using a standard urinary catheter in an outpatient setting. Assuming positive findings from the durability of response endpoint from the ENVISION Phase 3 study, UroGen anticipates completing its NDA submission for UGN-102 in September with a potential FDA decision as early as the first quarter of 2025.

On May 13, 2024 Turnstone Biologics Corp. ("Turnstone" or the "Company") (Nasdaq: TSBX), a clinical-stage biotechnology company developing a differentiated approach to treat and cure patients with solid tumors by pioneering selected tumor-infiltrating lymphocyte (Selected TIL) therapy, reported financial results for the first quarter ended March 31, 2024, and provided recent business highlights (Press release, Turnstone Biologics, MAY 13, 2024, View Source [SID1234643161]).

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"We are excited about the therapeutic potential of our pipeline of next-generation Selected TIL therapies," said Sammy Farah, M.B.A., Ph.D., Turnstone’s President and Chief Executive Officer. "The next frontier for TIL therapy is to extend its therapeutic activity in additional solid tumor indications beyond melanoma. Our differentiated approach includes a proprietary ‘cell selection’ step which is designed to generate a TIL product that is dominated by tumor-reactive T cells. We believe this process is crucial to develop potent TIL-based therapies such as Turnstone’s Selected TILs to address harder-to-treat, lower mutational burden cancers, such as colorectal cancer and other immunologically cold tumors. This year, we look forward to generating clinical data to highlight the differentiation of our platform and support further advancement of our lead asset, TIDAL-01, which is currently being evaluated in several Phase 1 studies in multiple solid tumor indications. We remain on track and plan to provide a TIDAL-01 clinical update mid-year in connection with our next quarterly financial results"

First Quarter 2024 and Recent Business Highlights

Continued TIDAL-01 Development in Multiple Phase 1 Clinical Trials. The Company is continuing the development of TIDAL-01, Turnstone’s lead Selected TIL therapy. TIDAL-01 is designed to potentially expand the applicability of TIL therapy into solid tumor types where first-generation TILs have not been effective by employing an unbiased identification and functional screening process to isolate and selectively expand the greatest breadth of tumor-reactive TILs from the patient’s tumor. TIDAL-01 is currently being evaluated in colorectal cancer, head and neck squamous cell carcinoma, uveal melanoma, breast cancer, and cutaneous melanoma across Turnstone’s multi-site STARLING trial and investigator-sponsored trials in collaboration with H. Lee Moffitt Cancer Center. Furthermore, Turnstone recently secured additional dedicated cleanroom capacity at Moffit’s on-site cGMP facility for manufacturing of TIDAL-01 for the STARLING trial with IND clearance from the FDA. Turnstone expects to provide an initial/preliminary clinical update from the Phase 1 studies around mid-year.

Executed a $20M Non-Dilutive Revolving Credit Facility. In April, Turnstone secured a revolving credit facility from Banc of California that allows Turnstone to draw on an aggregate amount up to $20 million. The proceeds from the facility, if drawn, will be utilized to support ongoing development of the Company’s pipeline and clinical trials.

Strengthened Company’s Board of Directors. In April, Turnstone announced the appointment of William Waddill to its Board of Directors. Mr. Waddill brings more than three decades of financial and operational expertise in the biotechnology space, and proven leadership in industry organizations. The Company also announced that Patrick Machado has stepped down as a member of its Board of Directors.

First Quarter 2024 Financial Results

Cash, Cash Equivalents and Short-Term Investments: As of March 31, 2024, cash, cash equivalents and short-term investments were $77.8 million. The Company expects that the combined cash, cash equivalents and short-term investments will be sufficient to fund its operations into the third quarter of 2025.

Research and Development (R&D) Expenses: R&D expenses for the three months ended March 31, 2024, were $15.8 million, compared to $15.7 million for the same period in 2023. The increase was due primarily to an increase of $2.4 million in manufacturing expenses and $0.2 million in personnel related costs as we ramp up TIDAL-01 clinical trials offset by a decrease of $0.9 million in clinical and regulatory costs and $1.6 million in pre-clinical research and development costs due to the termination of the Takeda Agreement and winding down activities related to the RIVAL-01 platform during the three months ended March 31, 2023.

General and Administrative (G&A) Expenses: G&A expenses for the three months ended March 31, 2024, were $4.9 million, compared to $4.0 million for the same period in 2023. The increase was due primarily to an increase in professional service costs of $1.1 million related to the increased costs of operating as a public company offset by a decrease in personnel costs of $0.2 million.

Net Loss: Net loss for the three months ended March 31, 2024, was $19.6 million, compared to net income of $0.1 million for the same period in 2023. The decrease was primarily due to the recognition of deferred revenue from the termination of the Takeda Agreement recorded in Collaboration Revenue for the three months ended 2023 compared to no Collaboration Revenue recognized for the same period in 2024.

On May 13, 2024 TScan Therapeutics, Inc. (Nasdaq: TCRX), a clinical-stage biopharmaceutical company focused on the development of T cell receptor (TCR)-engineered T cell (TCR-T) therapies for the treatment of patients with cancer, reported financial results for the first quarter ended March 31, 2024, and provided a corporate update (Press release, TScan Therapeutics, MAY 13, 2024, View Source [SID1234643160]).

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"During the first quarter we advanced our clinical pipeline, most recently marked by the dosing of the first patient in our Phase 1 solid tumor program. This is a significant milestone on the way to developing enhanced, customized, multiplex TCR-T therapy. We continue to prioritize screening patients for this study to enable rapid enrollment, and I am pleased to announce that we are on track to sharing initial data later this year," said Gavin MacBeath, Ph.D., Chief Executive Officer. "At the same time, we remain focused on enrolling and following patients in our heme malignancies study. We plan to complete Phase 1 enrollment and open expansion cohorts at the proposed recommended Phase 2 dose in the third quarter of 2024 and provide a data update near the end of 2024. With the recent closing of our public offering, TScan is well-funded to execute on anticipated milestones into the fourth quarter of 2026."

Recent Corporate Highlights

•
The Company recently announced that the first patient has been dosed in its Phase 1 clinical trial evaluating TCR-T therapy for the treatment of various solid tumors. The Company is initially dosing patients with singleplex therapy to establish safety prior to initiating dosing with multiplex TCR-T (T-Plex). Patients are prospectively assigned to a treatment cohort based on expression of cancer-associated antigens and human leukocyte antigens (HLAs) in their tumor samples. The first patient, who has metastatic melanoma, was dosed with TSC-203-A0201, a TCR-T targeting PReferentially expressed Antigen in MElanoma (PRAME) on HLA-A*02:01. The Company expects robust patient enrollment as over 60 patients have completed all biomarker testing in the screening protocol. Of these patients, approximately 55% qualify for at least one TCR-T in the ImmunoBank and approximately 30% could qualify for multiplex therapy.

•
The Company recently provided an update on its Phase 1 heme malignancies program designed to treat residual disease and prevent relapse in patients with acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), or myelodysplastic syndromes (MDS). The update included additional follow-up on all eight treatment-arm patients as well as data on two additional control-arm patients. With a median follow-up of >10 months, all eight patients treated with TSC-100 or TSC-101 remain relapse-free with no detectable disease. No dose-limiting toxicities were observed. In contrast, two control-arm patients relapsed approximately six months post-transplant and one of these patients died approximately three months later. A third control-arm patient required clinical intervention because of concerns of impending relapse, and a fourth control-arm patient died post-transplant.

•
In April 2024, the Company announced the closing of an upsized $167.8 million underwritten public offering with participation from new and existing high-quality healthcare investors. The financing extends the Company’s cash runaway into the fourth quarter of 2026. The Company intends to use the net proceeds to

advance its heme and solid tumor programs and expand and optimize its manufacturing capabilities, as well as for general corporate purposes.

•
The Company announced two upcoming poster presentations at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting being held May 31–June 4 both in Chicago and virtually. Details on the respective presentations can be found here.

•
The Company recently presented at two major medical meetings:

o
American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 27th Annual Meeting
o
American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2024

Presentation materials from the meetings can be found on the "Publications" tab of TScan’s website at tscan.com.

Upcoming Anticipated Milestones

Heme Malignancies Program: TScan’s two lead TCR-T therapy candidates, TSC-100 and TSC-101, are designed to treat residual disease and prevent relapse in patients with AML, ALL, or MDS undergoing allogeneic hematopoietic cell transplantation (HCT) (NCT05473910).

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Opening of expansion cohorts at the proposed recommended Phase 2 dose level to further characterize safety and evaluate translational and efficacy endpoints is planned for the third quarter of 2024.
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Completion of Phase 1 enrollment and reporting of one-year clinical and translational data on initial patients is anticipated in the second half of 2024.
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Expects to initiate registration trial pending feedback from regulatory authorities and report two-year clinical and translational data in 2025.

Solid Tumor Program: TScan continues to expand the ImmunoBank, a collection of therapeutic TCR-Ts that target different cancer-associated antigens presented on diverse HLA types. TScan’s strategy is to treat patients with multiple TCR-Ts to overcome tumor heterogeneity and prevent resistance that may arise from either target or HLA loss (screening protocol: NCT05812027; treatment protocol: NCT05973487).

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Phase 1 solid tumor clinical study has been initiated; first patient dosed in early May, with three additional patients enrolled and manufacturing underway.
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Initial data expected in the second half of 2024.
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Additional investigational new drug (IND) filings planned to continue to expand the ImmunoBank.
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Long-term duration of response data for multiplex therapy anticipated in 2025.

First Quarter 2024 Financial Results

Revenue: Revenue for the first quarter of 2024 was $0.6 million, compared to $6.8 million for the first quarter of 2023. The decrease was primarily due to the timing of research activities pursuant to the Company’s collaboration agreements. Revenue for the first quarter of 2024 was related solely to the collaboration agreement with Amgen which commenced in May 2023. Revenue for the first quarter of 2023 was related solely to the collaboration agreement with Novartis, which concluded in March 2023.

R&D Expenses: Research and development expenses for the first quarter of 2024 were $24.9 million, compared to $21.8 million for the first quarter of 2023. The increase of $3.1 million was primarily driven by an increase in personnel expenses due to additional headcount in support of expanded research and development activities, as well as an increase in clinical studies expense associated with the ongoing enrollment of our Phase 1 heme study and start-up activities for the Phase 1 solid tumor clinical trial. Research and development expenses included non-cash stock compensation expense of $1.1 million and $0.4 million for the first quarter of 2024 and 2023, respectively.

G&A Expenses: General and administrative expenses for the first quarter of 2024 were $7.1 million, compared to $7.8 million for the first quarter of 2023. The decrease of $0.7 million was primarily driven by lower facility-related and personnel costs. General and administrative expenses included non-cash stock compensation expense of $0.9 million and $0.7 million for the first quarter of 2024 and 2023, respectively.

Net Loss: Net loss was $30.1 million for the first quarter of 2024, compared to $22.6 million for the first quarter of 2023, and included net interest income of $1.2 million and $0.2 million, respectively.

Cash Position: Cash, cash equivalents, and marketable securities as of March 31, 2024, were $162.8 million, excluding $5.0 million of restricted cash. The Company believes that its existing cash resources along with the $161.4 million net proceeds received from its April 2024 underwritten public offering will be sufficient to fund its current operating plan into the fourth quarter of 2026.

Share Count: As of March 31, 2024, the Company had issued and outstanding shares of 47,904,737, which consists of 43,628,149 shares of voting common stock and 4,276,588 shares of non-voting common stock, and outstanding pre-funded warrants to purchase 47,010,526 shares of voting common stock at an exercise price of $0.0001 per share.

Subsequent to the end of the first quarter of 2024, in connection with its April 2024 underwritten public offering, the Company issued an additional 4,958,068 shares of voting common stock, and pre-funded warrants to purchase up to 18,577,419 shares of voting common stock with an exercise price of $0.0001 per share.

On May 13, 2024 Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a fully-integrated biopharmaceutical company with marketed products and a pipeline of development candidates, reported financial results for the first quarter ended March 31, 2024, and provided an overview of recent operational highlights (Press release, TONIX Pharmaceuticals, MAY 13, 2024, View Source [SID1234643159]).

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"Our near-term priority continues to be the submission of our New Drug Application (NDA) for Tonmya (cyclobenzaprine HCl sublingual tablets) for the management of fibromyalgia, while continuing to build out our commercial strategy for the anticipated product launch in the event of FDA approval, which we currently estimate to occur in the second half of 2025," said Seth Lederman, M.D., Chief Executive Officer of Tonix.

Dr. Lederman added, "The well-known treatment-limiting side effects of the three currently approved drugs have led to widespread patient dissatisfaction, creating what we believe is a significant opportunity for a new therapeutic. Tonmya has a differentiated mechanism of action and is generally free of common side effects associated with the currently approved products, including weight gain, fatigue, insomnia, increased blood pressure, gastrointestinal issues or sexual dysfunction. As such, we believe Tonmya, if approved, could become the treatment of choice for the approximately 10 million people in the U.S. suffering the debilitating effects of fibromyalgia."

The Company is also advancing other key pipeline programs including those for immunology, obesity, eating disorders, infectious and rare diseases, many through a capital efficient strategy involving partnerships, grants and in-kind contributions.

Recent Highlights – Key Product Candidates*

Central Nervous System (CNS) Pipeline

Tonmya (also known as TNX-102 SL; cyclobenzaprine HCl sublingual tablets): a centrally-acting, non-opioid, small molecule analgesic taken once-daily at bedtime for the management of fibromyalgia (FM).

In January 2024, Tonix presented additional safety and tolerability data from the pivotal Phase 3 RESILIENT study that showed Tonmya treatment was not associated with increases in systolic or diastolic blood pressure or body weight, nor were there any reported sexual side effects. The Company had previously announced in December 2023 that the Phase 3 RESILIENT study, a registration-quality, double-blind, placebo-controlled study evaluating Tonyma met its pre-specified primary endpoint in the second of two positive Phase 3 clinical trials, significantly reducing daily pain compared to placebo (p-value=0.00005) in participants with fibromyalgia. Statistically significant and clinically meaningful results were also seen in all pre-specified key secondary endpoints including those related to improving sleep quality, reducing fatigue, and improving patient global ratings and overall fibromyalgia symptoms and function. Tonmya was well tolerated with an adverse event profile comparable to prior studies and no new safety signals observed. In addition, Tonmya therapy showed activity on improving female sexual function relative to placebo with a nominal p-value=0.010 by the Changes in Sexual Functioning Questionnaire short-form, female version.
Tonix plans to submit an NDA to the FDA in the second half of 2024 for Tonmya for the management of fibromyalgia. In February 2024, Tonix announced the engagement of Rho, Inc. as our contract research organization (CRO) to support NDA submission.
In February 2024, Tonix announced statistically significant results from its clinical pharmacokinetic (PK) bridging study of Tonmya in healthy adult male and female ethnic Japanese and Chinese volunteers. Results indicate that key PK parameters of cyclobenzaprine are comparable in ethnic Japanese and Chinese volunteers to Caucasian volunteers from a prior PK study. Tonmya was generally well tolerated in the ethnic Japanese and Chinese healthy volunteers. The company expects these data to fulfill the requirement for a bridging study, and enables Tonix to rely on Phase 3 studies RESILIENT and RELIEF results to support regulatory filings for clinical studies in Japan and China where cyclobenzaprine is a new chemical entity (NCE). Tonix holds issued patents for market exclusivity rights of Tonmya in Japan, China, Hong Kong and Taiwan.
In March 2024, Tonix announced the selection of two contract manufacturing organizations (CMOs), including Almac Pharma Services, as dual supply sources for the potential launch and commercialization of Tonmya in the U.S.
In March 2024, Tonix selected EVERSANA, a leading provider of commercialization services to the global life sciences industry, to support the launch strategy and commercial planning of Tonmya in the U.S.
Tonix presented additional efficacy data from RESILIENT at the 6th International Congress on Controversies in Fibromyalgia in Brussels, Belgium, March 7-8, 2024. The data showed that Tonmya treatment resulted in an improvement in cognitive dysfunction, or ‘brain fog’, measured by the change in the Fibromyalgia Impact Questionnaire-Revised (FIQ-R) memory item. The FIQ-R cognitive item showed nominal improvement in Tonmya-treated patients vs placebo-treated patients with a nominal p-value=0.001 and effect size of 0.31.
TNX-102 SL for the treatment of acute stress reaction (ASR) and acute stress disorder (ASD), and prophylaxis against development of posttraumatic stress disorder (PTSD)

In February 2024, the Company announced the FDA cleared the Investigational New Drug (IND) application for the Phase 2 investigator-initiated OASIS trial to evaluate TNX-102 SL in reducing the severity of ASR and the frequency of ASD and PTSD. The trial is sponsored by the University of North Carolina Institute for Trauma Recovery and supported by a $3 million grant from the U.S. Department of Defense, which was awarded in September 2023. The proposed Phase 2, Optimizing Acute Stress Reaction Interventions with TNX-102 SL (OASIS) study will examine the safety and efficacy of TNX-102 SL to reduce adverse posttraumatic neuropsychiatric sequelae among patients presenting to the emergency department (ED) after a motor vehicle collision. The study will enroll approximately 180 trauma survivors at ED study sites in the U.S. Participants will be randomized in the ED to receive a two-week course of either TNX-102 SL 5.6 mg or placebo.
Tonix anticipates the Phase 2 OASIS trial will initiate in the second quarter of 2024.
TNX-102 SL for the treatment of Fibromyalgia-Type Long COVID, also known as Post-Acute Sequelae of COVID-19 (PASC)

In January 2024, the Company announced the online publication of a research paper in the Journal Pain. The article titled, "Chronic Overlapping Pain Conditions Increase the Risk of Long COVID Features, Regardless of Acute COVID Status," by Bergmans, et al.1, found that patients with pre-existing chronic overlapping pain conditions (COPCs) had an increased risk of being diagnosed with symptoms of Long COVID1. Faculty at the University of Michigan directed the research. Commentary on the article titled, "A step towards better understanding chronic overlapping pain conditions" by Fitzcharles, et al,2 is in the same issue of the journal. COPCs include fibromyalgia, chronic fatigue syndrome, migraine headache, irritable bowel syndrome, endometriosis and low back pain. These results contribute to a growing body of evidence that common symptoms of Long COVID in many patients are at least partly driven by central nervous system mechanisms.
TNX-1300 (recombinant double mutant cocaine esterase): biologic for life-threatening cocaine intoxication

Tonix expects to initiate a Phase 2 clinical study of TNX-1300 for the treatment of cocaine intoxication in emergency rooms in the second quarter of 2024. In 2022, Tonix was awarded a Cooperative Agreement grant from the National Institutes of Health (NIH)’s National Institute of Drug Abuse (NIDA) to support development of TNX-1300.
TNX-1300 has been granted Breakthrough Therapy designation by the FDA.
TNX-1900 (intranasal potentiated oxytocin): small peptide in development through investigator-initiated studies for adolescent obesity, binge eating disorder, bone health in autism and social anxiety disorder (SAD).

TNX-1900 continues to be studied in four ongoing investigator-initiated Phase 2 studies as follows: Massachusetts General Hospital (MGH): (1) Phase 2 study for binge-eating disorder (BED); (2) Phase 2 study for adolescent obesity; (3) Phase 2 study for improving bone health in children with autism spectrum disorder (BOX); and at University of Washington, (4) Phase 2 study for social anxiety disorder (SAD). The BED study and the adolescent obesity study will investigate whether TNX-1900 has effects on eating behaviors in specialized populations.
Rare Disease Pipeline

TNX-2900 (intranasal potentiated oxytocin): small peptide for the treatment of Prader-Willi syndrome (PWS)

In March 2024, Tonix announced that it received Rare Pediatric Disease designation from the FDA for TNX-2900 for the treatment of PWS. Tonix has an IND to support clinical development of TNX-2900 to treat PWS in children and adolescents. The planned Phase 2 study is a dose-finding study involving approximately 36 PWS patients divided into four groups with approximately nine per group. One group will receive placebo and three groups will receive different dosage regimens of TNX-2900. TNX-2900 for the treatment of PWS was granted Orphan Drug designation by the FDA in 2022. PWS is a genetic disorder that affects several body systems, with cognitive and behavioral symptoms including pathological over-eating beginning in childhood and leading to severe metabolic sequelae.
Immunology Pipeline

TNX-1500 (anti-CD40L Fc-modified humanized monoclonal antibody): third generation anti-CD40L monoclonal antibody for prophylaxis of organ transplant rejection and treatment of autoimmune disorders.

The first proposed indication for TNX-1500 is prophylaxis of organ rejection in adult patients receiving a kidney transplant; but multiple additional indications are possible, including autoimmune diseases. Two peer reviewed publications described the work with TNX-1500 at the Massachusetts General Hospital (MGH) on allogeneic transplants in animals.3,4
Preclinical studies have shown that TNX-1500 maintains the activity of first-generation monoclonal antibodies (mAbs), yet with reduced risk of thrombotic complications.3-5 Modeling studies from animal pharmacokinetic data3 predict a half-life of greater than three weeks for TNX-1500 in humans, which supports a monthly i.v. dosing regimen. This analysis together with TNX-1500’s activity and tolerability in animals, suggests that the protein engineering of TNX-1500’s Fc region has achieved its design goals.
In February 2024, Tonix announced the completion of the clinical stage of its Phase 1 single ascending dose study of TNX-1500 in healthy volunteers. The primary objectives of the study are to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of intravenous TNX-1500. This first-in-human study is intended to support dosing in a planned Phase 2 trial in kidney transplant recipients.
In March of 2024, the MGH announced the first transplant of a genetically modified pig kidney into a living patient in collaboration with eGenesis, which produced the pig donors and used an anti-CD40L mAb from another company.5 Some of the pre-clinical work that supported the living human transplant was performed in collaboration with Tonix and used TNX-1500.6 The patient was able to return home after the transplant, but died after approximately two months.7
Marketed Products – Recent Highlights

As of April 1, 2024, Tonix completed the transition to becoming a fully integrated pharmaceutical company. Tonix Pharmaceuticals has implemented personnel, systems and contracts required to support a commercial organization and has assumed responsibility for distribution, selling and marketing of Zembrace SymTouch and Tosymra, as well as supply chain, regulatory and quality control of the two products.
Facilities – Recent Highlights

In the fourth quarter of 2023, Tonix engaged CBRE, an international real estate brokerage firm, to potentially find a strategic partner for, or buyer of, its Advanced Development Center (ADC) to align with the Company’s current business objectives and priorities. At this time, the Company does not have a commitment in place to sell the building. ADC, located in the New Bedford business park in Dartmouth, Massachusetts, is an approximately 45,000 square foot BSL-2 facility intended for clinical scale manufacturing of live-virus vaccines and biologics.
*All of Tonix’s product candidates are investigational new drugs or biologics and none have been approved for any indication.

Tonmya is conditionally accepted by the U.S. Food and Drug Administration (FDA) as the tradename for TNX-102 SL for the management of fibromyalgia. Tonmya has not been approved for any indication.

1 Bergmans RS, et al. PAIN. 2023. DOI: 10.1097/j.pain.0000000000003110.

2 Fitzcharles M-A, et al. PAIN. 2023. DOI: 10.1097/j.pain.0000000000003129.

3 Lassiter G., et al. Am J Transplantation. 2023. View Source

4 Miura S., et al. Am J Transplantation. 2023. View Source

5 Massachusetts General Hospital press release. March 21, 2024. "World’s First Genetically Edited Pig Kidney Transplant into Living Recipient Performed at Massachusetts General Hospital." www.massgeneral.org/news/press-release/worlds-first-genetically-edited-pig-kidney-transplant-into-living-recipient (accessed March 29, 2024)

6 Anand, R.P., et al Nature. 622, 393–401 (2023). View Source

7 Stoico, N. Boston Globe. May 11, 2023. "Mass Man who received first kidney transplant from genetically engineered pig has died, family says".

Recent Highlights – Financial

As of March 31, 2024, Tonix had $7.0 million of cash and cash equivalents, compared to $24.9 million as of December 31, 2023. Net cash used in operations was approximately $17.6 million for first quarter 2024, compared to net cash used in operations of $32.9 million for the same period in 2023.

On April 1, 2024, the Company closed a financing with existing healthcare-focused institutional investors for upfront gross proceeds of approximately $4.4 million through a registered direct offering.

First Quarter 2024 Financial Results

Net product revenue for the first quarter 2024 was approximately $2.5 million. Net product revenue consisted of combined net sales of Zembrace SymTouch and Tosymra, which were acquired from Upsher-Smith Laboratories, LLC on June 30, 2023. Cost of Sales for the first quarter 2024 was approximately $1.7 million.

Research and development expenses for the first quarter 2024 were $12.9 million, compared to $26.5 million for the same period in 2023. This decrease is predominantly due to decreased clinical, non-clinical and manufacturing expenses.

General and administrative expenses for the first quarter 2024 were $9.3 million, compared to $7.4 million for the same period in 2023. The increase was primarily due to sales and marketing and the transition services expenses associated with the Company’s recently acquired marketed products offset by a decrease in financial reporting expenses.

Net loss was $(14.9) million, or $(0.18) per share, basic and diluted, for the first quarter 2024, compared to net loss of $(33.0) million, or $(3.21) per share, basic and diluted, for the same period in 2023. The basic and diluted weighted average common shares outstanding for the first quarter 2024 was 80,879,108 compared to 10,268,500 shares for the same period in 2023.