On August 3, 2023, Replimune Group, Inc. (the "Company") reported to have entered into a Sales Agreement (the "Sales Agreement") with Leerink Partners LLC (formerly known as SVB Securities LLC) (the "Agent"), pursuant to which the Company may sell, from time to time, at its option, up to an aggregate of $250.0 million of shares of the Company’s common stock, $0.001 par value per share (the "Shares"), through the Agent, as the Company’s sales agent (Filing, 8-K, Replimune, AUG 3, 2023, View Source [SID1234633773]).

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Any Shares to be offered and sold under the Sales Agreement will be issued and sold (i) by methods deemed to be an "at the market offering" as defined in Rule 415(a)(4) promulgated under the Securities Act of 1933, as amended, or if authorized by the Company, in negotiated transactions or block trades, and (ii) pursuant to an automatically effective registration statement on Form S-3 filed by the Company with the Securities and Exchange Commission on August 3, 2023 for an offering of various securities, including shares of the Company’s common stock, preferred stock, debt securities, warrants and/or units for sale to the public in one or more public offerings.

Subject to the terms of the Sales Agreement, the Agent will use reasonable efforts to sell the Shares from time to time, based upon the Company’s instructions (including any price, time or size limits or other customary parameters or conditions the Company may impose). The Company cannot provide any assurances that it will issue any Shares pursuant to the Sales Agreement. The Company will pay the Agent a commission of up to 3.0% of the gross proceeds from the sale of the Shares, if any. The Company has also agreed to provide the Agent with customary indemnification rights.

Pursuant to the Sales Agreement, the parties mutually agreed to terminate that certain sales agreement, dated June 23, 2022, by and between the Company and the Agent (as amended, the "2022 Sales Agreement") with respect to the Company’s previous at-the-market offering program (the "2022 ATM Program").

This Current Report on Form 8-K shall not constitute an offer to sell or the solicitation of an offer to buy the Shares nor shall there be any sale of the Shares in any state in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state.

The foregoing description of the Sales Agreement does not purport to be complete and is qualified in its entirety by reference to the full text of the Sales Agreement, which is attached hereto as Exhibit 1.1 and is incorporated by reference herein.

On August 3, 2023 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported financial results for the second quarter of 2023 and provided a business update (Press release, Regeneron, AUG 3, 2023, View Source [SID1234633772]).

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"Regeneron delivered strong financial results in the second quarter of 2023 through increasingly diversified revenue streams, and we remain well-positioned for long-term growth," said Leonard S. Schleifer, M.D., Ph.D., Board Co-Chair, President and Chief Executive Officer of Regeneron. "In the past months, we have continued to advance our pipeline, in particular aflibercept 8 mg which we are progressing towards a potential FDA decision in the third quarter and for which we shared unprecedented two-year results in the pivotal PHOTON trial demonstrating durable vision gains at extended dosing intervals in patients with diabetic macular edema."

Financial Highlights

($ in millions, except per share data) Q2 2023 Q2 2022 % Change
Total revenues $ 3,158 $ 2,857 11 %
GAAP net income $ 968 $ 852 14 %
GAAP net income per share – diluted $ 8.50 $ 7.47 14 %
Non-GAAP net income(a) $ 1,182 $ 1,127 5 %
Non-GAAP net income per share – diluted(a) $ 10.24 $ 9.77 5 %

"I am pleased with the performance of our business in the second quarter of 2023, including incremental pipeline progress and exceptional commercial execution," said Robert E. Landry, Executive Vice President, Finance and Chief Financial Officer of Regeneron. "We continue to prioritize internal investments while allocating additional capital to opportunistic share repurchases and potential business development."

Business Highlights

Key Pipeline Progress
Regeneron has approximately 35 product candidates in clinical development, including a number of marketed products for which it is investigating additional indications. Updates from the clinical pipeline include:

Aflibercept 8 mg

In June 2023, the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) for the Biologics License Application (BLA) for aflibercept 8 mg for the treatment of patients with neovascular age-related macular degeneration (wet AMD), DME, and diabetic retinopathy (DR). The CRL was issued solely due to unresolved observations resulting from an FDA inspection at a third-party contract manufacturing organization, Catalent, that the Company engaged to complete vial-filling for aflibercept 8 mg. The CRL did not identify any issues with the aflibercept 8 mg clinical efficacy or safety profile, trial design, labeling, or drug substance manufacturing, and no additional clinical data or trials have been requested. The FDA has informed the Company and Catalent that certain manufacturing data and other information are required from Catalent to allow the FDA to approve aflibercept 8 mg; the Company expects that these data and information will be submitted to the FDA by mid-August 2023. The FDA has stated that it intends to prioritize the review of this submission, and therefore the Company anticipates the FDA will take action on the aflibercept 8 mg BLA during the third quarter of 2023.
In June 2023, the Company announced top-line, two-year (96 weeks) data for aflibercept 8 mg from the pivotal PHOTON trial in patients with DME. The longer-term data among aflibercept 8 mg patients who completed the trial demonstrated that the vast majority of patients were able to maintain or further extend the dosing intervals through two years. In addition, visual gains for aflibercept 8 mg remained consistent with those observed in the first year of the trial. In PHOTON, the safety of aflibercept 8 mg continued to be similar to EYLEA through two years and remained consistent with the known safety profile of EYLEA from previous clinical trials for DME. Results from the PHOTON study were presented at the American Society of Retina Specialists annual meeting in July 2023.
The two-year data from the pivotal PULSAR trial for aflibercept 8 mg in wet AMD continue to be expected in the third quarter of 2023.
In May 2023, Bayer announced that it initiated a Phase 3 study to evaluate the efficacy and safety of aflibercept 8 mg at extended dosing intervals compared to the standard of care, EYLEA, in macular edema following retinal vein occlusion (RVO).
Dupixent (dupilumab)

The FDA granted Breakthrough Therapy designation for uncontrolled COPD with an eosiniphilic phenotype based on the positive results of the Phase 3 BOREAS study. Based on ongoing discussions with the FDA, the Company expects that in addition to the BOREAS study results, data from the replicate Phase 3 NOTUS study will be needed to support an sBLA, and such data requirements remain under discussion with the FDA. The Company expects final results for the NOTUS study in mid-2024.
The Company and Sanofi presented positive Phase 3 results from the BOREAS trial in adults currently on maximal standard-of-care inhaled therapy (triple therapy) with uncontrolled COPD and evidence of type 2 inflammation at the 2023 American Thoracic Society International Conference. The results were also published in the New England Journal of Medicine.
In June 2023, the Ministry of Health, Labour and Welfare (MHLW) in Japan approved Dupixent for the treatment of adult patients with prurigo nodularis.
Oncology Programs

The Company announced promising data from three independent expansion cohorts of a Phase 1 trial for fianlimab, an antibody to LAG-3, in combination with Libtayo (cemiplimab) in adults with advanced melanoma, which were also presented at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. These results demonstrated that the combination led to clinically meaningful and durable results across multiple advanced melanoma patient populations. The safety profile of the combination was generally consistent with the safety profile of Libtayo monotherapy and other anti-PD-(L)1 agents, except for higher rates of adrenal insufficiency, which were successfully managed with steroid replacement.
The Company also presented updated positive data from two expansion dose cohorts from a pivotal trial for linvoseltamab, a bispecific antibody targeting BCMA and CD3, in patients with heavily pre-treated, relapsed/refractory multiple myeloma at the ASCO (Free ASCO Whitepaper) Annual Meeting. No new safety signals were identified.
The Company is investigating multiple CD28 costimulatory bispecific antibodies, including PSMAxCD28, EGFRxCD28, MUC16xCD28, and CD22xCD28, in ongoing Phase 1 trials in a variety of tumor settings in combination with Libtayo, or in combination with corresponding CD3 bispecifics. In the ongoing study of REGN5678, a costimulatory bispecific antibody targeting PSMA and CD28 in advanced prostate cancer, the Company has observed antitumor activity in combination with Libtayo as well as with REGN5678 monotherapy. In the Libtayo combination cohort, there have now been two immune-mediated Grade 5 adverse events (death), including one in July 2023. As a result, the Company has discontinued enrollment of patients receiving the combination of REGN5678 and full-dose Libtayo, and plans to explore REGN5678 combinations with lower doses of Libtayo. The Company also plans to enroll patients in a REGN5678 monotherapy cohort, as well as in combination with other immunotherapy modalities. Other costimulatory bispecific development programs continue their respective dose-escalation studies.
Itepekimab, an antibody to IL-33

The Phase 3 program investigating itepekimab in patients with COPD who are former smokers passed a recent interim futility analysis. The analysis was conducted by an Independent Data Monitoring Committee, and the Company and Sanofi remain blinded to the data. Results from the AERIFY-1 and AERIFY-2 studies are expected to be reported in 2025.

Corporate Update

The United States Supreme Court issued a unanimous opinion, ending a nearly decade-long patent dispute related to Praluent (alirocumab). The decision affirms the United States Court of Appeals for the Federal Circuit’s opinion, which held that Amgen’s asserted U.S. PCSK9 patent claims were invalid.
Second Quarter 2023 Financial Results

Revenues

($ in millions) Q2 2023 Q2 2022 % Change
Net product sales:
EYLEA – U.S. $ 1,500 $ 1,621 (7 %)
Libtayo – U.S. 130 91 43 %
Libtayo – ROW 80 — *
Praluent – U.S. 41 31 32 %
Evkeeza – U.S. 19 11 73 %
Inmazeb – U.S. 2 — *
Total net product sales 1,772 1,754 1 %

Collaboration revenue:
Sanofi 944 678 39 %
Bayer 377 358 5 %
Other (4 ) 8 *
Other revenue 69 59 17 %
Total revenues $ 3,158 $ 2,857 11 %

* Percentage not meaningful.
Net product sales of EYLEA in the U.S. decreased in the second quarter of 2023, compared to the second quarter of 2022, primarily due to a lower net selling price driven by changing market dynamics, including increased competition.

Sanofi collaboration revenue increased in the second quarter of 2023, compared to the second quarter of 2022, primarily due to the Company’s share of profits from commercialization of antibodies, which were $751 million in the second quarter of 2023, compared to $497 million in the second quarter of 2022. The change in the Company’s share of profits from commercialization of antibodies was driven by higher profits associated with an increase in Dupixent sales.

Refer to Table 4 for a summary of collaboration revenue.

Operating Expenses

GAAP %
Change
Non-GAAP(a) %
Change
($ in millions) Q2 2023 Q2 2022 Q2 2023 Q2 2022
Research and development (R&D) $ 1,085 $ 794 37 % $ 974 $ 690 41 %
Acquired in-process research and development (IPR&D) $ — $ 197 (100 %) * * n/a
Selling, general, and administrative (SG&A) $ 652 $ 476 37 % $ 562 $ 418 34 %
Cost of goods sold (COGS) $ 192 $ 149 29 % $ 163 $ 137 19 %
Cost of collaboration and contract manufacturing (COCM) $ 213 $ 148 44 % * * n/a
Other operating (income) expense, net $ (1 ) $ (17 ) (94 %) * * n/a

* GAAP and non-GAAP amounts are equivalent as no non-GAAP adjustments have been recorded.
GAAP and non-GAAP R&D expenses increased in the second quarter of 2023, compared to the second quarter of 2022, driven by additional costs incurred in connection with higher headcount and headcount-related costs, the advancement of the Company’s late-stage pipeline, the impact of the 2022 amendments to the Sanofi collaboration agreements, and increased manufacturing activity associated with the Company’s earlier-stage product candidates.
Acquired IPR&D in the second quarter of 2022 included a $195 million charge related to the Company’s acquisition of Checkmate Pharmaceuticals, Inc.
GAAP and non-GAAP SG&A expenses increased in the second quarter of 2023, compared to the second quarter of 2022, primarily due to an increase in commercialization-related expenses for Libtayo outside the U.S. (as effective July 1, 2022, the Company became solely responsible for the commercialization of Libtayo worldwide), higher headcount and headcount-related costs, and higher contributions to an independent not-for-profit patient assistance organization.
COCM expenses increased in the second quarter of 2023, compared to the second quarter of 2022, primarily due to the recognition of costs in connection with manufacturing commercial supplies of Dupixent.
Other Financial Information

GAAP other income (expense) included the recognition of net unrealized losses on equity securities of $31 million in the second quarter of 2023, compared to $164 million in the second quarter of 2022. GAAP and Non-GAAP other income (expense) also included interest income of $118 million in the second quarter of 2023, compared to $28 million in the second quarter of 2022.

In the second quarter of 2023, the Company’s GAAP effective tax rate (ETR) was 10.6%, compared to 11.5% in the second quarter of 2022. In the second quarter of 2023, the non-GAAP ETR was 12.2%, compared to 13.6% in the second quarter of 2022.

GAAP net income per diluted share was $8.50 in the second quarter of 2023, compared to $7.47 in the second quarter of 2022. Non-GAAP net income per diluted share was $10.24 in the second quarter of 2023, compared to $9.77 in the second quarter of 2022. A reconciliation of the Company’s GAAP to non-GAAP results is included in Table 3 of this press release.

During the second quarter of 2023, the Company repurchased shares of its common stock and recorded the cost of the shares, or $723 million, as Treasury Stock. As of June 30, 2023, an aggregate of $2.3 billion remained available for share repurchases under the Company’s share repurchase program.

On August 3, 2023 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and financial results for the second quarter ending June 30, 2023 (Press release, PTC Therapeutics, AUG 3, 2023, View Source [SID1234633771]).

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"I am extremely proud of the revenue growth in the first half of 2023 providing us confidence that we will meet our full-year total revenue guidance," said Matthew Klein, M.D., Chief Executive Officer, PTC Therapeutics, Inc. "In addition, the great results from the APHENITY and PIVOT-HD trials position us well for future growth."

Key Corporate Updates:

Second quarter 2023 revenue for the Duchenne muscular dystrophy (DMD) franchise was $162 million, representing 21% year-over-year growth.
Translarna (ataluren) quarterly net product revenue was $96 million, with growth coming from treatment of new patients and continued geographic expansion.
Emflaza (deflazacort) quarterly net product revenue was $66 million, driven by new patients, and high compliance.
Key Clinical and Regulatory Updates:

The primary endpoint of blood phenylalanine reduction in the APHENITY trial for sepiapterin in PKU was achieved, with highly statistically significant and clinically meaningful results.
PTC expects to file an NDA for sepiapterin in the fourth quarter of 2023, pending FDA feedback.
All key objectives were met in the 12-week interim data analysis of the PIVOT-HD trial of PTC518 in Huntington’s disease patients.
PTC expects to submit the BLA for Upstaza in the third quarter of 2023.
PTC expects a CHMP opinion on the Type II variation to support the conversion of the conditional marketing authorization for Translarna to a standard marketing authorization in the third quarter of 2023.
PTC expects additional regulatory meetings in the second half of 2023 including:
Type C meeting with the FDA for vatiquinone in FA
Type C meeting with the FDA for Translarna in DMD
Second Quarter 2023 Financial Highlights:

Total revenues were $213.8 million for the second quarter of 2023, compared to $165.5 million for the second quarter of 2022.
Total revenues include net product revenue across the commercial portfolio of $174.6 million for the second quarter of 2023, compared to $143.7 million for the second quarter of 2022. Total revenues also include royalty and manufacturing revenue of $39.2 million for the second quarter of 2023, compared to $21.8 million for the second quarter of 2022.
Translarna net product revenues were $96.5 million for the second quarter of 2023, compared to $77.0 million for the second quarter of 2022. These results were driven by treatment of new patients and continued geographic expansion.
Emflaza net product revenues were $65.7 million for the second quarter of 2023, compared to $56.8 million for the second quarter of 2022. These results reflect new patients and high compliance.
Roche reported Evrysdi 2023 year-to-date sales of approximately CHF 705 million, resulting in royalty revenue of $36.9 million to PTC for the second quarter of 2023, as compared to $21.8 million for the second quarter of 2022.
Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $185.9 million for the second quarter of 2023, compared to $157.3 million for the second quarter of 2022. The increase primarily reflects additional investment in advancement of the clinical pipeline.
Non-GAAP R&D expenses were $170.3 million for the second quarter of 2023, excluding $15.5 million in non-cash, stock-based compensation expense, compared to $143.5 million for the second quarter of 2022, excluding $13.8 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $88.4 million for the second quarter of 2023, compared to $79.9 million for the second quarter of 2022. The increase reflects our continued investment to support commercial activities, including expanding our commercial portfolio.
Non-GAAP SG&A expenses were $74.6 million for the second quarter of 2023, excluding $13.8 million in non-cash, stock-based compensation expense, compared to $66.0 million for the second quarter of 2022, excluding $13.9 million in non-cash, stock-based compensation expense.
During the second quarter of 2023, PTC announced the discontinuation of preclinical and early research programs in gene therapy and a reduction in workforce as part of a strategic portfolio prioritization, which resulted in a one-time charge of approximately $8.0 million recorded to R&D and SG&A expense.
The change in the fair value of contingent consideration was a gain of $128.9 million for the second quarter of 2023, compared to a gain of $15.2 million for the second quarter of 2022. The change in fair value of contingent consideration is related to the fair valuation of potential future consideration to be paid to former equity holders of Agilis Biotherapeutics, Inc. (Agilis) in connection with PTC’s acquisition of Agilis, which closed in August 2018. As a result of the discontinuation of the Friedreich ataxia and Angelman syndrome gene therapy programs, PTC determined that the fair value for all of the contingent consideration payable related to Friedreich ataxia and Angelman syndrome was $0 and recorded a gain of $129.8 million, which is the primary driver of the overall gain during the quarter. An intangible asset impairment of $217.8 million was recorded in the second quarter of 2023 which also related to the discontinuation of Friedreich ataxia and Angelman syndrome gene therapy programs. The net impact of these gene therapy program discontinuations was non-cash expense of $88 million recorded within total operating expenses.
The net loss was $198.9 million for the second quarter of 2023, compared to a net loss of $152.1 million for the second quarter of 2022.
Cash, cash equivalents, and marketable securities was $337.9 million on June 30, 2023, compared to $410.7 million at December 31, 2022.
Shares issued and outstanding as of June 30, 2023, were 75,318,233.
PTC Updates Full Year 2023 Financial Guidance as Follows:

PTC anticipates total revenues for the full year 2023 to be between $940 million and $1.0 billion.
PTC anticipates net product revenues for the DMD franchise for the full year 2023 to be between $545 and $575 million.
PTC anticipates GAAP R&D and SG&A expense for the full year 2023 to be between $930 million and $980 million.
PTC anticipates Non-GAAP R&D and SG&A expense for the full year to be between $810 million and $860 million, excluding estimated non-cash stock-based compensation expense of $120 million.
PTC also anticipates up to $62 million of one-time expenses, paid in cash or equity ($37 million of which was incurred during the first half of 2023), upon achievement of potential clinical and regulatory success-based milestones from previous acquisitions and expenses associated with a rights exchange agreement.

On August 3, 2023 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported financial results for the second quarter ended June 30, 2023 and provided a business update (Press release, Protara Therapeutics, AUG 3, 2023, View Source [SID1234633770]).

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"Following the positive preliminary data from the dose escalation portion of the ADVANCED-1 trial of TARA-002 in patients with high-grade non-muscle invasive bladder cancer (NMIBC), we have seen rapid enrollment in the expansion portion of the trial and anticipate sharing preliminary results in the first half of 2024," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We are building on this momentum, and plan to initiate two additional trials for TARA-002 later this year while continuing to maintain a disciplined approach to investments. We are in a solid financial position with cash runway into 2025."

Recent Highlights

TARA-002 in NMIBC

In April 2023 at the American Urological Association Annual Meeting, the Company announced positive preliminary results from the Phase 1a dose-escalation component of the ADVANCED-1 clinical trial of TARA-002, its investigational cell-based therapy, for the treatment of patients with high-grade NMIBC. The clinical data indicate that TARA-002 was generally well tolerated and anti-tumor activity was observed, including tumor regression in all three evaluable patients with carcinoma in situ (CIS), including one heavily pre-treated Bacillus Calmette-Guérin (BCG)-unresponsive patient who achieved a complete response (CR).

In May 2023, the Company dosed the first patients in its Phase 1b ADVANCED-1EXP study, an open-label expansion trial evaluating intravesical TARA-002 at the 40KE1 dose in 12 CIS patients, including BCG-naïve, BCG-unresponsive, and BCG-inadequately treated patients. Dosing continues to progress in the trial, with preliminary results expected in the first half of 2024. The primary endpoint of the trial is the CR rate at three months.

In the fourth quarter of 2023, the Company plans to initiate ADVANCED-2, a Phase 1b/2 open-label trial evaluating intravesical TARA-002 in up to 102 patients with high-grade CIS, including 27 patients with CIS (± Ta/T1), BCG-Naïve, or BCG-experienced, who have not received intravesical BCG for at least 24 months prior to CIS diagnosis, and 75 patients with BCG-unresponsive CIS (± Ta/T1).
TARA-002 in Lymphatic Malformations (LMs)

In April 2023, the Company received regulatory clearance from the U.S. Food and Drug Administration (FDA) to commence STARBORN-1, a Phase 2 clinical trial of TARA-002 in pediatric patients with macrocystic and mixed-cystic LMs. Trial start-up activities are well underway at the ten pediatric centers of excellence participating in the trial, and initiation is expected in the fourth quarter of 2023.
IV Choline Chloride Program

Protara is concluding its prospective prevalence study to enhance understanding of the incidence of choline deficiency in patients dependent on parenteral nutrition. The Company continues to engage with the FDA and plans to use both regulatory feedback and results from the prospective study to inform next steps for the IV Choline Chloride development program.
Second Quarter 2023 Financial Results

As of June 30, 2023, cash, cash equivalents and restricted cash were $80.4 million. The Company expects its current cash and cash equivalents will be sufficient to fund its planned operations into 2025.
Research and development expenses for the second quarter of 2023 increased to $7.2 million from $3.1 million during the second quarter of 2022, primarily reflecting an increase in expenses related to clinical and non-clinical trial activities for TARA-002.
General and administrative expenses for the second quarter of 2023 decreased to $4.9 million from $5.6 million for the prior year period, primarily due to lower employee related expenses.
For the second quarter of 2023, Protara reported a net loss of $11.3 million, or $1.00 per share, compared with a net loss of $8.5 million, or $0.76 per share, for the same period in 2022. Net loss for the second quarter of 2023 included approximately $1.6 million of stock-based compensation expenses.
About TARA-002

TARA-002 is an investigational cell therapy in development for the treatment of NMIBC and of LMs, for which it has been granted Rare Pediatric Disease Designation by the U.S. Food and Drug Administration. TARA-002 was developed from the same master cell bank of genetically distinct group A Streptococcus pyogenes as OK-432, a broad immunopotentiator marketed as Picibanil in Japan and approved in Taiwan by Chugai Pharmaceutical Co., Ltd. Protara has successfully shown manufacturing comparability between TARA-002 and OK-432.

When TARA-002 is administered, it is hypothesized that innate and adaptive immune cells within the cyst or tumor are activated and produce a strong immune cascade. Neutrophils, monocytes, and lymphocytes infiltrate the abnormal cells and various cytokines, including interleukins IL-2, IL-6, IL-8, IL-10, IL-12, interferon (IFN)-gamma, and tumor necrosis factor (TNF)-alpha are secreted by immune cells to induce a strong inflammatory reaction and destroy the abnormal cells.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

Bladder cancer is the 6th most common cancer in the United States, with NMIBC representing approximately 80% of bladder cancer diagnoses. Approximately 65,000 patients are diagnosed with NMIBC in the United States each year. NMIBC is cancer found in the tissue that lines the inner surface of the bladder that has not spread into the bladder muscle.

About Lymphatic Malformations (LMs)

LMs are rare, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain into the venous system. Most LMs are present in the head and neck region and are diagnosed in early childhood during the period of active lymphatic growth, with more than 50% detected at birth and 90% diagnosed before the age of 3 years. The most common morbidities and serious manifestations of the disease include compression of the upper aerodigestive tract, including airway obstruction requiring intubation and possible tracheostomy dependence; intralesional bleeding; impingement on critical structures, including nerves, vessels, lymphatics; recurrent infection, and cosmetic and other functional disabilities.

About IV Choline Chloride

IV Choline Chloride is an investigational, intravenous (IV) phospholipid substrate replacement therapy initially in development for patients receiving parenteral nutrition (PN). Choline is a known important substrate for phospholipids that are critical for healthy liver function. Because PN patients cannot sufficiently absorb adequate levels of choline and no available PN formulations contain sufficient amounts of choline to correct this deficiency, PN patients often experience a prolonged progression to hepatic failure and death, with the only known intervention being a dual small bowel/liver transplant. IV Choline Chloride has been granted Orphan Drug Designation by the FDA for the prevention of choline deficiency in PN patients.

On August 3, 2023 Protagonist Therapeutics (Nasdaq: PTGX) ("Protagonist" or "the Company") reported financial results for the second quarter ended June 30, 2023, and provided a corporate update (Press release, Protagonist, AUG 3, 2023, View Source [SID1234633768]).

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"The second quarter of 2023 was transformative for Protagonist, marked by the disclosure of strongly positive data from FRONTIER 1, a Phase 2b study of JNJ-2113, at the World Congress of Dermatology, and the decision by our partner, Janssen, to move forward into a Phase 3 clinical program in psoriasis and a Phase 2b study in ulcerative colitis," said Dinesh V. Patel, Ph.D., President and CEO of Protagonist. "JNJ-2113 is a first-in-class and only oral IL-23 receptor antagonist peptide that has the potential to lead the field of oral therapy for psoriasis. Today, a significant number of people living with moderate-to-severe plaque psoriasis are eligible for, but are still not receiving, advanced therapies, and JNJ-2113 can potentially offer a highly convenient, effective and safe solution to a broad category of eligible individuals."

"We have also made impressive strides forward in the clinical development of our fully owned asset, rusfertide, in polycythemia vera," Dr. Patel continued. "The Phase 2 REVIVE study results were presented to a large audience as a late breaker at EHA (Free EHA Whitepaper)2023. The Phase 3 VERIFY clinical trial is continuing to enroll study participants at sites globally, with enrollment completion projected by the end of the first quarter of 2024. Our cash position remains strong, with estimated cash runway through end of 2025. This estimate does not include the potential $200 million in milestones associated with a successful Phase 3 study, NDA filing, and approval of JNJ-2113 in psoriasis."

Q2 and Recent Corporate Highlights

· Positive Phase 2b FRONTIER 1 topline results were presented at the World Congress of Dermatology in Singapore in July 2023. All primary and secondary efficacy endpoints were achieved in the study, which evaluated five different dosing regimens of JNJ-2113 in adult patients with moderate-to-severe plaque psoriasis.

· JNJ-2113 is a novel oral IL-23R antagonist peptide which binds with high affinity to the IL-23 receptor.

· In the FRONTIER 1 study, a greater proportion of patients who received JNJ-2113 achieved Psoriasis Area and Severity Index (PASI) 75 (primary endpoint), as well as PASI 90 and PASI 100 (75, 90 and 100 percent improvement in skin lesions as measured by PASI, respectively), compared to placebo, at week 16.

1

· 78.6 percent, 59.5 percent and 40.5 percent of adult patients who received JNJ-2113 at 100 mg twice daily achieved PASI 75, 90 and 100, respectively, at week 16.

· Five treatment groups were evaluated in the study, ranging from 25 mg once daily to 100 mg twice daily.

· Treatment was generally well tolerated, and the proportions of patients with adverse events were comparable between patient groups.

· The proportion of participants experiencing one or more adverse events was 52.4 percent in the combined JNJ-2113 group and 51.2 percent in the placebo group.

· JNJ-2113 is advancing into Phase 3 development for moderate-to-severe plaque psoriasis and in a Phase 2b clinical trial for adults living with ulcerative colitis.

· Pre-clinical and Phase 1 data for JNJ-2113 were presented at the International Societies for Investigative Dermatology meeting in Tokyo, Japan in May 2023, showing selective systemic IL-23 pathway inhibition in pre-clinical rat models and systemic pharmacodynamic activity in healthy human volunteers in a Phase 1 trial.

· Positive topline results from the blinded, placebo-controlled, randomized withdrawal portion of the Phase 2 REVIVE study of rusfertide in polycythemia vera were presented in Frankfurt, Germany in June 2023 at EHA (Free EHA Whitepaper)2023.

· The Company completed a $115 million equity financing in April 2023.

Second Quarter 2023 Financial Results

· Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities as of June 30, 2023, were $313.4 million.

· License and Collaboration Revenue: License and collaboration revenue was zero for the three and six months ended June 30, 2023 as we completed our performance obligation associated with the Janssen License and Collaboration Agreement as of June 30, 2022. License and collaboration revenue for the three and six months ended June 30, 2022 was $0.9 million and $26.6 million, respectively. The six months to June 30, 2022 included a one-time $25.0 million milestone earned by the Company following the dosing of the third patient in the Janssen Phase 2b FRONTIER 1 clinical trial of JNJ-2113.

· Research and Development ("R&D") Expenses: R&D expenses were $33.2 million and $60.6 million for the three and six months ended June 30, 2023, respectively, as compared to $34.6 million and $70.9 million for the same periods in 2022. The decreases in R&D expenses from prior year periods was primarily due to decreases in PN-943 expenses and costs related to the completion of PN-232 Phase 1 trials, partially offset by an increase in rusfertide expenses related primarily to the Phase 3 VERIFY clinical trial.

· General and Administrative ("G&A") Expenses: G&A expenses were $9.2 million and $17.8 million for the three and six months ended June 30, 2023, respectively, as compared to $7.7 million and $18.2 million for the same periods in 2022. The increase in G&A expenses from the prior year quarter was primarily due to increases in payroll and stock-based compensation and general expenses. The decrease in G&A expenses from the prior year was primarily due to one-time costs incurred in the first quarter of 2022.

· Net Loss: Net loss was $38.5 million, or $0.68 per share, for the three months ended June 30, 2023 as compared to a net loss of $41.0 million, or $0.84 per share, for the three months ended June 30, 2022. Net loss was $72.2 million, or $1.34 per share, for the six months ended June 30, 2023 as compared to a net loss of $62.0 million, or $1.27 per share, for the six months ended June 30, 2022.