On August 7, 2023 IO Biotech, Inc. (Nasdaq: IOBT), a clinical biopharmaceutical company developing novel, immune-modulating cancer vaccines based on its T-win technology platform, reported that it has entered into a definitive securities purchase agreement for a private placement that is expected to result in gross proceeds of approximately $75 million, before deducting offering expenses (Press release, IO Biotech, AUG 7, 2023, View Source [SID1234633876]). The private placement includes participation from new and existing investors, including Lundbeckfonden BioCapital; Kurma Growth Opportunities Fund; Vivo Capital; Armistice Capital; Marshall Wace; Samsara BioCapital; Novo Holdings; Stonepine Capital Management; PFM Health Sciences, LP; HBM Healthcare Investments (Cayman) Limited; Pivotal Life Sciences; Sunstone Life Science Ventures; Logos Capital; Altamont Pharmaceutical Holdings, LLC; and The Red Hook Fund LP; among others. Members of the company’s management team also participated in this transaction.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The continued strong support that we have received from our existing investors and the enthusiasm from an impressive group of new healthcare investors are testaments to the potential of IO102-IO103, to the strength of the data generated to date from the Phase 1/2 study in metastatic melanoma and from our ongoing Phase 2 basket study (IOB-022/KND38)," said Mai-Britt Zocca, PhD, President and CEO of IO Biotech. "With the proceeds of this financing, we expect to have sufficient capital to fund our operations into the fourth quarter of 2025, supporting the continued development of IO102-IO103 through critical clinical milestones from our pivotal Phase 3 trial in first-line advanced melanoma, as well as from our Phase 2 studies."

Under the securities purchase agreement, the investors have agreed to purchase 37,065,647 shares of the company’s common stock and accompanying warrants to purchase up to an aggregate of 37,065,647 shares of common stock, at a combined purchase price of $2.025 per share and accompanying warrant. Each accompanying warrant will represent the right to purchase one share of the company’s common stock at an exercise price of $2.47 per share. The warrants will be exercisable for a period of three years and six months following the date of issuance.

Morgan Stanley and Piper Sandler acted as joint placement agents for the financing.

The offer and sale of the foregoing securities are being made in a transaction not involving a public offering and the securities have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), or applicable state securities laws. Accordingly, the securities may not be reoffered or resold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws.

This press release does not constitute an offer to sell or the solicitation of an offer to buy the securities, nor shall there be any sale of the securities in any state in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such state.

About IO102-IO103

IO102-IO103 is an investigational immune-modulating cancer vaccine designed to target the immunosuppressive mechanisms mediated by the proteins indoleamine 2,3-dioxygenase (IDO) and PD-L1. The company is currently conducting a Phase 3 trial (IOB-013/KN-D18; NCT05155254) evaluating IO102-IO103 in combination with pembrolizumab in first-line advanced melanoma patients. The company is also conducting a Phase 2 basket trial (IOB-022/KN-D38; NCT05077709), a non-comparative, open label trial to investigate the safety and efficacy of IO102-IO103 in combination with pembrolizumab for the first-line treatment of each of the following advanced cancers: non-small cell lung cancer (NSCLC) and squamous cell carcinoma of the head and neck (SCCHN).

On August 7, 2023 Ikena Oncology, Inc. (Nasdaq: IKNA, "Ikena"), a targeted oncology company forging new territory in patient-directed cancer treatment, and Pionyr Immunotherapeutics, Inc. (Pionyr), a privately-held, clinical-stage biotechnology company, reported the closing of Ikena’s acquisition of Pionyr in an all-stock transaction (Press release, Ikena Oncology, AUG 7, 2023, View Source [SID1234633875]). Ikena acquired all of Pionyr’s assets, including approximately $43 million in net cash, in exchange for shares of IKNA stock, in a combination of common stock and non-voting convertible preferred stock priced at $7.15 per share.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Ikena has made incredible strides this year, and we are glad to welcome the Pionyr shareholders to the Ikena team as we head into a pivotal time for our targeted oncology programs," said Mark Manfredi, Ph.D., Chief Executive Officer of Ikena. "The capital from this acquisition allows us to accelerate and expand our clinical efforts in IK-930 and further support our comprehensive clinical plan for IK-595 as we continue to build value through both depth and breadth in our pipeline. We are looking forward to sharing more updates from across our business and pipeline, including initial clinical data from IK-930 later this year."

The transaction further strengthens Ikena’s financial position as the Company advances development of its targeted oncology programs. The new capital will enable Ikena to expand and accelerate its targeted oncology programs, including both IK-930, Ikena’s clinical program targeting the Hippo pathway with initial clinical data expected this year, and IK-595, a MEK-RAF complexing inhibitor expected to have an IND submitted this year. The new Ikena shareholders include New Enterprise Associates, SV Health Investors, Sofinnova Investments, Vida Ventures, and Gilead Sciences. Gilead Sciences acquired an equity stake in Pionyr through their 2020 option-to-acquire agreement.

"We have been highly rigorous and selective in our search for a buyer. Our attraction to Ikena was driven by not only the potential of their targeted oncology programs, but by their company culture and the way their team has managed capital and developed their pipeline," commented Steven P. James, Pionyr’s Chief Executive Officer. "On behalf of our investors, board of directors, and the Pionyr executive team, I want to thank and congratulate the entire Pionyr team for their efforts."

Further details on the transaction are described below.

Transaction and Approval

•
The transaction was unanimously approved by the boards of directors from both companies and closed following signing of the definitive agreement

•
Ikena’s acquisition of Pionyr was structured as a stock-for-stock transaction whereby all of Pionyr’s outstanding common stock was exchanged for a combination of Ikena common stock and non-voting convertible preferred stock

•
Ikena acquired all of Pionyr’s assets, including cash, cash equivalents, and investments as well as all intellectual property and clinical and preclinical assets

Pionyr Programs

•
Ikena plans to include Pionyr’s programs as part of Ikena’s partnering portfolio, and to pursue strategic business development opportunities, including out-licensing

•
The transaction includes contingent value rights (CVRs) providing the legacy Pionyr shareholders with rights to 50% of the net proceeds, outside of royalties, for any potential monetization of the Pionyr programs within the next two years

Additional Details

•
Pionyr shareholders were collectively issued approximately 1.8 million shares of Ikena common stock with the remainder of the shares issued as Ikena non-voting convertible preferred stock

•
Pionyr’s valuation for the transaction was determined solely by net cash available at closing

•
The transaction price of $7.15 per share represents a premium to both Ikena’s twenty-day volume-weighted average ending on the transaction date and its most recent underwritten public offering

•
As a result of the transaction, the Pionyr shareholders collectively hold approximately 12% of Ikena’s outstanding shares

•
Ikena’s management team and board of directors remain unchanged

On August 7, 2023 Gritstone bio, Inc. (Nasdaq: GRTS), a clinical-stage biotechnology company that aims to develop the world’s most potent vaccines, reported that the company has joined the ctMoniTR Project, a collaborative research initiative providing foundational evidence to inform future use of change in circulating tumor DNA (ctDNA) as an early indicator of long-term clinical benefit from therapeutics in cancer patients (Press release, Gritstone Bio, AUG 7, 2023, View Source [SID1234633873]). The ctMoniTR Project is led by Friends of Cancer Research (Friends).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"ctDNA represents a highly promising biomarker that could transform the way we develop novel cancer immunotherapies," said Andrew Allen, M.D., Ph.D., Co-founder, President, and Chief Executive Officer of Gritstone bio. "As cancer drug development has evolved, the field has seen the immense impact that biomarkers can offer when it comes to identifying effective novel treatments early on, which can make an enormous difference in the lives of patients. We’re pleased to serve as a partner on the ctMoniTR Project, which will help empirically inform the clinical applications where ctDNA exhibits the greatest potential to accelerate novel drug development."

Through the ctMoniTR Project, Gritstone and other partners contribute data and collaborate with Friends to help develop data-driven evidence of how ctDNA can inform treatment decisions, support drug development, and inform regulatory decision-making. To date, Friends and its partners have collected data from more than 25 studies representing over 3,000 cancer patients, 16 additional treatments, and several cancer types.

"The use of ctDNA as a tool to predict clinical outcomes with novel forms of immunotherapy is accelerating at a rapid pace, and the data emerging from this growing field point to ctDNA potentially serving as a more precise and accurate predictor of outcomes than current radiology-based measures," added Karin Jooss, Ph.D., Executive Vice President and Head of R&D at Gritstone. "Through ctMonitor, we aim to help the field better understand and validate this new, powerful tool. We are thankful to Friends for their leadership of this unique project and hope our collective efforts can help bring new therapies to patients safely and efficiently."

For more information on the ctMoniTR Project, visit: View Source

On August 7, 2023 FibroGen, Inc. (NASDAQ: FGEN) reported financial results for the second quarter 2023 and provided an update on the Company’s recent developments (Press release, FibroGen, AUG 7, 2023, View Source [SID1234633872]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We saw another record quarter of roxadustat sales in China, and we recently submitted the sNDA to the China Health Authority for chemotherapy-induced anemia," said Thane Wettig, Interim Chief Executive Officer, FibroGen. "As I assume the leadership role, I am very optimistic about our future. Within the next year, we have three pamrevlumab read-outs and key milestones for our early-stage pipeline, which combined with our deeply experienced team and strong cash position, set us up well for delivering value to patients and shareholders."

Recent Developments and Key Events of Second Quarter 2023:

Appointed Thane Wettig as Interim Chief Executive Officer.
Entered into exclusive license for FOR46 (now FG-3246), a first-in-class CD46-targeting ADC for the treatment of metastatic castration-resistant prostate cancer (mCRPC).
Reported positive topline data from Company’s Phase 3 clinical study of roxadustat for treatment of anemia in patients receiving concurrent chemotherapy treatment for non-myeloid malignancies in China.
Filed Supplemental New Drug Application (sNDA) with China Health Authority for roxadustat in patients with chemotherapy-induced anemia.
Announced a non-dilutive term loan facility with investment funds managed by Morgan Stanley Tactical Value (MSTV), which resulted in proceeds to FibroGen of $75 million.
Reported negative topline results from MATTERHORN Phase 3 clinical study of roxadustat for treatment of anemia in patients with transfusion-dependent lower risk myelodysplastic syndromes (MDS).
Reported negative topline results from the LELANTOS-1 Phase 3 study of pamrevlumab for the treatment of non-ambulatory patients with Duchenne muscular dystrophy (DMD).
Reported negative topline results from the ZEPHYRUS-1 Phase 3 study of pamrevlumab in patients with idiopathic pulmonary fibrosis (IPF). Discontinued ZEPHYRUS-2 Phase 3 study of pamrevlumab in patients with IPF.
Implemented a cost reduction plan, resulting in an expected reduction of total annualized U.S. GAAP expenses of $100-120 million.
China Performance:

Achieved second quarter net product revenue under U.S. GAAP from the sale of roxadustat in China of $23.9 million compared to $23.3 million in the second quarter of 2022.
Achieved second quarter total roxadustat net sales in China1 by FibroGen and the distribution entity (JDE) jointly owned by FibroGen and AstraZeneca of $76.4 million, compared to $53.1 million in the second quarter of 2022, an increase of 44%, driven by over 40% growth in volume.
Roxadustat continues to be the number one brand based on value share in the anemia of chronic kidney disease market in China.
Upcoming Milestones:

Pamrevlumab

Topline data from the LELANTOS-2 Phase 3 study of pamrevlumab in ambulatory DMD patients expected in 3Q 2023.
Topline data from the LAPIS Phase 3 study of pamrevlumab in locally advanced unresectable pancreatic cancer (LAPC) expected in 1Q 2024.
Topline data from the PanCAN Precision PromiseSM Phase 2/3 study of pamrevlumab in metastatic pancreatic cancer expected in 1H 2024.
Early-Stage Oncology Pipeline

Anticipate the initiation of a Phase 2 trial of FG-3246, a first-in-class ADC targeting a novel epitope on CD46 for mCRPC in 2H 2024.
Anticipate the filing of two INDs: FG-3165 (anti-Gal9 antibody) in 1Q 2024 and FG-3175 (anti-CCR8 antibody) in 2H 2024.
Financial:

Total revenue for the second quarter of 2023 was $44.3 million, as compared to $29.8 million for the second quarter of 2022, an increase of 49%.
Net loss for the second quarter of 2023 was $87.7 million, or $0.90 net loss per basic and diluted share, compared to a net loss of $72.6 million, or $0.78 net loss per basic and diluted share one year ago.
Research and development expenses for the second quarter of 2023 included a one-time, non-cash charge of acquired in-process research and development expenses of $24.6 million, or $0.25 impact to net loss per basic and diluted share, resulting from the recent exclusive license for FG-3246 from Fortis Therapeutics.
At June 30, 2023, cash – defined as cash, cash equivalents, investments, and accounts receivable – was $361.3 million, including proceeds received during the quarter from the Company’s use of its at-the-market equity facility and the closing of the recently announced term loan.
We expect our cash, cash equivalents, investments, and accounts receivable to be sufficient to fund our operating plans into 2026.
_______________________
1 Total roxadustat net sales in China includes sales made by the distribution entity as well as FibroGen China’s direct sales, each to its own distributors. The distribution entity jointly owned by AstraZeneca and FibroGen is not consolidated into FibroGen’s financial statements.

Conference Call and Webcast Details
FibroGen will host a conference call and webcast today, Monday, August 7, 2023, at 5:00 PM Eastern Time to discuss financial results and provide a business update. Interested parties may access a live audio webcast of the conference call via the "Investor Relations" page of the Company’s website at www.fibrogen.com. To access the call by phone, please go to this link (registration link), and you will be provided with dial in details. To avoid delays, we encourage participants to dial in to the conference call fifteen minutes ahead of the scheduled start time. A replay of the webcast will also be available for a limited time at the following link (webcast replay).

About Pamrevlumab
Pamrevlumab is a potential first-in-class antibody being developed by FibroGen to inhibit the activity of connective tissue growth factor (CTGF), a common factor in fibrotic and proliferative disorders characterized by persistent and excessive scarring that can lead to organ dysfunction and failure. Pamrevlumab is in Phase 3 clinical development for the treatment of locally advanced unresectable pancreatic cancer (LAPC), ambulatory Duchenne muscular dystrophy (DMD), and in Phase 2/3 for the treatment of metastatic pancreatic cancer. The U.S. Food and Drug Administration has granted Orphan Drug Designation, and Fast Track designation to pamrevlumab for the treatment of patients with DMD and LAPC. The U.S. Food and Drug Administration has also granted Rare Pediatric Disease Designation to pamrevlumab for the treatment of patients with DMD. Pamrevlumab has demonstrated a safety and tolerability profile that has supported ongoing clinical investigation in DMD, LAPC, and metastatic pancreatic cancer. Pamrevlumab is an investigational drug and not approved for marketing by any regulatory authority. For information about our pamrevlumab studies please visit www.clinicaltrials.gov.

About Roxadustat
Roxadustat, an oral medication, is the first in a new class of medicines comprising HIF-PH inhibitors that promote erythropoiesis, or red blood cell production, through increased endogenous production of erythropoietin, improved iron absorption and mobilization, and downregulation of hepcidin. Roxadustat is in clinical development for chemotherapy-induced anemia (CIA) in China.

Roxadustat is approved in China, Europe, Japan, and numerous other countries for the treatment of anemia of CKD in adult patients on dialysis (DD) and not on dialysis (NDD). Several other licensing applications for roxadustat have been submitted by partners, Astellas and AstraZeneca, to regulatory authorities across the globe, and are currently under review. Astellas and FibroGen are collaborating on the development and commercialization of roxadustat for the potential treatment of anemia in territories including Japan, Europe, Turkey, Russia and the Commonwealth of Independent States, the Middle East, and South Africa. FibroGen and AstraZeneca are collaborating on the development and commercialization of roxadustat for the potential treatment of anemia in the U.S., China, and other markets not licensed to Astellas.

On August 7, 2023 Day One Biopharmaceuticals (Nasdaq: DAWN) ("Day One" or the "Company"), a clinical-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, reported its second quarter 2023 financial results and highlighted recent corporate achievements (Press release, Day One, AUG 7, 2023, View Source [SID1234633871]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Day One had a remarkable second quarter, with the initiation of the rolling submission of the tovorafenib NDA, followed by an oral presentation at ASCO (Free ASCO Whitepaper) with updated data that we anticipate will support our regulatory application to the FDA," said Jeremy Bender, Ph.D., chief executive officer of Day One. "The majority of children with relapsed or progressive pLGG need new treatment options. With a strong balance sheet, we believe we are well positioned to achieve our key milestones while working towards expanding our pipeline with other innovative therapies."

Program Highlights

•
On June 4, 2023, Day One announced new clinical data from the registrational Phase 2 FIREFLY-1 trial evaluating the investigational agent tovorafenib in relapsed or progressive pLGG in an oral presentation at the 2023 ASCO (Free ASCO Whitepaper) Annual Meeting. These new data, with a data cutoff of December 22, 2022, included:

RANO-HGG (n=69) data:

•
67% (n=46) ORR by RANO-HGG, the primary endpoint of the trial
•
93% CBR (complete response (CR) + partial response (PR)/unconfirmed partial response (uPR) + stable disease (SD))
o
6% (n=4) CR
o
61% (n=42) PR, including 3 uPR
o
26% (n=18) SD
•
At the time of data cutoff, the median duration of response (DOR) based on RANO-HGG criteria was not yet reached (95% CI: 9.0 months, not estimable)
Page 1 of 6

Exhibit 99.1

Among a total of 77 treated patients:

•
The median duration of tovorafenib treatment was 10.8 months, with 74% (n=57) of patients on treatment at the time of data cutoff

Safety data, based on the 136 patients treated in both Arm 1 and Arm 2 of FIREFLY-1, indicated monotherapy tovorafenib to be generally well-tolerated. The vast majority of adverse events were Grade 1 or Grade 2, with most common side effects reported related to tovorafenib being change in hair color (71%), fatigue (50%), vomiting (43%), maculopapular rash (41%) and headache (39%). The most commonly reported lab abnormalities were CPK elevation, anemia, hypophosphatemia and AST elevation. Nearly all of the lab abnormalities had no clinical manifestations and did not require clinical intervention or change in study treatment.

The Company also shared the evaluation of responses by Response Assessment in Pediatric Neuro-Oncology Low-Grade Glioma (RAPNO-LGG) and Response Assessment Neuro-Oncology Low-Grade Glioma (RANO-LGG). Those results include:

*RAPNO-LGG data (n=69):

•
51% (n=35) ORR by RAPNO-LGG
o
25% (n=17) PR including 4 uPR
o
26% (n=18) minor response (MR) including 4 unconfirmed MR (uMR)
o
36% (n=25) patients with SD
•
The median time to response was 5.5 months for confirmed responses
•
At the time of data cutoff, Independent Review Committee (IRC)-assessed median DOR based on confirmed RAPNO-LGG responses is 12 months (95% CI: 11.2, not estimable)
*Pending adjudication

RANO-LGG (n=76) data:

•
49% (n=37) ORR by RANO-LGG
o
26% (n= 20) PR including 8 uPR
o
22% (n= 17) MR including 2 uMR
o
34% (n=26) patients with SD
•
The median time to response was 4.2 months for confirmed responses
•
At the time of data cutoff, the IRC-assessed median DOR based on confirmed RANO-LGG responses is 14.4 months (95% CI: 8.4, not estimable)

•
Two additional posters were presented on June 5, 2023 during the ASCO (Free ASCO Whitepaper) Pediatric Oncology session, including a trial-in-progress poster for the FIREFLY-2 trial and a poster describing a healthcare resource utilization study conducted for pLGG patients.

•
Day One presented two posters at the 2023 American Society of Pediatric Oncology/Hematology Conference on May 10, 2023, focused on the pLGG burden of illness and healthcare utilization data.

•
The pivotal Phase 3 FIREFLY-2/LOGGIC clinical trial evaluating tovorafenib as a front-line therapy in patients aged 6 months to 25 years with pLGG continues to enroll in the United States, Canada, Europe, Australia and Asia, with approximately 100 sites planned.
Page 2 of 6

Exhibit 99.1

•
Patient enrollment continues in the Phase 1b/2 FIRELIGHT-1 trials evaluating tovorafenib as a monotherapy and as a combination with the Company’s investigational MEK inhibitor, pimasertib, in adults and adolescents with relapsed, progressive, or refractory solid tumors harboring MAPK pathway aberrations.

Corporate Highlights and Upcoming Milestones

•
In June 2023, Day One announced the successful closing of a public offering including the full exercise of the underwriters’ option to purchase additional shares, raising gross proceeds of $172.5 million which strengthens the Company’s balance sheet and extends cash runway into 2026.

•
The Company anticipates completing the rolling submission of the tovorafenib NDA by October 2023, following submission of an amended clinical study report (CSR) that will include safety and efficacy data from a planned June 2023 data cutoff.

Second Quarter 2023 Financial Highlights

•
Cash Position: Cash, cash equivalents and short-term investments totaled $442.9 million on June 30, 2023. Based on Day One’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations into 2026.

•
R&D Expenses: Research and development expenses were $32.2 million for the second quarter of 2023 compared to $22.6 million for the second quarter of 2022. The increase was primarily due to additional employee compensation costs, as well as clinical trial and manufacturing activities related to Day One’s lead product candidate, tovorafenib.

•
G&A Expenses: General and administrative expenses were $17.1 million for the second quarter of 2023 compared to $14.2 million for the second quarter of 2022. The increase was primarily due to additional employee compensation costs, as well as the ongoing build-out of commercial capabilities.

•
Net Loss: Net loss totaled $45.9 million for the second quarter of 2023 with non-cash stock compensation expense of $9.5 million, compared to $36.5 million for the second quarter of 2022 with non-cash stock compensation expense of $5.6 million.

Upcoming Events

•
2023 Wedbush PacGrow Healthcare Conference, August 8-9, 2023

•
Morgan Stanley 21st Annual Global Healthcare Conference, September 11-13, 2023

Page 3 of 6

Exhibit 99.1

About Tovorafenib

Tovorafenib is an investigational, oral, brain-penetrant, highly-selective type II pan-RAF kinase inhibitor designed to target a key enzyme in the MAPK signaling pathway, which is being investigated in primary brain tumors or brain metastases of solid tumors. Tovorafenib has been studied in over 325 patients to date. Currently tovorafenib is under evaluation in a pivotal Phase 2 clinical trial (FIREFLY-1) among pediatric, adolescent and young adult patients with relapsed or progressive pLGG, which is an area of considerable unmet need with no approved therapies for the vast majority of patients. The pivotal Phase 3 FIREFLY-2/LOGGIC clinical trial is evaluating tovorafenib as a front-line therapy versus standard of care chemotherapy. Tovorafenib is also being evaluated alone or as a combination therapy for adolescent and adult patient populations with relapsed or progressive solid tumors with MAPK pathway aberrations (FIRELIGHT-1).

Tovorafenib has been granted Breakthrough Therapy and Rare Pediatric Disease designations by the U.S. Food and Drug Administration (FDA) for the treatment of patients with pLGG harboring an activating RAF alteration. Tovorafenib has also received Orphan Drug designation from the FDA for the treatment of malignant glioma, and from the European Commission for the treatment of glioma.