On March 1, 2022 Bold Therapeutics, a clinical-stage biopharmaceutical company, reported that they have successfully completed the Phase 1b (dose-escalation) portion of its seamless adaptive oncology trial of BOLD-100 in combination with FOLFOX in the treatment of advanced gastrointestinal cancers (colorectal, pancreatic, gastric and bile duct) (Press release, Bold Therapeutics, MAR 1, 2022, View Source [SID1234609309]).

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Bold Therapeutics Successfully Completes Phase 1b Trial and Advances into Global Phase 2 Trial of BOLD-100 in the Treatment of Advanced GI Cancers
Bold Therapeutics Successfully Completes Phase 1b Trial and Advances into Global Phase 2 Trial of BOLD-100 in the Treatment of Advanced GI Cancers
BOLD-100 is a first-in-class ruthenium-based small molecule therapeutic that (1) alters the unfolded protein response (UPR) through selective GRP78 inhibition; and (2) induces reactive oxygen species (ROS) which causes DNA damage and cell cycle arrest. Collectively, these effects result in cell death in both sensitive and resistant solid and liquid cancers and in combination with a wide range of existing anti-cancer therapies. The FDA previously granted BOLD-100 Orphan Drug Designations (ODDs) in both Gastric and Pancreatic cancers, and Bold Therapeutics anticipates leveraging clinical data from the Phase 1b portion of its trial for one or more Breakthrough Therapy Designations (BTDs) in 2022.

The Phase 1b data – which Bold Therapeutics anticipates presenting at an upcoming cancer conference – indicate that (1) BOLD-100 can be safely combined with FOLFOX chemotherapy at a dose of 625 mg / m2 (the highest dose level tested), with no new Grade 3 or 4 treatment-emergent adverse events; and (2) patients can safely remain on treatment for an extended number of treatment cycles. Based on this strong safety and tolerability profile, the Study Steering Committee unanimously endorsed proceeding immediately into the Phase 2 (dose-expansion) portion of the study.

"Completing our Phase 1b trial with a strong safety profile for BOLD-100 is a significant achievement for Bold Therapeutics – and while it is too early to say anything definitive, preliminary efficacy data is undoubtedly encouraging," said Jim Pankovich, EVP, Clinical Development. "Despite substantial headwinds from the ongoing COVID-19 pandemic, we were nevertheless able to successfully enroll and treat patients at our six clinical sites in Canada, and I wish to recognize the patients for their contribution to this study as well as the persistent and resilient efforts of our investigators."

The Phase 2 (dose-expansion) portion of the seamless adaptive trial of BOLD-100 will enroll 80 additional patients at 13 investigational sites worldwide: 6 sites in Canada; 2 sites in the U.S.; and 5 sites in South Korea. Interim and complete Phase 2 data is expected by year-end 2022 and late 2023, respectively.

"As BOLD-100 advances into Phase 2, Bold Therapeutics crosses another significant value inflection point," stated Glenn Walthall, Chairman of the Board of Bold Therapeutics. "As Bold Therapeutics’ largest institutional investor, we are encouraged with the results that we’ve seen thus far and optimistic that BOLD-100 may significantly improve outcomes in these difficult-to-treat cancers that are often refractory to conventional treatment options. Consistent with preclinical observations, a number of patients in the study who had previously failed on FOLFOX alone suddenly responded when BOLD-100 was added to the treatment regimen – a result that can only be described as remarkable."

Bold Therapeutics executed a regional option agreement with an undisclosed biopharmaceutical company in South Korea in 2020 and is actively seeking development partners in other territories. Bold Therapeutics is also seeking investors for a data-driven institutional Series B round to be closed later in 2022, likely concurrent with interim Phase 2 results.

"I am exceptionally proud of the agile and industrious Bold Therapeutics team without whom this success would not be possible," added E. Russell McAllister, CEO of Bold Therapeutics. "Through innovative programs like NRC-IRAP, the Canadian government has provided Bold Therapeutics with substantial support in advancing our scientific understanding of BOLD-100 that not only allowed us to advance in the treatment of gastrointestinal cancer indications, but also opened up promising areas for future development. As a result, they share this win with us. The strong results from this Phase 1b trial and the overall accumulation of data on BOLD-100 continue to excite support for the development of this innovative therapeutic for patients with a wide range of advanced cancers."

On March 1, 2022 Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) reported that management will present at Cowen’s 42nd Annual Health Care Conference on Tuesday, March 8, 2022 at 2:50 p.m. ET (Press release, Vertex Pharmaceuticals, MAR 1, 2022, https://investors.vrtx.com/news-releases/news-release-details/vertex-present-cowens-42nd-annual-health-care-conference-march-8 [SID1234609395]).

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A live webcast of management’s remarks will be available through Vertex’s website, www.vrtx.com in the "Investors" section under the "News and Events" page. A replay of the conference webcast will be archived on the company’s website.

On March 1, 2022 Forma Therapeutics Holdings, Inc.(Nasdaq: FMTX), a clinical-stage biopharmaceutical company focused on sickle cell disease, prostate cancer and other rare hematologic diseases and cancers, reported financial results for the year ended December 31, 2021 (Press release, Forma Therapeutics, MAR 1, 2022, View Source [SID1234609220]). The company also highlighted recent progress and upcoming milestones for its pipeline programs.

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"2021 was a year of continued growth for Forma as we completed our comprehensive Phase I etavopivat trial, and further advanced the importance of assessing red blood cell health," said Frank Lee, president and chief executive officer of Forma. "2022 represents a year of expansion for Forma, expanding the breadth of the etavopivat development plan into new indications, proof of concept readout for FT-7051 in metastatic prostate cancer, and a new development candidate emerging from our research pipeline."

Key Business and Clinical Highlights

Pyruvate Kinase-R (PKR) Program in Sickle Cell Disease (SCD):

•Comprehensive etavopivat Phase I trial completed. Open label extension (OLE) results for 15 patients administered etavopivat 400 mg once daily for up to 12 weeks with a data cutoff as of November 23, 2021 were presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December 2021. Improvements were observed in measures of hematologic and hemolytic response, and biomarkers of red blood cell (RBC) health, including oxygenation and deformability, as well as systemic biomarkers of SCD. Etavopivat administered for up to 12 weeks reduced anemia by significantly raising and sustaining hemoglobin levels and also significantly increased the lifespan of RBCs with decreased hemolysis. In addition, an analysis of all patients in the 12-week open label cohort showed a decreasing trend in vaso-occlusive crises (VOCs) requiring hospitalization when compared to the rate 12 months prior to trial entry. Etavopivat was well tolerated in the trial and safety was consistent with underlying SCD.
CBP/p300 Program in Metastatic Castrate Resistant Prostate Cancer (mCRPC):

•FT-7051 well tolerated with signs of clinical activity in initial Phase I clinical trial results. Initial results from eight men in the trial were presented at the NCI/AACR/EORTC Virtual AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) in October 2021. Pharmacokinetic (PK) analysis of FT-7051 documented rapid absorption, with drug concentrations that approached the predicted efficacious dose based on estimates from preclinical models. In addition, skin biopsies demonstrated a reduction in a marker of activity in the CBP/p300 pathway. The majority of treatment-emergent adverse events (TEAEs) observed were mild or moderate with no events leading to treatment discontinuation. The first evaluable patient completing 12 weeks of treatment demonstrated a >80% decline in prostate-specific antigen (PSA80) from baseline at 16 weeks with stable disease.
IDH1 Program in Acute Myeloid Leukemia (AML)

•First Phase II results of olutasidenib used in combination with a chemotherapy were presented at the ASH (Free ASH Whitepaper) annual meeting in December 2021. The trial included patients who had not yet received therapy and were candidates for azacitidine as a first-line treatment, and also patients with relapsed/refractory (R/R) AML that had prior therapy with a hypomethylating agent (HMA) or an IDH1 inhibitor. The results support the potential of olutasidenib as the basis of combination therapy in patients with AML who have not achieved a durable response from prior therapy. Olutasidenib was well tolerated in the trial in combination with azacitidine and the combination had a safety profile largely consistent with that of olutasidenib alone. Forma is progressing a new drug application (NDA) for the treatment of R/R AML.
Corporate

•Appointed Ifeyinwa (Ify) Osunkwo, MD, MPH, as the company’s inaugural chief patient officer and senior vice president. Dr. Osunkwo will be responsible for realizing Forma’s vision to transform the lives of patients, including improving access and care through partnerships with global patient and community stakeholders.
•Launched formabridge and grants program. Through formabridge grants, Forma has committed $1 million in funding for promising and innovative initiatives that address unmet needs in transition from pediatric to adult care in SCD.
•Upcoming investor conference participation. Forma will participate in the Oppenheimer Healthcare Conference taking place March 15-16, 2022. The presentation webcast will be available in the "News & Investors" section of Forma’s website at www.FormaTherapeutics.com.
•Virtual research and development (R&D) review to be held in May, 2022. The company will provide an overview of its internal research pipeline strategy and review compounds in clinical and pre-clinical development. The live webcast will be available in the "News & Investors" section of Forma’s website www.FormaTherapeutics.com.
Upcoming Milestones

•Patient enrollment in global pivotal Phase II/III trial of etavopivat for the treatment of SCD, the Hibiscus Study. The first interim analysis (IA1) in the Hibiscus Study is expected to be reached by the end of 2022, with dose selection for the Phase III portion of the trial.
•Etavopivat development plans expanding. Forma began a Phase II trial in transfusion dependent SCD and both transfusion dependent and non-transfusion dependent thalassemia in late 2021, with initial results expected in late 2022. During 2022, Forma plans to begin clinical trials in pediatric SCD and low risk myelodysplastic syndrome (MDS).

•Additional FT-7051 clinical trial results in mCRPC. Men with mCRPC continue to be enrolled in the dose escalation portion of the Phase I trial. Forma plans to present updated results from the trial in mid-2022.
•Possibility of COVID-19 impact remains. The COVID-19 pandemic remains a factor in the successful completion of these milestones and ongoing clinical trials. Many clinical trials across the biopharma industry, including Forma’s, have been impacted by the COVID-19 pandemic. Clinical trial sites implementing new policies in response to COVID-19 have impacted enrollment of clinical trials or and the ability to access sites participating in clinical trials.
Financial Results

•Cash Position: Cash, cash equivalents and marketable securities were $490.3 million as of December 31, 2021, as compared to $645.6 million as of December 31, 2020. Current cash runway is projected through the third quarter of 2024.
•R&D Expenses: R&D expenses were $37.0 million and $125.7 million for the quarter and year ended December 31, 2021, compared to $24.9 million and $93.4 million for the quarter and year ended December 31, 2020. The increase was primarily attributable to the conduct of etavopivat Phase II/III and Phase I trials in SCD patients, as well as start-up costs related to the thalassemia trial, manufacturing activities, and increases in research and development staff, equity-based compensation, and investment in preclinical programs.
•General and Administrative (G&A) Expenses: G&A expenses were $13.2 million and $48.3 million for the quarter and year ended December 31, 2021, compared to $7.9 million and $30.8 million for the quarter and year ended December 31, 2020. The increase was primarily attributable to increases in equity-based compensation, personnel-related costs related to executive and staff hiring, professional fees, and insurance related expenses.
•Net Loss: Net loss was $50.1 million and $173.0 million for the quarter and year ended December 31, 2021, compared to net loss of $28.6 million and $70.4 million for the quarter and year ended December 31, 2020.

Forma will conduct a conference call and webcast March 1, 2022 at 8:00 a.m. Eastern Daylight Time (EDT) to discuss year end 2021 results and business updates. The call can be accessed by dialing (833) 301-1146 in the U.S., and (914) 987-7386 internationally, with conference ID 3322907.

The live webcast will be available in the "News & Investors" section of Forma’s website www.FormaTherapeutics.com.

On March 1, 2022 Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for cancer and autoimmune and inflammatory diseases, reported that members of its management team will participate at the following investor conferences in March 2022 (Press release, Alpine Immune Sciences, MAR 1, 2022, View Source [SID1234609250]):

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Cowen’s 42nd Annual Health Care Conference
Date: Monday, March 7, 2022
Time: 2:50 p.m. ET/11:50 a.m. PT
Event: Fireside Chat

Oppenheimer 32nd Annual Healthcare Conference
Date: Thursday, March 17, 2022
Time: 2:40 p.m. ET/11:40 a.m. PT
Event: Fireside Chat

Webcasts of the Cowen and Oppenheimer fireside chats will be available online in the investor relations section of the company’s website at View Source A replay of the fireside chats will be available on the company website for 90 days following the webcast.

On March 1, 2022 Revolution Medicines, Inc. (Nasdaq: RVMD), a clinical-stage oncology company developing novel targeted therapies for RAS-addicted cancers, reported that the company will participate in the Cowen 42nd Annual Health Care Conference(Press release, Revolution Medicines, MAR 1, 2022, View Source [SID1234609276]). Mark A. Goldsmith, M.D., Ph.D., chief executive officer and chairman of Revolution Medicines, will be the featured participant in a fireside chat at the event .

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Details of the company’s participation are as follows:

Cowen 42nd Annual Health Care Conference
Conference Date: March 7-9, 2022
Fireside Chat Time/Date: 11:10 a.m. Pacific on Monday, March 7, 2022
Format: Virtual conference; webcast available
To access the live webcast of the fireside chat, please visit the "Events & Presentations" page of Revolution Medicines’ website at View Source Additionally, a replay of the webcast will be available on the "Events & Presentations" page of the Revolution Medicines website for at least 14 days following the conference.