On October 28, 2021 Diffusion Pharmaceuticals Inc. (NASDAQ: DFFN) ("Diffusion" or the "Company"), an innovative biopharmaceutical company developing novel therapies that enhance the body’s ability to deliver oxygen to areas where it is needed most, reported that it will participate in the 2021 Alliance Global Partners Biotech & Specialty Pharma Conference taking place November 10, 2021. Diffusion will also be participating in one-on-one meetings (Press release, Diffusion Pharmaceuticals, OCT 28, 2021, View Source [SID1234592095]).

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Interested investors can schedule meetings with management from 8:30 a.m. to 5:00 p.m. on November 10. Those who wish to request a meeting with Diffusion should contact their Alliance Global Partners salesperson.

On October 28, 2021 Cellectis S.A. (NASDAQ: CLLS – EURONEXT GROWTH: ALCLS) (the "Company"), a gene-editing platform company with clinical-stage immuno-oncology programs using allogeneic chimeric antigen receptor (CAR)-T cells and gene therapy programs for genetic diseases, reported that it will report its financial results for the third quarter and first nine-month period ending September 30, 2021, on Thursday, November 4, 2021, after the close of the US market (Press release, Cellectis, OCT 28, 2021, View Source [SID1234592094]). The announcement will be followed by a conference call at 8:00 AM EDT / 2:00 PM CET on Friday, November 5, 2021, prior to the open of the US market.

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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

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The live dial-in information for the conference call is:

In addition, a replay of the call will be available until November 19th, by calling +1 877-660-6853 (Toll Free US & Canada); +1 201-612-7415 (Toll Free International).

On October 28, 2021 Cardinal Health (NYSE: CAH) reported it has been awarded a $750,000 contract by the U.S. Food and Drug Administration (FDA) to implement an 18-month real world evidence (RWE) study as part of the agency’s efforts to advance the applicability of RWE in regulatory decision-making (Press release, Cardinal Health, OCT 28, 2021, View Source [SID1234592093]).

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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

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The funding, which will support the project "Assessment of a Novel Methodology for Endpoints Assessing Response to Lymphoma Treatment in Real-World Studies," will evaluate the accuracy of real-world data (RWD) for lymphoma tumor response compared to blinded independent central review, the gold standard in randomized clinical trials. Within this research, Cardinal Health will work with the FDA Oncology Center of Excellence to assess tumor response in the clinical care setting.

"The utilization of real world evidence can ultimately lead to improving the patient experience by providing quicker access to safer therapies, but only if the potential is fully realized by bridging the measures used in clinical trials to those observed in real world patients," said Eli Phillips, Jr., PharmD, JD, vice president of Regulatory Sciences and Insights & Engagement, Cardinal Health. "I’m proud that Cardinal Health’s commitment to advancing patient care through the use of real world evidence for regulatory objectives has been recognized by the FDA with this award to further our research."

Since Congress passed the 21st Century Cures Act in 2016, the FDA has placed increased focus on evaluating opportunities to use RWD to support regulatory decision making, including supporting the expansion of indications for currently approved drugs. However, despite potential utility of RWD usage, the field currently lacks standardization in tumor response assessment that provide for reproducibility of results. This research could provide important insights to inform RWD study designs or study methodology for such a purpose.

On October 28, 2021 Blueprint Medicines Corporation (NASDAQ:BPMC) reported financial results and provided a business update for the third quarter ended September 30, 2021 (Press release, Blueprint Medicines, OCT 28, 2021, View Source [SID1234592092]).

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"We successfully executed across our entire portfolio in the third quarter, expanding the commercial adoption of AYVAKIT and GAVRETO and further advancing our next wave of therapeutic candidates toward potentially transformative proof-of-concept datasets," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "We are particularly encouraged by the launch momentum for AYVAKIT in advanced systemic mastocytosis, where we see broad prescriber demand across both academic and community centers and in patients regardless of prior therapy. In addition, we are pleased by the progress across our clinical portfolio, with topline data from the PIONEER trial of AYVAKIT in non-advanced systemic mastocytosis expected in mid-2022, strong patient interest fueling enrollment in the ongoing SYMPHONY trial of our EGFR inhibitor BLU-945, and multiple trial initiations on-track for the months ahead. Overall, this commercial and clinical progress across our portfolio, combined with the strength of our financial position and diversity of revenue, provides a robust foundation for growth and continuous innovation."

Third Quarter 2021 Highlights and Recent Progress

AYVAKIT/AYVAKYT (avapritinib): systemic mastocytosis (SM) and gastrointestinal stromal tumor (GIST)

Recorded $17.3 million in net product revenue during the third quarter of 2021, an increase of 104 percent over the second quarter, for AYVAKIT/AYVAKYT.
GAVRETO (pralsetinib): RET-altered cancers

Transferred responsibilities associated with the booking of U.S. product sales of GAVRETO to our collaboration partner Roche on July 1, 2021 and began to recognize the company’s share of U.S. revenue in the GAVRETO profit and loss. As previously reported by Roche, net end user product revenues for GAVRETO were $5.5 million.
Received, via our collaboration with Roche, a positive opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for GAVRETO for the treatment of adults with RET fusion-positive advanced non-small cell lung cancer (NSCLC). If approved, GAVRETO will be the first and only targeted treatment approved by the EMA that includes first-line treatment of people with RET fusion-positive advanced NSCLC.
Key Upcoming Milestones

The company expects to achieve the following near-term milestones:

Initiate a Phase 1 trial of BLU-701 in patients with EGFR-driven NSCLC in the fourth quarter of 2021.
Present preclinical data supporting combination of BLU-945 and BLU-701 in EGFR-driven NSCLC at a medical conference in early 2022.
Initiate a Phase 1 trial of BLU-222, a CDK2 inhibitor targeting cyclin-E aberrant cancers, in the first quarter of 2022.
Disclose topline data for the registration-enabling Part 2 of the PIONEER trial of AYVAKIT in non-advanced systemic mastocytosis in mid-2022.
Initiate a Phase 1 trial of BLU-852, a MAP4K1 inhibitor for the treatment of advanced cancers, in 2022. BLU-852 is the first development candidate nominated under Blueprint Medicines’ cancer immunotherapy collaboration with Roche.
Third Quarter 2021 Financial Results

Revenues: Revenues were $24.2 million for the third quarter of 2021, including $17.3 million of net product revenues from sales of AYVAKIT/AYVAKYT and $6.9 million in collaboration revenues. Blueprint recorded revenues of $745.1 million in the third quarter of 2020, including $6.1 million of net product revenues from sales of AYVAKIT, $0.2 million of net product revenues from sales of GAVRETO and $738.8 million in collaboration revenues.
Cost of Sales: Cost of sales was $3.8 million for the third quarter of 2021, as compared to $0.1 million for the third quarter of 2020. Cost of sales included manufacturing costs associated with our product sales as well as costs associated with the sale of drug product to our collaboration partners. The increase in cost of product sales was primarily driven by lower margin product sales to our collaboration partners during the third quarter of 2021.
Collaboration Loss Sharing: Collaboration loss sharing was $3.3 million for the third quarter of 2021 under our collaboration with Roche for GAVRETO.
R&D Expenses: Research and development expenses were $84.4 million for the third quarter of 2021, as compared to $74.2 million for the third quarter of 2020. This increase was primarily due to a decrease in reimbursement from the global development cost sharing arrangement under our collaboration with Roche for GAVRETO and increased costs related to early discovery efforts. Research and development expenses included $10.3 million in stock-based compensation expenses for the third quarter of 2021.
SG&A Expenses: Selling, general and administrative expenses were $49.8 million for the third quarter of 2021, as compared to $37.4 million for the third quarter of 2020. This increase was primarily due to increased costs associated with expanding our commercial infrastructure for commercialization of AYVAKIT/AYVAKYT and GAVRETO, partially offset by reimbursement under our collaboration with Roche for GAVRETO. General and administrative expenses included $13.7 million in stock-based compensation expenses for the third quarter of 2021.
Net Income (Loss): Net loss was $117.2 million for the third quarter of 2021, or a net loss per share of $2.00, as compared to a net income of $634.0 million for the third quarter of 2020, or a diluted net income per share of $11.16.
Cash Position: As of September 30, 2021, cash, cash equivalents and investments were $1,293.8 million, as compared to $1,549.7 million as of December 31, 2020.
Financial Guidance

Based on product revenue growth and strong collaboration execution with multiple milestone payments anticipated during the fourth quarter of 2021, the company expects to report full year 2021 total revenues in the range of $170-$180 million.

Conference Call Information

Blueprint Medicines will host a live conference call and webcast at 8:30 a.m. ET today to discuss third quarter 2021 financial results and recent business activities. The conference call may be accessed by dialing 844-200-6205 (domestic) or 929-526-1599 (international) and referring to conference ID 963004. A webcast of the call will be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.

On October 28, 2021 Rafael Pharmaceuticals, Inc. ("Rafael" or the "Company"), a company focused on the growing field of cancer metabolism-based therapeutics, reported that the AVENGER 500 Phase 3 clinical trial evaluating the efficacy and safety of CPI-613 (devimistat) in combination with modified FOLFIRINOX (mFFX) as a first-line therapy in patients with metastatic adenocarcinoma of the pancreas did not meet its primary endpoint of overall survival (Press release, Rafael Pharmaceuticals, OCT 28, 2021, View Source [SID1234592091]).

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In this multi-national Phase 3 randomized clinical trial, 528 patients with metastatic adenocarcinoma of the pancreas who had no prior therapy were randomized to receive either devimistat in combination with modified FOLFIRINOX (mFFX) or FOLFIRINOX, a current standard of care chemotherapy regimen. Devimistat given with mFFX did not significantly improve overall survival (HR=0.95, p=0.66). The median overall survival in the devimistat and mFFX arm was 11.1 months, compared to 11.7 months in the FOLFIRINOX arm.

"These cancers are incredibly difficult to treat, with few to no effective treatments available, but Rafael took the risk because we will always fight for our patients," said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. "While we are disappointed by the outcomes of these well-designed and well-executed studies, we remain committed to furthering our research and development in cancer metabolism for the treatment of hard-to-treat cancers, as our other studies continue. I personally want to express my heartfelt appreciation to the patients, their loved ones, the researchers and principal investigators for their trust and support. I am also incredibly grateful to my team, who works tirelessly for the patients we treat."

"Pancreatic cancer carries a high mortality rate and is extremely difficult to treat, but promising earlier clinical data encouraged us to move into this advanced phase trial with devimistat. While these are not the results we all hoped for, we will not give up, and we are hopeful that devimistat with its novel mechanism of action will demonstrate efficacy in other studies," said Philip Philip, M.D., Ph.D., F.R.C.P, principal investigator and medical oncologist at the Karmanos Cancer Institute at Wayne State University. "We are working diligently to further analyze the data and determine the best plan of action to further assess the drug’s capabilities in the clinic."

Devimistat is also being evaluated in a Phase 3 study, ARMADA 2000, in patients with relapsed or refractory acute myeloid leukemia (AML). Following a prespecified interim analysis, the independent data monitoring committee has recommended that the trial be stopped due to lack of efficacy.

"Earlier clinical trial data demonstrated positive results, despite the challenges we face in treating AML," said Jorge Cortes, M.D., director of the Georgia Cancer Center at Augusta University. "We were all hoping for positive results, and while this trial did not meet our expectations, we will continue to test devimistat in other studies."

The company will work with investigators to complete a full evaluation of the data on both trials.

About CPI-613 (Devimistat)
CPI-613 (devimistat) is a first-in-class clinical lead compound of Rafael, which targets enzymes that are involved in cancer cell energy metabolism and are located in the mitochondria of cancer cells. Devimistat is designed to target the mitochondrial tricarboxylic acid (TCA) cycle, a process essential to tumor cell multiplication and survival, selectively in cancer cells. Devimistat substantially increases cellular stress and the sensitivity of cancer cells to a diverse range of chemotherapeutic agents. This synergy allows for potential combinations of devimistat with lower doses of these generally toxic drugs to be more effective with lower patient side effects. Combination with devimistat represents a diverse range of opportunities to substantially improve patients’ benefit in many different cancers. The U.S. Food and Drug Administration (FDA) has given Rafael approval to initiate pivotal Phase 3 clinical trials in pancreatic cancer (AVENGER 500) and acute myeloid leukemia (ARMADA 2000) and has designated devimistat as an orphan drug for the treatment of pancreatic cancer, acute myeloid leukemia, myelodysplastic syndrome, peripheral T-cell lymphoma, soft tissue sarcoma, Burkitt’s lymphoma and biliary tract cancer. The European Medicines Agency (EMA) has granted orphan drug designation to devimistat for pancreatic cancer, acute myeloid leukemia and Burkitt’s lymphoma.