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Verastem Oncology Reports Third Quarter 2020 Financial Results and Highlights Recent Company Progress

On November 6, 2020 Verastem, Inc. (Nasdaq:VSTM) (also known as Verastem Oncology), a biopharmaceutical company committed to advancing new medicines for patients battling cancer, reported financial results for the three months ending September 30, 2020, and provided an overview of recent corporate highlights (Press release, Verastem, NOV 6, 2020, View Source [SID1234570541]).

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"The third quarter of 2020 was marked most notably by the sale of the COPIKTRA (duvelisib) franchise to Secura Bio in a deal valued at up to $311 million, plus royalties. This strategic transaction allows us to focus our resources and efforts on advancing the VS-6766 and defactinib combination program in KRAS mutant solid tumors and provides us with a cash runway until at least 2024," said Brian Stuglik, Chief Executive Officer of Verastem Oncology. "Looking ahead to the remainder of the year, we remain on track to commence two new company-sponsored, registration-directed Phase 2 clinical trials by year end, one in low-grade serous ovarian cancer (LGSOC) and one in KRAS mutant non-small cell lung cancer (NSCLC)."

Third Quarter 2020 and Recent Highlights

Presented Updated Data from the Phase 1/2 FRAME Study in Patients with LGSOC. In mid-September, Verastem reported positive updated results from the ongoing investigator-initiated Phase 1/2 FRAME study coinciding with a virtual oral presentation by Dr. Udai Banerji, Institute of Cancer Research and The Royal Marsden, at the 2nd Annual RAS-Targeted Drug Development (RTDD) Summit. The FRAME study is evaluating VS-6766, Verastem’s RAF/MEK inhibitor, in combination with defactinib, its FAK inhibitor, in patients with LGSOC. The results demonstrated that the novel, intermittent, combination dosing schedule used in the FRAME study continues to show encouraging clinical activity, durability and a favorable safety profile in patients with KRAS mutant LGSOC, including patients who had previously progressed following treatment with a MEK inhibitor.

New Data Published in The Lancet Oncology Supports Potential of VS-6766. An investigator-initiated Phase 1 study evaluating the intermittent dosing schedule of VS-6766 was published in the November issue of The Lancet Oncology. Tolerability and antitumor activity were observed across various cancers with RAS/RAF/MEK pathway mutations. The dose escalation study was the first to evaluate a dual RAF/MEK inhibitor using innovative intermittent dosing schedules in patients harboring RAS/RAF pathway mutations.

On Track to Commence Phase 2 Registration-Directed Trials in Lead Indications This Year. Following a meeting with the U.S. Food and Drug Administration (FDA), Verastem reported that the FDA is supportive of its adaptive study design for the planned Phase 2 registration-directed trial evaluating VS-6766 and defactinib in patients with recurrent LGSOC. Verastem expects to commence registration-directed clinical trials in both recurrent LGSOC and KRAS mutant non-small cell lung cancer by the end of 2020. Assuming a positive outcome from these registration-directed trials, Verastem expects to submit New Drug Applications to the FDA requesting accelerated approval for VS-6766 alone or in combination with defactinib in both LGSOC and KRAS mutant NSCLC.

Closed COPIKTRA Sale to Secura Bio in a Deal Totaling $311 Million, Plus Royalties. Verastem recently announced the closing of a strategic transaction selling global commercial and development rights to COPIKTRA in all oncology indications to Secura Bio, Inc. The transaction, which carries a total deal value of up to $311 million, plus royalties, provides Verastem with a cash runway until at least 2024 and will allow the Company to focus its resources and efforts on the clinical development of VS-6766 and defactinib in KRAS mutant solid tumors.

Presented New Preclinical Research Demonstrating Synergy and Tumor Regression with VS-6766 in Combination with G12C Inhibitors. In a virtual poster presentation, also at the RTDD Summit, Verastem highlighted new preclinical researchwhere VS-6766 showed synergy with KRAS-G12C inhibitors in reducing cancer cell viability across a panel of KRAS-G12C mutant NSCLC and colorectal cancer (CRC) cell lines. This enhanced cellular anti-cancer activity of the combination correlated with deeper and more durable inhibition of ERK pathway signaling compared to G12C inhibition alone. The anti-tumor effects of VS-6766 were stronger than the effects of trametinib at a comparable dose.
Third Quarter 2020 Financial Results

Total Revenue for the three months ending September 30, 2020 (2020 Quarter) was $78.6 million, compared to $9.0 million for the three months ending September 30, 2019 (2019 Quarter).

Sale of COPIKTRA license and related assets revenue for the 2020 Quarter was $70.0 million, compared to $0.0 for the 2019 Quarter. The 2020 Quarter was comprised of a $70.0 million upfront payment recognized as part of the COPIKTRA sale to Secura Bio Inc.

License and collaboration revenue for the 2020 Quarter was $2.8 million, compared to $5.0 million for the 2019 Quarter. The 2019 Quarter included a $5.0 million upfront payment received pursuant to a license and collaboration agreement executed between Verastem Oncology and Sanofi in July 2019. The 2020 Quarter was primarily comprised of $2.5 million for Sanofi achieving two development milestones under the license and collaboration agreement.

Net product revenue for the 2020 Quarter was $5.8 million, compared to $4.0 million for the 2019 Quarter.

Cost of sales as a result of the sale of COPIKTRA license and related assets for the 2020 Quarter was $31.2 million, compared to $0.0 million for the 2019 Quarter. The 2020 Quarter comprised of the intangible asset, certain duvelisib inventory, net duvelisib contract prepaid balances and certain manufacturing equipment for the amounts of $19.2 million, $6.0 million, $5.8 million, and $0.2 million, respectively, delivered to Secura Bio Inc. as part of the COPIKTRA sale.

Total research and development (R&D) and selling, general and administrative (SG&A) expenses for the 2020 Quarter were $31.6 million, compared to $34.4 million for the 2019 Quarter.

R&D expense for the 2020 Quarter was $11.0 million, compared to $12.2 million for the 2019 Quarter. The decrease of $1.2 million, or 10%, was primarily related to a decrease in contract research organization costs and lower employee related expense.

SG&A expense for the 2020 Quarter was $20.6 million, compared to $22.2 million for the 2019 Quarter. The decrease of $1.6 million, or 7%, primarily resulted from the company’s shift in strategic direction which led to lower commercial program and employee related expense. The 2020 Quarter includes $3.5 million of nonrecurring transaction expenses directly attributable to the COPIKTRA sale to Secura Bio Inc.

Net income (loss) for the 2020 Quarter was $13.1 million, or $0.08 per share (basic and diluted), compared to $(30.1) million, or $(0.41) per share (basic and diluted), for the 2019 Quarter.

For the 2020 Quarter, non-GAAP adjusted net income was $18.8 million, or $0.11 per share (diluted), compared to non-GAAP adjusted net loss of $26.2 million, or $0.35 per share (diluted), for the 2019 Quarter. Please refer to the GAAP to Non-GAAP Reconciliation attached to this press release.

Verastem Oncology ended the third quarter of 2020 with cash, cash equivalents and short-term investments of $205.7 million.

Financial Guidance and Outlook

With the proceeds from the sale of COPIKTRA, Verastem has a cash runway until at least 2024 to deliver on the current programs for VS-6766 and defactinib, including clinical and regulatory milestones and development in LGSOC and KRAS mutant NSCLC. Verastem expects its 2020 operating expenses to be approximately 40% lower than its 2019 operating expenses. As a result of its new strategic direction and operating plans, along with the sale of the COPIKTRA franchise during the third quarter 2020 and associated transition activities, the Company expects total operating expenses for the full year 2020 to be in the range of $80 million to $90 million. Beginning in 2021 Verastem expects its annual operating expenses to be approximately $50 million.

Use of Non-GAAP Financial Measures

To supplement Verastem Oncology’s condensed consolidated financial statements, which are prepared and presented in accordance with generally accepted accounting principles in the United States (GAAP), the Company uses the following non-GAAP financial measures in this press release: non-GAAP adjusted net loss and non-GAAP net loss per share. These non-GAAP financial measures exclude certain amounts or expenses from the corresponding financial measures determined in accordance with GAAP. Management believes this non-GAAP information is useful for investors, taken in conjunction with the Company’s GAAP financial statements, because it provides greater transparency and period-over-period comparability with respect to the Company’s operating performance and can enhance investors’ ability to identify operating trends in the Company’s business. Management uses these measures, among other factors, to assess and analyze operational results and trends and to make financial and operational decisions. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of the Company’s operating results as reported under GAAP, not in isolation or as a substitute for, or superior to, financial information prepared and presented in accordance with GAAP. In addition, these non-GAAP financial measures are unlikely to be comparable with non-GAAP information provided by other companies. The determination of the amounts that are excluded from non-GAAP financial measures is a matter of management judgment and depends upon, among other factors, the nature of the underlying expense or income amounts. Reconciliations between these non-GAAP financial measures and the most comparable GAAP financial measures for the three months ended March 31, 2020 and 2019 are included in the tables accompanying this press release after the unaudited condensed consolidated financial statements.

About VS-6766

VS-6766 (formerly known as CH5126766, CKI27 and RO5126766) is a unique inhibitor of the RAF/MEK signaling pathway. In contrast to other MEK inhibitors in development, VS-6766 blocks both MEK kinase activity and the ability of RAF to phosphorylate MEK. This unique mechanism allows VS-6766 to block MEK signaling without the compensatory activation of MEK that appears to limit the efficacy of other inhibitors.

About Defactinib

Defactinib (VS-6063) is an oral small molecule inhibitor of FAK and PYK2 that is currently being evaluated as a potential combination therapy for various solid tumors. The Company has received Orphan Drug designation for defactinib in ovarian cancer and mesothelioma in the US, EU and Australia. Preclinical research by Verastem Oncology scientists and collaborators at world-renowned research institutions has described the effect of FAK inhibition to enhance immune response by decreasing immuno-suppressive cells, increasing cytotoxic T cells, and reducing stromal density, which allows tumor-killing immune cells to enter the tumor.i,ii

About the VS-6766/Defactinib Combination

RAS mutant tumors are present in ~30% of all human cancers, have historically presented a difficult treatment challenge and are often associated with significantly worse prognosis. Challenges associated with identifying new treatment options for these types of cancers include resistance to single agents, identifying tolerable combination regimens with MEK inhibitors and new RAS inhibitors in development addressing only a minority of all RAS mutated cancers.

The combination of VS-6766 and defactinib has been found to be clinically active in patients with KRAS mt tumors. In an ongoing investigator-initiated Phase 1/2 FRAME study, the combination of VS-6766 and defactinib is being evaluated in patients with LGSOC, KRASmt NSCLC and colorectal cancer (CRC). Updated interim data from this study presented at the 2nd Annual RAS-Targeted Drug Development Summit in September 2020 demonstrated a 56% overall response rate and long duration of therapy among patients with KRAS-G12 mt LGSOC. Based on an observation of higher response rates seen in NSCLC patients with KRAS-G12V mutations in the study, Verastem will also be further exploring the role of VS-6766 and defactinib in KRAS-G12V NSCLC. The FRAME study was expanded in August 2020 to include new cohorts in pancreatic cancer, KRASmt endometrial cancer and KRAS-G12V NSCLC.

Entry into a Material Definitive Agreement.

On November 6, 2020, GT Biopharma, Inc. (the "Company") reported that it entered into a Securities Purchase Agreement with two purchasers (individually, a "Purchaser," and collectively, the "Purchasers") pursuant to which the Company has issued to the Purchasers Convertible Debentures in an aggregate principal amount of $250,000 (the "Debentures"), which Debentures are convertible into the Company’s common stock (the "Common Stock") at a price of $0.20 per share (Filing, 8-K, GT Biopharma, NOV 6, 2020, View Source [SID1234570455]).

The issuance of the Debentures was made in reliance on the exemption provided by Section 4(a)(2) of the Securities Act of 1933, as amended (the "Securities Act"), for the offer and sale of securities not involving a public offering and Regulation D promulgated under the Securities Act.

The foregoing summaries of the Securities Purchase Agreement, and the Debentures are qualified in their entirety by reference to the full text of the agreements, which are attached hereto as Exhibits 10.1 and 4.1, respectively, and are incorporated herein by reference.

Penumbra, Inc. to Present at the Canaccord Genuity Virtual MedTech & Diagnostics Forum

On November 6, 2020 Penumbra, Inc. (NYSE: PEN) reported that its management team is scheduled to present at the Canaccord Genuity Virtual MedTech & Diagnostics Forum on Thursday, November 19, 2020 (Press release, Penumbra, NOV 6, 2020, View Source;diagnostics-forum-301167651.html [SID1234570277]).

Event: Canaccord Genuity Virtual MedTech & Diagnostics Forum
Date: Thursday, November 19, 2020
Time: 1:30pm ET / 10:30am PT

A webcast of the presentation will be available by visiting the investors’ section of the company’s website at www.penumbrainc.com. The webcast will be available on the company’s website for at least two weeks following the event.

A better test for the tumor-targeting of CAR-T therapies

On November 6, 2020 Ludwig Cancer Research scientists reported that have developed a method to significantly improve the preclinical evaluation of chimeric antigen-receptor (CAR) T cell therapies, in which the immune system’s T cells are extracted from a patient, engineered to target a specific tumor-associated molecule and then grown and reinfused for cancer treatment (Press release, Ludwig Institute For Cancer Research, NOV 6, 2020, View Source [SID1234570273]). Published in the Journal of Experimental Medicine, the study also reports the construction and evaluation of co-engineered CAR-T cells and applies the method to examine their effects on tumors in a mouse model of the skin cancer melanoma.

Though CAR-T therapies have been approved for blood cancers, their application to solid tumors has proved challenging. This is in part because the complex microenvironments of solid tumors dampen immune responses in a variety of ways, not least through the recruitment of various suppressive immune cells.

"Most of the studies on CAR-T therapies have been done using human T cells in mice that lack their own immune system—because if they had one, it would attack the human CAR-T cells," said Melita Irving, a researcher at the Ludwig Institute for Cancer Research Lausanne Branch who led the study with Ludwig Lausanne Director George Coukos. "But the tumor microenvironment can have a huge impact on your T cell product, so we’re really interested in doing studies using engineered mouse T cells in immunocompetent mice. This allows us to observe the dynamic interplay between the immune system and the CAR-T cells that we transfer."

Trouble is that mouse T cells are difficult to engineer and expand optimally in culture in the numbers required to rigorously model CAR-T cell therapies. For the current study, the Ludwig Lausanne team first developed a protocol to overcome those difficulties. It involved, among other things, the sequential use of three immune signaling molecules known as interleukins (IL-2, 7 and 15) in the cultivation and expansion of the engineered T cells.

They showed that CAR-T cells cultured using the protocol are markedly activated when exposed to their target. The cells also show signs of being more youthful and have molecular features common to memory T cells that grow briskly when stimulated by their targets.

"This means that when you transfer these cells, they’re very robust cells and can really expand quickly to control tumors," said Irving.

Irving, Coukos and colleagues then engineered their cultured mouse T cells to co-express, along with the chimeric antigen receptor, the IL-15 protein—which promotes the formation of memory T cells. They then examined the efficacy of these "fourth generation" (4G) CAR-T cells against a mouse model of melanoma and compared their activity to that of ordinary mouse CAR-T cells generated using the novel protocol.

"We saw better tumor control by the IL-15 expressing CAR-T cells and better proliferation and persistence of the CAR-T cells themselves," said Irving. The 4G CAR-T cells were also less prone to programmed cell death and expressed lower levels of a cell surface protein named PD-1, which promotes T cell suicide.

Their analysis revealed that the 4G CAR-T cells were not just killing cancer cells more efficiently. They were also reprogramming the microenvironment of the tumor to augment such killing. Their use resulted in an activation of natural killer cells—which target cancer cells—in the tumor microenvironment, and a marked decline in M2 macrophages, which suppress anti-tumor immune responses and support tumor growth.

"We hope that our publication of this protocol will help the T cell engineering community in general and enable a more robust preclinical evaluation of T cell therapies," said Irving.

This study was supported by Ludwig Cancer Research, the European Research Council, the Biltema Foundation and Oncosuisse.

In addition to his Ludwig post, George Coukos directs the Department of Oncology at the University Hospital of Lausanne (CHUV-UNIL) and co-directs the Swiss Cancer Center, Léman. Melita Irving is also a group leader within the department of oncology, UNIL CHUV.

SCYNEXIS Reports Third Quarter 2020 Financial Results and Provides Company Update

On November 6, 2020 SCYNEXIS, Inc. (NASDAQ: SCYX), a biotechnology company pioneering innovative medicines to potentially overcome and prevent difficult-to-treat and drug-resistant infections, reported financial results for the third quarter ended on September 30, 2020 and provided an update on recent clinical and corporate developments.

"We submitted our NDA for ibrexafungerp for the treatment of vaginal yeast infections ahead of schedule and, with approval anticipated in mid-2021, we continue to progress towards our transition into a fully integrated commercial-stage antifungal company with an initial focus on women’s health," said Marco Taglietti, M.D., President and Chief Executive Officer of SCYNEXIS. "In parallel, we are also advancing clinical trials evaluating ibrexafungerp’s potential to treat invasive fungal infections in the hospital setting, with additional data read-outs expected in 2021."

Ibrexafungerp Update

SCYNEXIS recently announced the submission of its NDA to the U.S. Food and Drug Administration (FDA) for oral ibrexafungerp for the treatment of VVC, also known as vaginal yeast infection. The submission occurred in October and SCYNEXIS expects to receive FDA feedback on the acceptability of this submission in December. As a qualified infectious disease product (QIDP), ibrexafungerp is expected to receive a six-month priority review following NDA acceptance with a PDUFA date anticipated in mid-2021.

Enrollment is ongoing in the Phase 3 CANDLE study, investigating the efficacy and safety of oral ibrexafungerp for the prevention of recurrent VVC, for which there is no approved therapy in the U.S. Pending successful completion of this trial, SCYNEXIS anticipates top-line results and the submission of a supplemental NDA for this indication in the second half of 2021.

Enrollment is ongoing in our refractory invasive fungal infections (rIFI) program, which comprises two open-label Phase 3 studies (FURI and CARES). Similar to the two interim analyses of previously reported data, SCYNEXIS intends to analyze the outcomes from the next cohort of patients that have completed their treatment course in both FURI and CARES studies and announce these findings when available.

Enrollment is ongoing in the Phase 2 SCYNERGIA study for patients with invasive aspergillosis evaluating oral ibrexafungerp in combination with voriconazole. Top-line data from this study are expected in the second half of 2021.

Pre-clinical activities are also ongoing towards the development of a liposomal intravenous formulation of ibrexafungerp.

Data presentations. In October 2020, SCYNEXIS presented ibrexafungerp data at two scientific conferences. The first was the Nurse Practitioners in Women’s Health (NPWH) held virtually on October 15-17, where SCYNEXIS showcased in vitro activity of ibrexafungerp against fluconazole-susceptible and -resistant Candida species, as well as data from the Phase 3 VANISH-303 trial in VVC. The second October conference was IDWeek 2020 held virtually on October 21-25. At this conference SCYNEXIS presented an interim analysis from its ongoing Phase 3 FURI trial in refractory infections and pre-clinical data highlighting the potential for ibrexafungerp use in invasive fungal infections. In August 2020, SCYNEXIS presented its full data set from the Phase 3 VANISH-303 VVC trial at the Infectious Diseases Society for Obstetrics and Gynecology (IDSOG). Some of the posters presented are available here.
Corporate Developments

On July 17, 2020, SCYNEXIS executed a 1-for-10 reverse split of its issued and outstanding common stock.
Third Quarter Financial Results

Cash and cash equivalents totaled $29.5 million as of September 30, 2020, compared to $48.4 million in cash, cash equivalents, and short-term investments at December 31, 2019.

Research and development expense for the three months ended September 30, 2020 decreased to $8.0 million from $9.3 million for the three months ended September 30, 2019. The decrease of $1.2 million, or 13%, for the three months ended September 30, 2020, was primarily driven by a decrease of $2.0 million in clinical development expense, and a decrease of $0.5 million in preclinical expense, offset in part by an increase in regulatory expense of $0.6 million, an increase of $0.3 million in chemistry, manufacturing, and controls (CMC) expense, and a net increase in other research and development expense of $0.4 million.

Selling, general and administrative expenses for the quarter ended September 30, 2020 increased to $3.5 million from $2.5 million for the quarter ended September 30, 2019. The increase of $1.0 million, or 40%, for the three months ended September 30, 2020 was primarily driven by a $0.8 million increase in professional fees and commercial related expenses recognized during the three months ended September 30, 2020.

Total other income was $12.4 million for the quarter ended September 30, 2020, compared to total other income of $3.8 million for the quarter ended September 30, 2019. During the quarter ended September 30, 2020 and 2019, SCYNEXIS recognized non-cash gains of $7.8 million and $1.8 million, respectively, on the fair value adjustment of the warrant liabilities and during the quarter ended September 30, 2020 and 2019, recognized non-cash gains of $5.3 million and $2.3 million on the fair value adjustment of the derivative liabilities, respectively.

Net income for the quarter ended September 30, 2020 was $0.9 million, or $0.09 per basic and ($0.28) per diluted share, compared to a net loss of $7.9 million, or ($1.43) per basic and ($1.45) per diluted share for the quarter ended September 30, 2019.

About Ibrexafungerp

Ibrexafungerp [pronounced eye-BREX-ah-FUN-jerp] is an investigational antifungal agent and the first representative of a novel class of structurally-distinct glucan synthase inhibitors, triterpenoids. This agent combines the well-established activity of glucan synthase inhibitors with the potential flexibility of having oral and intravenous (IV) formulations. Ibrexafungerp is currently in development for the treatment of fungal infections caused primarily by Candida (including C. auris) and Aspergillus species. It has demonstrated broad-spectrum antifungal activity, in vitro and in vivo, against multidrug-resistant pathogens, including azole- and echinocandin-resistant strains. The FDA has granted Qualified Infectious Disease Product (QIDP) and Fast Track designations for the formulations of ibrexafungerp for the indications of invasive candidiasis (IC) (including candidemia), invasive aspergillosis (IA) and VVC, and has granted Orphan Drug Designation for the IC and IA indications. Ibrexafungerp is formerly known as SCY-078. (Press release, Scynexis, NOV 6, 2020, View Source [SID1234570270])