On May 11, 2021 Aptevo Therapeutics Inc. ("Aptevo" or "the Company") (NASDAQ:APVO), a clinical-stage biotechnology company focused on developing novel immuno-oncology therapeutics based on its proprietary ADAPTIR and ADAPTIR-FLEX platform technologies, reported its financial results for the quarter ended March 31, 2021 (Press release, Aptevo Therapeutics, MAY 11, 2021, View Source [SID1234579700]).

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"We are pleased to report progress in the clinical trial of our ADAPTIR platform candidate APVO436, with the dose limiting toxicity (DLT) evaluation in cohorts 1 through 9 now completed and 44 patients enrolled to date in cohorts 1-10. We remain confident in the clinical impact potential of APVO436 and our platform technologies, and look forward to sharing interim data readouts from our ongoing APVO436 study later this year," said Mr. Marvin White, President and CEO of Aptevo. "The transaction with HCR, which we completed in March 2021, provided significant non-dilutive funding, which adds to our cash runway and helps fund the company through the next twelve months."

First Quarter 2021 Financial Results Summary

As announced in March, Aptevo received $35 million ("the Investment Amount") from its sale of the right to royalty payments made by Pfizer Inc. ("Pfizer") with respect to net sales of RUXIENCE to an entity managed by HealthCare Royalty Management, LLC ("HCR"). Aptevo is eligible to receive additional payments in aggregate of up to an additional $32.5 million based on the achievement of sales milestones in 2021, 2022, and 2023 (collectively, the "Milestone Amounts"). The Royalty Purchase Agreement further provides that, once HCR reaches aggregate royalty payments totaling 190% of the Investment Amount plus the Milestone Amounts to the extent paid by HCR to the Company, Aptevo will be entitled to receive 50% of any additional royalty payments by Pfizer thereafter. In connection with this royalty purchase agreement, Aptevo amended its term loan agreement with MidCap Financial and used $10 million of the proceeds received to pay down outstanding principal.

Cash Position: Aptevo had cash and cash equivalents as of March 31, 2021 totaling $58.8 million, including restricted cash of $1.3 million. The restricted cash is expected to be released over the next twelve months.

Royalty Revenue: Royalty revenue was $2.4 million for the three months ended March 31, 2021, related to the royalty from Pfizer on global net sales of RUXIENCE, a biosimilar to the drug RITUXAN, launched by Pfizer in early 2020. RUXIENCE is a trademark of Pfizer; RITUXAN is a trademark of Biogen. Due to our continuing involvement under the Definitive Agreement originally between Trubion and Wyeth, we continue to recognize royalty revenue on net sales of RUXIENCE and record the royalty payments to HCR as a reduction of the liability when paid. As such payments are made to HCR, the balance of the liability will be effectively repaid over the life of the Royalty Purchase Agreement.

Research and Development Expenses: Research and development expenses increased to $5.4 million for the three months ended March 31, 2021 from $4.0 million for the three months ended March 31, 2020 as we continue to invest in the APVO436 clinical trial and our preclinical candidates, including ALG.APV-527, APVO603 and APVO442.

General and Administrative Expenses: For the three months ended March 31, 2021, general and administrative expenses increased to $3.9 million from $3.6 million for March 31, 2020, with higher costs for professional services.

Other Expense, Net: Other expense, net consists primarily of interest on debt and costs related to debt extinguishment. Other expense, net was $0.8 million for the three months ended March 31, 2021 and $2.4 million for the three months ended March 31, 2020. A slight increase in interest expense this quarter was offset by a significant decrease due to a loss on extinguishment of debt of $2.1 million recognized in the first quarter of 2020 when we repaid our previous loan to MidCap Financial using the proceeds from the sale of Aptevo BioTherapeutics LLC.

Discontinued Operations: Income from discontinued operations was $0.4 million for the three months ended March 31, 2021 and $12.9 million for the three months ended March 31, 2020. For the three months ended March 31, 2021, we collected a deferred payment of $0.2 million from Medexus related to fourth quarter 2020 IXINITY sales and $0.2 million from the Saol Therapeutics, related to the 2017 sale of the Hyperimmune Business to them. For the three months ended March 31, 2020, we recognized net income from discontinued operations totaling $12.9 million. This included the gain on the sale of Aptevo BioTherapeutics LLC of $14.3 million and net operating losses from Aptevo BioTherapeutics LLC of $1.6 million related to the period prior to the sale on February 28, 2020.

Net Income (Loss): Aptevo’s net loss for the three-month period ended March 31, 2021 was $(7.3) million or $(1.64) per share, as compared to a net income of $2.9 million or $0.89 per share for the corresponding period in 2019. Our net loss from continuing operations for the first quarter of 2021 was $(7.6) million compared to $(10.0) million in the first quarter of 2020.

Liability Related to Sale of Future Royalties: We treat the Royalty Purchase Agreement with HCR as a debt financing, amortized under the effective interest rate method over the estimated life of the related expected royalty stream. The liabilities related to sale of future royalties and the debt amortization are based on our current estimates of future royalties expected to be paid over the life of the arrangement. We will periodically assess the expected royalty payments using projections from external sources. To the extent our estimates of future royalty payments are greater or less than previous estimates or the estimated timing of such payments is materially different than previous estimates, we will adjust the effective interest rate and recognize related non-cash interest expense on a prospective basis. We are not obligated to repay the proceeds received under the Royalty Purchase Agreement with HCR.

On May 11, 2021 Ultimovacs ASA ("Ultimovacs") (OSE ULTI), a clinical stage leader in immune stimulatory vaccines for cancer, reported the publication in Frontiers in Immunology of its positive long-term Overall Survival (OS) data from the Phase I trial evaluating the Company’s universal cancer vaccine, UV1, in combination with checkpoint inhibitor ipilimumab in patients with metastatic malignant melanoma (Press release, Ultimovacs, MAY 11, 2021, View Source [SID1234579716]). As published in the journal, in addition to the achievement of the primary endpoints of safety and tolerability, 50% of the patients were still alive at the data cut-off, supporting the combination of the Company’s proprietary UV1 vaccine with ipilimumab, a CTLA-4 checkpoint inhibitor and standard-of-care treatment, in this late-stage patient population.

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"Publishing our clinical trial data in such a prestigious peer-reviewed publication adds validation for UV1 as a promising therapeutic option for cancer patients. As we continue to evaluate UV1 in various combinations and indications, it is valuable to gain increased international recognition from the clinical community for this study," stated Jens Bjørheim, Chief Medical Officer at Ultimovacs. "Historical data on the use of ipilimumab as monotherapy in malignant melanoma have shown a 5-year survival rate below 20%, therefore the results published today reinforce UV1’s potential in this indication."

The data published in Frontiers in Immunology covers 4.8 years of follow-up on the total of 12 metastatic malignant melanoma patients that were enrolled in the Phase I trial. As reported in the journal, the OS was 50% at 4.8 years, which was confirmed by the results of 5 years of follow-up announced by the Company in December 2020.

Building on these promising Phase I results, Ultimovacs is currently enrolling INITIUM, its Phase II clinical trial evaluating UV1 in combination with ipilimumab and nivolumab in patients with metastatic malignant melanoma. The company expects to announce data on the trial’s primary endpoint in 2H2022. In addition, Ultimovacs has an ongoing and fully-enrolled Phase I trial evaluating UV1 in combination with pembrolizumab, a PD-1 checkpoint inhibitor, as a first line treatment in metastatic malignant melanoma patients.

The publication in Frontiers in Immunology can be found under doi: 10.3389/fimmu.2021.663865.

About UV1

UV1 is a peptide-based vaccine inducing a specific T cell response against the universal cancer antigen telomerase. UV1 is being developed as an "off-the-shelf" therapeutic cancer vaccine which may serve as a platform for use in combination with other immunotherapy which requires an ongoing T cell response for their mode of action. To date, UV1 has been tested in four phase I clinical trials in a total of 82 patients and maintained a positive safety and tolerability profile as well as encouraging signals of efficacy.

About UV1 Clinical Programs

As a universal cancer vaccine, UV1’s unique mechanism of action has the potential to be applicable across most cancer types. The clinical development of the UV1 vaccine includes four randomized, multinational, Phase II combination trials: INITIUM, NIPU, DOVACC and FOCUS, recruiting over 500 patients in total. The INITIUM trial is an Ultimovacs-sponsored clinical trial recruiting 154 patients with metastatic malignant melanoma to evaluate UV1 in combination with ipilimumab and nivolumab as first-line treatment. The NIPU study is testing UV1 in combination with checkpoint inhibitors ipilimumab and nivolumab as second-line treatment in 118 patients with advanced malignant pleural mesothelioma, a rare lung cancer. The study is sponsored by Oslo University Hospital and Bristol-Myers Squibb is providing the checkpoint inhibitors for this study. The DOVACC study is sponsored by the Nordic Society of Gynaecological Oncology. In total, 184 patients with high-grade ovarian cancer will be enrolled to evaluate UV1 in combination with durvalumab and olaparib, both provided by AstraZeneca. FOCUS is an investigator-sponsored, randomized clinical trial enrolling 75 patients with metastatic head and neck cancer receiving pembrolizumab as standard of care, and will evaluate the impact of adding UV1 to this regimen. Ultimovacs anticipates announcing data on the primary endpoints for the NIPU and INITIUM studies in 2H2022 and for the DOVACC and FOCUS studies in 2023.

On May 11, 2021 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 ended March 31, 2021, highlighted recent progress and outlined key corporate objectives (Press release, Verastem, MAY 11, 2021, View Source [SID1234579808]).

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"During the first quarter of 2021, we expanded the selection portion of the Phase 2 RAMP 201 study to include all recurrent low-grade serous ovarian cancer types. This decision was based on the positive updated data from the LGSOC cohort of the Phase 1/2 FRAME which continues to show strong response rates across both KRAS mutant and wild-type recurrent LGSOC, along with robust durability and a favorable tolerability profile," said Brian Stuglik, Chief Executive Officer of Verastem Oncology. "We closed the first quarter with just over $127 million in cash, cash equivalent and investments, leaving us well positioned to execute on our two ongoing Phase 2 studies evaluating VS-6766 and defactinib in LGSOC and KRAS G12V non-small cell lung cancer (NSCLC), as well as our other key corporate objectives."

Recent Corporate Highlights

LGSOC

Reported updated data from the LGSOC cohort of the ongoing, investigator-sponsored Phase 1/2 FRAME study evaluating VS-6766 in combination with defactinib in patients with recurrent LGSOC. Combination continues to demonstrate activity, durability and a favorable tolerability profile, including in patients who have progressed following treatment with a MEK inhibitor.

Overall response rate (ORR) across all patients was 52% (11 of 21 patients).

ORR for patients with KRAS mutant LGSOC was 70% (7 of 10 patients).

ORR for patients with wild type LGSOC was 44% (4 of 9 patients).

The most common side effects were Grade 1/2 rash, creatine kinase elevation, nausea, hyperbilirubinemia and diarrhea, which were reversible.
Company-sponsored, registration-directed Phase 2 study (RAMP 201) underway investigating VS-6766 alone and in combination with defactinib for the treatment of recurrent LGSOC. Study recently expanded to include both KRAS mutant and KRAS wild-type patients in the selection phase to determine the optimal go-forward regimen for both types of LGSOC.
KRAS G12V Mutant NSCLC

Company-sponsored, registration-directed Phase 2 study (RAMP 202) underway investigating VS-6766 alone and in combination with defactinib for the treatment of patients with KRAS G12V mutant NSCLC.
Upcoming Milestones and Key Priorities for 2021-2022

LGSOC

Updated data from Phase 1/2 FRAME study to be submitted for presentation at a major medical meeting during the second half of 2021.
Complete selection portion of RAMP 201 during first half of 2022; commence expansion portion.
G12V NSCLC

Complete selection portion of RAMP 202 during first half of 2022; commence expansion portion.
First Quarter 2021 Financial Results

Verastem Oncology ended the first quarter 2021 with cash, cash equivalents and investments of $127.1 million.

Total revenue for the three months ended March 31, 2021 (2021 Quarter) was $1.0 million, compared to $5.1 million for the three months ended March 31, 2020 (2020 Quarter).

Total operating expenses for the 2021 Quarter were $15.1 million, compared to $31.4 million for the 2020 Quarter.

Selling, general and administrative expenses for the 2021 Quarter were $6.2 million, compared to $19.6 million for the 2020 Quarter. The decrease of $13.4 million, or 68.4%, primarily resulted from the Company’s shift in strategic direction and COPIKTRA sale to Secura Bio, Inc., which led to lower employee related expenses and consulting and professional fees.

Research and development expense for the 2021 Quarter was $8.9 million, compared to $10.9 million for the 2020 Quarter. The decrease of $2.0 million, or 18.3%, was primarily related to the upfront non-refundable payment of $3.0 million to Chugai Pharmaceutical Co., Ltd for the VS-6766 license in the 2020 Quarter and decreased contract research organization costs, partially offset by increased drug substance and drug product costs and increased investigator sponsored trial expenses.

Net loss for the 2021 Quarter was $15.0 million, or $0.09 per share (basic and diluted), compared to $38.0 million, or $0.35 per share (basic and diluted), for the 2020 Quarter.

For the 2021 Quarter, non-GAAP adjusted net loss was $12.4 million, or $0.07 per share (diluted), compared to non-GAAP adjusted net loss of $21.3 million, or $0.20 per share (diluted), for the 2020 Quarter. Please refer to the GAAP to Non-GAAP Reconciliation attached to this press release.

Financial Guidance and Outlook

With the proceeds from the sale of COPIKTRA, Verastem Oncology expects that it will have 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 Oncology expects its 2021 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, 2021 and 2020 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 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.1,2

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, KRAS mt NSCLC and colorectal cancer (CRC). The FRAME study was expanded to include new cohorts in pancreatic cancer, KRASmt endometrioid cancer and KRAS-G12V NSCLC. Verastem Oncology is also supporting an investigator-initiated Phase 2 trial evaluating VS-6766 with defactinib in patients with metastatic uveal melanoma.

Verastem Oncology has initiated Phase 2 registration-directed trials of VS-6766 with defactinib in patients with recurrent LGSOC and in patients with recurrent KRAS-G12V NSCLC as part of its RAMP (Raf And Mek Program).

On May 10, 2021 Genmab reported the initiation of a share buy-back program to mitigate dilution from warrant exercises and to honor our commitments under our Restricted Stock Units program (Press release, Genmab, MAY 10, 2021, View Source [SID1234579510]).

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The share buy-back program is expected to be completed no later than June 30, 2021 and comprises up to 200,000 shares.

The following transactions were executed under the program from May 3, 2021 to May 7, 2021:

Details of each transaction are included as an appendix to this announcement.

Following these transactions, Genmab holds 236,006 shares as treasury shares, corresponding to 0.36% of the total share capital and voting rights.

The share buy-back program is undertaken in accordance with Regulation (EU) No. 596/2014 (‘MAR’) and the Commission Delegated Regulation (EU) 2016/1052, also referred to as the "Safe Harbour Regulation." Further details on the terms of the share buy-back program can be found in our company announcement no. 11 dated February 23, 2021.

On May 10, 2021 Codiak BioSciences, Inc. (Nasdaq: CDAK), a clinical-stage biopharmaceutical company focused on pioneering the development of exosome-based therapeutics as a new class of medicines, reported the appointment of Jennifer Wheler, M.D., an industry leader in targeted oncology and immunotherapy drug development, as Chief Medical Officer (Press release, Codiak Biosciences, MAY 10, 2021, View Source [SID1234579550]). Dr. Wheler, who joins the company’s Executive Leadership Team and reports to president and CEO Douglas Williams, Ph.D., will provide leadership and direction for Codiak’s pipeline of clinical development programs through early-stage trials to global regulatory submissions.

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Benny Sorensen, M.D., Ph.D., who has led the preclinical to clinical translational work and clinical strategy for Codiak for the past five years, advancing exoSTINGTM and exoIL-12TM through IND/CTA and into the current Phase 1 trials will become Senior Vice President, Strategic Projects. Dr. Sorensen will report to president and CEO Douglas Williams and continue as a member of Codiak’s Executive Leadership Team. He will help direct business development and other strategic external partnering projects and continue to provide support for the ongoing clinical trials.

"In the past year, our clinical team, led by Benny, has initiated two clinical programs and we expect to file an IND for a third program later this year, all of which are evaluating the potential for engineered exosomes to engage the immune system in novel ways to attack intractable cancers. With the anticipated advancement and intensified focus in oncology, we believe it is an ideal time for us to expand our team for the next phase of growth," said Dr. Williams. "As a clinical oncologist with many years of patient treatment and drug development experience, Jennifer knows this space incredibly well and will bring valuable leadership and deep domain expertise to the outstanding team that we already have in place."

Dr. Wheler brings more than 20 years of experience in industry and academic clinical research to Codiak, including a strong track record of successful early-stage oncology drug development. A board-certified oncologist, she served most recently as chief medical officer of Bicara Therapeutics, where she designed and led the first-in-human Phase I/II trial for the company’s bi-functional antibody candidate, including building the clinical trial infrastructure, clinical operations team and KOL network. Previously, Dr. Wheler led first-in-human studies for novel immuno-oncology assets at Novartis Institutes for BioMedical Research. From 2006 to 2015, she worked in the Department of Investigational Cancer Therapeutics at the University of Texas MD Anderson Cancer Center where she served as Principal Investigator on more than 30 Phase 1 trials, including one of the first published trials of randomized data demonstrating benefit for treating patients with therapy matched to their tumor’s molecular profile. She is co-author on more than 145 peer-reviewed publications. Dr. Wheler completed fellowships in breast cancer medicine at Memorial Sloan Kettering Cancer Center and in oncology at Yale Cancer Center and her internal medicine residency at Columbia University Irving Medical Center and New York-Presbyterian Hospital. She received her A.B. from Princeton University and her M.D. from Weill Cornell Medical College in New York. With a background in the arts, Dr. Wheler also founded Collage, a nonprofit organization that brings personalized and innovative art programs to patients living with cancer.

"Codiak has a rich pipeline of first-in-class molecules that leverages a deep understanding of exosome biology and engineering. These exquisitely constructed molecules combine targeted and immunomodulatory approaches that have previously been elusive—the potential to impact the lives of patients with cancer and other diseases is immense," said Dr. Wheler. "As an oncologist who has spent much of my career investigating new drugs designed to improve outcomes for patients, I see this as an tremendous opportunity: the science is compelling, the early clinical data support the desired product profile and the team is superb. I am excited to lend my experience to help lead the current clinical programs and work to guide the next programs into the clinic."

"I’m excited to welcome Jennifer to Codiak," said Dr. Sorensen. "With two oncology-focused programs now in the clinic and a third on track for IND this year, her background and experience will be incredibly valuable as we continue to advance these candidates. I look forward to collaborating with her as Codiak remains focused on moving the exoSTING and exoIL-12 clinical trials toward expected data read-outs later this year and prepares for the planned exoASO-STAT6 IND submission."