On March 7, 2022 Targovax ASA (OSE: TRVX), a clinical-stage immuno-oncology company developing immune activators to target hard-to-treat solid tumors, and Agenus (NASDAQ: AGEN), an immuno-oncology company with an extensive pipeline of checkpoint antibodies, adjuvants, and vaccines designed to activate immune response to cancers and infections, reported that they have entered into a clinical collaboration and supply agreement to combine Targovax’s TG mutant KRAS cancer vaccines with Agenus´s clinically validated and FDA approved QS-21 STIMULON adjuvant (Press release, Targovax, MAR 7, 2022, View Source [SID1234609599]).

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The TG vaccines are peptide mixtures designed to elicit polyvalent immune responses against the most frequent mutations in the RAS gene family, which drive up to 30% of all cancers. TG01 has shown promise in phase 1/2 clinical trials, and Targovax has recently been awarded two prestigious research grants providing up to NOK 18m in funding to advance the TG program. Cancer vaccines require combination with an adjuvant in order to stimulate antigen processing and T-cell priming, and Targovax has selected QS-21 STIMULON as the adjuvant of choice for continued clinical development.

QS-21 STIMULON is a purified, natural saponin proprietary to Agenus. It has been widely studied in over 120 clinical trials and consistently demonstrated powerful antibody and cell-mediated immune responses as well as a favorable safety profile. QS-21 STIMULON is a critical component of multiple vaccines, including GSK’s FDA approved Shingrix shingles vaccine with protection exceeding 9 years, and the world’s first malaria vaccine Mosquirix.

Under the collaboration with Targovax, Agenus will supply QS-21 STIMULON and scientific support for up to three initial TG01 clinical trials in different cancer indications. If the clinical results meet certain pre-specified performance targets, the collaboration will be extended into an exclusive pivotal development and commercialization agreement where Agenus will be entitled to tiered double-digit royalties on future TG vaccine sales.

Dr. Garo H. Armen, Chief Executive Officer of Agenus Inc., said: "We look forward to collaborating with Targovax to extend the benefit of QS-21 STIMULON to new patient populations such as those harboring KRAS-mutated cancers. It highlights the broad applicability of our QS-21 STIMULON technology and could unlock a sizeable commercial opportunity in KRAS-mutated cancers."

Dr. Erik Digman Wiklund, Chief Executive Officer of Targovax ASA, added: "QS-21 STIMULON is arguably the most clinically and commercially successful FDA approved adjuvant for protein- and peptide-based vaccines, and has proven highly effective in eliciting immune responses. We expect that this unique adjuvant will further enhance the efficacy of our TG01 and TG02 products, and see it as a critical component of our development strategy as we prepare to bring the TG vaccines back into the clinic in multiple mutant KRAS cancers."

On March 7, 2022 Jounce Therapeutics Presented the Corporate Presentation (Press release, Jounce Therapeutics, MAR 7, 2022, View Source [SID1234609619]).

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On March 7, 2022 Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage biopharmaceutical company developing disruptive therapeutics for the treatment of patients with cancer reported that an abstract for SBP-101, a proprietary polyamine analogue, has been accepted for poster presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper), which will be held April 8-13, 2022 (Press release, Panbela Therapeutics, MAR 7, 2022, View Source [SID1234609582]). The work reflects the Company’s on-going collaboration with Johns Hopkins University School of Medicine.

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Details of the presentation are as follows:

Poster Presentation

Title: The potential of spermine analogue SBP-101 (diethyl dihydroxyhomospermine) as a polyamine metabolism modulator in ovarian cancer

Session Category: Experimental and Molecular Therapeutics
Session Title: Small Molecule Therapeutic Agents Abstract #: 5488
Additional meeting information can be found on the AACR (Free AACR Whitepaper) website: Abstracts | AACR (Free AACR Whitepaper) Annual Meeting 2022 | April 8-13, 2022 | New Orleans

The abstract and poster will also be available on the Company’s website at View Source once the information has been released by AACR (Free AACR Whitepaper)

About SBP-101

SBP-101 is a proprietary polyamine analogue designed to induce polyamine metabolic inhibition (PMI) by exploiting an observed high affinity of the compound for pancreatic ductal adenocarcinoma and other tumors. The molecule has shown signals of tumor growth inhibition in clinical studies of US and Australian metastatic pancreatic cancer patients, demonstrating a median overall survival (OS) of 12.0 months which is not yet final, and an objective response rate (ORR) of 48%, both exceeding what is seen typically with the standard of care of gemcitabine + nab-paclitaxel suggesting potential complementary activity with the existing FDA-approved standard chemotherapy regimen. In data evaluated from clinical studies to date, SBP-101 has not shown exacerbation of bone marrow suppression and peripheral neuropathy, which can be chemotherapy-related adverse events. Serious visual adverse events have been evaluated and patients with a history of retinopathy or at risk of retinal detachment will be excluded from future SBP-101 studies. The safety data and PMI profile observed in the current Panbela sponsored clinical trial provides support for continued evaluation of SBP-101 in a randomized clinical trial. For more information, please visit View Source .

On March 7, 2022 Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported financial results for the full year ended December 31, 2021, and highlighted recent progress and upcoming milestones for its pipeline programs (Press release, Scholar Rock, MAR 7, 2022, View Source [SID1234609600]).

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"We are excited by the momentum and engagement around our ongoing Phase 3 SAPPHIRE trial of apitegromab in spinal muscular atrophy, as well as our DRAGON Phase 1 trial of SRK-181 in solid tumors. These programs have significant potential to address unmet patient need in a differentiated way, as well as demonstrate the tremendous potential of Scholar Rock’s platform, which focuses on inhibiting the activation of latent growth factors," said Nagesh Mahanthappa, Interim Chief Executive Officer of Scholar Rock. "While we progress these clinical programs, we also continue to build upon our world-leading expertise in selectively targeting the activation mechanisms of the TGFβ-superfamily growth factors and advance our preclinical pipeline focused on diseases such as fibrosis and regulation of iron metabolism. We look forward to providing more details about these programs at scientific meetings and through publications in the coming months."

Company Updates and Upcoming Milestones

Apitegromab is a selective inhibitor of myostatin activation being developed as the potential first muscle-directed therapy for the treatment of spinal muscular atrophy (SMA).

Conducting SAPPHIRE Phase 3 Clinical Trial Evaluating Apitegromab in Patients with Non-Ambulatory Type 2 and 3 SMA. The randomized, double-blind, placebo-controlled Phase 3 clinical trial is evaluating apitegromab as add-on therapy for patients on either nusinersen or risdiplam. The study design plans for approximately 156 patients aged 2-12 years old with non-ambulatory Type 2 and 3 SMA to be enrolled in the main efficacy population. Patients will be randomized 1:1:1 to receive for 12 months either apitegromab 20 mg/kg, apitegromab 10 mg/kg, or placebo by intravenous (IV) infusion every 4 weeks in addition to background SMN treatment.
Data from TOPAZ Phase 2 Open Label Extension Trial Expected to be Presented by mid-2022. As of February 28, 2022, 55 of 57 patients remain enrolled in the long-term extension trial of apitegromab in Type 2 and Type 3 SMA.
SRK-181 is a selective inhibitor of latent TGFβ1 activation being developed with the aim of overcoming primary resistance to and increasing the number of patients who may benefit from checkpoint inhibitor therapy.

Advancing Part B of the DRAGON Phase 1 POC Trial for SRK-181. Based on the safety and pharmacokinetic data from Part A of the DRAGON Phase 1 trial, Scholar Rock is conducting the Part B dose expansion portion of the trial, in which SRK-181 is dosed at 1500 mg every three weeks (Q3W) in patients receiving an approved anti-PD-(L)1 therapy dosed Q3W, or 1000 mg every two weeks (Q2W) in patients receiving an approved anti-PD-(L)1 therapy dosed Q2W. Part B consists of multiple proof-of-concept cohorts focused upon evaluating the ability of SRK-181 to overcome primary resistance to anti-PD-(L)1 therapy. Each cohort will enroll up to 40 patients with various solid tumors, including urothelial carcinoma (UC), cutaneous melanoma (MEL), non-small cell lung cancer (NSCLC), clear cell renal cell carcinoma (ccRCC), and other solid tumors. The biomarker strategy in part B of DRAGON will explore early signs of SRK-181 activity, including target engagement and pathway modulation. This will include measuring effects on both circulating and tumor immune contexture, such as CD8+ T cell infiltration and reductions in myeloid-derived suppressor cell (MDSC) populations as well as analysis of TGFβ-related pathway signaling. Early efficacy and safety data are anticipated in 2022.
Full Year 2021 Financial Results

For the full year ended December 31, 2021, net loss was $131.8 million or $3.59 per share compared to a net loss of $86.5.0 million or $2.81 per share for the year ended December 31, 2020.

Revenue was $18.8 million for the year ended December 31, 2021 compared to $15.4 million for the year ended December 31, 2020. Revenue was related to the Gilead fibrosis-focused research collaboration, which concluded in December 2021.
Research and development expense was $108.5 million for the year ended December 31, 2021 compared to $74.1 million for the year ended December 31, 2020. The increase year-over-year was primarily attributable to planned spend associated with apitegromab development, including costs associated with clinical drug supply manufacturing and costs associated with our SAPPHIRE trial, as well as higher personnel-related costs.
General and administrative expense was $40.3 million for the year ended December 31, 2021 compared to $28.2 million for the year ended December 31, 2020. The increase year-over-year was primarily attributable to professional services and higher personnel-related costs, including filling key positions essential to progressing research, development and pre-commercial activities.
As of December 31, 2021, Scholar Rock had cash, cash equivalents, and marketable securities of approximately $253.0 million, which is expected to fund the Company’s operations into mid-2023. "Continued operational excellence is an important priority for us in 2022 as we advance several programs across the continuum of drug development. As we continue to progress our SMA program with our Phase 3 SAPPHIRE trial, we are also encouraged that so many of the patients who participated in the TOPAZ study are still opting to receive apitegromab. We look forward to obtaining further insights into longer-term treatment outcomes and sharing that data in the coming months. In our immuno-oncology program, the DRAGON study has entered Part B and could produce meaningful data later this year and our research colleagues are pushing forward on several fronts as we continue to generate exciting programs from our scientific platform," said Ted Myles, COO and CFO of Scholar Rock.

On March 7, 2022 Arctoris, a tech-enabled biopharma company, and Evariste Technologies, an AI-drug discovery company, reported they have formed a joint venture to identify novel small molecule kinase inhibitors for treatment of patients with Non-Small Cell Lung Cancer (NSCLC) (Press release, Lifescience Newswire, MAR 7, 2022, View Source [SID1234609620]).

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Current treatment options for NSCLC are limited, especially in advanced stages. It has been shown that the proto-oncogene cMet is mutated or upregulated in approximately 5% of all NSCLC cases. While cMET has been successfully targeted by two recently approved drugs (Tepotinib, Capmatinib), rapid development of resistance has been reported and there is a clear need for improved second-generation cMET inhibitors to overcome resistance.

The two companies are combining their platforms for AI-guided and robotics-powered drug discovery to develop a set of novel kinase inhibitors against cMET. The partnership will bring together two highly synergistic approaches – quantitative decision making and state-of-the-art generative chemistry, combined with real-time biological and biochemical profiling and data generation, to significantly accelerate the design-make-test-analyze cycle. The two companies will also use their strong links to leading centres for NSCLC treatment to leverage clinical insights, inform their discovery and development efforts and directly address clinically relevant liabilities limiting the effectiveness of currently available therapies.

"We are really excited to be working with Arctoris on this project. There is a huge need for next generation cMET inhibitors for NSCLC. This is a cancer that affects millions globally, and we hope that we can bring meaningful benefit to some of these lives in the near future," shares Dr. Nicholas Firth, CEO of Evariste Technologies.

Arctoris CEO Martin-Immanuel Bittner MD DPhil FRSA commented on the joint venture, "Together with our partners at Evariste, we are developing novel treatment options in NSCLC against a fully validated target, where first generation inhibitors can be improved on in a clinically meaningful way. Combining patient-derived insights on resistance and toxicity patterns with AI-powered molecule design and our robotic platform, Ulysses, we aim to develop superior next generation inhibitors within a significantly accelerated time frame."

The collaboration between Arctoris and Evariste is already underway and has identified novel, active chemical matter. "We look forward to keeping our community updated about the progress we are making within the joint venture between Evariste Technologies and Arctoris. We have had an incredible start already, and we look forward to continuing our work to develop better treatments options for patients worldwide", shares Bittner.