On August 5, 2019 SELLAS Life Sciences Group, Inc. (Nasdaq: SLS) ("SELLAS" or the "Company"), a clinical-stage biopharmaceutical company focused on the development of novel cancer immunotherapies for a broad range of cancer indications, reported completion of enrollment in a Phase 2 randomized investigator-sponsored trial (IST) of nelipepimut-S (NPS) in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF) in women with ductal carcinoma in situ (DCIS) of the breast who are HLA-A2+ or A3+ positive, express HER2 at IHC 1+, 2+, or 3+ levels, and are pre- or post-menopausal (Press release, Sellas Life Sciences, AUG 5, 2019, View Source [SID1234538133]).

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"We are pleased to announce completion of enrollment in the Phase 2 VADIS trial, an important milestone for our NPS clinical program. The premise of the VADIS study is quite innovative, as it will provide valuable data and give us the opportunity to gauge in a controlled, randomized setting whether NPS can effectively induce an antitumor immune response in DCIS patients. We believe NPS could serve as an earlier stage treatment for women with breast cancer and hope to gain through this study further insights on the immunobiological mechanism underlying the clinical activity of NPS. The VADIS results could inform us as to potential synergies between NPS and standard therapies in women with DCIS. We are excited to move NPS another step closer to our goal of improving the therapeutic options for breast cancer patients by potentially serving as an early stage treatment for patients with DCIS. We look forward to seeing the initial data by the end of 2019," said Angelos M. Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS.

"We are delighted to have completed enrollment in the VADIS study, which will test for an array of sophisticated histologic, immunodynamic and molecular markers of immune responses following treatment with NPS, including induction of HER2-specific cytotoxic T lymphocyte (CTL) and epitope spreading, the latter being the herald of clinical efficacy for a successful peptide vaccine," said Elizabeth A. Mittendorf, MD, PhD, Rob and Karen Hale Distinguished Chair in Surgical Oncology, Director of Research, Breast Surgical Oncology Brigham and Women’s Hospital, Director, Breast Immuno-Oncology Program Dana-Farber/Brigham and Women’s Cancer Center, and the Principal Investigator of the Phase 2 VADIS trial. "VADIS is poised to inform us on the design of future treatment strategies for DCIS, which remains an unmet medical need, including combinations of NPS with standard therapies in a broad population," concluded Dr. Mittendorf.

About the Phase 2 VADIS Trial

This Phase 2 randomized trial is sponsored and operationalized by the National Cancer Institute (NCI) to study NPS’ potential clinical effects in earlier-stage disease. Patients are randomized to receive, prior to surgery, either GM-CSF followed by NPS two weeks later or GM-CSF alone. The primary endpoint of the trial is the difference in the frequency of newly induced NPS-cytotoxic T lymphocytes (CTL; CD8+ T-cell) in peripheral blood between the two arms of the study, using a dextramer assay. Secondary endpoints to be compared between the two arms include the nature and incidence of adverse events and in vivo immune response to NPS, in addition to other select histologic and molecular biomarkers. Initial data from this trial are expected by the end of 2019.

About DCIS

DCIS is defined by the NCI as a noninvasive condition in which abnormal cells are found in the lining of a breast duct and have not spread outside the duct to other tissues in the breast. DCIS is the most common type of breast neoplasm with malignant potential. In some cases, DCIS may become invasive cancer and spread to other tissues and, currently, it is not possible to know which lesions could become invasive. Current treatment options for DCIS include breast-conserving surgery and radiation therapy with or without tamoxifen, breast-conserving surgery without radiation therapy, or total mastectomy with or without tamoxifen. Tamoxifen is given in cases with hormone receptor positivity only. No targeted or immune therapies have shown any definitive clinical activity in DCIS to date. The current standard treatment aims at forestalling the progression of DCIS to invasive cancer. In approximately 15-25% of cases progression does occur. DCIS is diagnosed in more than 60,000 women each year in the United States, comprising 1 in 5 newly diagnosed cases of breast cancer.

On August 5, 2019 Harpoon Therapeutics, Inc. (Nasdaq: HARP), a clinical-stage immunotherapy company developing a novel class of T cell engagers, reported financial results for the second quarter and six months ended June 30, 2019 and provided a corporate update (Press release, Harpoon Therapeutics, AUG 5, 2019, View Source [SID1234538132]).

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"I am pleased with the exciting progress Harpoon has made so far in 2019 with two T cell engagers, HPN424 and HPN536, in the clinic as planned," said Gerald McMahon, Ph.D., President and Chief Executive Officer of Harpoon Therapeutics. "The dose escalation portion of the clinical trial for our lead product candidate, HPN424, continues to advance and we expect to present an interim dataset during the first half of 2020 at an appropriate medical meeting. Our confidence in our TriTAC platform continues to solidify as we learn more about its capabilities and promise."

"Consistent with the TriTAC mechanism of action, we observed T cell activation and cytokine induction with HPN424 treatment, which prompted us to explore the use of dexamethasone as a premedication to limit potential adverse events," said Natalie Sacks, M.D., Chief Medical Officer of Harpoon Therapeutics. "We have found that the addition of weekly dexamethasone premedication, tapered over several weeks, has successfully limited adverse events. When patients completed the scheduled taper, they continued to receive weekly HPN424, without dexamethasone, with no complications observed thus far. This strategy has allowed us to proceed with dose escalations of HPN424 and further advance this promising potential therapy."

Second Quarter 2019 Business Highlights and Other Recent Developments

HPN424, Harpoon’s lead product candidate in development as a potential treatment for prostate cancer, continues to enroll patients in a Phase 1 clinical trial. The treatment regimen has been modified to include premedication with dexamethasone, tapered over several weeks. Several patients have completed the scheduled taper, and have successfully continued treatment with HPN424 in the absence of dexamethasone. Enrollment is ongoing, with patients now being treated in the seventh dose-escalation cohort. Pharmacokinetics observed to date continue to support once-weekly dosing of HPN424. Harpoon plans to present interim results at a medical meeting in the first half of 2020.

In April, Harpoon advanced its second TriTAC, HPN536, a mesothelin-targeting T cell engager, into the clinic and dosed the first patient in a Phase 1/2a clinical trial for ovarian and other mesothelin-expressing solid tumors. Patient enrollment continues as planned and two dosing cohorts have been completed. The study consists of two phases, an initial dose escalation phase of approximately 20 ovarian cancer patients, followed by an expansion phase of up to three additional parallel cohorts of 20 patients each with ovarian, pancreatic and mesothelioma cancer. The study is collecting data to evaluate the safety, tolerability, pharmacokinetics and activity of HPN536. For additional information about the trial, please visit clinicaltrials.gov using the identifier NCT03872206.

Anticipated Milestones

HPN424 – present interim Phase 1 data in the first half of 2020 at a medical conference and initiate expansion cohort in 2020

HPN536 – present proof of concept data in 2020

HPN217 – submit IND by the end of 2019 and initiate Phase 1 trial in the first quarter of 2020

HPN328 – initiate Phase 1 trial in 2020

Second Quarter Financial Results

Harpoon Therapeutics ended the second quarter of 2019 with $133.9 million in cash, cash equivalents, and marketable securities compared to $89.5 million as of December 31, 2018. The increase was due to approximately $71 million in net proceeds from Harpoon’s initial public offering, completed in February 2019, partially offset by cash used in operations.

Net loss for the second quarter ended June 30, 2019 was $11.8 million compared to $6.0 million for the second quarter ended June 30, 2018. The net loss for the six months ended June 30, 2019 was $25.4 million compared to $10.9 million in the first six months of the prior year.

Revenue for the second quarter of 2019 was $1.1 million compared to $1.1 million for the second quarter of 2018. For the six months ended June 30, 2019, revenue was $2.1 million compared to $2.6 million for the six months ended June 30, 2018. For the six months ended June 30, 2019, the decrease was due to an upfront payment of $0.5 million recognized in the first quarter of 2018 related to our license agreement with Werewolf Therapeutics, Inc. During both the three and six month periods, revenue primarily consisted of the amortized portion of the deferred $17.0 million upfront payment received in October 2017 under the collaboration agreement with AbbVie.

Research and development (R&D) expense for the second quarter of 2019 was $10.0 million compared to $6.2 million for the second quarter of 2018. For the six months ended June 30, 2019, R&D expense was $19.4 million, compared to $11.7 million for the six months ended June 30, 2018. The increase for both periods primarily arose from clinical development expenses and an increase in personnel-related expenses, which included conducting preclinical studies, the continuation of the clinical trials for HPN424 and HPN536, and manufacturing activities for four TriTAC product candidates in various stages of development.

General and administrative (G&A) expense for the second quarter of 2019 was $3.7 million compared to $1.0 million for the second quarter of 2018. G&A expense for the six months ended June 30, 2019 was $9.6 million compared to $1.9 million for the six months ended June 30, 2018. The increase for both periods was due to an increase in consulting and accounting services primarily related to the 2018 audit, legal fees, headcount, and other professional services to support our ongoing operations as a public company.

On August 5, 2019 Eagle Pharmaceuticals, Inc. ("Eagle" or the "Company") (Nasdaq: EGRX) reported a clinical development plan to support the submission of a New Drug Application (NDA) for the Company’s innovative fulvestrant formulation (Press release, Eagle Pharmaceuticals, AUG 5, 2019, View Source [SID1234538131]). Fulvestrant, an estrogen receptor antagonist with no agonist properties, is approved by the U.S. Food and Drug Administration (FDA) for the treatment of advanced hormone-related breast cancers. The therapeutic effect of fulvestrant relies on its ability to inhibit estrogen receptors (ER) in cancer cells by binding to and downregulating, or blocking, the ER in breast cancer cells. Recent studies have shown that higher residual ER availability is associated with early disease progression.

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Eagle’s original formulation of fulvestrant was studied in a clinical trial conducted in 2018 in healthy post-menopausal women. The study was conducted in 600 subjects over 140 days; 300 subjects received the branded product FASLODEX and 300 subjects received Eagle’s formulation. A detailed review of the study data led to the hypothesis that the unique properties of Eagle’s formulation would potentially allow for greater inhibition of estrogen receptors. Based on this hypothesis, Eagle has recently completed additional work designed to further enhance its proprietary drug formulation.

In March and June 2019, Eagle met with the FDA and mutually agreed to a clinical program that could provide an efficient approval pathway for the Company’s fulvestrant formulation. The main goal of the clinical research program is to determine if the unique properties of Eagle’s fulvestrant formulation will result in greater inhibition of estrogen receptors, potentially leading to improved efficacy outcomes, including lower disease progression rates, compared to current treatment options.

Eagle intends to begin a pilot study shortly in healthy female volunteers to evaluate the pharmacokinetics and safety of its novel formulation. Once the pilot study results are reviewed, a clinical pivotal trial designed to evaluate fulvestrant exposure and estrogen receptor inhibition based upon the parameters determined with the FDA will be conducted in a target patient population. Depending on recruitment rates and other factors, Eagle believes the pivotal study could be completed within approximately 12 months of commencing enrollment.

"The opportunity to develop a new treatment option that has the potential to better address the epidemic of breast cancer affecting millions of women in the U.S. and worldwide is an important priority for Eagle. Following a thorough review of the data from our previous study, we believe that our fulvestrant product has a unique profile that may allow it to achieve a greater level of estrogen receptor inhibition, and we are encouraged by the guidance provided by FDA to develop a clinical path to further explore the potential of our novel formulation. If successful, the Eagle product could provide a meaningful improvement and a new option to treat this breast cancer patient population," stated Scott Tarriff, Chief Executive Officer of Eagle Pharmaceuticals.

Breast cancer is the most commonly diagnosed cancer in women, with more than 2.8 million breast cancer survivors in the U.S. today; approximately 290,000 women are diagnosed in the U.S. annually. Hormone receptor-positive (HR+) breast cancer is the most common clinical subtype, with the estrogen receptor (ER) being expressed in approximately 70% of those diagnosed.

About Fulvestrant

Fulvestrant is indicated as a monotherapy treatment of hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced breast cancer in postmenopausal women not previously treated with endocrine therapy, or HR-positive advanced breast cancer in postmenopausal women with disease progression following endocrine therapy, or as a combination therapy for the treatment of: (1) HR-positive, HER2-negative advanced or metastatic breast cancer in postmenopausal women in combination with ribociclib as initial endocrine based therapy or following disease progression on endocrine therapy, or (2) HR-positive, HER2-negative advanced or metastatic breast cancer in combination with palbociclib or abemaciclib in women with disease progression after endocrine therapy.

On August 5, 2019 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported financial results for the second quarter 2019 and provided an operational update (Press release, Bellicum Pharmaceuticals, AUG 5, 2019, View Source [SID1234538130]).

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"Thus far in 2019, Bellicum has made significant progress in each of its development programs," said Rick Fair, President and Chief Executive Officer of Bellicum Pharmaceuticals. "We presented encouraging interim data at ASCO (Free ASCO Whitepaper) on our BPX-601 GoCAR-T product candidate, advanced towards a Phase 1 study for BPX-603, our dual-switch HER2-targeted GoCAR-T product candidate, and announced that rivo-cel achieved the primary endpoint in its European registrational study. Looking forward, we have strategically prioritized our GoCAR-T programs and plan to enroll these trials to evaluate how our technology may help extend the impact of CAR-T therapies to the treatment of solid tumors."

PROGRAM HIGHLIGHTS AND CURRENT UPDATES

BPX-601 GoCAR-T

Bellicum presented updated safety and activity data for BPX-601 from a Phase 1/2 study in patients with metastatic pancreatic cancer expressing prostate stem cell antigen (PSCA) at the 2019 American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. The data showed a favorable safety profile—with no dose-limiting toxicities—and provided further evidence that GoCAR-T technology boosts expansion and persistence of CAR-T cells in patients. Of 13 patients evaluable for efficacy treated with BPX-601 and a single dose of rimiducid, 8 patients (62%) achieved stable disease, including 3 with tumor shrinkage of 10% to 24%. As a next step in the study, Bellicum is currently enrolling an additional cohort to evaluate repeat rimiducid dosing to re-activate iMC over time, which is intended to deepen and extend the treatment effect. Initial results from this cohort are expected in late 2019 or early 2020.
Controllable Dual-Switch GoCAR-T Product Candidates

Bellicum believes that its next-generation dual-switch GoCAR-T technology may enhance efficacy relative to current generation CAR-T therapy through iMC activation while enabling clinicians to manage certain treatment-emergent toxicities with CaspaCIDe. The company expects IND clearance for BPX-603, a dual-switch GoCAR-T targeting HER2-expressing solid tumors, later this year. The company also expects to submit an IND application for BPX-802, a dual-switch GoCAR-T product candidate targeting an antigen expressed in hematological malignancies.
Rivo-cel

In July, the company announced that rivo-cel achieved the primary endpoint (Event Free Survival at 180 days) and all secondary endpoints in its BP-004 European registrational trial. Data from this trial is expected to form the basis of anticipated submissions of European Marketing Authorisation Applications (MAAs) for rivo-cel and rimiducid in support of potential regulatory approval. The company is actively seeking a partnership for the continued development and commercialization of rivo-cel.
Second Quarter 2019 Financial Results

Cash Position and Guidance: Bellicum reported cash, restricted cash and investments totaling $60.6 million as of June 30, 2019, compared to $98.0 million at December 31, 2018. Based on current operating plans, Bellicum expects that current cash resources will be sufficient to meet operating requirements through at least the end of 2019. During the second quarter, Bellicum utilized its at-the-market financing facility selling 1.2 million shares for net cash proceeds of $4.4 million.

R&D Expenses: Research and development (R&D) expenses were $19.9 million for the second quarter of 2019, compared to $18.4 million for the second quarter of 2018. The higher expenses in the second quarter of 2019 resulted primarily from higher expenditures related to the GoCAR-T platform including initiation of additional clinical sites and costs related to IND filing. R&D expenses for the six months ended June 30, 2019 were $36.7 million compared to $34.9 million for the comparable period in the prior year.

G&A Expenses: General and administrative (G&A) expenses were $7.5 million for the second quarter of 2019 compared to $5.4 million during the comparable period in 2018. The higher expenses in the second quarter 2019 relative to the comparable period in 2018 were primarily due to increased personnel related costs and commercialization preparation activities. G&A expenses for the six months ended June 30, 2019 were $15.1 million compared to $11.1 million for the first six months of 2018.

Net Loss: Bellicum reported a net loss of $26.9 million for the second quarter of 2019 compared to a net loss of $24.2 million for the second quarter of 2018. The results included non-cash, share-based compensation charges of $2.0 million and $3.6 million for the second quarter of 2019 and 2018, respectively. Net loss for the six months ended June 30, 2019 was $51.5 million compared to a loss of $47.0 million for the six months ended June 30, 2018.

Shares Outstanding: At July 31, 2019, Bellicum had 46,254,163 shares of common stock outstanding.

About BPX-601

BPX-601, the company’s first GoCAR-T product candidate, incorporates iMC, Bellicum’s inducible co-activation domain. iMC (inducible MyD88/CD40) is designed to provide a powerful boost to T cell proliferation and persistence and enable the CAR-T to override key immune inhibitory mechanisms, including PD-1 and TGF-beta. BPX-601 is being evaluated as a treatment for solid tumors expressing prostate stem cell antigen (PSCA), including pancreatic, gastric, and prostate cancers.

About Rivo-cel (BPX-501)

Rivo-celTM (rivogenlecleucel) is an allogeneic polyclonal T cell product designed to reduce the rate of relapse of leukemia following a stem cell transplant. The cell treatment contains a diverse repertoire of T cells, which may contribute to a robust graft vs. leukemia effect. Rivo-cel’s antiviral benefits may also reduce morbidity and mortality in patients susceptible to infection following a transplant. The product’s CaspaCIDe safety switch enables this approach by allowing physicians to reduce the number of alloreactive cells in the event of uncontrolled GvHD. Rivo-cel addresses a major unmet need in adult and pediatric leukemia, lymphoma and genetic blood disease patients following a haploidentical stem cell transplant.

On August 5, 2019 Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) reported financial results for its fiscal 2019 third quarter ended June 30, 2019 (Press release, Arrowhead Research Corporation, AUG 5, 2019, View Source [SID1234538129]). The company is hosting a conference call at 4:30 p.m. EDT to discuss results.

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Conference Call and Webcast Details

Investors may access a live audio webcast on the Company’s website at View Source For analysts that wish to participate in the conference call, please dial 855-215-6159 or 315-625-6887 and provide Conference ID 3249189.

A replay of the webcast will be available on the company’s website approximately two hours after the conclusion of the call and will remain available for 90 days. An audio replay will also be available approximately two hours after the conclusion of the call and will be available for 3 days. To access the audio replay, dial 855-859-2056 or 404-537-3406 and provide Conference ID 3249189.

Selected Fiscal 2019 Third Quarter and Recent Events

Received U.S. Food and Drug Administration (FDA) clearance to begin an adaptive design Phase 2/3 trial, called SEQUOIA, with the potential to serve as a pivotal registrational study of ARO-AAT, Arrowhead’s second generation subcutaneously administered RNAi therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency

Secured first regulatory clearance in the United Kingdom for the ARO-AAT 2002 study, a pilot open-label, multi-dose, Phase 2 study to assess changes in a novel histological

activity scale in response to ARO-AAT over time in patients with alpha-1 antitrypsin deficiency associated liver disease

Expanded the AROHBV1001 Phase 1/2 study to include a new triple combination cohort that includes: JNJ-3989, formerly ARO-HBV; JNJ-6379, Janssen’s investigational orally administered capsid assembly modulator of the class that forms normal capsid structures; and, a nucleos(t)ide analog, or NUC

In connection with the start of dosing of this cohort, Arrowhead earned a $25 million milestone payment from Janssen

Received orphan drug designation from FDA for ARO-APOC3 for the treatment of familial chylomicronemia syndrome

Received FDA Fast Track designation for ARO-AAT

Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier

Received orphan drug designation from FDA for ARO-ANG3 for the treatment of homozygous familial hypercholesterolemia

Completed discovery and development work on ARO-HSD, a previously undisclosed liver-targeted candidate targeting HSD17B13, a hydroxysteroid dehydrogenase involved in the metabolism of hormones, fatty acids and bile acids, that is now in IND-enabling GLP-toxicology studies, and, pending success in the tox program, on schedule for a CTA filing at the end of 2019

Started IND-enabling GLP-toxicology studies for ARO-HIF2, designed to inhibit the production of HIF-2a for the treatment of clear cell renal cell carcinoma, to support, pending success in the tox program, a CTA filing at the end of 2019

Completed dosing in the single-ascending dose portions of the Phase 1 studies of Arrowhead’s two wholly-owned cardiometabolic candidates, ARO-APOC3 and ARO-ANG3, and progressed towards the multiple-dose portions of the Phase 1 studies in various patient populations