On May 3, 2018 C4 Therapeutics (C4T) reported that Andrew Phillips, Ph.D. has been appointed as President and Chief Executive Officer (Press release, C4 Therapeutics, MAY 3, 2018, View Source [SID1234526071]). Dr. Phillips has been leading C4T’s scientific efforts as Chief Scientific Officer since January 2016 and assumed corporate leadership responsibilities with his added appointment as President in September 2016. In conjunction with Dr. Phillips appointment as CEO, Stewart Fisher, Ph.D., has been promoted to Chief Scientific Officer.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Andy has played a key role in C4T’s growth and has helped define who we are today as a Company," said Marc Cohen, co-founder and Executive Chairman of C4T. "In addition to being a world-class scientist, Andy has provided exemplary leadership in recruiting a phenomenal management team, developing a robust internal pipeline of projects, and nurturing our strategic alliances with Roche and Calico. I look forward to working with Andy as CEO as we continue to build C4T’s targeted protein degradation platform and advance degraders into the clinic to address areas of high unmet medical need."

Prior to joining C4T, Dr. Phillips served as Senior Director for the Center for Development of Therapeutics at the Broad Institute of MIT and Harvard, where he led overall therapeutic efforts and provided strategic leadership for a number of major partnerships. Previously, he was a Full Professor of Chemistry at Yale University, where he received the ACS Cope Scholar Award for his research accomplishments which included the development of small molecules aimed at modulating ‘undruggable’ targets. Prior to this, he was a Full Professor of Chemistry and Biochemistry at the University of Colorado at Boulder, where his efforts in complex molecule synthesis and targeting protein-protein interactions garnered a number of awards, including an Alfred P. Sloan Research Fellowship, an Eli Lilly Grantee Award, and a National Science Foundation CAREER Award.

Dr. Phillips received a B.Sc. (Hons) in biochemistry and a Ph.D. in biochemistry and chemistry from the University of Canterbury in New Zealand and completed a postdoctoral fellowship in organic chemistry at the University of Pittsburgh.

On May 3, 2018 Spectrum Pharmaceuticals, Inc. (NasdaqGS: SPPI), a biotechnology company with fully integrated commercial and drug development operations with a primary focus in hematology and oncology, reported financial results for the three-month period ended March 31, 2018 (Press release, Spectrum Pharmaceuticals, MAY 3, 2018, View Source [SID1234526089]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We had a productive first quarter as we continued to advance our lead pipeline drugs poziotinib and ROLONTIS," said Joe Turgeon, President and Chief Executive Officer of Spectrum Pharmaceuticals. "We were honored to have poziotinib data appear in Nature Medicine, be presented at AACR (Free AACR Whitepaper), and announce our strengthened IP with MD Anderson in addition to releasing ROLONTIS topline data. In the next few months, we look forward to announcing important clinical data from both therapies at key scientific meetings."

Clinical Program Update:

Poziotinib, an irreversible tyrosine kinase inhibitor:

The company has initiated a multi-center study which is currently enrolling non-small cell lung cancer (NSCLC) patients. This trial will enroll up to 87 patients with EGFR exon 20 insertion mutations and up to 87 patients with HER2 exon 20 insertion mutations at leading cancer centers throughout U.S. The study will evaluate objective response rate (ORR) as the primary endpoint, disease control rate (DCR), duration of response (DOR), progression free survival (PFS), quality of life (QOL) and safety as additional endpoints.
An investigator sponsored trial is ongoing at the University of Texas MD Anderson Cancer Center in NSCLC patients with exon 20 mutations in EGFR or HER2. The 50 patient EGFR cohort is fully enrolled.
In an April 2018 Nature Medicine publication, updated data from the first 11 NSCLC patients with EGFR exon 20 mutations receiving poziotinib in MD Anderson’s Phase 2 clinical trial showed a confirmed objective response rate of 64 percent. As noted in the publication, the median progression-free survival had not been reached, with a median follow up of 6.6 months. The safety profile was consistent with those previously described for poziotinib and other TKIs. The company expects additional data from this study at the World Conference on Lung Cancer, in Toronto (September 23-26, 2018).
Data presented at AACR (Free AACR Whitepaper) in April 2018, demonstrated pre-clinical and early clinical activity of poziotinib in HER2 exon 20 mutant NSCLC, suggesting poziotinib could be a promising agent for the numerous cancer types driven by HER2 exon 20 mutations.
The company is planning a basket trial to study poziotinib in exon 20 mutations across several solid tumors.
The company has entered into an exclusive licensing agreement with MD Anderson which strengthens and extends intellectual property on poziotinib. The filed patents, if granted, will extend until 2037.
ROLONTIS(eflapegrastim), a novel long-acting GCSF:

A registrational Phase 3 study, ADVANCE, was initiated under a special protocol assessment with the FDA last year to evaluate ROLONTIS in the management of chemotherapy-induced neutropenia.
The company announced the ADVANCE study met the primary efficacy endpoint of non-inferiority in duration of severe neutropenia between ROLONTIS and pegfilgrastim. The adverse event profile was similar between the two treatment arms. Phase 3 data abstract to be released by American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) followed by an oral presentation at Multinational Association of Supportive Care in Cancer (MASCC) in June 2018.
The company has completely enrolled RECOVER, an international Phase 3 study that has a design similar to ADVANCE.
Spectrum is working toward a pre-BLA meeting with the FDA to ensure alignment in preparation for a planned Q4 BLA submission.
Financial Guidance

The company has also raised guidance for 2018. Expected 2018 revenue will be between $95 million to $115 million, up from previously projected revenue of $90 million to $110 million. Additionally, the company expects its current cash and marketable securities to be sufficient to fund operations into 2020.

Three-Month Period Ended March 31, 2018 (All numbers are approximate)

GAAP Results

Total product sales were $28.1 million in the first quarter of 2018. Product sales in the first quarter included: FOLOTYN (pralatrexate injection) net sales of $12.7 million, EVOMELA (melphalan) for injection net sales of $8.1 million, BELEODAQ (belinostat) for injection net sales of $2.7 million, ZEVALIN (ibritumomab tiuxetan) net sales of $3.0 million, MARQIBO (vinCRIStine sulfate LIPOSOME injection) net sales of $0.9 million, and FUSILEV (levoleucovorin) net sales of $0.6 million.

Spectrum recorded net loss of $15.8 million, or $0.16 per basic and diluted share in the three-month period ended March 31, 2018, compared to net loss of $23.5 million, or $0.30 per basic and diluted share in the comparable period in 2017. Total research and development expenses were $17.9 million in the quarter, as compared to $14.8 million in the same period in 2017. Selling, general and administrative expenses were $24.1 million in the quarter, compared to $19.1 million in the same period in 2017.

Non-GAAP Results

Spectrum recorded non-GAAP net loss of $14.7 million, or $0.15 per basic and diluted share in the three-month period ended March 31, 2018, compared to non-GAAP net loss of $11.4 million, or $0.14 per basic and diluted share in the comparable period in 2017. Non-GAAP research and development expenses were $17.1 million, as compared to $14.3 million in the same period of 2017. Non-GAAP selling, general and administrative expenses were $20.4 million, as compared to $15.7 million in the same period in 2017.

Conference Call

Thursday, May 3, 2018 @ 4:30 p.m. Eastern/1:30 p.m. Pacific

Domestic: (877) 837-3910, Conference ID# 8765418

International: (973) 796-5077, Conference ID# 8765418

This conference call will also be webcast. Listeners may access the webcast, which will be available on the investor relations page of Spectrum Pharmaceuticals’ website: www.sppirx.com on May 3, 2018 at 4:30 p.m. Eastern/1:30 p.m. Pacific.

On May 3, 2018 Ferring Pharmaceuticals reported the signing of an agreement giving the company the option to secure global commercialisation rights to nadofaragene firadenovec/Syn3 (rAd-IFN/Syn3), a novel gene therapy being developed by FKD Therapies Oy (FKD) as a treatment for patients with high-grade non-muscle invasive bladder cancer (NMIBC), who are unresponsive to Bacillus Calmette-Guérin (BCG) therapy (Press release, Ferring Pharmaceuticals, MAY 3, 2018, View Source [SID1234551851]). This option is exercisable on marketing approval from the US FDA. Ferring will create a new US oncology division with the specialist knowledge and presence to introduce novel advanced therapies to the market.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

rAd-IFN/Syn3 is currently undergoing Phase 3 development in the US under the sponsorship of Finnish gene therapy specialists FKD. The results of the earlier Phase 2 trial, published in the Journal of Clinical Oncology, reported 35% of BCG unresponsive NMIBC bladder cancer patients given one dose of rAd-IFN/Syn3 every three months, were free of high-grade disease at one year1. The ongoing Phase 3 study is designed to establish the efficacy and safety of the product. rAd-IFN/Syn3 has been awarded Fast Track and Breakthrough Therapy designations by the FDA.

"We are excited about the potential to commercialise rAd-IFN/Syn3, a novel gene therapy for bladder cancer patients," said Michel Pettigrew, President of the Executive Board and Chief Operating Officer, Ferring Pharmaceuticals. "The gene therapy sector is growing rapidly and building a presence in this specialised area is a very positive opportunity for Ferring."

Bladder cancer is one of the most frequently occurring cancers with an estimated 430,000 new cases being reported worldwide each year2. It is the fourth most common cancer in men in the US3 and is the most expensive cancer to treat on a life-time basis, with a high burden on patients, their relatives and healthcare systems4. In high-grade NMIBC patients, BCG is the gold standard treatment and although effective, over 60% of cases eventually re-occur5, 6. The outcome for such patients is poor, with total cystectomy (complete removal of the bladder) to prevent the cancer spreading to other organs generally being the next treatment option. As such, the BCG unresponsive population is one of high unmet clinical need.

"Today, bladder cancer patients have very limited medical options and new treatments that delay or prevent total removal of the bladder and improve clinical outcomes are urgently needed for patients," said Professor Klaus Dugi, Chief Medical Officer, Ferring Pharmaceuticals. "Phase 2 clinical results for rAd-IFN/Syn3 were very encouraging and we look forward to the Phase 3 data."

Gene therapy is one of a new class of therapeutic treatments known as advanced therapy medicinal products. rAd-IFN/Syn3 is built on adenoviral vector technology, a non-integrating vector, and results in enhanced expression of the therapeutic protein interferon alfa 2b. To date, it has completed three clinical trials in the US.

About rAd-IFN/Syn3

rAd-IFN/Syn3 (nadofaragene firadenovec/Syn3) is an investigational gene therapy consisting of an adenovirus containing the gene interferon alfa-2b. It is administered by catheter into the bladder, where the virus enters the cells of the bladder wall. Inside the cells, the virus breaks down leaving the active gene to do its work. The internal gene/DNA machinery of the cells picks up the gene and translates its DNA sequence, resulting in the cells secreting high quantities of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This novel gene therapy approach turns the patient’s own bladder wall cells into multiple interferon microfactories, enhancing the body’s natural defences against the cancer. The Phase 3 trial for rAd-IFN/Syn3 opened in 2016 with up to 150 patients to be enrolled across 35 centers in the US.

G1 Therapeutics has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission .

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

On May 3, 2018 Vical Incorporated (Nasdaq:VICL) reported financial results for the three months ended March 31, 2018. Net loss for the first quarter of 2018 was $6.3 million, or $0.29 per share, compared with a net loss of $2.8 million, or $0.25 per share, for the first quarter of 2017 (Press release, Vical, MAY 3, 2018, View Source [SID1234526014]). Revenues for the first quarter of 2018 were $0.7 million, compared with revenues of $3.2 million for the first quarter of 2017, reflecting revenues from Astellas Pharma Inc. for services performed under ASP0113 collaborative agreements.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Vical had cash and investments of $58.3 million at March 31, 2018. The Company’s cash burn for the first quarter of 2018 was $4.6 million, which was consistent with the Company’s full year 2018 guidance of between $20 million and $24 million.

Program updates include:

VCL-HB01 HSV-2 Therapeutic Vaccine

Vical expects to announce top-line results from a Phase 2 study of its HSV-2 therapeutic vaccine during the second quarter of 2018. The last subject in the study has now completed nine months of surveillance; the primary endpoint of annualized recurrence rate will be calculated based on recurrences that are both clinically- and virologically-confirmed. This endpoint provides important information on the number of genital lesion recurrences over time in this chronic disease setting and is clinically meaningful for both patients and treating physicians. The Phase 2 study is being conducted in HSV-2 seropositive healthy adult subjects, 18 to 50 years of age who are randomized 2:1 to receive either vaccine or placebo.
VL-2397 Antifungal Drug

During the first quarter of 2018, Vical initiated a Phase 2 trial comparing VL‑2397 to standard first-line treatment for invasive aspergillosis in immunocompromised adults with acute leukemia or recipients of an allogeneic hematopoietic cell transplant (ClinicalTrials.gov Identifier: NCT03327727). The Company intends to conduct the trial in approximately 40 major cancer and transplantation centers in North America, Europe and Asia. The FDA has advised that VL‑2397 would be eligible for a Limited Use Indication (LUI) approval for the treatment of invasive aspergillosis for patients with limited treatment options. The FDA has also granted Vical Qualified Infectious Disease Product (QIDP), Orphan Drug and Fast Track designations for VL‑2397 for the treatment of invasive aspergillosis. Only one new class of antifungal therapy has been approved in the last 30 years. VL-2397 has a novel mechanism of antifungal action and could be the first therapeutic in a new class of antifungals. VL-2397 was isolated from a leaf litter fungus in a Malaysian national park, and was in‑licensed from Astellas in 2015.
Hepatitis B Virus (HBV) Therapeutic Drug

The Company is pursuing preclinical development of a novel treatment for chronic HBV infection based on its DNA and lipid-delivery technologies. The initial aim of this program will be to demonstrate proof of concept for inhibiting HBV infection in an in vivo model. This preclinical development effort is being conducted in collaboration with Vical’s partner, AnGes, Inc. of Osaka, Japan.
Vical will conduct a conference call and webcast today, May 3, at noon Eastern Time, to discuss the Company’s financial results and program updates. The call and webcast are open on a listen-only basis to any interested parties. To listen to the conference call, dial in approximately ten minutes before the scheduled call to (323)794-2567 (preferred), or (888)278-8469 (toll-free), and reference confirmation code 1443635. A replay of the call will be available for 48 hours beginning about two hours after the call. To listen to the replay, dial (719)457-0820 (preferred) or (888)203-1112 (toll-free) and enter replay passcode 1443635. The webcast will also be available live and archived through the events page at www.vical.com. For further information, contact Vical’s Investor Relations department by phone at (858)646-1127 or by e-mail at [email protected].