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Pieris Pharmaceuticals Reports 2018 Year-End Cash Position and Provides Corporate Update

On March 12, 2019 Pieris Pharmaceuticals, Inc. (NASDAQ: PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for respiratory diseases, cancer and other indications, reported its cash position for the fiscal year ended December 31, 2018 and provided an update on the Company’s recent and future developments (Press release, Pieris Pharmaceuticals, MAR 12, 2019, View Source [SID1234534237]).

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”2018 was an important year for the development of our two core franchises: respiratory diseases and immuno-oncology. On the respiratory front, we announced that PRS-060, an inhaled IL-4 receptor alpha antagonist for moderate-to-severe asthma that we are developing with AstraZeneca and our first respiratory program to enter the clinic, was safe and well-tolerated in healthy volunteers in a single ascending dose phase 1 study. We also initiated a multiple ascending dose phase 1 study of PRS-060 in mild asthmatics, in addition to launching discovery efforts for two proprietary respiratory programs and two additional respiratory programs as part of the AstraZeneca collaboration. On the immuno-oncology front, we continue to enroll and dose patients in the phase 1 dose-escalation study of PRS-343, a 4-1BB/HER2 bispecific for HER2-positive solid tumors, and the phase 1 combination study of PRS-343 with atezolizumab. We also advanced PRS-344, a PD-L1/4-1BB bispecific drug candidate we are developing with Servier, into IND-enabling studies and plan to file an IND application for that program later this year,” said Stephen S. Yoder, President and CEO of Pieris. ”We believe that the tireless effort of our team and partners across our clinical and preclinical pipeline last year has created an opportunity for many catalysts this year.”

PRS-060: Pieris continues to enroll subjects with mild asthma and elevated levels of fractional exhaled nitric oxide (FeNO) in a multiple ascending dose phase 1 study of PRS-060, an inhaled IL-4 receptor alpha antagonist for moderate-to-severe asthma. This study is evaluating the safety, tolerability and FeNO-reducing potential of PRS-060 versus placebo. The data from the PRS-060 phase 1 studies will be presented at upcoming medical meetings, including detailed data from the PRS-060 phase 1 single-ascending dose study, for which the Company reported topline data last year. PRS-060 is the lead candidate in Pieris’ respiratory collaboration with AstraZeneca. Pieris is sponsoring the phase 1 studies and AstraZeneca is funding the costs. Assuming successful completion of the ongoing phase 1 study, AstraZeneca would sponsor and fund the phase 2a study, after which Pieris will have options to co-develop and, subsequently, to co-commercialize the drug candidate.
Respiratory Pipeline: Pieris initiated an additional discovery-stage program in its alliance with AstraZeneca in the fourth quarter, bringing the total number of active programs to three; AstraZeneca may initiate up to two additional programs within the alliance. The Company also continues to advance the two proprietary discovery-stage respiratory programs initiated last year and intends to initiate additional proprietary respiratory programs in 2019.
PRS-343: Pieris continues to enroll and treat patients in a phase 1 dose-escalation study of PRS-343, a 4-1BB/HER2 bispecific for HER2-positive solid tumors, and intends to report comprehensive data from the study later this year. The Company also continues to enroll the dose-escalation phase 1 study of PRS-343 in combination with atezolizumab and intends to report data from this trial later this year.
PRS-344: Pieris has exercised its opt-in right to co-develop and retain U.S. rights for PRS-344, a PD-L1/4-1BB antibody-Anticalin bispecific molecule, and achieved two preclinical milestones as part of the development of the drug candidate, receiving milestone payments totaling €2.0 million from Servier. In collaboration with Servier, Pieris plans to file an IND application for the candidate this year. PRS-344 is one of five bispecific programs that Pieris is developing as part of its immuno-oncology alliance with Servier and is the first program within the alliance scheduled to enter clinical development.
Seattle Genetics Collaboration: Pieris has generated and characterized the first tumor-targeting bispecific for further evaluation and development by Seattle Genetics as part of the companies’ three-program immuno-oncology collaboration.
PRS-080: Pieris has completed dosing all patients in the phase 2a multiple ascending dose study of PRS-080, a half-life-optimized hepcidin antagonist for anemia. This study is intended to evaluate the safety, tolerability, and pharmacological activity of 5 once-weekly doses of PRS-080 as well as the effect of repeated dosing on hemoglobin levels in this patient population. Pieris intends to present the full data set from this study in the first half of 2019. In addition, following delivery of a final study report, ASKA will decide whether to exercise its option to develop and commercialize PRS-080 in Japan and other Asian territories.
Cash Position: Cash, cash equivalents and investments totaled $128.1 million as of December 31, 2018, compared to a cash, cash equivalents and investments balance of $82.6 million as of December 31, 2017. The increase was driven primarily by the $47.2 million in net proceeds from the Company’s February 2018 equity financing, the $30.0 million in upfront payments received as part of the Seattle Genetics immuno-oncology collaboration, and the $12.5 million milestone payment from AstraZeneca that was triggered during the fourth quarter of 2017 and received during the first quarter of 2018.
Additional Financials: Our 2018 full-year audited financials will be released with our Annual Report on Form 10-K, expected to be filed by Monday, March 18, 2019.
Conference Call:

Pieris management will host a conference call beginning at 8:00 AM Eastern Daylight Time on Tuesday, March 12, 2019, to provide a corporate update. Individuals can join the call by dialing +1-877-407-8920 (US & Canada) or +1-412-902-1010 (International). An archived replay of the call will be available by dialing +1-877-660-6853 (US & Canada) or +1-201-612-7415 (International) and providing the Conference ID #: 13661472.

RadioMedix and Vect-Horus Announce Signing of a Letter of Intent to Co-Develop a Radio-Theranostic Agent for Glioblastoma

On March 12, 2019 Vect-Horus and RadioMedix reported the signing of a Letter of Intent (LOI) to establish an agreement for the co-development of a Vect-Horus theranostic agent for the diagnostic and radiotherapy of Glioblastoma Multiforme (GBM) of the brain (Press release, RadioMedix, MAR 12, 2019, View Source [SID1234534235]).

This partnership will marry the expertise of Vect-Horus in targeting tumors with its technology VECTrans and the know-how of RadioMedix in developing and conducting pre-clinical evaluation and clinical trials with radiopharmaceuticals.

GBM is the most common and most aggressive malignant type of primary brain tumors and is a serious and life-threatening condition. The theranostic agent targets the Low-Density Lipoprotein Receptor (LDLR), which is highly expressed on many cancer cells, including glioblastoma. Positron Emission Tomography (PET) imaging and biodistribution studies in human glioblastoma xenograft and orthotopic models have shown a significant accumulation of the agent within the tumor.

"We are pleased that our technology, VECTrans in combination with expertise of RadioMedix will serve to advance an innovative and promising approach for glioblastoma treatment." said Alexandre Tokay, CEO of Vect-Horus "This co-development project is fully aligned with our strategy to expand the use of our technology in cancer indications with high unmet medical needs."
"We look forward to very compelling outcomes from our co-development project, as we seek to create new technologies for the benefit of cancer patients, by giving doctors and hospitals more imaging and treatment options." Said D.r Jamal Temsamani, Director of Drug Development of Vect-Horus.

"Development of a highly sensitive and effective theranostic approach for GBM has always been among our top priorities. Our initial evaluation of VECTrans technology appears to be extremely promising." said D.r Ebrahim Delpassand, CEO of RadioMedix. "Our plan is to utilize VECTrans technology to initially develop an accurate molecular diagnostic PET/CT probe to precisely map the tumor involvement in the brain. This will significantly improvethe precision of the surgical resection of the tumor. The treatment will then be followed by targeted alpha-emitter or beta-emitter radionuclide therapy using the same technology to eradicate any residual malignant cells at the molecular level" added Dr. Delpassand.

"RadioMedix has strong interests and all expertise needed to develop the VECTrans–based radio-theranostic agents for GBM", said Izabela Tworowska, Ph.D., CSO of RadioMedix. "Targeting LDLR can improve the blood-brain barrier (BBB) permeability of the drug and selectively deliver the isotope payload to GBM cancer cells."

Synlogic Reports Fourth Quarter and Full Year 2018 Financial Results and Provides Business Update

On March 12, 2019 Synlogic, Inc. (Nasdaq: SYBX), a clinical stage company applying synthetic biology to probiotics to develop novel, living medicines, reported its financial results for the fourth quarter and full year ended December 31, 2018 (Press release, Synlogic, MAR 12, 2019, View Source [SID1234534234]).

"2019 is an exciting year for Synlogic as we continue to advance out platform and programs," said Aoife Brennan, M.B., Ch.B. Synlogic’s president and chief executive officer. "Data expected mid-year from clinical trials of SYNB1020 and SYNB1618, in patients with hyperammonemia and phenylketonuria, respectively, will guide our plans for future development of our Synthetic Biotic medicines in these areas and additional rare metabolic diseases. We are also exploring new applications of our platform as we advance our first immuno-oncology program toward submission of an Investigational New Drug (IND) application and into clinical studies. Furthermore, with our increased capability to manufacture clinical trial material, we are now in a position to advance our programs through development as expeditiously as possible."

2019 Priorities
Pipeline

Presentation of top-line data from Phase 1b/2a study of SYNB1020 in patients with cirrhosis and elevated blood ammonia expected by mid-year. The double-blind, placebo-controlled study is designed to assess safety and tolerability, the ability of orally administered SYNB1020 to lower blood ammonia as well as its effect on several exploratory measures associated with hyperammonemia in this population. These data will provide valuable information for both platform development and the development path for Synlogic’s hyperammonemia program.
Presentation of top-line data from study to evaluate SYNB1618 in patients with PKU expectedby mid-year. The second part of this double-blind, placebo-controlled study is designed to assess safety and tolerability of orally administered SYNB1618 in a single dose (N=4) and multiple dose cohort (N=10) of patients with phenylketonuria (PKU). Additional endpoints of the study will explore the production of biomarkers of SYNB1618 activity that are expected to provide information as to the differences in pharmacodynamics of the Synthetic Biotic medicine in patients versus healthy volunteers.
Advancement of SYNB1891 immuno-oncology program candidate to enable filing of an IND application in the second half of the year. SYNB1891 is an intra-tumorally administered Synthetic Biotic medicine engineered to produce cyclic di-AMP (CDA), an agonist of the STING pathway, that is designed to serve as a dual innate activator of the immune system as a potential treatment for solid tumors.
Continued progress and refinement of manufacturing and process development. Synlogic is developing and manufacturing solid oral Synthetic Biotic formulations suitable for Phase 2 clinical trials and beyond.
Advancement of new research programs to expand product pipeline.
Presentation and publication of data at major scientific and medical meetings.Synlogic is committed to publishing and presenting data that demonstrate the breadth of Synlogic’s Synthetic Biotic platform.
Advancement of collaboration. On March 6, 2019, Synlogic announcedthat its collaboration with AbbVie to develop a Synthetic Biotic medicine for the treatment of inflammatory bowel disease (IBD) had advanced into lead optimization triggering a milestone payment to Synlogic. The two companies will work together to develop and enable the selection of a suitable candidate for entry into IND-enabling studies.
Corporate

Continued strengthening of Synlogic’s leadership. In February 2019, Synlogic announced the appointment of Patricia N. Hurter, Ph.D.,Senior Vice President of Pharmaceutical and Preclinical Sciences at Vertex Pharmaceuticals, Inc.,to its board of directors. Synlogic is also conducting a search to fill the position of Chief Medical Officer vacated by Dr. Brennan.
Continued exploration of additional strategic opportunities. Synlogic expects to develop additional strategic collaborations to expand the reach of the Synthetic Biotic platform.
Fourth Quarter 2018 Financial Results
For the three months ended December 31, 2018, Synlogic reported a consolidated net loss of $11.9 million, or $0.47 per share, compared to a net loss of $11.7 million, or $0.74 per share, for the corresponding period in 2017.

Research and development expenses were $8.9 million for the three months ended December 31, 2018 compared to $7.7 million for the corresponding period in 2017. The increase was primarily due to an increase in compensation-related expenses associated with increased headcount and increases in expenses related to the lease of a larger facility at 301 Binney Street in Cambridge, Massachusetts, which Synlogic occupied in January 2018.

General and administrative expenses for the three months ended December 31, 2018 were $4.0 million compared to $4.3 million for the corresponding period in 2017. The decrease was primarily due to decreases in professional fees such as audit, legal and tax services, partially offset by increases in compensation-related expenses associated with increased headcount and increases in expenses related to the lease of Synlogic’s facility at 301 Binney Street.

Revenue was $0.1 million for the three months ended December 31, 2018 and December 31, 2017. Revenue is associated with the payments received for services performed under the Synlogic’s collaboration with AbbVie to develop a Synthetic Biotic medicine for the treatment of IBD.

As of December 31, 2018, Synlogic had cash, cash equivalents, and short-term investments of $122.7 million.

Full Year 2018 Financial Results
For the year ended December 31, 2018, consolidated net loss was $48.4 million, or $2.03 per share, compared to a consolidated net loss of $40.4 million, or $6.00 per share, for the year ended December 31, 2017. Revenues were $2.5 million for the year ended December 31, 2018, compared to $2.4 million for the same period in 2017. Total operating expenses were $53.8 million for the year ended December 31, 2018, compared to $43.3 million for the same period in 2017. The increase in operating expenses was primarily due to compensation-related expenses associated with increased headcount, increased expenses related to the lease of Synlogic’s facility at 301 Binney Street and increased external costs associated with development of Synlogic’s Synthetic Biotic programs, including process and formulation development, pre-clinical and clinical studies.

Conference Call & Webcast Information
Synlogic will host a conference call and live webcast today at 8:00 a.m. ET today, Tuesday, March 12, 2019. To access the live webcast, please visit the "Event Calendar" page within the Investors and Media section of the Synlogic website. Alternatively, investors may listen to the call by dialing +1 (844) 815-2882 from locations in the United States or +1 (213) 660-0926 from outside the United States. The conference ID number is 2181868. For those unable to participate in the conference call or webcast, a replay will be available for 30 days on the Investors and Media section of the Synlogic website.

Bellicum Pharmaceuticals Provides Operational Update and Reports Financial Results for the Fourth Quarter and Year Ended December 31, 2018

On March, 12 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 fourth quarter and full year ended December 31, 2018 (Press release, Bellicum Pharmaceuticals, MAR 12, 2019, View Source [SID1234534233]).

"We believe that Bellicum is well-positioned for an exciting and eventful 2019 as a result of our investments to strengthen our clinical development and executive teams over the past 24 months," said Bellicum’s President & CEO Rick Fair. "Based on promising preclinical and initial clinical data on BPX-601, we have prioritized GoCAR-T candidates incorporating our iMC activation switch in our clinical development strategy. In addition to progressing our Phase 1/2 trial of BPX-601, we intend to submit IND applications for two novel, dual-switch GoCAR-T clinical programs in 2019. For rivo-cel, we anticipate topline Phase 2 results in pediatric patients in the second quarter of 2019, which will form the basis for European Marketing Authorisation Application (MAA) submissions planned for late 2019."

PROGRAM HIGHLIGHTS AND CURRENT UPDATES

Bellicum Advances and Expands Clinical Program for GoCAR-T Platform

BPX-601

Bellicum completed enrollment in the dose-escalation portion of its ongoing Phase 1/2 study of BPX-601 in patients with advanced pancreatic cancer expressing prostate stem cell antigen (PSCA). Bellicum presented positive initial clinical data from Part 1 of the study at the ESMO (Free ESMO Whitepaper) Immuno-Oncology Congress in December 2018 and the Gastrointestinal Cancers Symposium in January 2019 that showed a promising safety profile. In several patients reported, enhanced cell expansion, prolonged cell persistence, and early evidence of clinical activity and disease control were observed. The trial protocol has been amended to add the complete lymphodepletion conditioning regimen cyclophosphamide/fludarabine (Cy/Flu) and to include patients with gastric and prostate cancers. Once this safety cohort has been completed, Bellicum plans to incorporate 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 these cohorts are expected in 2019.
Controllable Dual-Switch Candidates

BPX-603 is Bellicum’s first controllable dual-switch GoCAR-T product candidate, which incorporates both the iMC activation switch and the CaspaCIDe safety switch. BPX-603 is designed to target solid tumors that express the human epidermal growth factor receptor 2 antigen (HER2). HER2 is a validated antigen for cancer therapies, and academic CAR-T cell clinical studies have shown evidence of antitumor activity. These CAR-T approaches targeting HER2 have been limited by modest clinical efficacy and off-tumor/on-target toxicity. Bellicum believes that its dual-switch GoCAR-T technology may be uniquely suited to improve upon these earlier efforts by driving greater efficacy through iMC activation while enabling clinicians to manage any treatment-emergent toxicities with CaspaCIDe. Bellicum expects to submit an IND application for BPX-603 and to initiate a clinical trial in 2019. BPX-802 is a dual-switch GoCAR-T product candidate targeting an antigen expressed in hematological malignancies. Bellicum expects to submit an IND application for BPX-802 in late 2019.
Company Prepares for Rivo-cel Registration in E.U. for Pediatric Patients and Initiates Global Trial for Adult and Adolescent Patients

Bellicum presented late interim analyses of the pediatric study BP-004 at ASH (Free ASH Whitepaper) in December 2018. The results demonstrated that 90.9% of rivo-cel treated patients experienced Event Free Survival (EFS) at 180 days compared to 87.7% of patients undergoing matched unrelated donor (MUD) HSCT. Non-inferiority on EFS versus MUD HSCT is the primary endpoint for European regulatory review. The results also demonstrated that rivo-cel treated patients had high rates of relapse-free (82.9%) and overall (94.4%) survival with a median follow up of 20 months. Lastly, the results demonstrated a best overall response rate of 70% in patients that developed advanced or steroid-refractory GvHD when treated with rimiducid, with the majority being complete responses. The Company expects topline results from the BP-004 study in the second quarter of 2019 and intends to submit MAAs for rivo-cel and rimiducid in late 2019. The Company recently initiated THRIVE, a pivotal randomized global Phase 2/3 clinical trial of rivo-cel in adult and adolescent patients 12 years and older with intermediate- and high-risk acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). In the Phase 3 portion of the trial, patients will be randomized to receive either a/b T cell and B-cell depleted haploidentical HSCT plus rivo-cel without GvHD prophylaxis, or a haploidentical HSCT followed by cyclophosphamide post-transplant, commonly referred to as the "Baltimore regimen," which is the current standard of care for haplo-HSCT in the U.S. and Europe.
CORPORATE UPDATES

Company Makes Key Additions to Management Team and Board of Directors; Forms Scientific and Clinical Advisory Board

Atabak Mokari was appointed Chief Financial Officer. He brings 20 years of financial leadership in life sciences, medtech and healthcare investment banking, and joined the Company from IRIDEX Corporation, a NASDAQ-listed medical technology company where he served as Chief Financial Officer and VP Corporate Development. Aaron Foster, Ph.D., was promoted to Senior Vice President, Head of Research, and will oversee the Company’s research organization. Dr. Foster was previously Vice President, Translational Research & New Product Development. Prior to joining Bellicum in 2012, he was Assistant Professor at Baylor College of Medicine at the Center for Cell & Gene Therapy, where he led a group researching adoptive T cell therapies, cancer vaccines, and nanotherapeutics. Concurrently, David Spencer, Ph.D., stepped down from his operating role as Chief Technology Officer, and has assumed an advisory position on the Company’s Scientific Advisory Board. Judith Klimovsky, M.D., joined the Board of Directors, replacing Frank McGuyer. Dr. Klimovsky currently serves as Executive Vice President & Chief Development Officer of Genmab, an international biotechnology company developing antibody therapeutics for cancer, where she leads the company’s product development efforts. Company formed a new Scientific and Clinical Advisory Board comprised of the following individuals:
— Malcom Brenner, M.D., Ph.D. (Baylor College of Medicine)
— Marco Davila, M.D., Ph.D. (Moffitt Cancer Center)
— Gianpietro Dotti, M.D. (University of North Carolina Lineberger Comprehensive Cancer Center and Professor for the Department of Microbiology and Immunology at UNC-Chapel Hill)
— Daniel Powell Jr., Ph.D. (University of Pennsylvania, Perelman School of Medicine)
— Naiyer Rizvi, M.D. (Columbia University Irving Medical Center)
— Charles Sentman, Ph.D. (Dartmouth, Geisel School of Medicine)
— David Spencer, Ph.D. (Co-Founder, Bellicum Pharmaceuticals)
Fourth Quarter and Full Year 2018 Financial Results

Cash Position and Guidance: Bellicum reported cash, restricted cash and investments totaling $98.0 million as of December 31, 2018, compared to $106.5 million at December 31, 2017. Based on current operating plans, Bellicum expects that current cash resources will be sufficient to meet operating requirements through the end of 2019.

R&D Expenses: Research and development expenses were $19.8 million and $71.2 million for the fourth quarter and year ended December 31, 2018, respectively, compared to $14.3 million and $65.7 million during the comparable periods in 2017. The higher expenses in the fourth quarter and full year 2018 compared to respective periods in 2017 were primarily due to an increase in costs related to Bellicum’s GoCAR-T product platform and general research and development expenses, partially offset by a decrease in expenditures related to rivo-cel.

G&A Expenses: General and administrative expenses were $7.0 million and $25.0 million for the fourth quarter and year ended December 31, 2018, respectively, compared to $5.1 million and $21.0 million during the comparable periods in 2017. The higher expenses in the fourth quarter and full year 2018 compared to respective periods in 2017 were primarily due to increased personnel related costs due to hiring additional employees.

Net Loss: Bellicum reported a net loss of $27.2 million for the fourth quarter of 2018 and $98.0 million for the year ended December 31, 2018, compared to a net loss of $21.9 million and $91.8 million for the comparable periods in 2017. The results included non-cash, share-based compensation charges of $3.0 million and $13.8 million for the fourth quarter and year ended December 31, 2018, respectively, and $3.4 million and $13.6 million for the comparable periods in 2017.

Shares Outstanding:

At December 31, 2018, Bellicum had 43,564,596 shares of common stock outstanding.

Upcoming Near-term Potential Milestones

BPX-601 – Expect to report data from additional cohorts in ongoing Phase 1/2 clinical trial in 2019
— Cy/Flu regimen cohort: mid-year 2019
— Repeat rimiducid dosing cohort: late 2019 BPX-603 – Expect IND submission and clinical trial initiation in 2019 BPX-802 – Expect IND submission in late 2019 Rivo-cel:
— Expect topline results from the BP-004 study in the second quarter of 2019
— Plan to submit MAAs for rivo-cel and rimiducid in late 2019
Conference Call and Webcast

Bellicum management will host a webcast and conference call at 5:00 p.m. Eastern today to discuss the financial results. To access the call, participants should dial 877-407-3103 (U.S. domestic) and 201-493-6791 (international) at least 10 minutes prior to the start of the call. The event will be webcast live and can also be accessed in the Investors & Media section of bellicum.com. An archived version of the webcast will also be available for replay in the Investors & Media section of the Bellicum website following the call.

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-cel (rivogenlecleucel) is an allogeneic polyclonal T-cell product designed to accelerate immune recovery after HSCT and to reduce 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 anti-infective benefits may also reduce morbidity and mortality, as patients are highly 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 inherited blood disease patients following a haploidentical stem cell transplant.

Advaxis Reports First Quarter Fiscal 2019 Financial Results and Provides Clinical Pipeline Update

On March 12, 2019 Advaxis, Inc. (NASDAQ: ADXS), a late-stage biotechnology company focused on the discovery, development and commercialization of immunotherapy products, reported an update on its clinical pipeline and financial results for the first quarter ended January 31, 2019 (Press release, Advaxis, MAR 12, 2019, View Source [SID1234534225]).

Updates on the progress of the Company’s clinical pipeline include:

Cancer Type-Focused Hotspot/Off-the-Shelf Neoantigen Therapies (ADXS-HOT) – The Company initiated its first clinical trial using a novel and proprietary approach to cancer immunotherapy that targets hotspot mutations, cancer testis antigens and oncofetal antigens. The first drug candidate from this program, ADXS-503, is designed to treat all types of non-small cell lung cancer and is now enrolling patients. Safety, tolerability and immune correlative data from this Phase1/2 study are anticipated by the end of June 2019.
Personalized, Neoantigen-Directed Therapy (ADXS-NEO) – The Company continues to enroll patients in a Phase 1 dose-escalation study with its personalized antigen delivery program using whole-exome sequencing of a patient’s tumor to identify personal neoantigens. Early immune response data from the first cohort of this study were presented last month at the Immuno-Oncology 360o Conference, and safety, tolerability and immune correlative data from the first two cohorts will be presented at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting on March 31, 2019.
Prostate Cancer (ADXS-PSA) – Previously reported data from a Phase 1/2 study of ADXS-PSA in combination with KEYTRUDA (pembrolizumab) demonstrated a manageable safety profile (mostly grade 1-2 treatment-related adverse events), in a cohort of 37 heavily pretreated metastatic castration-resistant prostate cancer patients and showed a greater level of clinical activity compared to monotherapy. The Company will be presenting updated clinical and biomarker data on this program at the AACR (Free AACR Whitepaper) Annual Meeting on April 1, 2019.
Cervical Cancer (ADXS-HPV) – In January 2019 the Company announced that the U.S. Food and Drug Administration (FDA) placed a partial clinical hold on the Company’s Phase 3 AIM2CERV clinical trial for axalimogene filolisbac (AXAL) in high-risk locally advanced cervical cancer. The partial hold relates to FDA’s request for additional information pertaining to certain AXAL chemistry, manufacturing and controls (CMC) matters. The FDA did not cite any safety issues related to the trial and all currently enrolled patients are continuing to receive treatment. The Company has submitted its initial response to the request for additional CMC information and is currently in discussions with the Agency. In parallel, Advaxis is in discussions with the Agency regarding the Company’s request, made late in 2018, to include a second interim analysis for efficacy. The Company is working diligently to reach a resolution with the Agency on both of these matters.
Management Commentary

"We reached an important milestone with our ADXS-HOT program last month when we enrolled the first patient in the ADXS-503 Phase 1/2 clinical trial. This is the first clinical trial initiated using a drug construct from our ADXS-HOT program, for which we have designed over 10 different drug constructs for various cancer types," said Kenneth A. Berlin, president and chief executive officer of Advaxis. "We continue to be very excited about our ADXS-HOT program and anticipate filing two additional INDs for drug constructs from this program during 2019."

He added, "We expect 2019 will be an important and eventful year for Advaxis due to the amount of information we anticipate generating from our programs and we look forward to reporting data throughout the year. In order to ensure our various programs progress to data readout, we are continually evaluating ways to increase our cash runway by controlling expenses and generating cash from potential out-licensing and/or financing transactions."

Financial Results for First Quarter Ended January 31, 2019

Research and development expenses for the first quarter of fiscal year 2019 were $6.7 million, compared with $16.8 million for the first quarter of fiscal year 2018. The $10.1 million decrease was primarily attributable to costs incurred during the last fiscal year related to the Company’s Marketing Authorization Application in Europe and cost controls initiated in the latter part of fiscal year 2018. Additionally, there was a decrease in clinical trial expenses resulting from the partial clinical hold on AIM2CERV and winding down of several older studies.

General and administrative expenses for the first quarter of fiscal year 2019 were $2.7 million, compared with $5.9 million for the first quarter of fiscal year 2018. The $3.2 million decrease was primarily attributable to professional and consulting fees relating to external strategy and program assessment work performed for the Company during fiscal year 2018 that did not recur in fiscal year 2019, in addition to improved cost controls initiated in the latter part of fiscal year 2018.

As previously reported, in December 2018 the Company received notice from Amgen of its intent to terminate its collaboration in the ADXS-NEO program. As a result, the Company adjusted its measure of progress for its performance obligations under the collaboration agreement and, based on the modified service period, reported incremental revenue of $15.6 million in the first quarter of fiscal year 2019. Net income for the first quarter of fiscal year 2019 was $12.8 million or $0.18 per share, compared with a net loss for the first quarter of fiscal year 2018 of $20.5 million or $0.49 per share. Net cash used during the first quarter ended January 31, 2019 was $12.4 million.