On May 11, 2018 Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket"), a leading U.S.-based multi-platform gene therapy company, reported financial results for the quarter ended March 31, 2018, and provided an update on the Company’s recent achievements, as well as upcoming milestones (Press release, Rocket Pharmaceuticals, MAY 11, 2018, View Source;p=irol-newsArticle&ID=2348660 [SID1234526542]).

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"2018 is off to a great start for Rocket. During the first quarter, we continued to make excellent progress advancing our pipeline of lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy programs towards key value inflection points. We look forward to presenting updated patient data from the Fanconi Anemia program later this month, and to disclosing our AAV-based gene therapy program by the end of the year," said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "We remain on track for multiple regulatory filings across our pipeline over the next 12 months. As a result, there is the potential for four programs to be in the clinic in 2019, with clinical data for up to two programs."

Dr. Shah continued, "While near-term milestones are certainly important, Rocket’s long-term strategy is based on a sustainable and integrated multi-platform approach. We remain focused on our mission of continued innovation to deliver best-in-class curative cell and gene therapies to patients living with devastating diseases."

Anticipated Milestones Over the Next 12 -18 Months

Fanconi Anemia (FA): Updated patient data from the ongoing Phase 1/2 study of RP-L102 conducted with CIEMAT is expected over the next 12 months. The next update will be provided at the American Society of Gene and Cell Therapy’s (ASGCT) (Free ASGCT Whitepaper) 21st Annual Meeting in Chicago. The oral presentation is scheduled during the Presidential Symposium on Friday, May 18, 2018, at 2:45 p.m. Central Time.
Leukocyte Adhesion Deficiency-I (LAD-I): An Investigational Medicinal Product Dossier (IMPD) is expected to be filed in the fourth quarter of 2018. Clinical data is expected over the next 12-18 months.
Pyruvate Kinase Deficiency (PKD): An IMPD application is expected to be filed in early 2019.
Infantile Malignant Osteopetrosis (IMO): Preclinical studies continue to advance in support of filing a first-in-human clinical study.
AAV: Disclosure of the disease indication and preclinical data is planned for the second half of 2018.
Upcoming Conferences

Bank of America Merrill Lynch Health Care Conference 2018. Rocket is scheduled to present on Tuesday, May 15, at 9:20 a.m. Pacific Time.
UBS Global Healthcare Conference. Rocket is scheduled to present on Wednesday, May 23, at 9:30 a.m. Eastern Time.
Jefferies Global Healthcare Conference. Rocket is scheduled to present on Thursday, June 7, at 2:00 p.m. Eastern Time.
First Quarter 2018 Financial Results

Cash position. Cash, cash equivalents and short-term investments as of March 31, 2018, were $182.7 million, which includes a $52.0 million fully convertible debenture which expires in 2021.
R&D expenses. Research and development expenses were $5.7 million for the quarter ended March 31, 2018, compared to $2.3 million for the quarter ended March 31, 2017.
G&A expenses. General and administrative expenses were $8.7 million for the quarter ended March 31, 2018, compared to $0.6 million for the quarter ended March 31, 2017. The increase in G&A expenses was primarily due to one-time merger related expenses of $5.3 million.
Net loss. Net loss was $15.3 million or $(0.42) per share (basic and diluted) for the quarter ended March 31, 2018, compared to $2.7 million or $(0.39) per share (basic and diluted) for the quarter ended March 31, 2017.
Shares outstanding. Approximately 39.4 million shares of common stock were outstanding as of March 31, 2018.
Financial Guidance

Cash position. Based on its current operating plan, Rocket expects its cash, cash equivalents and short-term investments as of March 31, 2018, will be sufficient to run its operations into 2020.

On May 11, 2018 Trillium Therapeutics Inc. (NASDAQ/TSX: TRIL), a clinical stage immuno-oncology company developing innovative therapies for the treatment of cancer, reported financial and operating results for the three months ended March 31, 2018 (Press release, Trillium Therapeutics, MAY 11, 2018, View Source [SID1234526543]).

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"Following the initial signals of monotherapy responses reported at ASH (Free ASH Whitepaper) last year, we have increasingly focused both our TTI-621 clinical trials on patients with T-cell malignancies throughout the first quarter of 2018" said Dr. Niclas Stiernholm, president and CEO of Trillium Therapeutics. "We have also been preparing to launch our second clinical CD47 program, TTI-622. Having both an IgG1 and an IgG4 SIRPaFc fusion protein in clinical testing should allow us to address important scientific questions related to the impact of the Fc region in various clinical scenarios, including combination therapy."

2018 First Quarter Highlights:

Reported refinements to both our phase 1 trials of TTI-621 to focus near-term efforts on patients with cutaneous T-cell lymphoma (CTCL) and peripheral T-cell lymphoma (PTCL). This action builds on the monotherapy results of TTI-621 presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2017 where weekly infusions of TTI-621 were shown to be well tolerated and intratumoral injection was observed to reduce local lesions in 9 out of 10 patients with mycosis fungoides, a common type of CTCL.
The U.S. Food and Drug Administration granted an Orphan Drug Designation to TTI-621 for the treatment of cutaneous T-cell lymphoma. Orphan Drug Designation qualifies the sponsor of the drug candidate for various development incentives, which include an exemption from fees under the Prescription Drug User Fee Act and a seven-year marketing exclusivity period following approval.
Presented preclinical TTI-622 data at the 2018 AACR (Free AACR Whitepaper) Annual Meeting demonstrating that TTI-622 induces the phagocytosis of a broad panel of tumor cells derived from patients with both hematological and solid tumors. As a monotherapy, TTI-622 treatment resulted in decreased tumor growth and improved survival in a B cell lymphoma xenograft model, as well as enhanced the efficacy of cetuximab (anti-EGFR) and daratumumab (anti-CD38) antibodies in solid and hematological xenograft models, respectively. We expect to enroll the first patient in a Phase 1 clinical trial of TTI-622 in Q2 2018.
First Quarter 2018 Financial Results

As of March 31, 2018, Trillium had cash and cash equivalents and marketable securities, and working capital of $73.9 million and $61.7 million, respectively, compared to $81.8 million and $68.9 million, respectively at December 31, 2017. The decrease in cash and cash equivalents and marketable securities, and working capital was due mainly to cash used in operations of approximately $9.5 million.

Net loss for the three months ended March 31, 2018 of $8.6 million was lower than the loss of $11.5 million for the three months ended March 31, 2017. The net loss was lower due mainly to a net foreign currency gain of $1.6 million for the three months ended March 31, 2018, compared to a net foreign currency loss of $0.4 million in the prior year period. Research and development expenses decreased by $0.9 million in 2018 as a result of lower manufacturing activity for TTI-621 and TTI-622. These decreases were partially offset by higher clinical trial expenses.

On May 11, 2018 Advaxis, Inc. (NASDAQ: ADXS), a late-stage biotechnology company focused on the discovery, development and commercialization of immunotherapy products, reported on recently presented preclinical data demonstrating the anti-cancer potential of their Lm vector that presents tumor neoantigens, and is being evaluated in the ADXS-NEO program (Press release, Advaxis, MAY 11, 2018, View Source [SID1234526525]). ADXS-NEO is derived from the Company’s proprietary Lm Technology and is being developed in partnership with Amgen.

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These preclinical findings were discussed in poster presentations at the recent American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. Additionally, portions of these data were presented by Amgen at a podium presentation during the European Neoantigen Summit 2018.

The first study, as discussed in a poster presentation at AACR (Free AACR Whitepaper) entitled "Neoantigens that fail to elicit measurable T cell responses following peptide immunization can control tumor growth when delivered using a Listeria-based immunotherapy platform," showed that ADXS-NEO generates T cell responses against neoantigen peptides that control tumor growth, even when they were identified as "non-immunogenic" using a conventional peptide-adjuvant immunization. The poster is available here: View Source

In the second study, discussed in a poster presentation at AACR (Free AACR Whitepaper) entitled "Targeting frameshift mutations with a Listeria monocytogenes immunotherapy drives neoantigen-specific antitumor immunity in MC38 and CT26 mouse tumor models," Advaxis’ Lm platform was shown to target frameshift mutations and generate T cells to multiple neoantigens per frameshift in MC38 and CT26 mouse tumor models. The poster is available here: View Source

"The results of these studies highlight the therapeutic potential of targeting neoantigens and suggest that ADXS-NEO could impact the way we approach treatment across a range of cancers," said Ken Berlin, President and CEO of Advaxis. "We look forward to our continued collaboration with Amgen, and to moving into the clinic with this product candidate."

"These preclinical findings provide foundational rationale suggesting that ADXS-NEO has the potential to generate immune responses against multiple neoantigens with the ability to control tumor growth. This is a personalized approach that uses a patient’s own immune system to recognize and eliminate cancer cells harboring multiple mutations that caused their malignancy," said Robert G. Petit, Ph.D., Chief Scientific Officer and Executive Vice President of Advaxis. "We also saw potent immune responses targeting frameshift mutations and control of tumor growth via multiple complementary mechanisms. This is important because frameshift mutations are observed to generate up to nine times more neoantigens per mutation than in-frame mutations" according to Turajlic, et al.

About ADXS-NEO

ADXS-NEO is a personalized Listeria monocytogenes (Lm)-based immunotherapy designed to generate immune response against mutation-derived tumor-specific neoantigens. The program focuses on creating a customized treatment with neoantigens specifically selected based on a biopsy of the patient’s tumor. The approach enables the development of tailored immune-therapies. ADXS-NEO is being evaluated in collaboration with Amgen.

On May 11, 2018 Cellectar Biosciences (Nasdaq: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported recent corporate highlights and financial results for the three months ended March 31, 2018 (Press release, Cellectar Biosciences, MAY 11, 2018, View Source [SID1234526526]).

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Recent Corporate Highlights

·Received orphan drug designation and rare pediatric disease designation from the U.S. Food and Drug Administration (FDA) for CLR 131 to treat neuroblastoma.

·Received orphan drug designation from the FDA for CLR 131 to treat rhabdomyosarcoma, a rare pediatric cancer.

·Presented Phase 1 study results at the 12th World Congress of the World Federation of Nuclear Medicine and Biology demonstrating that CLR 124 is able to cross the blood-brain barrier and achieve uptake in brain tumors. The company believes these data have positive read through for CLR 131 which varies only by the radionuclide delivered.

·Initiated the diffuse large B-cell lymphoma cohort of the company’s Phase 2 clinical trial of CLR 131. This cohort is the fourth and final in the study for patients with R/R B-cell hematologic cancers.

·Initiated cohort 5 in the Phase 1 study of CLR 131 in highly pretreated R/R multiple myeloma patients. This is the first cohort in the trial to use a fractionated dosing schedule.

·Presented two late-breaking poster presentations at the AACR (Free AACR Whitepaper) Annual Meeting. The posters highlighted the potential benefits of fractionated dosing regimens of CLR 131 and the ability of the company’s Phospholipid Drug Conjugates (PDCs) to provide improved targeting of tumor cells and the intracellular trafficking of these molecules.

·Granted seminal U.S. patent for phospholipid-ether analogs covering composition of matter and method of use for proprietary PDCs in combination with anti-cancer agents.

·Issued U.S. patent entitled "Alkylphosphocholine analogs for multiple myeloma imaging and therapy," covering the use of CLR 131 in multiple MM and received a composition of matter patent in Japan.

"The first quarter and recent weeks brought significant progress on both the clinical and regulatory fronts. We advanced both Phase 1 and Phase 2 studies for CLR 131, presented new data at major scientific conferences, and received orphan drug designation and rare pediatric disease designation from the FDA to treat neuroblastoma in pediatric patients, as well as an orphan drug designation to treat rhabdomyosarcoma," said James Caruso, president and CEO of Cellectar Biosciences. "We are particularly excited to begin our upcoming Phase 1 study to explore CLR 131 as a treatment option for children with life-threatening rare pediatric cancers."

First Quarter 2018 Financial Results

Research and development expense for the first quarter of 2018 was $2.1 million, compared with $1.9 million for the first quarter of 2017. The year over year increase is attributable to higher preclinical and clinical project costs, manufacturing, and general research and development costs.

General and administrative expense for the first quarter of 2018 was $1.3 million, compared with $1.0 million for the first quarter of 2017. The year over year increase is attributable to higher consulting, legal and marketing fees, as well as one-time personnel costs incurred in connection with the decision to outsource our manufacturing.

The net loss attributable to common stockholders for the first quarter of 2018 was $3.5 million, or $0.21 per share based on 16.8 million shares outstanding, compared with a net loss attributable to common stockholders for the first quarter of 2017 of $2.9 million, or $0.24 per share based on 12.0 million shares outstanding.

Cash and cash equivalents as of March 31, 2018 were $6.8 million, compared with $10.0 million as of December 31, 2017.

On May 11, 2018 Hutchison China MediTech Limited ("Chi-Med") (AIM/Nasdaq: HCM) reported that Christian Hogg, Chief Executive Officer, will present at the Bank of America Merrill Lynch 2018 Health Care Conference on Wednesday, May 16, 2018 at 1:00 pm PDT in Las Vegas (Press release, Hutchison China MediTech, MAY 11, 2018, View Source [SID1234526527]).

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The presentation and Q&A session will be webcast live and can be accessed at www.chi-med.com in the Shareholder Information section under "Events, Circulars & Forms." Investors interested in listening to the live webcast should log on before the start time to download any software required. A replay of the event will be available shortly thereafter, for 90 days.