On August 7, 2019 Medigene AG (FSE: MDG1, Frankfurt, Prime Standard), a clinical stage immuno-oncology company focusing on the development of T cell immunotherapies, reported on their business performance and financial results for the first half of 2019 (Press release, MediGene, AUG 7, 2019, View Source [SID1234538320]). In the first six months of 2019, the Company made additional advances in their immunotherapy programs and reported a significant revenue increase, primarily due to the partnerships with bluebird bio and Roivant/Cytovant. Medigene confirms the financial guidance for the fiscal year 2019.

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"In the first half of 2019, we reported on clinical progress for three TCR therapy projects. Importantly, we initiated patient treatment in our ongoing study with Medigene’s first TCR therapy, MDG1011. We also commenced preparations for the Phase I trial of our second TCR therapy, MDG1021, with Leiden University Medical Center, scheduled to start in 2020. Additionally, our partner bluebird bio announced its intention to start a Phase I clinical trial in 2020 with the first TCR from our partnership. Also, Medigene was able to present promising clinical interim results from our DC vaccine trial," said Prof. Dolores Schendel, CEO/CSO of Medigene AG. "In addition, we established an important strategic partnership with Roivant/Cytovant for cellular therapies in Asia which will enable us to bring much needed treatment alternatives to this patient population. We are very pleased with the progress we have made this year and look forward to making further advancements with our proprietary immunotherapy programs as well as with those of our partners in the coming months."

Major events since the beginning of 2019:
– In June, Medigene presented an interim analysis from its ongoing Phase I/II clinical trial with dendritic cell (DC) vaccines in acute myeloid leukemia (AML) patients at the congress of the European Hematology Association (EHA) (Free EHA Whitepaper). The completion of the ongoing trial is scheduled for the end of 2019, following a two-year treatment period.
– In May, Medigene entered into a clinical trial agreement for its second T cell receptor (TCR) immunotherapy MDG1021 with Leiden University Medical Center. The Phase I clinical trial using an HA-1-specific TCR is scheduled to start in 2020 as Medigene’s second company-sponsored clinical TCR-T trial.
– In May, Medigene’s partner bluebird bio presented preclinical data for the first TCR candidate from the Medigene collaboration. The data demonstrated a high antigen sensitivity of the MAGE-A4-TCR, with strong recognition of tumor cell lines and durable tumor elimination in a subcutaneous in vivo melanoma model. Additionally, the data showed functional responses in both CD8+ and CD4+ T cell populations, demonstrating that addition of the CD8 co-receptor will not be needed for use of this MAGE-A4-TCR in solid tumors. bluebird bio intends to commence clinical development of the TCR targeting the MAGE-A4 tumor antigen in 2020; bluebird bio may potentially combine this highly active TCR with other technologies to enhance T cell function in solid tumor microenvironments in next generation programs.
– In April, Medigene entered into a license and cooperation agreement with the Roivant group on behalf of the newly founded Cytovant Sciences for research and discovery of cell therapies in Asia. Medigene granted Cytovant exclusive licenses to develop, manufacture, and commercialize Medigene’s TCR-based immunotherapy targeting the NY-ESO-1 antigen as well as a DC vaccine targeting WT-1 and PRAME. In addition, Roivant/Cytovant and Medigene entered into a strategic collaboration and discovery agreement for TCR immunotherapies for two additional targets. Medigene will be responsible for the generation and delivery of the TCR constructs and Cytovant will ultimately assume sole responsibility for the development and commercialization of these TCR therapies; the TCRs will be tailored specifically for Asian patients. Under the terms of the transaction, Medigene received an upfront payment of USD10 million and is eligible to receive development, regulatory, and commercial milestone payments, which in aggregate could total over USD1 billion for the four products across multiple indications.
– In April, Medigene sold the remaining rights and stock of Veregen to Aresus Pharma and thereby completed its transformation into a pure-play immunotherapy company.
– In February, Medigene dosed the first patient in the Phase I/II clinical trial of Medigene’s first T cell immunotherapy (TCR-T) MDG1011 to treat acute myeloid leukemia, myelodysplastic syndrome and multiple myeloma.
– In January, Medigene licensed a co-stimulatory receptor from Helmholtz Zentrum Munich to enhance TCR therapies for solid tumors.

Key figures in the first half of 2019:
– Revenue from the core immunotherapies business grew by 47% to Eur4,926k (6M-2018: Eur3,358 k) due to the partnership with bluebird bio and a new partnership with Roivant/Cytovant.
– Total revenue increased by 33% to Eur5,640k (6M-2018: Eur4,253k).
– Research and development expenses increased as planned by 26% to Eur10,922k (6M-2018: Eur8,669 k) due to an extension of the preclinical and, in particular, the clinical development and manufacturing activities for Medigene’s immunotherapy programs.
– EBITDA loss increased by 77% to Eur13,208 k (6M-2018: Eur7,479k); the increase of EBITDA loss was 14% (Eur8,532 k) when excluding the Veregen effect in the non-core business.
– Cash and cash equivalents and time deposits were Eur65,256 k as at June 30, 2019 (December 31, 2018: Eur71,408 k).

Financial guidance 2019:
Medigene confirmed the financial guidance issued in the 3M-2019 quarterly report:
– The Company anticipates generating total revenue of Eur10 – 11m in 2019.
– Due to anticipated advances in development projects, Medigene is projecting research and development expenses of Eur24 – 29m and forecasts an EBITDA loss of Eur23 – 28m.
– This assessment does not include potential future milestone payments or cash flows from existing or future partnerships or transactions, as the timing and extent of such events depends to a large extent on external parties and therefore cannot be reliably predicted by Medigene.
– Based on its current planning, the Company has sufficient financial resources to fund business operations beyond the planning horizon of two years.

Immunotherapy Outlook:
Current Phase I/II clinical trial with Medigene’s first TCR therapy MDG1011
Medigene commenced the Phase I/II clinical trial of its TCR-based T cell therapy MDG1011 and began treating patients in the first quarter of 2019. There are currently six active study centers and Medigene anticipates opening additional sites as previously guided. In 2019, the focus of the trial will be patient recruitment in the first dosing cohorts to assess the safety and tolerability of the treatment with MDG1011.

Planned Phase I/II clinical trial with Medigene’s second TCR therapy MDG1021
Based on the Clinical Trial Agreement entered into with the Leiden University Medical Center (LUMC) in May 2019, Medigene plans to conduct a Phase I clinical trial with Medigene’s TCR therapy MDG1021, targeting the HA-1 antigen. Medigene will assess the safety, feasibility and preliminary efficacy of MDG1021 in patients with relapsed or persistent hematologic malignancies following allogeneic hematological stem cell transplantation. The study is scheduled to start in 2020 as Medigene’s second company-sponsored clinical TCR-T trial after preparation for the trial is completed.

Development of additional TCR candidates
Now that a robust platform for the discovery and characterization of new TCR candidates has been fully established, building a solid pipeline of potential TCR-development candidates is an important goal to secure future clinical programs for both internal and existing or future partners.

In 2019, in addition to the MDG1011 clinical trial, Medigene will therefore work on characterizing new TCR candidates for future clinical trials under the responsibility and funding of Medigene and collecting preclinical data to prepare further clinical TCR trials.

Optimization of future TCR therapies for solid tumors
The chimeric co-stimulator receptor (PD-1/4-1BB molecule) licensed from HMGU will be assessed in combination with Medigene’s tumor-specific T cells (TCR-Ts) in preclinical models in order to optimize future TCR therapies for solid tumors.

TCR partnerships
In addition, Medigene continues its successful collaboration with bluebird bio and expects to make further progress on TCR candidate discovery. bluebird bio announced to commence clinical development in solid tumors with the first TCR from this partnership (a TCR targeting MAGE-A4) in 2020.

Within the framework of the partnership entered into with Roivant/Cytovant, Medigene will now, together with the collaboration partner, undertake the preparations to generate TCR constructs tailored specifically to Asian patients using its proprietary TCR discovery platform.

Dendritic cell vaccines (DCs)
Medigene will continue the current Phase I/II clinical trial for DC vaccines for the treatment of acute myeloid leukemia (AML) as planned and bring it to a conclusion at the end of 2019. The final data will be available towards the end of 2019/beginning of 2020.

The full version of the quarterly statement 6M-2019 can be downloaded here: View Source

Conference call and webcast: A telephone conference (webcast) in English will be held today at 3:00 pm CET (Munich/Frankfurt) / 9:00 am EST (New York) and transmitted live via webcast. Access and transmission of the synchronized presentation slides and a recording of the presentation is available on the homepage of Medigene at View Sourcewebcasts/

On August 7, 2019 Lipocine Inc. (NASDAQ: LPCN), a clinical-stage biopharmaceutical company focused on metabolic and endocrine disorders, reported financial results for the second quarter and six months ended June 30, 2019, and provided a corporate update (Press release, Lipocine, AUG 7, 2019, View Source [SID1234538314]).

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Second Quarter and Recent Corporate Highlights

·Filed a New Drug Application ("NDA") for TLANDO as testosterone replacement therapy ("TRT") in adult males for conditions associated with a deficiency of endogenous testosterone, also known as hypogonadism.
oFDA has assigned November 9, 2019 as the Prescription Drug User Fee Act ("PDUFA") goal date.
·Received clearance from the U.S. Food and Drug Administration ("FDA") to clinically investigate LPCN 1144 in an expanded target population of adult male non-alcoholic steatohepatitis ("NASH") patients.
oFDA has waived the limitation of only testing in NASH subjects with total testosterone levels below 300 ng/dL (threshold for hypogonadism).
·Initiated the LiFT ("Liver Fat intervention with oral Testosterone") LPCN 1144 Phase 2 clinical study, a paired-biopsy study in confirmed pre-cirrhotic NASH subjects.
·Filed suit against Clarus Therapeutics, Inc. ("Clarus") in the United States District Court ("Delaware") alleging that Clarus’s JATENZO product infringes six of Lipocine’s U.S. patents.
·Joined the Russell Microcap Index effective July 1, 2019 as a result of the Russell indexes annual reconstitution.

"During the second quarter, we continued to advance our pipeline, with important milestones achieved for both TLANDO and LPCN 1144," said Dr. Mahesh Patel, Chairman, President and Chief Executive Officer of Lipocine. "The acceptance of the NDA for TLANDO for treating hypogonadism in males puts us on track for potential approval of this product in the fourth quarter of 2019. TLANDO is designed as a fixed dose oral TRT. For LPCN 1144, we were excited to initiate the LiFT study in biopsy confirmed NASH subjects, and based on our observed meaningful liver fat reduction in hypogonadal males, receive FDA clearance to expand our target patient population in adult NASH males regardless of their hypogonadal status. We believe this increases LPCN 1144’s potential utility in a broader NASH population who reportedly have low testosterone."

Second Quarter Ended June 30, 2019 Financial Results

Lipocine reported a net loss of $3.4 million, or ($0.14) per diluted share, for the quarter ended June 30, 2019, compared with a net loss of $3.3 million, or ($0.15) per diluted share, in the quarter ended June 30, 2018.

Research and development expenses were $2.0 million for the quarter ended June 30, 2019, compared with $1.5 million for the quarter ended June 30, 2018. The increase in research and development expenses was primarily due to increases in outside service costs related to the second quarter initiation of the LiFT LPCN 1144 Phase 2 clinical study in confirmed pre-cirrhotic NASH subjects and increased contract research organization and outside consulting costs for TLANDO in connection with completion of the ABPM study and the filing of the NDA.

General and administrative expenses were $1.4 million for the quarter ended June 30, 2019, compared with $1.7 million for the quarter ended June 30, 2018. The decrease in general and administrative was primarily due to decreased personnel costs, a decrease in severance compensation, a decrease in stock compensation expense and a decrease in bonus expense. The decreases were offset by increases in other general and administrative expenses including an increase in professional fees, an increase in corporate insurance and an increase in marketing expenses.

As of June 30, 2019, the Company had total cash, cash equivalents and marketable securities aggregating $19.5 million, compared to $20.3 million at December 31, 2018. Of the total cash, cash equivalents and marketable securities balances, $5.0 million is restricted under our agreement with Silicon Valley Bank until TLANDO receives approval.

Six Months Ended June 30, 2019 Financial Results

Lipocine reported a net loss of $6.7 million, or ($0.28) per diluted share, for the six months ended June 30, 2019, compared with a net loss of $6.0 million, or ($0.28) per diluted share, in the six months ended June 30, 2018.

Research and development expenses were $3.9 million for the six months ended June 30, 2019, compared with $2.9 million for the six months ended June 30, 2018. The increase/decrease in research and development expenses was primarily due to increased contract research organization and outside consulting costs for TLANDO in connection with completion of the ABPM study and the filing of the NDA, increased manufacturing costs for TLANDO, increased outside service costs related to the second quarter initiation of the LiFT LPCN 1144 Phase 2 clinical study in confirmed pre-cirrhotic NASH subjects, increased outside contract research organization costs and sample storage costs related to TLANDO XR (LPCN 1111) offset by decreased contract manufacturing costs for LPCN 1107 and decreased personnel costs.

General and administrative expenses were $2.6 million for the six months ended June 30, 2019, compared with $3.4 million for the six months ended June 30, 2018. The decrease in general and administrative was primarily due to decreased personnel costs, which includes a decrease in salaries and related benefits mainly from the elimination of our commercial sales and marketing team in 2018, a decrease in severance compensation, a decrease in stock compensation expense and a decrease in bonus expense of $12,000. The decreases were offset by increases in other general and administrative expenses including an increase in professional fees and an increase in corporate insurance.

On August 7, 2019 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) therapies for cancer, reported financial results for the quarter ended June 30, 2019 (Press release, Allogene, AUG 7, 2019, View Source [SID1234538313]).

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"The second quarter was an important one on many fronts, from advancing our pipeline with the clearance of our second investigational new drug application, to designing our state-of-the-art manufacturing facility and the continued onboarding of highly-skilled employees who are passionate about bringing allogeneic cell therapy to patients," said David Chang, M.D., Ph.D., President, Chief Executive Officer and Co-Founder of Allogene. "Our teams are focused on advancing our allogeneic platform, which includes our first company-sponsored clinical trial with ALLO-501 for patients with relapsed/refractory non-Hodgkin lymphoma. We are pleased with how this dose escalation study is progressing, which includes the use of our selective lymphodepletion strategy anchored around our proprietary anti-CD52 antibody, ALLO-647."
Recent Highlights
ALLO-501 (anti-CD19 AlloCAR T)

The ALLO-501 Phase 1 portion of the ALPHA trial for patients with relapsed/refractory non-Hodgkin lymphoma (NHL) was initiated in Q2 2019. The trial is designed to assess the safety and tolerability at increasing dose levels of ALLO-501 in the most common NHL subtypes of relapsed/refractory large B-cell lymphoma, including diffuse large B-cell lymphoma (DLBCL) and follicular lymphoma (FL). Five sites with expertise in CAR T are open for enrollment. The company remains on track to release topline data from the ongoing Phase 1 ALPHA trial in the first half of 2020.

The Company continues to progress the planned second generation of ALLO-501, which is devoid of the rituximab off-switch, through preclinical development and plans to introduce this next generation prior to the start of the Phase 2 registrational study.
ALLO-715 (anti-BCMA AlloCAR T)

An Investigational New Drug (IND) application for ALLO-715, a wholly-owned CAR T product candidate targeting B cell maturation antigen (BCMA) for relapsed/refractory multiple myeloma, was cleared by the U.S. Food & Drug Administration (FDA) in May 2019. The Company remains on track to initiate a Phase 1 trial in the second half of 2019.

The Phase 1 ALLO-715 UNIVERSAL trial is designed to assess the safety and tolerability at increasing dose levels of ALLO-715 to identify an optimal dose of ALLO-715 for the potential Phase 2 study. This trial will utilize ALLO-647, the Company’s proprietary anti-CD52 monoclonal antibody, as a part of the lymphodepletion regimen. The trial also includes the potential for exploratory cohorts that will allow study of additional lymphodepletion regimens, including one that only uses ALLO-647 without fludarabine and cyclophosphamide.

Additional Pipeline Updates

UCART19 (Servier-Sponsored Program in Collaboration with Allogene) – Servier has re-initiated recruitment for the CALM and PALL trials in relapsed/refractory acute lymphoblastic leukemia. UCART19 is expected to be advanced to potential registrational trials in 2020.
Corporate Highlights

The Company recently announced the appointment of Rafael G. Amado, M.D. as Executive Vice President of Research and Development and Chief Medical Officer. In this new position, Dr. Amado will lead the Company’s clinical and research functions with the goal of rapidly advancing our pipeline of allogeneic CAR T therapies for hematologic and solid tumors. This appointment reunites Dr. Amado with many former colleagues, including David Chang, M.D., Ph.D., President, Chief Executive Officer and Co-Founder.
Second Quarter Financial Results

As of June 30, 2019, Allogene had $650.2 million in cash, cash equivalents, and investments, compared to $721.4 million as of December 31, 2018.

Research and development expenses were $31.8 million for the second quarter of 2019, which includes $4.7 million of non-cash stock-based compensation expense, compared to $122.5 million for the second quarter of 2018. The second quarter of 2018 included a non-cash charge of $109.4 million related to in process research and development acquired as a result of the Pfizer asset acquisition.

General and administrative expenses were $14.2 million for the second quarter of 2019, which includes $6.7 million of on-cash stock-based compensation expense, compared to $12.5 million for the second quarter of 2018.

Net loss for the second quarter of 2019 was $41.2 million, or $0.41 per share, including non-cash stock-based compensation expense of $11.5 million, compared to a net loss of $134.9 million, or $43.82 per share for the second quarter of 2018.

The Company continues to expect full-year 2019 net losses to be between $200 million and $210 million dollars, including estimated non-cash stock-based compensation expense of $45 million to $50 million and excluding any impact from potential business development activities.
Conference Call and Webcast Details

Allogene will host a live conference call and webcast today at 5:30 AM Pacific Time/8:30 AM Eastern Time to discuss financial results and provide a business update. To access the live conference call by telephone, please dial 1 (866) 940-5062 (U.S.) or 1 (409) 216-0618 (International). The conference ID number for the live call is 4851687. The webcast will be made available on the Company’s website at www.allogene.com under the Investors tab in the News and Events section. Following the live audio webcast, a replay will be available on the Company’s website for approximately 30 days.

On August 7, 2019 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported that Troy Wilson, Ph.D., J.D., President and Chief Executive Officer, is scheduled to present at the 2019 Wedbush PacGrow Healthcare Conference on Wednesday, August 14, 2019 at 12:45 p.m. ET / 9:45 a.m. PT (Press release, Kura Oncology, AUG 7, 2019, View Source [SID1234538312]). The conference will be held August 13-14, 2019 in New York City.

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A live audio webcast of the presentation will be available in the Investors section of Kura’s website at www.kuraoncology.com, with an archived replay available for 30 days following the event.

On August 7, 2019 Horizon Therapeutics plc (Nasdaq: HZNP) reported its second-quarter 2019 financial results and increased its full-year 2019 net sales and adjusted EBITDA guidance (Press release, Horizon Pharma, AUG 7, 2019, View Source [SID1234538311]).

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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

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"The second quarter was another quarter of outstanding execution and strategic progress," said Timothy Walbert, chairman, president and chief executive officer, Horizon. "We generated double-digit net sales growth in our orphan and rheumatology segment, driven by continued momentum from KRYSTEXXA, our medicine for uncontrolled gout and our main growth driver. In addition, we recently submitted teprotumumab for U.S. FDA approval, another milestone toward delivering the first FDA-approved treatment to people living with active thyroid eye disease."