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MorphoSys Announces Intention to Submit Marketing Authorization Application for Tafasitamab to European Medicines Agency

On August 6, 2019 MorphoSys AG (FSE: MOR; Prime Standard Segment; MDAX & TecDAX; NASDAQ: MOR) reported its intention to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) based on its phase 2 L-MIND study of tafasitamab (MOR208) and lenalidomide in relapsed or refractory diffuse large B cell lymphoma (r/r DLBCL) (Press release, MorphoSys, AUG 6, 2019, View Source [SID1234538247]). A letter of intent was submitted to EMA in early July and MorphoSys plans to complete the MAA submission by mid-2020.

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L-MIND is a single-arm, open-label phase 2 study investigating the antibody tafasitamab in combination with lenalidomide in patients with relapsed or refractory diffuse large B cell lymphoma who are not eligible for high-dose chemotherapy and autologous stem cell transplantation. In July 2019, the company presented positive primary analysis data from L- MIND at the ICML conference in Lugano. These data will form the basis for the planned filing package of the MAA that, if granted, allows for earliest approval in Europe in 2021.

"We are excited about the progress we are making with our plan for a possible regulatory approval for tafasitamab in Europe," said Dr. Malte Peters, Chief Development Officer at MorphoSys. "The preceding constructive interaction with the European regulatory authorities has given us the confidence to pursue an MAA submission for approval in Europe based on L-MIND. This complements our current focus on seeking approval of tafasitamab in the U.S. on the basis of L-MIND, for which we will complete a BLA submission to the FDA by year-end."

The Spanish Sarcoma Group Will Lead a European Consortium to Evaluate the Ability of Lixte Biotechnology Holding’s LB-100 to Improve First Line Therapy for Advanced Soft Tissue Sarcomas.

On August 6, 2019 Lixte Biotechnology Holdings, Inc. (OTCQB: LIXT) reported that it signed a clinical trial agreement with the Spanish Sarcoma Group (Grupo Español de Investigación en Sarcomas, GEIS) to support a Phase 1b/randomized Phase 2 study of doxorubicin, the global standard for initial treatment of advanced soft tissue sarcomas (ASTS), versus doxorubicin plus LB-100 (Press release, Lixte Biotechnology, AUG 6, 2019, View Source [SID1234538246]).

Dr. John S. Kovach, CEO of Lixte, said, "We are pleased to support this investigator-initiated trial proposed by GEIS, a leader for many years in seeking improved therapies for ASTS. GEIS has a network of referral centers in Spain and across Europe that has an impressive track record of efficiently conducting innovative studies in ASTS. We believe GEIS and their EU collaborators are an excellent team to evaluate the potential benefit of adding Lixte’s non-cytotoxic inhibitor of DNA damage repair to standard treatment for this challenging disease. The goal is to enter the first patient in the last quarter of this year and to complete enrollment of approximately 170 patients over two years."

Dr. Kovach continued, "Doxorubicin alone has been the mainstay of first line treatment of ASTS for over 40 years with little therapeutic gain from adding cytotoxic compounds to or substituting other cytotoxic compounds for doxorubicin. In animal models, LB-100, an inhibitor of protein phosphatase2A (PP2A), consistently enhances the antitumor activity of doxorubicin without apparent increases in toxicity and, in a Phase 1 clinical trial, LB-100 alone was associated with stabilization of an advanced chondrosarcoma and fibrosarcoma for 6 and 9 months, respectively, without toxicity. LB-100 potentiates the effectiveness of agents like doxorubicin by inhibiting multiple steps required to repair chemotherapy induced DNA damage. We are excited to learn if the combination of LB-100 and doxorubicin will finally advance the effectiveness of ASTS therapy."

Dr. Javier Martin-Broto, Coordinating Investigator of the trial and medical oncologist at Virgen del Rocío University Hospital (Seville) commented, "Although there has been an increase in overall survival in advanced sarcoma in recent years, this gain has not been accompanied by advances in first line therapy. Anthracyclines, and specifically doxorubicin, is still the standard initial treatment. The growing list of negative phase III trials indicates to us that sarcoma therapy is in crisis. It is true that sarcoma encompasses more than 60 different subtypes and, for some of them, substantial advances have emerged. But it is also true that the most frequent sarcoma subtypes desperately need a turning point. One promising topic of research is the combination of doxorubicin with drugs that are able to impair the mechanisms of DNA repair. LB-100 has demonstrated synergistic action in in vivo preclinical mesenchymal tumors. GEIS will lead a European initiative to conduct a phase I/randomized II trial exploring the combination of doxorubicin plus LB-100 in first line of advanced soft tissue sarcomas".

United Therapeutics Corporation To Present At 2019 Wedbush PacGrow Healthcare Conference

On August 6, 2019 United Therapeutics Corporation (Nasdaq: UTHR) reported that Martine Rothblatt, Ph.D., Chairman and Chief Executive Officer of United Therapeutics, will provide an overview and update on the company’s business at the 2019 Wedbush PacGrow Healthcare Conference in New York City (Press release, United Therapeutics, AUG 6, 2019, View Source [SID1234538235]).

The presentation will take place on Tuesday, August 13, 2019, at 10:20 AM Eastern Time, and can be accessed via a live webcast on the United Therapeutics website at View Source under the "Investors" tab in the "Events and Presentations" section. An archived, recorded version of the presentation will be available approximately twenty-four hours after the presentation ends and can be accessed at the same location for 90 days. [uthr-g]

Palatin Technologies To Present At Canaccord Genuity’s 39th Annual Growth Conference

On August 6, 2019 Palatin Technologies, Inc. (NYSE American: PTN) reported that it will be presenting at Canaccord Genuity’s 39th Annual Growth Conference on Wednesday, August 7, 2019, at 10:30 am ET. The conference will be held at the InterContinental Hotel in Boston, MA (Press release, Palatin Technologies, AUG 6, 2019, View Source [SID1234538234]).

Carl Spana, Ph.D., President and Chief Executive Officer of Palatin Technologies, will provide an update on Palatin’s corporate and development programs, including:

Hypoactive Sexual Desire Disorder / Vyleesi (bremelanotide injection)
On June 21, 2019, the U.S. Food and Drug Administration (FDA) granted marketing approval of AMAG Pharmaceuticals, Inc’s New Drug Application (NDA) for Vyleesi, a melanocortin receptor agonist developed by Palatin, indicated for the treatment of acquired, generalized hypoactive sexual desire disorder (HSDD) in premenopausal women. The Vyleesi autoinjector is the first treatment for this patient population that can be self-administered as needed in anticipation of sexual activity.
The FDA’s approval of the NDA triggered a $60 million milestone payment to Palatin (received July 2019) under its North American license agreement with AMAG.
AMAG is expected to launch Vyleesi nationally in September 2019.
Discussions with multiple parties on potential commercial partnerships for territories outside North America, China and South Korea are advancing.
Melanocortin Anti-Inflammatory / Autoimmune Programs under development for the treatment of inflammatory and autoimmune diseases such as dry eye, uveitis, diabetic retinopathy and inflammatory bowel diseases (ulcerative colitis)
A Phase 2 proof-of-concept clinical study for PL-8177 with a systemic formulation in NIU patients is anticipated to commence in the first quarter of calendar year 2020.
A Phase 2 proof-of-concept clinical study for PL-8177 with an oral formulation in ulcerative colitis patients is anticipated to commence in the first quarter of calendar year 2020.
A Phase 2 clinical study for PL-9643 in dry eye disease is currently anticipated to commence in the first quarter of calendar year 2020. A recently completed Type B pre-IND (Investigational New Drug) meeting with the FDA established the development pathway for activities, including CMC, non-clinical studies and clinical studies, required for an NDA (New Drug Application) submission.
Cash and cash equivalents at June 30, 2019, on a pro forma basis, which includes the $60 million milestone payment by exclusive North American licensee, AMAG Pharmaceuticals, Inc., is approximately $102 million dollars.

Audentes Therapeutics Reports Second Quarter 2019 Financial Results and Provides Corporate Update

On August 6, 2019 Audentes Therapeutics, Inc. (Nasdaq: BOLD), a leading AAV-based genetic medicines company focused on developing and commercializing innovative products for serious rare neuromuscular diseases, reported its financial results for the second quarter ended June 30, 2019 and provided an update on the company’s recent achievements and anticipated upcoming milestones (Press release, Audentes Therapeutics, AUG 6, 2019, View Source [SID1234538233]).

"It is a very busy time at Audentes, and we are excited by the significant progress we have made across our portfolio," stated Matthew R. Patterson, Chairman and Chief Executive Officer. "Following collaborative interactions with FDA and EMA, we have initiated enrollment of eight additional XLMTM patients into an ASPIRO pivotal expansion cohort, which is designed to confirm the safety and efficacy profile of AT132 at a dose of 3×1014 vg/kg. We are optimistic that these data will support the submission of a BLA for AT132 in mid-2020."

Mr. Patterson continued, "We remain on track for a third quarter IND submission of AT845 for the treatment of Pompe disease. And importantly, we’ve made substantial progress advancing our DMD program. We expect patient dosing with NCH’s AT702 construct to commence as planned in the fourth quarter of 2019 and to submit an IND in the first quarter of 2020 for the Audentes AT702 construct, which is designed to serve as the platform for our rapid expansion into additional DMD genotypes next year. Together with our work in myotonic dystrophy, we believe this rich development pipeline positions us well for continued growth and industry leadership in the field of AAV-based genetic medicines for neuromuscular disease."

Recent Achievements & Upcoming Key Events

AT132 for XLMTM:

Positive data update from ASPIRO, the Phase 1/2 clinical trial of AT132 for the treatment of XLMTM, presented at the 22nd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) in May 2019.
3×1014 vg/kg selected as optimal dose.
Following collaborative interactions with the FDA and EMA, initiated enrollment of eight additional XLMTM patients into the ASPIRO pivotal expansion cohort, designed to confirm the safety and efficacy profile of AT132 at a dose of 3×1014 vg/kg.
Enrollment in the pivotal expansion cohort is expected to be complete in the fall of 2019.
BLA submission for AT132 planned in mid-2020; MAA submission planned for the second half of 2020.
Next clinical data presentation planned at the 24th International Annual Congress of the World Muscle Society (WMS) in Copenhagen, Denmark, October 1-5, 2019.
Additional information on the ASPIRO Pivotal Expansion Cohort:

The pivotal expansion cohort is enrolling eight patients, consisting of four age-matched pairs (+/- 6 months), with one patient from each pair randomized to receive a single dose of AT132 (3×1014 vg/kg) or serve as a delayed treatment control. Delayed treatment control patients will be administered AT132 following the collection of 24-week data from the full pivotal cohort.
Patients will be followed for safety and efficacy for five years, with the primary analysis occurring 24 weeks post treatment.
Key inclusion criteria: less than 5 years of age or have been enrolled in INCEPTUS, on invasive ventilator support for 20-24 hours per day, unable to sit without assistance for at least 30 seconds.
The primary efficacy endpoint is defined as change from baseline in INCEPTUS, on invasive ventilator support for 20-24 hours per day, unable to sit without assistance for at least 30 seconds.
The primary efficacy endpoint is defined as change from baseline in hours of ventilatory support over time through week 24. As of the April 2019 ASPIRO data analysis, all nine patients treated achieved sustained and meaningful reductions in ventilatory support, with four patients successfully completely weaned off of mechanical ventilation.
AT845 for Pompe Disease:

Completed IND-enabling dose ranging and toxicology studies.
On track to submit IND in the third quarter of 2019.
AT702/AT751/AT753 for DMD:

Plan to commence patient dosing in the fourth quarter of 2019 with AT702 produced by NCH. AT702 is designed to induce exon 2 skipping to treat DMD caused by duplications of exon 2 (Dup2) and mutations in exons 1-5 of the dystrophin gene.
Dose ranging and toxicology studies underway to support a first quarter 2020 IND submission for the Audentes AT702 construct and transition the balance of the AT702 DMD program into a Phase 1/2 clinical study utilizing this product candidate.
The construct backbone of the Audentes AT702 product is designed to serve as a vectorized exon skipping platform for rapid expansion into additional DMD genotypes.
Preclinical work is underway to advance AT751 and AT753 to treat DMD patients with genotypes amenable to exons 51 and 53 skipping.
In combination, AT702, AT751 and AT753 have the potential to address more than 25% of DMD patients; plan to leverage our vectorized exon skipping platform to develop additional product candidates with the potential to address up to 80% of DMD patients over time.
AT466 for DM1:

Preclinical vector screening studies underway.
Plan to submit IND in 2020.
Manufacturing:

Advanced chemistry, manufacturing, and controls (CMC) BLA and MAA-readiness efforts for AT132.
State-of-the-art, internal, large-scale cGMP manufacturing facility provides sufficient capacity for AT132 global commercialization as well as the near-term clinical development of all pipeline programs.
Completed construction and commissioning of state-of-the-art, internal plasmid manufacturing facility to support production of nonclinical and cGMP-grade plasmids for all of our development programs, including the potential commercialization of AT132.
Corporate:

Appointed Edward R. Conner, M.D. as Senior Vice President and Chief Medical Officer. Ed is responsible for leading the company’s global clinical development strategy and oversees clinical development, clinical operations, regulatory affairs, medical affairs and patient advocacy.
Promoted Fulvio Mavilio, Ph.D. to Senior Vice President of Translational Science. Fulvio is responsible for advancing the company’s pipeline from discovery through to IND-enabling preclinical development and oversees discovery biology, pharmacology/toxicology, bioinformatics and bioanalytics.
Second Quarter 2019 Financial Results

Cash Position: As of June 30, 2019, cash, cash equivalents and marketable securities were $378.6 million.
Research and Development Expenses: Research and development expense was $37.3 million for the second quarter of 2019 compared to $26.3 million for the same period in 2018, an increase of $11.0 million. The increase was primarily attributable to higher direct program expenses for AT132 and AT845, along with additional R&D headcount to advance clinical and pre-clinical programs. Included in R&D expense for the three months ended June 30, 2019 was $3.2 million of non-cash stock-based compensation expense, compared to $2.3 million in the same period in 2018. For the six months ended June 30, 2019, research and development expense was $77.1 million compared to $46.2 million for the same period in 2018.
General and Administrative Expenses: General and administrative expense was $9.8 million for the second quarter of 2019 compared to $6.3 million for the same period in 2018. The increase was primarily attributable to headcount increases and infrastructure investment to support growth. Included in G&A expense for the three months ended June 30, 2019 was $2.9 million of non-cash stock-based compensation expense, compared to $1.8 million in the same period in 2018. For the six months ended June 30, 2019, general and administrative expense was $21.8 million compared to $12.8 million for the same period in 2018.
Net Loss: Net loss was $44.8 million for the second quarter of 2019 compared to $31.4 million for the same period in 2018. Basic and diluted net loss per share for the three months ended June 30, 2019, was $1.01 compared with $0.85 for the same period in 2018. For the six months ended June 30, 2019, net loss was $94.2 million compared to $56.9 million for the same period in 2018. Basic and diluted net loss per share for the six months ended June 30, 2019, was $2.14 compared with $1.59 for the same period in 2018.
Conference Call
At 4:30 p.m. Eastern Time today, Audentes management will host a conference call and a simultaneous webcast to discuss its second quarter 2019 financial results and provide a corporate update. To access a live webcast of the conference call, please visit the Events & Presentations page within the Investors + Media section of the Audentes website at www.audentestx.com. Alternatively, please call (833) 659-8620 (U.S.) or (409) 767-9247 (international) and dial the conference ID# 4244738 to access the call.

A replay of the webcast will be available on the Audentes website for approximately 30 days.