Scynexis has filed a 10-K – Annual report [Section 13 and 15(d), not S-K Item 405] with the U.S. Securities and Exchange Commission (Filing, 10-K, Scynexis, 2018, MAR 13, 2018, View Source [SID1234524715]).

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On March 13, 2018 MorphoSys Reported Updated Data from L-MIND Study of MOR208 plus Lenalidomide in Aggressive Lymphoma (r/r DLBCL) (Press release, MorphoSys, MAR 13, 2018, View Source [SID1234556340]).

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– New data from the ongoing L-MIND trial of MOR208 plus lenalidomide in relapsed/refractory DLBCL patients ineligible for high-dose chemotherapy and autologous stem cell transplantation confirm earlier data reported from this trial

– 81 patients enrolled, 68 available for efficacy assessment at cut-off date

– Preliminary median progression-free survival (PFS) not reached, preliminary PFS rate at 12 months of 50.4%

– Overall response rate (ORR) of 49% with 29 out of 33 responses ongoing, complete response (CR) rate of 31%

– Data show that MOR208 in combination with lenalidomide has been well tolerated in the study: no unexpected toxicities were observed for the treatment combination and no infusion-related reactions were reported for MOR208

– MorphoSys continues to have productive discussions with the FDA under the current breakthrough therapy designation on the path to market for MOR208, including the possibility of an expedited regulatory submission and approval for MOR208 based primarily on the L-MIND study.

MorphoSys AG (FSE: MOR; Prime Standard Segment, TecDAX; OTC: MPSYY) today reported updated data from the ongoing single-arm phase 2 clinical trial known as L-MIND. L-MIND is designed to investigate the antibody MOR208 plus lenalidomide in patients with relapsed or refractory diffuse large B cell lymphoma (r/r DLBCL) who are not eligible for high-dose chemotherapy (HDC) and autologous stem cell transplantation (ASCT). MOR208 is an investigational Fc-engineered monoclonal antibody directed against CD19 and is currently in clinical development in blood cancer indications.

The L-MIND study enrolled patients with r/r DLBCL, who are ineligible for HDC and ASCT, after up to three prior lines of therapy, with at least one prior therapy including an anti-CD20 targeting therapy (such as rituximab). In November 2017, the trial completed patient enrollment with 81 patients. The updated interim data reported today (cut-off date December 12, 2017) included all 81 patients enrolled in the L-MIND trial, 68 of whom were available for efficacy assessment by the investigators at the time of data cut-off. Patients enrolled had a median age of 72 years and had received a median of two prior treatment lines.

Data reported today, with a median observation time of 8.3 months, showed a response in 33 out of 68 patients (overall response rate (ORR) 49%) and a complete response (CR) in 31% of the patients. The preliminary progression-free survival (PFS) rate at 12 months was 50.4% (95% confidence interval 40 – 67%) and the preliminary median PFS had not been reached (95% confidence interval: 4.3 months-not reached). 29 out of 33 responses (88%) were ongoing at the time of data-cut off. Median time to response was 1.8 months, median time to complete response was 3.6 months.

No unexpected toxicities were observed for the treatment combination and no infusion-related reactions (IRRs) were reported for MOR208. The most frequent adverse events with a toxicity grading of 3 or higher were neutropenia, thrombocytopenia, febrile neutropenia and pneumonia, observed in 36%, 12%, 7% and 7% of patients, respectively.

The results reported today confirm and corroborate earlier interim data reported from this trial (Salles et al, ASH (Free ASH Whitepaper) 2017), which had been based on 51 patients enrolled, 44 of whom had been eligible for investigators’ efficacy assessment at the June 13, 2017 cut-off date.

"We are truly excited about this data and our productive discussions with FDA under the current breakthrough therapy designation on the path to market for MOR208, including the possibility of an expedited regulatory submission and approval for MOR208 based primarily on the L-MIND study. We look forward to continuing the analysis of maturing data from the L-MIND trial and to maintaining our interactions with the FDA," commented Dr. Malte Peters, Chief Development Officer of MorphoSys AG.

"There is a very high unmet medical need for patients with r/r DLBCL who, after having failed initial therapies, are ineligible for high-dose chemotherapy and autologous stem cell transplantation," said Dr. Simon Moroney, Chief Executive Officer of MorphoSys AG. "We are very encouraged by our most recent clinical data from the ongoing L-MIND trial, which support our plan to develop MOR208 in combination with lenalidomide as a chemo-free treatment option for this patient population."

About DLBCL
Diffuse large B-cell lymphoma (DLBCL) is the most frequent type of malignant lymphoma worldwide and accounts for approximately 30% of all non-Hodgkin lymphomas. Between 30% and 40% of all patients with DLBCL either fail to respond to or show a relapse to initial therapy. Patients who failed frontline therapy and are not eligible to high dose chemotherapy and autologous transplantation have a very poor outcome and require more therapeutic options.

About CD19 and MOR208
CD19 is broadly and homogeneously expressed across different B cell malignancies including DLBCL and CLL. CD19 has been reported to enhance B cell receptor (BCR) signaling, which is assumed important for B cell survival, making CD19 a potential target in B cell malignancies.
MOR208 (previously Xmab(R)5574) is an investigational humanized Fc-engineered monoclonal antibody directed against CD19. Fc-modification of MOR208 is intended to lead to a significant potentiation of antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP), thus aiming to improve a key mechanism of tumor cell killing. MOR208 has been observed in preclinical models to induce direct apoptosis by binding to CD19, which is assumed to be a crucial component for B cell receptor (BCR) signaling.
MorphoSys AG is clinically investigating MOR208 as a therapeutic option in B cell malignancies in a number of ongoing combination trials. An open-label phase 2 combination trial (L-MIND study) is investigating the safety and efficacy of MOR208 in combination with lenalidomide in patients with relapsed/refractory DLBCL who are not eligible for high-dose chemotherapy (HDC) and autologous stem cell transplantation (ASCT). Based on interim data from L-MIND, in October 2017 the FDA granted Breakthrough Therapy Designation for MOR208 plus lenalidomide in this patient population. The pivotal phase 2/3 B-MIND study is designed to investigate MOR208 in combination with the chemotherapeutic agent bendamustine in patients with relapsed/refractory DLBCL who are not eligible for high-dose chemotherapy (HDC) and autologous stem cell transplantation (ASCT) in comparison to the combination of the anti-CD20 antibody rituximab plus bendamustine. In addition, MOR208 is currently being investigated in patients with relapsed/refractory CLL/SLL after discontinuation of a prior Bruton tyrosine kinase (BTK) inhibitor therapy (e.g. ibrutinib) in combination with idelalisib or venetoclax.

On March 13, 2018 ERYTECH Pharma (Euronext Paris: ERYP – Nasdaq: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported that Chief Executive Officer, Gil Beyen, will present at the Cowen and Company 38th Annual Health Care Conference on Wednesday, March 14, 2018 at The Marriott Copley Place, in Boston, MA (Press release, ERYtech Pharma, MAR 13, 2018, View Source;p=RssLanding&cat=news&id=2337929 [SID1234524732]).

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Conference Details:

Conference: Cowen and Company 38th Annual Health Care Conference
Date: March 14, 2018
Presentation Time: 9:20 AM EDT / 14:20 CET

A live webcast of the presentation will be available online from the investor relations page of the company’s corporate website at View Source and via the below link: http://wsw.com/webcast/cowen46/eryp/

Following, the live webcast, an archive of the presentation will be available on the company website for 30 days, under the "Investors" section at investors.erytech.com.

On March 13, 2018 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported interim clinical data of BPX-501 in pediatric patients with acute myeloid leukemia (AML) and primary immunodeficiencies (PIDs). BPX-501 is an adjunct T cell therapy incorporating CaspaCIDe administered after haploidentical hematopoietic stem cell transplant (haplo-HSCT) for the treatment of hematologic cancers and inherited blood diseases (Press release, Bellicum Pharmaceuticals, MAR 13, 2018, View Source [SID1234524699]).

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Data from the ongoing BP-004 trial suggest that BPX-501 T cells may contribute to a durable anti-leukemic effect in patients with AML. In the study, 38 pediatric AML patients in their first (n=13) or second (n=25) complete response underwent a haplo-HSCT followed by treatment with BPX-501. With a median follow-up of one year, rates of relapse-free survival and overall survival were 91.5% and 97.3%, respectively. This compares to one-year rates reported in the literature in pediatric AML patients undergoing alternate-donor HSCT of 60% to 80%.

"The recurrence of cancer is one of the most serious risks to AML patients receiving a stem cell transplant. These impressive results in children with AML suggest that BPX-501 may be effectively reducing or eradicating residual cancer cells following a haplo-transplant procedure," commented Neena Kapoor, M.D., Director of the Blood and Marrow Transplantation Program at Children’s Hospital of Los Angeles and an investigator in the BP-004 trial.

Also from the BP-004 study, the Company reported high rates of disease-free survival and overall survival in pediatric patients with PIDs, including Severe Combined Immunodeficiency ("bubble boy disease"), Wiskott-Aldrich syndrome, Chronic Granulomatous Disease, and other rare immune deficiencies. Of 59 pediatric PID patients undergoing a haplo-HSCT and treatment with BPX-501, disease-free survival was reported at 88.1% and overall survival was reported at 88.6% with a median follow-up of one year.

Continued Dr. Kapoor: "Delayed immune reconstitution can lead to severe infectious complications, a major cause of morbidity and mortality in PID patients who undergo a T-depleted haplo-HSCT. BPX-501 donor T cells administered after a transplant support immune recovery in these patients, and the CaspaCIDe safety switch engineered into BPX-501 may provide a critical safety net to address the risk of uncontrolled GvHD from donor T cells."

Based on these clinical data, Bellicum is working with the investigators and the U.S. Food and Drug Administration to develop a protocol for a potential U.S. registration study in pediatric patients. Pending regulatory clearances, the Company expects to initiate the study by the end of 2018. These clinical data have been submitted for presentation at an upcoming medical meeting.

About BPX-501
BPX-501 is an adjunct T cell therapy administered after allogeneic HSCT, comprising genetically modified donor T cells incorporating Bellicum’s CaspaCIDe safety switch. It is designed to provide a safety net to eliminate alloreactive BPX-501 T cells (via administration of activator agent rimiducid) should uncontrollable GvHD or other T-cell mediated complications occur. This enables physicians to more safely perform stem cell transplants by administering BPX-501 engineered T cells to speed immune reconstitution, provide control over viral infections and enhance Graft-versus-leukemic effect without unacceptable GvHD risk. The ongoing BP-004 clinical study of BPX-501 is being conducted at transplant centers in the U.S. and Europe.

About CaspaCIDe
CaspaCIDe (also known as inducible Caspase-9, or iC9) is the Company’s safety switch, incorporated into certain Bellicum product candidates. The CaspaCIDe switch consists of the Chemical Induction of Dimerization, or CID, binding domain coupled to the signaling domain of Caspase-9, an enzyme that is an integral part of the apoptotic, cell death pathway. If a patient experiences a serious side effect, the activator agent, rimiducid, is administered to trigger dimerization and activation of the safety switch, which in turn leads to selective apoptosis of the CaspaCIDe-expressing cells.

On March 13, 2018 Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company that designs and develops innovative human enzyme therapeutics for patients with rare genetic diseases and cancer, today reported new repeat dose data from its Phase 1/2 open-label trial of pegzilarginase (AEB1102) in patients with Arginase 1 Deficiency (Press release, Aeglea BioTherapeutics, MAR 13, 2018, View Source [SID1234524967]). The Company also reported financial results for the fourth quarter and year ended December 31, 2017.

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"We have seen encouraging results after repeated doses of pegzilarginase in patients with Arginase 1 Deficiency," said Anthony G. Quinn, M.B Ch.B, Ph.D., interim chief executive officer of Aeglea. "Along with marked and sustained reductions in plasma arginine levels, we are seeing consistent reductions in the levels of other guanidino compounds beyond what appears to be achieved through current approaches to disease management. This is important given the potential contribution of guanidino compounds to the progressive hyperargininemia-related neurological abnormalities seen in this patient population. In addition to our developing understanding of the effects of lowering arginine levels in patients with Arginase 1 Deficiency, we have a good understanding of the safety profile in a larger population that includes patients from our cancer trials. We look forward to sharing additional updates on Arginase 1 Deficiency at the ACMG Annual Clinical Genetics Meeting in April 2018 and in the third quarter of 2018. These updates will include clinical insights from the two adult patients in the long-term extension study."

"Repeated doses of pegzilarginase lowered and maintained plasma arginine and guanidino compound metabolites beyond what was achievable with current standard of care, which consists of a protein-restricted diet and ammonia scavengers," said George Diaz, M.D., Ph.D, professor and chief, Division of Medical Genetics, Icahn School of Medicine at Mount Sinai, and co-author on the abstract. "Given that some patients benefit from the reductions in plasma arginine achieved with dietary protein restriction, it will be important to learn whether longer term reductions in plasma arginine with pegzilarginase beyond that achieved with diet translates into clinical benefit."

Clinical Updates

Arginase 1 Deficiency:

Aeglea presented repeat dose data from the Phase 1/2 clinical trial of pegzilarginase for two adult patients and single ascending dose data for one pediatric patient with Arginase 1 Deficiency at the 2018 Society for Inherited Metabolic Disorders (SIMD) Annual Meeting on March 12, 2018.

Sustained lowering of plasma arginine was achieved with repeated weekly IV dose of 0.04 mg/kg of pegzilarginase.

Arginine-derived metabolites elevated at baseline were rapidly decreased and reductions maintained with repeat doses of pegzilarginase.

Pegzilarginase was well tolerated with the exception of a single infusion-associated reaction in one pediatric patient who had anti-drug antibodies (ADA) and blunting of the expected reduction in plasma arginine after the second dose. The patient transitioned to the repeat dose part of the trial and received three further infusions. Although dosing was well tolerated with premedication and slower infusion rates, the patient withdrew consent due to the burden of balancing school and the clinical trial.

No marked or sustained increase in ADA titers were seen in the two adult Arginase 1 Deficiency patients or in the 48 cancer patients tested after dosing with pegzilarginase. Baseline ADA at low titer was detected in one of two adult Arginase 1 Deficiency patients and four of 48 cancer patients. There was no apparent effect of the presence of the ADA on arginine reduction or safety profile.

The effects of repeat dosing of pegzilarginase provides an opportunity to evaluate the clinical benefits of sustained reduction of plasma arginine beyond what can be achieved with standard of care therapy.

The Company expects to report pediatric and adult repeat dose data in patients with Arginase 1 Deficiency in the third quarter of 2018.
Cancer:

The first uveal and cutaneous melanoma patients were dosed with pegzilarginase in Aeglea’s open-label Phase 1 cohort expansions, with the intent to confirm the safety profile and Phase 2 dose and identify further signals of clinical activity.

The Company expects to report Phase 1 cohort expansion topline data, including safety and clinical activity, in the fourth quarter of 2018.
Upcoming Events

Aeglea will present a corporate update at the 17th Annual Needham Healthcare Conference on March 27, 2018 in New York.

Aeglea will present a poster with additional data on Arginase 1 Deficiency patients at the 2018 ACMG Annual Clinical Genetics Meeting on April 12, 2018 in Charlotte, North Carolina that will include additional clinical insights on baseline standardized assessments of neuromotor function and on short-term treatment with repeat doses of pegzilarginase.

Fourth Quarter and Full Year Financial Results

As of December 31, 2017, Aeglea had available cash, cash equivalents and marketable securities of $50.3 million. Based on Aeglea’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations through the third quarter of 2019.

Aeglea recognized grant revenues of $1.5 million in the fourth quarter of 2017, compared with $1.2 million in the fourth quarter of 2016. The grant revenues were the result of a $19.8 million research grant received from the Cancer Prevention and Research Institute of Texas (CPRIT). The revenue increase was primarily due to higher qualifying expenditures associated with the clinical trials for pegzilarginase in cancer patients in the fourth quarter of 2017 compared with the fourth quarter of 2016.

Grant revenues of $5.2 million were recognized in the year ended December 31, 2017, compared with $4.6 million in the year ended December 31, 2016. The increase was primarily due to higher qualifying expenditures associated with the clinical trials for pegzilarginase in cancer patients in 2017 compared with 2016.

Research and development expenses totaled $5.8 million for the fourth quarter of 2017, compared with $4.7 million for the fourth quarter of 2016. The increase was primarily due to expanded clinical activity for Aeglea’s lead product candidate, pegzilarginase, as Aeglea initiated three solid tumor single-agent cohort expansions and a Phase 1/2 combination trial in patients with small cell lung cancer.

Research and development expenses totaled $22.8 million for the year ended December 31, 2017, compared with $18.1 million for the year ended December 31, 2016. The increase was primarily associated with expanded manufacturing, regulatory, research, and clinical development capabilities, as Aeglea completed its Phase 1 dose escalation trial in patients with advanced solid tumors. Additionally, the Company initiated enrollment in three solid tumor single-agent cohort expansions and a Phase 1/2 combination trial in patients with small cell lung cancer.

General and administrative expenses totaled $2.3 million for the fourth quarter of 2017, compared with $2.0 million in the fourth quarter of 2016. This increase was primarily due to higher employee compensation costs.

General and administrative expenses totaled $10.1 million for the year ended December 31, 2017, compared with $8.4 million for the year ended December 31, 2016. This increase was primarily due to higher employee compensation, consulting, and facility costs.

Net loss totaled $6.5 million and $5.5 million for the fourth quarter of 2017 and 2016, respectively. Net loss totaled $27.2 million and $21.7 million for the years ended December 31, 2017 and 2016, respectively.

Conference Call & Webcast Details

Aeglea will hold a conference call on Tuesday, March 13, 2018 at 8:00 a.m. ET. To access the live conference call via phone, please dial (877) 709-8155 (toll free) within the United States, or +1 (201) 689-8881 internationally. A replay of the call will be available through March 20, 2018 by dialing (877) 660-6853 within the United States or +1 (201) 612-7415 internationally. The conference ID is 13677425.

To access the live and archived webcast of the presentation, please visit the Presentations & Events section of the Aeglea BioTherapeutics investor relations website. Please connect to the website at least 15 minutes prior to the presentation to allow for any software download that may be necessary.