On May 7, 2019 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors, reported additional positive interim safety and efficacy data from its ongoing open label, single arm Phase 1/2 study of Annamycin in Poland (Press release, Moleculin, MAY 7, 2019, View Source [SID1234535842]). After receiving a single starting dose of 120 mg/m2 in the first cohort of the dose escalation phase of the trial, 2 of 3 patients treated responded sufficiently to qualify for a potentially curative bone marrow transplant. The results for all 3 patients were reviewed by the Safety Review Committee, which determined that no drug-related adverse events were observed that would prevent advancing the trial to the next higher dose level of 150 mg/m2. To date in the European trial, one patient experienced grade 2 mucositis (which resolved to grade 1 within 2 days) and no other adverse events related to Annamycin have been reported. No additional patient data have been developed in the Company’s parallel US clinical trial, which is currently recruiting its second cohort to be given a dose level of 120 mg/m2 (the US trial started at a lower initial dose of 100 mg/m2).

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Moleculin Biotech, Inc. is a clinical-stage pharmaceutical company focused on the treatment of highly resistant cancers. (PRNewsfoto/Moleculin Biotech, Inc.)

"The progress in Europe, specifically Poland, continues to be encouraging," commented Walter Klemp, Moleculin’s Chairman and CEO. "In just a few months, we have completed the first cohort and we’ve seen a partial response from 2 out of 3 patients sufficient to qualify them for a potentially curative bone marrow transplant. It’s important to remember that these are relapsed or refractory patients for whom the standard of care failed. So, to see this kind of response at the starting dose level in the dose-ranging phase, we believe is quite remarkable. And, although this data is still preliminary and may not be indicative of the ultimate outcome of the trial, the improvement in activity at 120 mg/m2 in Poland as compared with the 100 mg/m2 cohort in the US is consistent with our expectations for Annamycin."

Mr. Klemp continued: "Recognizing that cardiotoxicity is a significant limitation of existing therapies, we are pleased that we continue to see no evidence of cardiotoxicity in any of the patients treated thus far. Specifically, there was no observed reduction in left ventricle ejection fraction, which is the standard metric for acute cardiotoxicity, nor any change in biomarkers that would indicate the potential for long-term cardiovascular impairment. This is an important step in the ongoing clinical study of Annamycin and toward our goal of ultimately demonstrating the drug’s safety and effectiveness to support regulatory approval in both the US and European Union."

Dr. Robert Shepard, Moleculin’s Chief Medical Officer for Annamycin added: "With the Polish trial now progressing to 150 mg/m2, we expect to see even better results. And, although 150 mg/m2 was the maximum tolerable dose established by the prior developer of Annamycin due to the incidence of mucositis in patients above that dose level, now that the cryotherapy protocol is well understood to mitigate the potential for dose-limiting mucositis, there is a good opportunity for dose levels to progress even beyond 150 mg/m2, so the potential to help patients is very exciting."

Study Design

The Company is studying Annamycin in both the US and Poland in open label, single arm clinical trials to assess the safety and efficacy of Annamycin for the treatment of adults with relapsed or refractory acute myeloid leukemia. The US and Polish trials have the same study design, consisting of a Phase 1 intended to establish a "Recommended Phase 2 Dose" ("RP2D"), to which the studies will then proceed. The Phase 1 studies provide for escalating doses in cohorts of 3 patients each, with each successive cohort receiving the next higher dose level until "dose limiting toxicities" prevent further increases. Cohort 1 in Poland received a dose of 120 mg/m2, and the results permit moving to 150 mg/m2. Cohort 1 in the US started at 100 mg/m2, and the results support moving to 120 mg/m2, and the Company is now seeking patients for the second cohort in both studies. (Because one patient in US cohort 1 did not complete the evaluation protocol, a fourth patient was added to complete that cohort.) Once the Company establishes an RP2D, the intent is for each trial to advance to a Phase 2 arm planned to assess the safety and efficacy of Annamycin in 21 additional patients.

We plan to report top-line results by cohort in each trial, with each announcement also including an update on the other trial. Top-line results will include reporting of any drug-related adverse events ("AEs") and assessment of cardiotoxicity, including Echo or MUGA scans measuring change in ejection fraction and measuring blood troponin level, which is considered a biomarker for potential long-term cardiovascular impairment. To date, one patient experienced grade 2 mucositis (which resolved to grade 1 within 2 days) and no other drug-related AEs have been reported. Also, no loss of ejection fraction or reduction in Troponin levels has been reported. Top-line results will also include the number of partial responses ("PRs"), complete responses ("CRs") and patients deemed capable of progressing to a potentially curative bone marrow transplant, which we term "bridge to transplant" ("BTs"), each of which is essentially a function of the magnitude of reduction in a patient’s bone marrow blasts. For purposes of these clinical trials, a CR means, primarily, that the patient’s bone marrow blasts reduced to 5% or less, a PR means the patient’s bone marrow blasts reduced, but not to the level of a CR, and a BT means patients are deemed capable of progressing to a potentially curative bone marrow transplant. To date, there have been no PRs, CRs or BTs in the US trial, which has treated 4 patients at 100 mg/m2, and 2 PRs and BTs out of 3 patients treated in the Polish trial at 120 mg/m2.

The US trial also differs from the Polish trial in that the FDA would like to review safety data relating to cardiotoxicity from patients treated prior to advancing beyond 120 mg/m2. The Company believes that the additional patient safety data gained from the Polish trial may also assist in the FDA’s review of cardiac safety.

On March 7, 2019 Trovagene, Inc. (Nasdaq: TROV), a clinical-stage oncology therapeutics company, using a precision medicine approach to develop drugs that target cell division (mitosis) for the treatment of leukemias, lymphomas and solid tumor cancers, reported company highlights and financial results for the first quarter ended March 31, 2019 (Press release, Trovagene, MAY 7, 2019, View Source [SID1234535858]). The company is issuing this press release in lieu of conducting a conference call.

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"We are pleased with our accomplishments year-to-date, and our onvansertib clinical development programs continue to advance as planned," said Dr. Thomas Adams, Chief Executive Officer and Chairman of Trovagene. "We began the year with presentation of data from our Phase 2 trial in metastatic Castration-Resistant Prostate Cancer (mCRPC) at the Genitourinary Cancers Symposium (ASCO-GU) and followed this with a presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) demonstrating activity of onvansertib in prostate cancer patients showing initial resistance to anti-androgen therapy. We also achieved a critical milestone in our ongoing Phase 1b/2 trial in Acute Myeloid Leukemia (AML), with a complete response (2 CR’s and 1 CRi) to treatment achieved in 3 of 6 (50%) of evaluable patients at the highest doses of onvansertib (27mg/m2 and 40mg/m2) evaluated in combination with standard-of-care chemotherapy, decitabine."

Dr. Adams added, "We achieved a number of key milestones in the first quarter of 2019, including: Successful completion of the first four dose levels, without any dose-limiting toxicities, in the Phase 1b segment of our AML trial; receipt of a "Study May Proceed" notification from the FDA for our Phase 1b/2 trial in metastatic Colorectal Cancer (mCRC) in January and subsequent agreement with PoC Capital to fund this trial."

The Company has advanced its business to-date in 2019, with the following activities and milestone achievements:

Announced Data Demonstrating Significant Synergy of Onvansertib in Combination with Venetoclax in Cell Model of Venetoclax Resistant AML
On April 23, 2019, Trovagene announced preclinical data that provides support for clinical evaluation of onvansertib in combination with venetoclax (Venclexta – AbbVie) in patients with difficult-to-treat relapsed or refractory acute myeloid leukemia (AML). Preclinical data showed that the combination demonstrated synergy (the combined effect of the two drugs is greater than the sum of their individual effects) with a significant decrease in tumor cell viability.

Announced Update to Phase 1b/2 AML Trial Data Presented at AACR (Free AACR Whitepaper) – Additional Patients Achieve Complete Response at Two Highest Dose Levels of Onvansertib
On April 5, 2019, Trovagene announced updates to the Phase1b/2 AML trial data presented at the AACR (Free AACR Whitepaper) conference on April 1, 2019. Complete response (2 CR’s and 1 CRi) to treatment with onvansertib in combination with decitabine was achieved in 3 of 6 (50%) of evaluable patients at the highest doses (27mg/m2 and 40mg/m2). The first complete response was achieved at the highest dose of onvansertib (40mg/m2) in combination with low-dose cytarabine (LDAC). Overall, approximately 90% clinical benefit has been achieved to-date in the trial and there have been no dose limiting toxicities observed. Dose escalation is continuing with enrollment in the onvansertib 60mg/m2 cohort.

Announced Early Data from Phase 2 Trial Indicates Activity of Onvansertib in Prostate Cancer Patients Showing Initial Resistance to Anti-Androgen Therapy
On April 2, 2019, Trovagene announced early data from its ongoing Phase 2 study evaluating onvansertib in combination with Zytiga in patients with mCRPC. Early prostate specific antigen ("PSA") response was observed when onvansertib is added to abiraterone (Zytiga) in 2 of 6 patients to-date; the first patient achieved the primary efficacy endpoint of disease stabilization. The PSA trajectory in the patient achieving the primary efficacy endpoint indicates alteration of the natural history of early signs of resistance to Zytiga. Patients with observed responses to-date harbor the highly aggressive androgen receptor variant (AR-V7) which is known to be resistant to treatment with Zytiga.

Announced Phase 1b/2 Dose Escalation Trial of Onvansertib in Relapsed/Refractory AML Demonstrates Safety, Tolerability and Relative Durability with Complete Responses at Highest Dose Levels
On April 1, 2019, Trovagene announced the presentation of new data from its ongoing Phase 1b/2 study evaluating onvansertib in combination with standard-of-care chemotherapy in AML. The greatest anti-leukemic activity has been observed in the onvansertib + decitabine arm, with complete response in 2 (1 CR and 1 CRi) of 4 (50%) of evaluable patients from the two highest dose levels. There have been no dose-limiting toxicities observed to-date and two-thirds of patients have completed ≥2 cycles of treatment, with 2 patients currently on treatment for more than 11 and 5 months, respectively. There has been a significant association observed between biomarker-positive patients and response to onvansertib treatment.

Announced Presentation Update on Phase 2 Study of Onvansertib in Combination with Zytiga in Patients with mCRPC at ASCO (Free ASCO Whitepaper)-GU
On February 14, 2019, Trovagene announced the presentation of a poster reviewing its ongoing Phase 2 study evaluating onvansertib in combination with Zytiga (abiraterone acetate)/prednisone in patients with metastatic Castration-Resistant Prostate Cancer (mCRPC) at the Genitourinary Cancers Symposium (ASCO-GU) in San Francisco, CA. The data featured demonstrates the safety and tolerability of onvansertib in combination with Zytiga which was confirmed in the safety lead-in portion of the trial that was completed prior to opening the trial to full enrollment. In addition, a second arm is planned with the goal of maximizing clinical activity by reducing the dosing schedule from the current 21-day cycle to a 14-day cycle.

Announced that Trovagene and PoC Capital Entered into an Agreement to Fund Clinical Development of Onvansertib in Metastatic Colorectal Cancer (mCRC)
On January 29, 2019, Trovagene announced an agreement with PoC Capital to fund its Phase 1b/2 trial of onvansertib in combination with FOLFIRI and Avastin in patients with mCRC with a KRAS mutation. Trovagene submitted an Investigational New Drug (IND) application and protocol to the FDA on December 19, 2018, and received a "study may proceed" notification from the FDA, 28-days later, on January 16, 2019. The trial is being conducted at two prestigious cancer centers in the U.S.; USC Norris Comprehensive Cancer Center and The Mayo Clinic.

Announced New Patent Issued for Combination of Onvansertib and Anti-Androgen Drugs to Treat Non-Metastatic and Metastatic Prostate Cancer
On January 23, 2019, Trovagene announced the issuance of a new patent (10,155,006), entitled Combination Therapies and Methods of Use Thereof for Treating Cancer, by the U.S. Patent and Trademark Office (USPTO). This patent broadens previously issued patent (9,566,280), by expanding the use of onvansertib to encompass combination therapies with any anti-androgen and androgen antagonist drug, such as Zytiga, Xtandi and Erleada for the treatment of metastatic and non-metastatic castrate-resistant prostate cancer. The issuance of this patent further strengthens Trovagene’s existing intellectual property portfolio obtained with the licensing of exclusive global development and commercialization rights to onvansertib from Nerviano Medical Sciences in March, 2017.

First Quarter 2019 Financial Results

Total operating expenses were approximately $4.1 million for the three months ended March 31, 2019, a decrease of $0.7 million from $4.8 million for the same period in 2018. The decrease in operating expenses is attributed to a reduction of $1.0 million in SG&A and $0.4 million for cost of revenues related to the disposition of the CLIA lab, and partially offset by an increase of $0.8 million in R&D costs.

Net cash used in operating activities in the first quarter of 2019 was approximately $3.4 million, compared to $2.9 million in the same period in 2018. The year-over-year increase of $0.5 million can be attributed primarily to funding used to advance the onvansertib clinical trials.

Research and development expenses increased by approximately $0.8 million to $2.6 million for the three months ended March 31, 2019 from $1.9 million for the same period in 2018. The overall increase in research and development expenses was primarily due to the increased outside service costs for clinical studies related to the development of our drug candidate, onvansertib. We expect increases in research and development costs as we advance the onvansertib clinical development programs in AML, mCRPC and mCRC.

Selling, general and administrative expenses decreased by approximately $1.0 million to $1.5 million for the three months ended March 31, 2019 from $2.5 million for the same period in 2018. The significant components of the decrease were primarily due to the reduction in salaries and staff costs and stock-based compensation.

The weighted average diluted shares of common stock outstanding used to calculate per share results for the three months ended March 31, 2019 was 4.1 million.

As of March 31, 2019, Trovagene had approximately $11.3 million of cash and cash equivalents

On May 7, 2019 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company developing first-in-class antibody product candidates focused on unmet medical needs in inflammation, reported operating results for the first quarter ended March 31, 2019 and provided pipeline updates (Press release, AnaptysBio, MAY 7, 2019, View Source [SID1234535886]).

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"2019 is set to be an important year for AnaptysBio, with multiple top-line data readouts from our wholly-owned pipeline, initiation of a new Phase 2b trial of etokimab in eosinophilic asthma and submission of an IND for our next development candidate in our wholly-owned pipeline," said Hamza Suria, president and chief executive officer of AnapytsBio. "We look forward to advancing each of these programs as we work to bring novel treatments to patients with severe inflammatory diseases."

Etokimab (ANB020 Anti-IL-33 Program)

In September 2018, the Company reported positive interim top-line data from its Phase 2a proof-of-concept clinical trial of etokimab in adult patients with severe eosinophilic asthma. The Company will present the full data from this trial at the 2019 European Academy of Allergy and Clinical Immunology (EAACI) Congress. AnaptysBio also plans to initiate a multi-dose Phase 2b randomized, double-blinded, placebo-controlled trial of etokimab in eosinophilic asthma in 2019.

The Company is conducting a Phase 2b randomized, double-blinded, placebo-controlled, multi-dose study in 300 adult patients with moderate-to-severe atopic dermatitis, also referred to as the ATLAS trial, to assess different dose levels and dosing frequencies of subcutaneously-administered etokimab, with top-line data expected in the second half of 2019.

AnaptysBio is conducting a randomized, placebo-controlled Phase 2 trial in approximately 100 adult patients with chronic rhinosinusitis with nasal polyps, also referred to as the ECLIPSE trial. Patients are being treated with two multi-dosing frequencies of subcutaneously-administered etokimab or placebo, each in combination with mometasone furoate nasal spray as background therapy. The Company anticipates top-line data from the ECLIPSE trial will be available in the second half of 2019.

ANB019 (Anti-IL-36 Receptor Program)

The Company is conducting a single arm, open-label Phase 2 trial of ANB019 in approximately 10 patients with generalized pustular psoriasis, or GPP, also known as the GALLOP trial, with top-line data expected in mid-2019.

The Company is conducting a randomized, placebo-controlled, multi-dose Phase 2 trial in 50 patients with palmoplantar pustulosis, or PPP, also known as the POPLAR trial, with top-line data anticipated in the first half of 2020.
First Quarter Financial Results

Cash, cash equivalents and investments totaled $484.2 million as of March 31, 2019 compared to $500.2 million as of December 31, 2018, for a decrease of $16.0 million. The decrease primarily relates to cash used for operating activities.

Research and development expenses were $20.6 million for the three months ended March 31, 2019, as compared to $11.8 million for the three months ended March 31, 2018. The increase was primarily due to continued advancement of the Company’s etokimab and ANB019 clinical programs and additional

personnel-related expenses including share-based compensation during the three months ended March 31, 2019.

General and administrative expenses were $4.1 million for the three months ended March 31, 2019, as compared to $3.9 million for the three months ended March 31, 2018. The increase was primarily attributable to additional personnel-related expenses, including share-based compensation.

Net loss was $22.1 million for the three months ended March 31, 2019, or a net loss per share of $0.82, as compared to a net loss of $15.1 million for the three months ended March 31, 2018, or a net loss per share of $0.63.
Financial Guidance
AnaptysBio expects that its cash, cash equivalents and investments will fund its current operating plan at least through the end of 2020.
About Etokimab
Etokimab, previously referred to as ANB020, is an antibody that potently binds and inhibits the activity of interleukin-33, or IL-33, a pro-inflammatory cytokine that multiple studies have indicated is a central mediator of atopic diseases, which AnaptysBio believes is broadly applicable to the treatment of atopic inflammatory disorders, such as atopic dermatitis, eosinophilic asthma, chronic rhinosinusitis with nasal polyps, or CRSwNP, and potentially other allergic conditions. Following completion of a healthy volunteer Phase 1 trial of etokimab, AnaptysBio continued clinical development of etokimab into a Phase 2a trial for moderate-to-severe adult atopic dermatitis and a placebo-controlled Phase 2a trial in severe adult eosinophilic asthma patients. AnaptysBio is conducting its ATLAS trial, a randomized, double-blinded, placebo-controlled multi-dose Phase 2b clinical trial of etokimab in 300 moderate-to-severe adult atopic dermatitis patients where top-line data is anticipated in the second half of 2019. The Company is conducting its ECLIPSE trial, a randomized, double-blinded, placebo-controlled Phase 2 trial of etokimab in approximately 100 adult patients with CRSwNP with top-line data anticipated in the second half of 2019. AnaptysBio also plans to initiate a randomized, double-blinded, placebo-controlled, multi-dose Phase 2b trial of etokimab in patients with eosinophilic asthma in 2019.
About ANB019
ANB019 is an antibody that inhibits the function of the interleukin-36-receptor, or IL-36R, which AnaptysBio plans to initially develop as a potential first-in-class therapy for patients suffering from generalized pustular psoriasis, or GPP, and palmoplantar pustulosis, or PPP. AnaptysBio has previously presented data from this Phase 1 clinical trial, which demonstrated favorable safety, pharmacokinetics and pharmacodynamic properties that supported advancement of ANB019 into Phase 2 studies. AnaptysBio is conducting its GALLOP trial, a Phase 2 study of ANB019 in GPP where top-line data is anticipated in mid-2019, and its POPLAR trial, a Phase 2 study in PPP where top-line data is anticipated in the first half of 2020.

On May 7, 2019 Onconova Therapeutics, Inc. (NASDAQ: ONTX), a Phase 3 stage biopharmaceutical company focused on discovering and developing novel small molecule drug candidates to treat cancer, with a primary focus on Myelodysplastic Syndromes (MDS), reported that the Company will release its first quarter 2019 financial results on Tuesday, May 14, 2019 before the market opens (Press release, Onconova, MAY 7, 2019, View Source [SID1234535802]). The Company will host a conference call on Tuesday, May 14, 2019, at 9 a.m. Eastern Time to discuss these results. Interested parties may access the call by dialing toll-free (855) 428-5741 from the U.S. or (210) 229-8823 internationally and using conference ID 4275108. The call will also be webcast live. Please click here to access the webcast on the Investor Relations page of the Company’s website. A replay will be available for 90 days.

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About Myelodysplastic Syndromes

Myelodysplastic syndromes (MDS) are conditions that can occur when the blood-forming cells in the bone marrow become dysfunctional and thus produce an inadequate number of circulating blood cells. It is frequently associated with the presence of blasts or leukemic cells in the marrow. This leads to low numbers of one or more types of circulating blood cells, and to the need for blood transfusions. In MDS, some of the cells in the bone marrow are abnormal (dysplastic) and may have genetic abnormalities associated with them. Different cell types can be affected, although the most common finding in MDS is a shortage of red blood cells (anemia). Patients with higher-risk MDS may progress to the development of acute leukemia.

On May 7, 2019 ExCellThera Inc., an advanced clinical stage biotechnology company delivering molecules and bioengineering solutions to expand stem and immune cells for therapeutic use, reported that it has entered into a manufacturing and collaboration agreement with Comprehensive Cell Solutions (CCS), a division of New York Blood Center (NYBC) for the clinical grade production of ExCellThera’s lead cell therapy product, ECT-001 (Press release, ExCellThera, MAY 7, 2019, View Source [SID1234535826]).

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The agreement provides ExCellThera access to New York Blood Center’s cGMP clean room manufacturing facilities in New York for the production of ECT-001, to be used in clinical trials principally in the United States. ExCellThera’s near term pipeline includes several ongoing studies and additional trials using ECT-001 to be initiated in the coming months.

"The New York Blood Center holds one of the world’s largest blood banks and has over 50 years of leadership in research and development, medical services and laboratory services. We are extremely pleased to enter into this multi-year agreement which will allow us to work closer together and leverage their leadership and expertise in this specialized field," said David Millette, Chief Operating and Financial Officer at ExCellThera. "ExCellThera expects a substantial scale-up in production to meet the needs of new trials coming online as ECT-001 advances through the final phases of its clinical development. This collaboration with NYBC will increase overall production capabilities by adding capacity to the existing manufacturing facilities in Montreal, Canada, while also providing redundancies, easing cross-border issues and providing further strategic collaboration opportunities."

"We are proud to partner with ExCellThera as they work to create groundbreaking curative treatments for patients suffering from blood diseases. Their work has the potential to dramatically increase access to stem cells for patients who need them and unleash the therapeutic promise of the hundreds of thousands of cord blood units stored worldwide," said Chief Medical and Scientific Officer at New York Blood Center, Dr. Beth H. Shaz. "New York Blood Center is the perfect fit for this endeavor. With its facilities, in the heart of the city’s life sciences corridor, NYBC can collaborate with leading international biotechnology companies doing critical work to advance medical research. As the creators of the world’s first and largest public cord bank, we’re excited about the opportunity to leverage our experience to support such an important mission."

The ECT-001 technology is a combination of a small molecule, UM171, and an optimized culture system. The technology, capable of expanding the number of stem and immune cells exponentially in as little as seven days, is used in novel curative blood transplant therapies for patients with blood malignancies and other diseases, allowing more rapid engraftment, greatly reduced incidence of transplant-related mortality, low risk of chronic graft-versus-host disease and low risk of relapse, resulting in better outcomes for patients. The FDA has granted ECT-001 orphan drug designation for the prevention of graft-versus-host disease and regenerative medicine advanced therapy designation in the treatment of hematologic malignancies.

The agreement also provides further strategic collaboration between the parties, including opportunities to explore additional avenues to work together in pursuit of a common goal of improving transplantation protocol and the livelihood of patients.