Entry into a Material Definitive Agreement

On September 26, 2019, vTv Therapeutics Inc. (the "Company"), reported that it has entered into a letter agreement (the "September 2019 Letter Agreement"), with MacAndrews & Forbes Group LLC (the "Investor"), for the Investor’s commitment to purchase, at the Company’s option, exercisable on demand during a one-year period after the date of the Letter Agreement (the "Investment Period"), the Company’s Class A common stock, par value $0.01 per share ("Common Stock") at a per share price of $1.46, which is equal to the closing price of the Common Stock for the trading day prior to the date of the September 2019 Letter Agreement (Filing, 8-K, vTv Therapeutics, SEP 30, 2019, View Source [SID1234539933]). The September 2019 Letter Agreement also permits the Investor to exercise an option to purchase Common Stock at the same price up to three times during the Investment Period. The aggregate amount of Common Stock that may be purchased by the Investor (whether at its or the Company’s option) pursuant to the September 2019 Letter Agreement is limited to $10.0 million.

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In consideration for the commitment of the Investor under the September 2019 Letter Agreement, the Investor will receive warrants (the "Warrants") to purchase 400,990 shares of the Company’s Common Stock, exercisable at a price of $1.68, which is 115% of the option price under the September 2019 Letter Agreement. The Warrants will be exercisable until September 26, 2026.

The obligation of the Investor to fund and the obligation of the Company to issue shares under the September 2019 Letter Agreement is subject to the execution of mutually acceptable definitive documentation at the time of a request for funding.

As of September 26, 2019, subsidiaries and affiliates of the Investor held 23,084,267 shares of the Company’s Class B Common Stock and 23,506,897 shares of the Company’s Class A Common Stock. As a result, the Investor’s holdings represent approximately 81.5% of the combined voting power of the Company’s outstanding common stock. One of the Company’s directors, Paul G. Savas, is also an employee of the Investor. The transactions described above were approved in accordance with the Company’s Related Person Transactions Policy.

The descriptions of the September 2019 Letter Agreement and the Warrants contained herein do not purport to be complete and are qualified in their entirety by reference to the September 2019 Letter Agreement and the Warrants, copies of which will be filed as exhibits to the Company’s Quarterly Report on Form 10-Q for the quarter ending September 30, 2019.

Synlogic to Present at the Chardan 3rd Annual Genetic Medicines Conference

On September 30, 2019 Synlogic, Inc. (NASDAQ: SYBX) reported that Aoife Brennan, M.B., B.Ch., Synlogic’s president and chief executive officer, will provide a corporate update at the Chardan 3rd Annual Genetic Medicines Conference on Monday, October 7 at 4:15 pm ET, in New York City (Press release, Synlogic, SEP 30, 2019, View Source [SID1234539932]).

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A live webcast of the presentation can be accessed under "Event Calendar" in the Investors & Media section of the Company’s website. An archived copy of the webcast will be available on the Synlogic website for approximately 30 days after the event.

New England Journal of Medicine publishes data showing improved survival with Jevtana® (cabazitaxel) over second androgen receptor-targeted agent in metastatic castration-resistant prostate cancer

On September 30, 2019 Sanofi Genzyme reported Data published in the New England Journal of Medicine showed that patients with metastatic castration-resistant prostate cancer (mCRPC) previously treated with docetaxel and who progressed within 12 months on an androgen receptor (AR)-targeted agent (abiraterone or enzalutamide) experienced significantly longer radiographic progression free survival (rPFS) with Jevtana (cabazitaxel) plus prednisone compared with abiraterone plus prednisone or enzalutamide (Press release, Sanofi Genzyme, SEP 30, 2019, View Source [SID1234539931]). Overall survival (OS) with Jevtana was also significantly longer. These findings from the CARD study were presented today in the Presidential Symposium of the 2019 European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in Barcelona, Spain.

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"In this study, treatment with Jevtana significantly improved radiographic progression free survival and overall survival compared with enzalutamide or abiraterone," said Professor Ronald de Wit from Erasmus MC University Hospital, Rotterdam, The Netherlands, and the lead investigator of the CARD study. "These results are exciting as they have the potential to impact treatment guidelines for metastatic prostate cancer and current clinical practice."

CARD is a randomized, open-label, treatment sequencing clinical study involving 62 sites across 13 European countries, enrolling 255 patients (median aged 70 years, 31% aged over 75 years) with mCRPC who were previously treated with docetaxel and who progressed within 12 months on an AR-targeted agent, in any order. These patients were randomized 1:1 to Jevtana (25 mg/m2 intravenously every three weeks, daily prednisone, and granulocyte colony-stimulating factor) versus abiraterone (1,000 mg plus prednisone, daily) or enzalutamide (160 mg daily; patients received abiraterone if they were previously treated with enzalutamide, or enzalutamide if they were previously treated with abiraterone).

CARD study met primary and secondary endpoints

The study’s primary endpoint was rPFS, which more than doubled with Jevtana treatment (N=129) compared to abiraterone or enzalutamide (N=126; median 8.0 vs 3.7 months; HR=0.54; 95% CI, 0.40–0.73; p<0.0001). Patients treated with Jevtana experienced an improvement in rPFS in all pre-specified subgroups, irrespective of the timing of the previous alternative AR-targeted agent, before or after docetaxel. Jevtana also significantly improved a key secondary endpoint, OS (median 13.6 vs 11.0 months; HR=0.64; 95% CI, 0.46–0.89; p=0.0078), reducing the risk of death from any cause by 36% compared with abiraterone or enzalutamide. Other key secondary endpoints all favored Jevtana: progression free survival (PFS) (median 4.4 vs 2.7 months; p<0.0001); confirmed prostate specific antigen (PSA) (35.7% vs 13.5%; p=0.0002) and tumor responses (36.5% vs 11.5%; p=0.004). Pain response (45.0% vs 19.3%; p<0.0001) and time to symptomatic skeletal events (not reached vs 16.7 months; p=0.0499) were also significantly improved with Jevtana treatment.

The incidence of grade ≥3 adverse events was (56.3% with Jevtana vs 52.4% with AR-targeted agents). Key grade ≥3 treatment-emergent adverse events with Jevtana versus AR-targeted agents were renal disorders (3.2% vs 8.1%), infections (7.9% vs 7.3%), musculoskeletal pain/discomfort (1.6% vs 5.6%), cardiac disorders (0.8% vs 4.8%), asthenic conditions (4.0% vs 2.4%), diarrhea (3.2% vs 0), peripheral neuropathy (3.2% vs 0) and febrile neutropenia (3.2% vs 0). Serious adverse event rates of any grade were similar for Jevtana treatment (38.9%) and treatment with an AR-targeted agent (38.7%). AEs led to death in 7 vs 14 patients (5.6% vs 11.3%) for Jevtana compared to AR-targeted agents. No new safety signals were observed.

About Prostate Cancer

Prostate cancer is a very heterogenous disease and one of the most common types of cancer in men.[i] Prostate cancer is the second leading cause of cancer related death among men in the United States2 and the third in Europe.3

Metastatic castration-resistant prostate cancer (mCRPC) is prostate cancer that has spread beyond the prostate gland and progressed despite androgen deprivation therapy.

About Jevtana (cabazitaxel)

Jevtana is a semi-synthetic taxane chemotherapy. Jevtana is a microtubule inhibitor that binds to tubulin. This leads to the stabilization of microtubules, which results in the inhibition of mitotic and interphase cellular functions.

U.S. INDICATION

JEVTANA is a prescription anti-cancer medicine used with the steroid medicine prednisone. JEVTANA is used to treat men with castration-resistant prostate cancer (prostate cancer that is resistant to medical or surgical treatments that lower testosterone) that has worsened (progressed) after treatment with other medicines, including docetaxel.

IMPORTANT SAFETY INFORMATION FOR U.S. PATIENTS

What is the most important information I should know about JEVTANA?

JEVTANA may cause serious side effects, including:

Low white blood cells, which can cause you to get serious infections, and may lead to death. Men who are 65 years or older may be more likely to have these problems. Your healthcare provider (HCP):

will do blood tests regularly to check your white blood cell counts during your treatment with JEVTANA.
may lower your dose of JEVTANA, change how often you receive it, or stop JEVTANA until your HCP decides that you have enough white blood cells.
may prescribe a medicine for you called G-CSF, to help prevent complications if your white blood cell count is too low.

Tell your HCP right away if you have any of these symptoms of infection during treatment with JEVTANA: fever (take your temperature often during treatment with JEVTANA), cough, burning during urination, or muscle aches.

Also, tell your HCP if you have any diarrhea during the time that your white blood cell count is low. Your HCP may prescribe treatment for you as needed.

Severe allergic reactions can happen within a few minutes after your infusion of JEVTANA starts, especially during the first and second infusions. Your HCP should prescribe medicines before each infusion to help prevent severe allergic reactions.

Tell your HCP right away if you have any of these symptoms of a severe allergic reaction during or soon after an infusion of JEVTANA: rash or itching, skin redness, feeling dizzy or faint, breathing problems, chest or throat tightness, or swelling of face.

JEVTANA can cause severe stomach and intestine problems, which may lead to death. You may need to go to the hospital for treatment.

Vomiting and diarrhea can happen when you receive JEVTANA. Severe vomiting and diarrhea with JEVTANA can lead to loss of too much body fluid (dehydration), or too much of your body salts (electrolytes). Death has happened from having severe diarrhea and losing too much body fluid or body salts with JEVTANA. Your HCP will prescribe medicines to prevent or treat vomiting and diarrhea, as needed with JEVTANA.

Tell your HCP if: you have vomiting or diarrhea, or if your symptoms get worse or do not get better. JEVTANA can cause a leak in the stomach or intestine, intestinal blockage, infection, and bleeding in the stomach or intestine. This can lead to death. Tell your HCP if you get any of these symptoms: severe stomach-area (abdomen) pain, constipation, fever, blood in your stool, or changes in the color of your stool.

Kidney failure may happen with JEVTANA, because of severe infection, loss of too much body fluid (dehydration), and other reasons, which may lead to death. Your HCP will check you for this problem and treat you if needed.

Tell your HCP if you develop these signs or symptoms: swelling of your face or body, or decrease in the amount of urine that your body makes each day or blood in your urine

Lung or breathing problems may happen with JEVTANA and may lead to death. Men who have lung disease before receiving JEVTANA may have a higher risk for developing lung or breathing problems with JEVTANA treatment. Your HCP will check you for this problem and treat you if needed.

Tell your HCP right away if you develop any new or worsening symptoms, including: trouble breathing, shortness of breath, chest pain, cough or fever.

Who should not receive JEVTANA?

Do not receive JEVTANA if: your white blood cell (neutrophil count) is too low, you have had a severe allergic reaction to cabazitaxel or other medicines that contain polysorbate 80 (ask your HCP if you are not sure), you have severe liver problems or you are pregnant. JEVTANA can harm your unborn baby or possibly cause loss of pregnancy.

What should I tell my HCP before receiving JEVTANA?

Before receiving JEVTANA, tell your HCP if you:

had allergic reactions in the past
are age 65 or older
have kidney or liver problems
have lung problems
are a male with a female partner who is able to become pregnant. Males should use effective birth control (contraception) during treatment with JEVTANA and for 3 months after your final dose of JEVTANA.

JEVTANA may cause fertility problems in males. This may affect your ability to father a child. Talk to your HCP if you have concerns about fertility.

Tell your HCP about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. JEVTANA can interact with many other medicines. Do not take any new medicines without asking your HCP first. Your HCP will tell you if it is safe to take the new medicine with JEVTANA.

What are the possible side effects of JEVTANA?

Common side effects of JEVTANA include:

Low red blood cell count (anemia), which is common with JEVTANA, but can sometimes also be serious. Your HCP will regularly check your red blood cell count. Symptoms of anemia include shortness of breath and tiredness.
Low blood platelet count, which is common with JEVTANA, but can sometimes also be serious. Tell your HCP if you have any unusual bruising or bleeding.
Inflammation of the bladder, which has happened in men who have previously received pelvic radiation therapy. Tell your HCP if you have blood in your urine, burning sensation during urination, or frequent or urgent need to urinate.
fever
diarrhea
tiredness
nausea
vomiting
constipation
weakness
back pain
numbness, tingling, burning or decreased sensation in your hands or feet
shortness of breath
stomach pain
change in your sense of taste
cough
joint pain
hair loss
decreased appetite

Tell your HCP if you have any side effect that bothers you or that does not go away. These are not all the possible side effects of JEVTANA. For more information, ask your HCP or pharmacist.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Please see full Prescribing Information / Patient Information, including Serious Side Effects.

Rakuten Medical Releases New, Post-hoc Analysis of Safety, Pharmacokinetic (PK), Immunogenicity and Biomarker Data from its Phase 1 & 1/2a Clinical Trials

On September 30, 2019 Rakuten Medical, a clinical-stage, global biotechnology company developing precision-targeted cancer therapies based on its proprietary, anti-cancer treatment platform, Illuminox, reported post-hoc analysis of safety, pharmacokinetic (PK), immunogenicity and exploratory biomarker data from its RM-1929 photoimmunotherapy (PIT) Phase 1 & 1/2a studies (Press release, Rakuten Medical, SEP 30, 2019, View Source [SID1234539930]). These early clinical trials evaluated the safety and anti-tumor activity of RM-1929 PIT in patients with locoregional, recurrent head and neck squamous cell carcinoma (rHNSCC).

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The key findings were presented during two poster presentations at the 2019 ESMO (Free ESMO Whitepaper) Congress held this week in Barcelona:

"Integrated safety, pharmacokinetics (PK) and immunogenicity data of RM-1929 photoimmunotherapy (PIT) in subjects with locoregional, recurrent head and neck squamous cell carcinoma (rHNSCC)" (Abstract 2954), presented by Jennifer M. Johnson, MD, PhD, FACP; Assistant Professor, Department of Medical Oncology and Associate Program Director, Hematology and Medical Oncology Fellowship, Thomas Jefferson University.

The assessment of safety and PK across the Phase 1 & 1/2a studies, when considered in connection with preliminary Phase 2a clinically meaningful anti-tumor activity reported at ASCO (Free ASCO Whitepaper) 2019, support the current global Phase 3 study of ASP-1929 Illuminox treatment in locoregional rHNSCC patients. Specifically:

Out of the 41 patients in the trial, 17 experienced at least one serious adverse event (SAE), and five experienced SAE considered related to study treatment; the majority being local to the treatment site.
RM-1929 PIT 640 mg/m2 dose achieved an AUC and T1/2, which is expected to achieve saturation of the epidermal growth factor receptors (EGFR). Repeated administration of RM-1929 PIT (cycle 2-4) given approximately four weeks apart, were comparable to those in cycle one, with a low presence of anti-drug antibodies (ADA).
"Intratumoral and peripheral exploratory biomarker analysis in patients with locoregional, recurrent head and neck squamous cell carcinoma (rHNSCC) treated with RM-1929 photoimmunotherapy" (Abstract 3725), presented by Jack Bui, MD, PhD, Pathology; University of California San Diego Health.

Immune-activation biomarkers following RM-1929 PIT treatment were evaluated in the Phase 1/2a study in patients with rHNSCC; conclusions indicated that certain subsets of patients displayed potential signs of immune activation following treatment, regardless of best target tumor response. More specifically:

RM-1929 PIT was associated with induction of PD-L1 expression in tumor and immune cells in eight out of 12 patients.
EGFR expression was maintained in residual tumors in 12 patients with pre- and post-treatment biopsies, indicating that target lesions may be candidates for additional PIT treatment cycles.
Peripheral blood immunophenotyping suggests possible activation of innate and adaptive immunity following treatment in 15 of 18 patients.
"Rakuten Medical is encouraged by these post-hoc analyses across our available early studies, as they further characterize the safety and PK, and provide insights into potential immune activation by RM-1929 PIT that could potentially enable further development in combination with other anti-cancer, immunotherapy treatment options," said Jeannie Hou, VP of Clinical Development at Rakuten Medical. "Our ASP-1929 Illuminox Phase 3 clinical trial is currently underway, which is another important step in the development of a possible new therapeutic option for head and neck cancer patients; and may provide those suffering from this devastating disease with an additional therapeutic choice."

Geron Announces Fast Track Designation Granted to Imetelstat for Relapsed/Refractory Myelofibrosis

On September 30, 2019 Geron Corporation (Nasdaq: GERN) reported that the United States Food and Drug Administration (FDA) has granted Fast Track designation to imetelstat for the treatment of adult patients with Intermediate-2 or High-risk myelofibrosis (MF) whose disease has relapsed after or is refractory to janus kinase (JAK) inhibitor treatment, or relapsed/refractory MF (Press release, Geron, SEP 30, 2019, View Source [SID1234539928]). The Fast Track designation includes patients with primary MF and MF developed after essential thrombocythemia or polycythemia vera. This is the same patient population that was studied in Geron’s IMbark Phase 2 clinical trial. There are currently no marketed drugs specifically approved for relapsed/refractory MF, representing a significant unmet medical need. Geron plans to conduct an End of Phase 2 meeting with the FDA by the end of the first quarter of 2020 to determine if there is a regulatory path forward for imetelstat in relapsed/refractory MF.

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The FDA’s Fast Track Program is designed to facilitate the development and expedite the review of new drugs that are intended to treat serious conditions and supported by data that demonstrate the potential to address an unmet medical need. Fast Track designation provides opportunities for frequent interactions with FDA review staff, including meetings to discuss the drug’s development plan and to ensure the collection of appropriate data needed to support approval. Through the Fast Track Program, a product candidate may be eligible for priority review, if supported by the clinical data, and for the ability to submit completed sections of a New Drug Application (NDA) on a rolling basis as data become available prior to completion of the full application.

About Myelofibrosis

Myelofibrosis (MF), a type of myeloproliferative neoplasm, is a chronic blood cancer in which abnormal or malignant precursor cells in the bone marrow proliferate rapidly, causing scar tissue to form (fibrosis). As a result, normal blood production in the bone marrow is impaired and may shift to other organs, such as the spleen and liver, which can cause them to enlarge substantially. People with MF may have abnormally low or high numbers of red blood cells, white blood cells or platelets in the blood or bone marrow. MF patients can also suffer from debilitating constitutional symptoms, such as fever, weight loss, night sweats, and itching (pruritus). MF patients have shortened survival, and their disease may transform to acute myeloid leukemia.

The number of people living with MF in the United States is estimated at 13,000 patients, with approximately 3,000 new cases diagnosed each year. There are currently only two drugs approved by the FDA for treating MF patients. The most widely used drug therapy for MF has a discontinuation rate of 75% after five years of treatment. Once patients discontinue treatment with this drug due to failure or lack of response, there are no specifically approved therapies, and the median overall survival for these MF patients is approximately 14 to 16 months, representing a significant unmet medical need.

About IMbark

IMbark is a Phase 2 clinical trial to evaluate two starting dose levels of imetelstat (either 4.7 mg/kg or 9.4 mg/kg administered by intravenous infusion every three weeks) in patients with Intermediate-2 or High-risk MF who have relapsed after or are refractory to prior treatment with a JAK inhibitor. The co-primary efficacy endpoints for the trial are spleen response rate and symptom response rate. Key secondary endpoints are safety and overall survival. IMbark is closed to new patient enrollment.

About Imetelstat

Imetelstat is a novel, first-in-class telomerase inhibitor exclusively owned by Geron and being developed in hematologic myeloid malignancies. Early clinical data suggest imetelstat may have disease-modifying activity through the suppression of malignant progenitor cell clone proliferation, which allows potential recovery of normal hematopoiesis. Ongoing clinical studies of imetelstat consist of IMerge, a Phase 2/3 trial in lower risk myelodysplastic syndromes (MDS), and IMbark, a Phase 2 trial in Intermediate-2 or High-risk myelofibrosis (MF). Imetelstat has been granted Fast Track designation by the United States Food and Drug Administration for both the treatment of patients with non-del(5q) lower risk MDS who are refractory or resistant to an erythropoiesis-stimulating agent and for patients with Intermediate-2 or High-risk MF whose disease has relapsed after or is refractory to janus kinase (JAK) inhibitor treatment.