On August 30, 2018 SOTIO, a biotechnology company owned by the PPF Group, reported the completion of the acquisition of Cytune Pharma by PPF (Press release, SOTIO, AUG 30, 2018, View Source [SID1234529225]). SOTIO will continue to develop the lead program SO-C101 (RLI-15) within its pipeline and intends to initiate first-in- human clinical trials in early 2019. SOTIO is spearheading all of PPF’s activities in the biotech sector and closely cooperated during Cytune’s acquisition process. All Cytune projects will be developed as part of SOTIO’s pipeline.

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Cytune Pharma develops IL-15 based therapies for the treatment of cancer. The lead molecule of Cytune’s pipeline, SO-C101 (RLI-15), is a human fusion protein of IL-15 and the high-affinity binding domain of IL-15Rα and acts as a specific IL-2/IL-15Rβγ agonist.

The deal announced in 2015 between PPF Group and Cytune involved investments into Cytune and a staged-acquisition of the company, which has now been completed with the acquisition of all outstanding shares of Cytune. Cytune becomes an important part of SOTIO’s activities in the biotech sector and will also closely collaborate with its affiliate Accord Research on the future development of the lead program SO-C101, and other earlier-stage targeted RLI-15 programs. SOTIO is planning to initiate a first-in-human study with SO-C101 in selected European countries and the US in early 2019. A key part of this early clinical program is the combination with immune checkpoint inhibitors.

"We are excited about the completion of the acquisition and welcome Cytune to the PPF Group. Cytune and SOTIO have been closely and very successfully collaborating over the course of the last few years and we very much look forward to initiating the first clinical trials with SO-C101 in a few months," commented Radek Spisek, CEO of SOTIO. "SO-C101 will become a core program of our pipeline, together with our clinical programs in ovarian, lung and prostate cancer based on our autologous dendritic cell therapy platform DCVAC."

"I’m proud that our products, originating from INSERM and the University of Nantes and supported by Bpifrance and Atlanpole Biotherapies, hold the potential to lead to new therapeutic options for the treatment of cancer patients. Such IL-2/IL-15Rβγ agonists represent a new and very promising class of drugs in the immune-oncology segment as they have shown to re-induce responses in patients after failure to checkpoint inhibitors treatment," adds David Bechard, President and COO of Cytune. "The continued financial support by PPF combined with SOTIO’s clinical development expertise will be instrumental in accelerating the development of SO-C101 in multiple clinical trials, ultimately for the benefit of cancer patients."

The financial terms of the transaction are not disclosed.

About SO-C101
Molecule SO-C101 (RLI-15) is a human fusion protein of IL-15 and the high-affinity binding domain of IL-15Rα which acts as a specific agonist of the intermediate-affinity IL-2/IL-15Rβγ. It is a novel immunotherapeutic approach with potential applications in a variety of oncology indications. In preclinical experiments, SO-C101 has been shown to stimulate and induce proliferation of immune effector cells, such as cytotoxic T cells and NK cells, without expanding the CD4+ T regulatory cells. Based on the preclinical experiments, SO-C101 is more potent and better tolerated compared to the unmodified IL-15 or IL-2. SO-C101 and other products based on this platform allow for combinations with other immunotherapeutic strategies, including checkpoint inhibitors.

On August 30, 2018 Bristol-Myers Squibb Company (NYSE: BMY) reported that the U.S. Food and Drug Administration (FDA) accepted its supplemental Biologics License Application (sBLA) for Sprycel (dasatinib) in combination with chemotherapy for the treatment of pediatric patients with newly diagnosed Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL) (Press release, Bristol-Myers Squibb, AUG 30, 2018, View Source [SID1234529168]). The FDA action date is December 29, 2018.

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"Sprycel was first established as an important treatment option for appropriate pediatric patients last year, when it was approved for the treatment of children with Ph+ chronic myeloid leukemia," said Jeffrey Jackson, Ph.D., development lead, hematology, Bristol-Myers Squibb. "This latest milestone in Ph+ ALL reinforces our commitment to researching the potential of Sprycel in different types of pediatric leukemia and to providing this vulnerable population with access to potential new therapies."

The application is based on data from CA180-372 (NCT01460160), an ongoing Phase 2 trial evaluating the addition of Sprycel to a chemotherapy regimen modeled on a Berlin-Frankfurt-Munster high-risk backbone in pediatric patients with newly diagnosed Ph+ ALL.

About Sprycel

Sprycel first received FDA approval in 2006 for the treatment of adults with Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) in chronic phase (CP) who are resistant or intolerant to prior therapy including imatinib. At that time, Sprycel also received FDA approval for adults with Ph+ acute lymphoblastic leukemia (ALL) who are resistant or intolerant to prior therapy. Sprycel is approved and marketed for these indications in more than 60 countries.

Sprycel is also an FDA-approved treatment for adults with newly diagnosed Ph+ CML-CP and is approved for this indication in more than 50 countries.

Both the FDA and the European Commission approved the expansion of Sprycel’s indication to include pediatric patients with Ph+ CML-CP in November 2017 and July 2018.

U.S. FDA-APPROVED INDICATIONS FOR SPRYCEL

SPRYCEL (dasatinib) is indicated for the treatment of adults with:

Newly diagnosed Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) in chronic phase
Chronic, accelerated, or myeloid or lymphoid blast phase Ph+ CML with resistance or intolerance to prior therapy including imatinib
Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) with resistance or intolerance to prior therapy
SPRYCEL is indicated for the treatment of pediatric patients with:

Ph+ CML in chronic phase
IMPORTANT SAFETY INFORMATION

Myelosuppression

Treatment with SPRYCEL is associated with severe (NCI CTCAE Grade 3/4) thrombocytopenia, neutropenia, and anemia, which occur earlier and more frequently in patients with advanced phase CML or Ph+ ALL than in patients with chronic phase CML. Myelosuppression was reported in patients with normal baseline laboratory values as well as in patients with pre-existing laboratory abnormalities.

In patients with chronic phase CML, perform complete blood counts (CBCs) every 2 weeks for 12 weeks, then every 3 months thereafter, or as clinically indicated
In patients with advanced phase CML or Ph+ ALL, perform CBCs weekly for the first 2 months and then monthly thereafter, or as clinically indicated
Myelosuppression is generally reversible and usually managed by withholding SPRYCEL temporarily and/or dose reduction
In clinical studies, myelosuppression may have also been managed by discontinuation of study therapy
Hematopoietic growth factor has been used in patients with resistant myelosuppression
Bleeding-Related Events

SPRYCEL can cause serious and fatal bleeding. In all CML or Ph+ ALL clinical studies, Grade ≥3 central nervous system (CNS) hemorrhages, including fatalities, occurred in <1% of patients receiving SPRYCEL. The incidence of Grade 3/4 hemorrhage, occurred in 5.8% of adult patients and generally required treatment interruptions and transfusions. The incidence of Grade 5 hemorrhage occurred in 0.4% of adult patients. The most frequent site of hemorrhage was gastrointestinal.

Most bleeding events in clinical studies were associated with severe thrombocytopenia
In addition to causing thrombocytopenia in human subjects, dasatinib caused platelet dysfunction in vitro
Concomitant medications that inhibit platelet function or anticoagulants may increase the risk of hemorrhage
Fluid Retention

SPRYCEL may cause fluid retention. After 5 years of follow-up in the adult randomized newly diagnosed chronic phase CML study (n=258), grade 3/4 fluid retention was reported in 5% of patients, including 3% of patients with grade 3/4 pleural effusion. In adult patients with newly diagnosed or imatinib resistant or intolerant chronic phase CML, grade 3/4 fluid retention occurred in 6% of patients treated with SPRYCEL at the recommended dose (n=548). In adult patients with advanced phase CML or Ph+ ALL treated with SPRYCEL at the recommended dose (n=304), grade 3/4 fluid retention was reported in 8% of patients, including grade 3/4 pleural effusion reported in 7% of patients. In pediatric patients with chronic phase CML cases of Grade 1 or 2 fluid retention were reported in 10.3% of patients.

Patients who develop symptoms of pleural effusion or other fluid retention, such as new or worsened dyspnea on exertion or at rest, pleuritic chest pain, or dry cough should be evaluated promptly with a chest x-ray or additional diagnostic imaging as appropriate
Fluid retention events were typically managed by supportive care measures that may include diuretics or short courses of steroids
Severe pleural effusion may require thoracentesis and oxygen therapy
Consider dose reduction or treatment interruption
Cardiovascular Events

SPRYCEL can cause cardiac dysfunction. After 5 years of follow-up in the randomized newly diagnosed chronic phase CML trial in adults (n=258), the following cardiac adverse reactions occurred:

Cardiac ischemic events (3.9% dasatinib vs 1.6% imatinib), cardiac related fluid retention (8.5% dasatinib vs 3.9% imatinib), and conduction system abnormalities, most commonly arrhythmia and palpitations (7.0% dasatinib vs 5.0% imatinib). Two cases (0.8%) of peripheral arterial occlusive disease occurred with imatinib and 2 (0.8%) transient ischemic attacks occurred with dasatinib
Monitor patients for signs or symptoms consistent with cardiac dysfunction and treat appropriately.

Pulmonary Arterial Hypertension (PAH)

SPRYCEL may increase the risk of developing PAH in adult and pediatric patients, which may occur any time after initiation, including after more than 1 year of treatment. Manifestations include dyspnea, fatigue, hypoxia, and fluid retention. PAH may be reversible on discontinuation of SPRYCEL.

Evaluate patients for signs and symptoms of underlying cardiopulmonary disease prior to initiating SPRYCEL and during treatment. If PAH is confirmed, SPRYCEL should be permanently discontinued
QT Prolongation

SPRYCEL may increase the risk of prolongation of QTc in patients including those with hypokalemia or hypomagnesemia, patients with congenital long QT syndrome, patients taking antiarrhythmic medicines or other medicinal products that lead to QT prolongation, and cumulative high-dose anthracycline therapy

Correct hypokalemia or hypomagnesemia prior to and during SPRYCEL administration
Severe Dermatologic Reactions

Cases of severe mucocutaneous dermatologic reactions, including Stevens-Johnson syndrome and erythema multiforme, have been reported in patients treated with SPRYCEL.

Discontinue permanently in patients who experience a severe mucocutaneous reaction during treatment if no other etiology can be identified
Tumor Lysis Syndrome (TLS)

TLS has been reported in patients with resistance to prior imatinib therapy, primarily in advanced phase disease.

Due to potential for TLS, maintain adequate hydration, correct uric acid levels prior to initiating therapy with SPRYCEL, and monitor electrolyte levels
Patients with advanced stage disease and/or high tumor burden may be at increased risk and should be monitored more frequently
Embryo-Fetal Toxicity

Based on limited human data, SPRYCEL can cause fetal harm when administered to a pregnant woman. Hydrops fetalis, fetal leukopenia and fetal thrombocytopenia have been reported with maternal exposure to SPRYCEL. Transplacental transfer of dasatinib has been measured in fetal plasma and amniotic fluid at concentrations comparable to those in maternal plasma.

Advise females of reproductive potential to avoid pregnancy, which may include the use of effective contraception, during treatment with SPRYCEL and for 30 days after the final dose
Effects on Growth and Development in Pediatric Patients

In pediatric trials of SPRYCEL in chronic phase CML after at least 2 years of treatment, adverse reactions associated with bone growth and development were reported in 5 (5.2%) patients, one of which was severe in intensity (Growth Retardation Grade 3). These 5 cases included cases of epiphyses delayed fusion, osteopenia, growth retardation, and gynecomastia. Of these 5 cases, 1 case of osteopenia and 1 case of gynecomastia resolved during treatment.

Lactation

No data are available regarding the presence of dasatinib in human milk, the effects of the drug on the breastfed child or the effects of the drug on milk production. However, dasatinib is present in the milk of lactating rats.

Because of the potential for serious adverse reactions in nursing children from SPRYCEL, breastfeeding is not recommended during treatment with SPRYCEL and for 2 weeks after the final dose
Drug Interactions

Strong CYP3A4 inhibitors: The coadministration with strong CYP3A inhibitors may increase dasatinib concentrations. Increased dasatinib concentrations may increase the risk of toxicity. Avoid concomitant use of strong CYP3A4 inhibitors. If concomitant administration of a strong CYP3A4 inhibitor cannot be avoided, consider a SPRYCEL dose reduction
Grapefruit juice may increase plasma concentrations of SPRYCEL and should be avoided
Strong CYP3A4 inducers: The coadministration of SPRYCEL with strong CYP3A inducers may decrease dasatinib concentrations. Decreased dasatinib concentrations may reduce efficacy. Consider alternative drugs with less enzyme induction potential. If concomitant administration of a strong CYP3A4 inducer cannot be avoided, consider a SPRYCEL dose increase
St. John’s wort may decrease plasma concentrations of SPRYCEL and should be avoided
Gastric Acid Reducing Agents: The coadministration of SPRYCEL with a gastric acid reducing agent may decrease the concentrations of dasatinib. Decreased dasatinib concentrations may reduce efficacy.

Do not administer H2 antagonists or proton pump inhibitors with SPRYCEL. Consider the use of antacids in place of H2 antagonists or proton pump inhibitors. Administer the antacid at least 2 hours prior to or 2 hours after the dose of SPRYCEL. Avoid simultaneous administration of SPRYCEL with antacids.
Adverse Reactions

The safety data reflects exposure to SPRYCEL at all doses tested in clinical studies (n=2809) including 324 adult patients with newly diagnosed chronic phase CML, 2388 adult patients with imatinib resistant or intolerant chronic or advanced phase CML or Ph+ ALL, and 97 pediatric patients with chronic phase CML.

The median duration of therapy in a total of 2712 SPRYCEL-treated adult patients was 19.2 months (range 0–93.2 months). Median duration of therapy in:

1618 adult patients with chronic phase CML was 29 months (range 0–92.9 months)
Median duration for 324 adult patients in the newly diagnosed chronic phase CML trial was approximately 60 months
1094 adult patients with advanced phase CML or Ph+ ALL was 6.2 months (range 0–93.2 months)
In two non-randomized trials in 97 pediatric patients with chronic phase CML (51 patients newly diagnosed and 46 patients resistant or intolerant to previous treatment with imatinib), the median duration of therapy was 51.1 months (range 1.9 to 99.6 months).

In the newly diagnosed adult chronic phase CML trial, after a minimum of 60 months of follow-up, the cumulative discontinuation rate for 258 patients was 39%.

In the overall population of 2712 adult SPRYCEL-treated patients, 88% of patients experienced adverse reactions at some time and 19% experienced adverse reactions leading to treatment discontinuation.

Among the 1618 adult SPRYCEL-treated patients with chronic phase CML, drug-related adverse reactions leading to discontinuation were reported in 329 (20.3%) patients.

In the adult newly diagnosed chronic phase CML trial, drug was discontinued for adverse reactions in 16% of SPRYCEL-treated patients with a minimum of 60 months of follow-up
Among the 1094 SPRYCEL-treated patients with advanced phase CML or Ph+ ALL, drug-related adverse reactions leading to discontinuation were reported in 191 (17.5%) patients.

Among the 97 pediatric subjects, drug-related adverse reactions leading to discontinuation were reported in 1 patient (1%).

Patients ≥65 years are more likely to experience the commonly reported adverse reactions of fatigue, pleural effusion, diarrhea, dyspnea, cough, lower gastrointestinal hemorrhage, and appetite disturbance, and more likely to experience the less frequently reported adverse reactions of abdominal distention, dizziness, pericardial effusion, congestive heart failure, hypertension, pulmonary edema and weight decrease, and should be monitored closely.

In adult newly diagnosed chronic phase CML patients:
Drug-related serious adverse reactions (SARs) were reported for 16.7% of patients. Serious adverse reactions reported in ≥5% of patients included pleural effusion (5%)
Grade 3/4 laboratory abnormalities included neutropenia (29%), thrombocytopenia (22%), anemia (13%), hypophosphatemia (7%), hypocalcemia (4%), elevated bilirubin (1%), and elevated creatinine (1%)
In adult patients resistant or intolerant to prior imatinib therapy:
Drug-related SARs were reported for 26.1% of SPRYCEL-treated patients treated at the recommended dose of 100 mg once daily in the randomized dose-optimization trial of patients with chronic phase CML resistant or intolerant to prior imatinib therapy. Serious adverse reactions reported in ≥5% of patients included pleural effusion (10%)
Grade 3/4 hematologic laboratory abnormalities in chronic phase CML patients resistant or intolerant to prior imatinib therapy who received SPRYCEL 100 mg once daily with a minimum follow up of 60 months included neutropenia (36%), thrombocytopenia (24%), and anemia (13%). Other grade 3/4 laboratory abnormalities included: hypophosphatemia (10%), and hypokalemia (2%)
Among chronic phase CML patients with resistance or intolerance to prior imatinib therapy, cumulative grade 3/4 cytopenias were similar at 2 and 5 years including: neutropenia (36% vs 36%), thrombocytopenia (23% vs 24%), and anemia (13% vs 13%)
Grade 3/4 elevations of transaminases or bilirubin and Grade 3/4 hypocalcemia, hypokalemia, and hypophosphatemia were reported in patients with all phases of CML
Elevations in transaminases or bilirubin were usually managed with dose reduction or interruption
Patients developing Grade 3/4 hypocalcemia during the course of SPRYCEL therapy often had recovery with oral calcium supplementation
In pediatric subjects with Ph+ CML in chronic phase
Drug-related SARs were reported for 14.4% of pediatric patients
In the pediatric studies, the rates of laboratory abnormalities were consistent with the known profile for laboratory parameters in adults
Most common adverse reactions (≥15%) in patients included myelosuppression, fluid retention events, diarrhea, headache, skin rash, hemorrhage, dyspnea, fatigue, nausea, and musculoskeletal pain

On August 30, 2018 Gilead Sciences, Inc. (Nasdaq: GILD) reported that John F. Milligan, PhD, Gilead’s President and Chief Executive Officer; Robin L. Washington, Gilead’s Executive Vice President and Chief Financial Officer; and John McHutchison, AO, MD, Gilead’s Chief Scientific Officer and Head of Research & Development, will participate in a fireside chat at the Morgan Stanley Global Healthcare Conference in New York on Wednesday, September 12 at 11:05 a.m. Eastern Time (Press release, Gilead Sciences, AUG 30, 2018, View Source [SID1234529187]).

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The audio portion of the fireside chat will be accessible live through the company’s Investors page at www.gilead.com/investors. Please connect to the company’s website at least 15 minutes prior to the start of the presentation to ensure adequate time for any software download that may be required to listen to the webcast. The replay will be available for 14 days following the presentation.

On August 30, 2018 Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), a clinical stage pharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors, reported financial results for the quarter ended June 30, 2018 and provided an update on its corporate activities and product pipeline (Press release, Crinetics Pharmaceuticals, AUG 30, 2018, View Source [SID1234529208]).

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"Crinetics has made major progress in 2018 with the company’s initial public offering, which raised net proceeds of $106.4 million," said Scott Struthers, Ph.D., Founder and Chief Executive Officer of Crinetics. "With our balance sheet substantially strengthened, we will focus on growing our operations and preparing for the initiation of our planned Phase 2 clinical trials for CRN00808 in acromegaly in early 2019, as well as developing our other pipeline programs."

Second Quarter 2018 and Subsequent Highlights

Completed initial public offering. In July 2018, Crinetics closed its initial public offering of 6,900,000 shares of common stock at a public offering price of $17.00 per share. Crinetics received approximately $106.4 million in net proceeds, after deducting underwriting discounts, commissions, and estimated offering expenses.

Awarded up to $3.2 million in SBIR grants for congenital hyperinsulinism and acromegaly. In June 2018, Crinetics was awarded up to approximately $3.2 million in Small Business Innovation Research (SBIR) grants from the National Institute of Diabetes and Digestive and Kidney Diseases of the National Institutes of Health (NIH) to fund the continued research and development of its nonpeptide, oral somatostatin agonists for congenital hyperinsulinemias (CHI) and acromegaly. Crinetics will be eligible to receive funding for up to approximately $1.9 million for CHI and $1.3 million for acromegaly.

Appointed Alan S. Krasner, M.D. as Chief Medical Officer. In June 2018, Crinetics appointed Alan S. Krasner, M.D. as Chief Medical Officer. Dr. Krasner joined Crinetics from Shire Pharmaceuticals where he was a Senior Medical Director and served as Global Development Lead for Natpara, the first recombinant human intact parathyroid hormone treatment for hypoparathyroidism. Prior to Shire, he worked at Biodel and Pfizer conducting clinical research at various stages of product development in diabetes and obesity.

Second Quarter 2018 Financial Results

Research and development expenses were $5.2 million and $9.9 million for the three and six months ended June 30, 2018, respectively, compared to $2.1 million and $4.1 million for the same periods in 2017. The increases were primarily attributable to increased manufacturing and development activities associated with clinical and preclinical programs and increased personnel-related costs due to the hiring of additional development personnel.

General and administrative expenses were $1.1 million and $2.4 million for the three and six months ended June 30, 2018, compared to $0.4 million and $1.0 million for the same periods in 2017. The increases were primarily due to increased spending on pre-commercialization activities and legal costs, as well as higher personnel-related costs to support the growth of operating activities.

Net loss for the three months ended June 30, 2018 was $5.6 million, compared to a net loss of $1.6 million for the three months ended June 30, 2017. For the six months ended June 30, 2018, the company’s net loss was $11.0 million compared to a net loss of $4.3 million for the six months ended June 30, 2017.

Cash, cash equivalents and short-term investments totaled $68.4 million as of June 30, 2018, which does not include the $106.4 million in net proceeds from the initial public offering in July 2018, compared with $14.2 million as of December 31, 2017.

As of August 24, 2018, the company had 24,024,231 common shares outstanding.

On August 30, 2018 Molecular Partners AG (SIX: MOLN), a clinical-stage biopharmaceutical company pioneering the use of DARPin therapeutics to treat serious diseases, reported its unaudited financial results for the first half-year 2018 (Press release, Molecular Partners, AUG 30, 2018, View Source [SID1234529588]). In the course of the semester, the company reported promising updated data on the phase 2 trial of its lead oncology asset MP0250. Moreover, on July 19, the company’s strategic partner Allergan announced positive phase 3 topline data for abicipar showing non-inferior efficacy results compared to Lucentis, utilizing a fixed quarterly dosing regimen compared with every four weeks dosing for Lucentis.

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"We are very pleased with our company’s clinical and business progress during the first half of 2018," said Patrick Amstutz, Chief Executive Officer of Molecular Partners. "We are focusing on our DARPin candidates in oncology, while our partner, Allergan, is moving forward with abicipar and has exercised all three options from our discovery alliance agreement, underscoring our mutual conviction regarding the promising potential of DARPin therapeutics in ophthalmology."

"We presented promising updates on clinical data for our lead oncology asset, MP0250, in multiple myeloma. Moreover, we have entered into a collaboration with AstraZeneca ensuring the free supply of Tagrisso for our second phase 2 study of MP0250 in EGFR-mutated non-small cell lung cancer, and we have dosed the first patients," added Andreas Harstrick, Chief Medical Officer of the company.

Updated data confirm promising progress of phase 2 study of MP0250 in multiple myeloma
In April, at the European Myeloma Network (EMN) Conference in Turin, as well as in June, at the 23rd Annual Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) in Stockholm, Molecular Partners presented updated preliminary results from the ongoing phase 2 study of its lead proprietary oncology candidate, MP0250. The ongoing, open-label phase 2 clinical study is examining the safety and efficacy of MP0250 in combination with bortezomib (Velcade) and dexamethasone in patients with relapsed/refractory multiple myeloma (RRMM). All patients had been pretreated with at least two lines of therapy, including an IMiD and bortezomib, and 50% were considered proteasome refractory. The study is being performed at nine centers in Germany, Poland and Italy.

At the data cutoff on May 21, 2018, five of eight evaluable patients achieved an objective response (4 patients with PR/partial response; 1 patient with VGPR/very good partial response). Responses were durable, with median time on treatment for responding patients of 22.5 weeks and the longest response then still ongoing at 41 weeks. Main adverse events were consistent with the known side effect profile of VEGF-targeting agents and of Velcade, respectively.

Overall, a total of at least 40 patients are planned to be treated in this phase 2 study. The company anticipates additional safety data and initial efficacy data to be disclosed before year-end 2018.

First patients dosed in second phase 2 study of MP0250, evaluating MP0250 and osimertinib (Tagrisso) in non-small cell lung cancer (NSCLC)
In April, Molecular Partners announced a collaboration with AstraZeneca (LON: AZN) to conduct a phase 1b/2 clinical study of MP0250 in combination with osimertinib (Tagrisso) in patients with EGFR-mutated non-small cell lung cancer (NSCLC) who were pre-treated with osimertinib. Under the collaboration agreement, AstraZeneca supplies osimertinib for the clinical study. The study is planned to enroll approximately 40 patients and will take place in the United States. The first patients in this phase 2 study were dosed and recruitment is ongoing. Initial safety data are expected by the end of 2018 with initial efficacy data to be disclosed in 2019.

MP0274 in HER2-positive solid tumors: Enrollment for phase 1 study ongoing
Molecular Partners has amended the protocol of the phase 1 study of MP0274, a multi- DARPin product candidate being developed for the treatment of HER2-positive solid tumors, to allow the enrollment of more patients at lower doses. In preclinical studies MP0274 induces a profound inhibition of specific downstream signaling pathways, and directly kills HER2-addicted tumor cells through the induction of apoptosis. This represents a new and differentiated mode of action as compared to current standard of care antibodies.

Enrollment and patient dosing of the phase 1 study is ongoing and the company continues to expect initial safety data in Q4 2018, with the first efficacy data expected in 2019.

Immuno-oncology: Preclinical data on the company’s DARPin "toolbox" and on MP0310
At the 2018 annual meeting of the American Association of Cancer Research (AACR) (Free AACR Whitepaper) in Chicago, Molecular Partners presented new preclinical data on MP0310 as well as its DARPin "toolbox". MP0310 binds to 4-1BB and FAP and is the first immuno-oncology DARPin candidate in development.

Preclinical data indicate that MP0310 can activate immune cells in the tumor microenvironment and not in the general circulation. This may translate into a better efficacy/safety profile than that seen with anti-4-1BB monoclonal antibodies.

Abicipar: Positive topline data announced for two pivotal phase 3 trials for patients with neovascular AMD, demonstrating the efficacy of an abicipar 12-week fixed dosing regimen with 50% fewer injections than Lucentis
On July 19, 2018, Allergan and Molecular Partners announced positive phase 3 topline data from two clinical trials of abicipar. Those trials, called SEQUOIA and CEDAR, demonstrated that both the 8-week and 12-week treatment regimens of abicipar met the pre-specified primary endpoint of non-inferiority to ranibizumab (Lucentis). SEQUOIA and CEDAR are identical global phase 3 studies designed to assess the efficacy and safety of abicipar compared with ranibizumab in treatment-naive patients with neovascular age-related macular degeneration (nAMD). The primary endpoint measured the proportion of treated patients with stable vision at week 52.

In the first year of both studies abicipar demonstrated similar efficacy, after 6 or 8 injections, to a regimen of 13 ranibizumab injections. The overall adverse events were similar among the three treatment arms. The incidence of intraocular inflammation was approximately 15% in the abicipar arms, higher than the rate seen in ranibizumab-treated patients, which was below 1% in both trials. To minimize inflammation, Allergan has further optimized the formulation of abicipar and is currently testing this formulation (MAPLE trial). The trial is currently recruiting patients, with a goal of 100 patients enrolled.

"We are very excited to see that the most advanced DARPin molecule, abicipar, has reached its primary endpoint in phase 3. This is a very important milestone for Molecular Partners and the DARPin technology in general," said Patrick Amstutz, CEO of Molecular Partners. "We are very pleased to see that abicipar can indeed help patients in need with less frequent dosing which was the key point when we generated abicipar in the first place," added Michael T. Stumpp, COO of Molecular Partners.

The SEQUOIA and CEDAR phase 3 clinical trials continue on a masked basis, now in their second year. Full data details of the primary endpoints and the secondary endpoints will be presented at an upcoming scientific conference. Allergan plans to file abicipar in the first half of 2019. Allergan will be requesting a meeting with the Food and Drug Administration (FDA) to discuss the corresponding BLA submission. The market launch of abicipar is foreseen for 2020.

Financial highlights: Increased clinical activities and build-out of organization
In the first half of 2018, Molecular Partners recognized total revenues of CHF 9.4 million (H1 2017: CHF 6.0 million) and incurred operating expenses of CHF 22.1 million (H1 2017: CHF 22.7 million) in line with expectations. This led to an operating loss of CHF 12.7 million for the first half-year (H1 2017: operating loss of CHF 16.7 million). The company recognized a net financing income of CHF 1.0 million (H1 2017: CHF -2.7 million), mainly driven by positive FX effects on the USD and EUR cash positions. This resulted in a net loss of CHF 11.7 million for the first half-year 2018 (H1 2017: CHF 19.4 million).

The net cash used from operating activities during the first half of 2018 was CHF 19.4 million (H1 2017: net cash used of CHF 20.5 million). Including time deposits, the cash and cash equivalents position decreased by CHF 8.9 million vs. year-end 2017 to CHF 122.4 million as of June 30, 2018 (December 31, 2017: CHF 131.3 million). The total shareholders’ equity, at CHF 116.3 million as of June 30, 2018, remained broadly unchanged (December 31, 2017: CHF 116.7 million).

As a result of the adoption of IFRS 15, deferred revenues as of December 31, 2017 of CHF 18.4 million were partly reclassified to equity (CHF 8.9 million) to reflect the accumulated past effect of the adoption as of January 1, 2018. The remaining portion of CHF 9.4 million was recognized as revenues in H1 2018.

As of June 30, 2018, the company employed 112 FTE, up 8% year-over-year as well as compared to year-end 2017. About 90% of the employees are employed in R&D-related functions.

"During the first half of 2018, Molecular Partners’ financial position continued to develop in line with our expectations. Our strong cash position provides us with financial flexibility to achieve multiple value-creating inflection points into 2020," said Andreas Emmenegger, Chief Financial Officer of Molecular Partners.

Pamela A. Trail appointed Chief Scientific Officer and member of the Executive Management
On June 21, 2018, Pamela A. Trail, Ph.D, was appointed Chief Scientific Officer of Molecular Partners and a new member of the Executive Management Team of the company. Dr. Trail served most recently as Vice President of Oncology Strategy and Program Direction at Regeneron Pharmaceuticals. She has over 30 years of experience in directing cancer drug discovery efforts at leading pharmaceutical companies worldwide. Dr. Trail holds a Ph.D. in Immunology and Virology from the University of Connecticut. With her addition the company significantly strengthens its leading research capabilities applying the DARPin platform to oncology drug development.

Michael T. Stumpp appointed Chief Operating Officer
On June 21, 2018, Michael T. Stumpp, Ph.D., a co-founder of Molecular Partners and formerly Chief Scientific Officer of the company, was appointed Chief Operating Officer of Molecular Partners. Dr. Stumpp was part of the research team at the University of Zurich that invented the DARPin technology. Since Molecular Partners’ inception, he has overseen the DARPin pipeline.

Business outlook and priorities
As pertains to the company’s proprietary oncology pipeline, Molecular Partners expects to report additional safety data and initial efficacy data from the phase 2 study of MP0250 in patients with multiple myeloma (MM) in 2018. The company also expects initial safety data from the phase 1b/2 study of MP0250 in NSCLC in 2018, having dosed the first patients. For MP0274, the proprietary, single-pathway DARPin drug candidate for the treatment of HER2-positive cancer, Molecular Partners expects initial safety data in Q4 2018 and first efficacy data in 2019.

The company will continue to advance its immuno-oncology pipeline and will present further research and preclinical data for its DARPin candidate MP0310 in 2018. In this promising field, Molecular Partners has reinforced its focus on activating agonists in a tumor-restricted way.

In ophthalmology, following the positive phase 3 topline results of abicipar announced by Allergan on July 19, 2018, Molecular Partners will continue to support Allergan in advancing abicipar through phase 3 studies in patients with neovascular AMD and in further optimizing the abicipar formulation in order to minimize inflammation. Molecular Partners will also continue to support Allergan in the launch of the phase 3 study for abicipar in DME with the improved abicipar formulation expected for 2019 as well as in advancing the three preclinical ophthalmology assets optioned-in from the existing research collaboration. Allergan anticipates a market launch for abicipar for the neovascular AMD indication in the year 2020.

Financial outlook 2018
As the first half of 2018 developed in line with management’s expectations, Molecular Partners is able to add more precision to the financial outlook 2018 which was provided with the company’s 2017 full-year results on February 8, 2018, as well as in the company’s quarterly management statement on April 26, 2018.

For the full year 2018, at constant exchange rates, the company expects total expenses at the lower end of the CHF 50-60 million range indicated, of which around CHF 6 million will be non-cash effective costs for share-based payments, IFRS pension accounting and depreciations. However, this guidance is subject to the progress of the pipeline, mainly driven by manufacturing costs, the speed of enrollment of patients in clinical studies and data from research and development projects.

No guidance can be provided with regard to net cash flow projections. Timelines and potential milestone payments from existing and potentially new partnerships are not disclosed.

R&D day in New York on December 6, 2018
Molecular Partners will host its 2nd R&D Update in New York City on December 6, 2018.
For details and to reserve a seat, please contact Susan Noonan at [email protected]​.

Investor documentation of H1 2018 results

The H1 2018 results presentation, the H1 2018 press release as well as the unaudited Financial Statements for H1 2018 and the company’s H1 2018 Report and additional information are available on the investors section of the company’s website.

Conference call and audio webcast
Molecular Partners will conduct a conference call and audio webcast of the company’s H1 2018 results on August 30, 2018, at 2:00pm CET (1:00pm GMT, 8:00am EST).
In order to register for the H1 2018 conference call, please dial the following numbers approximately 10 minutes before the start of the presentation:

Switzerland / Europe +41 (0) 58 310 5000
UK +44 (0) 207 107 0613
USA +1 (1) 631 570 5613
Participants will have the opportunity to ask questions after the presentation.

The H1 2018 audio webcast will be accessible, both live and as a replay, on the investors section of the company’s website www.molecularpartners.com​, along with the accompanying presentation slides.

*DARPin is a registered trademark owned by Molecular Partners AG.

Financial Calendar
November 1, 2018 – Q3 2018 Management Statement
December 6, 2018 – R&D Day in New York
February 7, 2019 – Publication of Full-year Results 2018 (unaudited)
March 15, 2019 – Expected Publication of Annual Report 2018
April 16, 2019 – Annual General Meeting
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About the DARPin Difference
DARPin therapeutics are a new class of protein therapeutics opening an extra dimension of multi-specificity and multi-functionality. DARPin candidates are potent, specific, safe and very versatile. They can engage more than 5 targets at once, offering potential benefits over those offered by conventional monoclonal antibodies or other currently available protein therapeutics.
The DARPin technology is a fast and cost-effective drug discovery engine, producing drug candidates with ideal properties for development and very high production yields.

With their good safety profile, low immunogenicity and long half-life in the bloodstream and the eye, DARPin therapeutics have the potential to advance modern medicine and significantly improve the treatment of serious diseases, including cancer and sight-threatening disorders. Molecular Partners is partnering with Allergan to advance clinical programs in ophthalmology, and is advancing a proprietary pipeline of DARPin drug candidates in oncology and immuno-oncology. The most advanced global product candidate is abicipar, a molecule currently in phase 3, in partnership with Allergan. Several DARPin molecules for various ophthalmic indications are also in development. The most advanced DARPin therapeutic candidate wholly owned by Molecular Partners, MP0250, is in phase 2 clinical development for the treatment of solid tumors and hematological tumors. MP0274, the second-most advanced DARPin drug candidate owned by Molecular Partners, has broad anti-HER activity; it inhibits HER1, HER2 and HER3-mediated downstream signaling via Her2, leading to induction of apoptosis. MP0274 is currently in phase 1. Molecular Partners is also advancing a growing preclinical pipeline that features several immuno-oncological development programs. DARPin is a registered trademark owned by Molecular Partners AG.