On January 8, 2018 Novavax, Inc. presented Investor Presentation (Presentation, Novavax, JAN 8, 2018, View Source [SID1234522978]).

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On January 8, 2018 Seattle Genetics, Inc. (NASDAQ: SGEN) reported the progress of its pipeline of antibody-drug conjugates (ADCs) at the 36th Annual J.P. Morgan Healthcare Conference (Press release, Seattle Genetics, JAN 8, 2018, View Source;p=RssLanding&cat=news&id=2325358 [SID1234523020]). Through both internal efforts and that of its collaborators, the company’s ADC technology is being employed in more than 20 programs in clinical trials, including multiple late-state development programs across hematologic malignancies and solid tumors.

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"ADCs continue to advance as an important therapeutic modality, both as single agents and as part of various combination regimens, across hematologic malignancies and solid tumors," said Clay Siegall, Ph.D., President and Chief Executive Officer of Seattle Genetics. "We are the industry leader in ADC technology driven by our scientific expertise in monoclonal antibodies, drug payloads and stable linker technologies. Our leadership is further illustrated by the continued clinical and commercial expansion of ADCETRIS (brentuximab vedotin), progress with our late-stage programs enfortumab vedotin and tisotumab vedotin, and the breadth of our pipeline of other ADCs and empowered antibodies. In addition, our collaborators are making significant advances with several programs using our technology. ADCs are an integral part of an evolving cancer treatment paradigm, and we are committed to bringing important new treatments to patients in need."

ADCETRIS, which pioneered a new class of ADCs, is commercially available in 70 countries worldwide and generated more than $600 million in global sales in 2017. On January 2, 2018, the company announced that the FDA accepted for filing a supplemental Biologics License Application (BLA) for ADCETRIS in combination with chemotherapy for the frontline treatment of patients with advanced classical Hodgkin lymphoma. The FDA granted Priority Review for the application, and the Prescription Drug User Fee Act (PDUFA) target action date is May 1, 2018. The submission of the supplemental BLA is based on positive results from a phase 3 clinical trial called ECHELON-1. In October 2017, the FDA granted Breakthrough Therapy Designation (BTD) for ADCETRIS in frontline advanced Hodgkin lymphoma based on the ECHELON-1 study results.

In addition to advancing ADCETRIS, Seattle Genetics and its collaborator Astellas have initiated a pivotal phase 2 clinical trial of enfortumab vedotin for patients with locally advanced or metastatic urothelial cancer who have been previously treated with checkpoint inhibitor (CPI) therapy. The study is designed to support potential registration under the FDA’s accelerated approval regulations. In addition, Seattle Genetics, in collaboration with its development partner Genmab, plans to initiate a phase 2 clinical trial of tisotumab vedotin for patients with recurrent and/or metastatic cervical cancer. This study is intended to support potential registration under the FDA’s accelerated approval regulations.

Seattle Genetics’ ADC technologies are also empowering several collaborator programs in late-stage clinical trials. These include:

GSK2857916, an ADC being developed by GlaxoSmithKline (GSK) for multiple myeloma. GSK recently reported encouraging data from the program at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December 2017;
Polatuzumab vedotin, an ADC being developed by Genentech/Roche. Positive results were presented at ASH (Free ASH Whitepaper) from a phase 2 trial in advanced-stage diffuse large B-cell lymphoma. A phase 3 trial is underway; and,
Depatuxizumab mafodotin, an ADC for glioblastoma in development by AbbVie. Encouraging data have been reported from this ADC, which is currently in a phase 3 clinical trial.
Polatuzumab vedotin and GSK2857916 have both received BTD from the FDA and PRIority MEDicines (PRIME) designations from the European Medicines Agency. These designations signify the importance of therapies such as these in addressing significant unmet medical need.

"Through our robust internal development efforts and our strong licensing and co-development agreements, we are extending the potential of ADCs globally. We look forward to future results of studies that include Seattle Genetics’ novel technologies both as monotherapies, as well as in combination with checkpoint inhibitors and other agents," said Dr. Siegall.

In 2018, Seattle Genetics anticipates several milestones, including:

Working with FDA towards the May 1 PDUFA action date for ADCETRIS in combination with chemotherapy for frontline treatment of patients with advanced classical Hodgkin lymphoma;
Reporting ECHELON-2 data of ADCETRIS in combination therapy in frontline CD30-expressing mature T-cell lymphoma (MTCL);
Continuing enrollment of the enfortumab vedotin (EV) pivotal trial in locally advanced or metastatic urothelial cancer patients previously treated with a checkpoint inhibitor;
Continuing enrollment of the EV phase 1b trial in combination with checkpoint inhibitors, for patients with locally advanced or metastatic urothelial cancer;
Initiating a phase 2 trial of tisotumab vedotin (TV) in recurrent and/or metastatic cervical cancer to potentially support registration; and,
Initiating a phase 2 trial of TV as part of a combination regimen for first-line cervical cancer and a phase 2 trial of TV in other solid tumor types;
Initiating multiple trials evaluating ladiratuzumab vedotin in combination with checkpoint inhibitors in metastatic triple negative breast cancer, as well as evaluation as a neoadjuvant therapy for early breast cancer as part of the I-SPY consortium.
ADCETRIS is currently not approved for the frontline treatment of MTCL or Hodgkin lymphoma.

On January 8, 2018 Molecular Partners AG (ticker: MOLN), a clinical-stage biopharmaceutical company developing a new class of drugs known as DARPin therapies*, reported initial safety and efficacy data from its ongoing phase 2 study of MP0250 in multiple myeloma (MM) (Press release, Molecular Partners, AUG 8, 2018, View Source [SID1234522938]).

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Eight patients have been treated in the first cohort of 8 mg/kg MP0250 and were included in the safety analysis set. No dose-limiting toxicities (DLTs) have been reported to date. Most frequent adverse events were Grade 1 and 2, except two Grade 3 adverse events (thrombocytopenia and transitory liver enzyme elevation) observed in two patients.

Of seven response evaluable patients receiving MP0250 in combination with bortezomib and dexamethasone, three patients showed partial response (PR) and one patient minimal response (MR). Responses were analyzed according to the International Myeloma Working Group (IMWG) criteria.

"We are very pleased with the good initial safety and tolerability data we observed in the first eight patients treated with MP0250 in combination with bortezomib and dexamethasone. Furthermore, we see promising responses within this first dose cohort. We look forward to evaluating additional patients at higher doses of MP0250 with bortezomib and dexamethasone to treat resistant multiple myeloma patients," commented Dr. Andreas Harstrick, Chief Medical Officer at Molecular Partners.

MP0250 is a proprietary DARPin drug candidate neutralizing VEGF and HGF and thus blocking key escape pathways and resistance. Increases in VEGF and HGF are associated with disease progression in multiple myeloma and have been linked to poor prognosis: they are known to be able to stimulate neovascularization, bone destruction, and myeloma proliferation, migration, and adhesion in the bone marrow. MP0250 shows activity in many preclinical tumor models including in multiple myeloma models in which it enhances the effects of bortezomib on inhibition of M protein production and bone lysis and reduces invasion of tumor cells. MP0250 has shown a favorable safety profile in a phase 1 clinical study in 45 patients in advanced solid tumors.

In the phase 2 MM study, the safety and efficacy of MP0250 is examined in combination with bortezomib (Velcade) and dexamethasone in patients with relapsed and refractory multiple myeloma (RRMM) who have failed standard therapies. The study is performed in Germany, Poland and Italy. A total of 40 patients are planned to be treated, 12 patients in the dose-escalation phase (Part 1) to establish a safe dose, and an additional 28 patients in the dose-expansion phase (Part 2) resulting in a total of 34 patients at the target dose. Additional safety and efficacy data are expected by the end of 2018.

About the DARPin Difference
DARPin therapeutics are a new class of protein therapeutics that open an extra dimension of multi-specificity and multi-functionality. DARPin candidates are potent, specific, safe and very versatile. They can engage more than five targets at once, offering potential benefits over those provided 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 therapies 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. 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 systemic DARPin molecule, MP0250, is in a clinical POC study in multiple myeloma. In addition, Molecular Partners will evaluate MP0250 for the treatment of solid tumors in a phase 1b/2 trial in patients with epidermal growth factor receptor (EGFR)-mutated non-small cell lung cancer (NSCLC). MP0274, the company’s second-most advanced DARPin drug candidate in oncology, has entered into phase 1 clinical development. With its broad anti-HER activity, MP0274 inhibits HER1-, HER2-, and HER3-mediated downstream signaling via Her2, leading to induction of apoptosis. 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.

On January 8, 2018 Sanofi and Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported that it will accelerate and expand investment for the clinical development of the PD-1 (programmed cell death protein 1) antibody cemiplimab in oncology and dupilumab in Type 2 allergic diseases (Press release, Sanofi Genzyme, JAN 8, 2018, View Source [SID1234522976]). Both of these breakthrough therapies have the potential to benefit a number of different patient populations and this strategic investment will enable the companies to evaluate cemiplimab and dupilumab in broad clinical development programs.

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Under the terms of the expansion, the investment in cemiplimab will be increased to $1.64 billion, an increase of approximately $1 billion over the initial 2015 agreement and Sanofi and Regeneron will continue to equally fund cemiplimab development. The companies will also continue their investment in other immuno-oncology programs under their existing Immuno-oncology Discovery Agreement. Investigational cemiplimab is being studied as monotherapy and in combination with other therapies in a wide range of cancers including advanced skin cancers, non-small cell lung cancer, cervical cancer and lymphomas, with more studies in other indications planned to begin in 2018. The companies expect to submit U.S. and EU regulatory applications for cemiplimab in advanced cutaneous squamous cell carcinoma in the first quarter of 2018.

The additional investment in the dupilumab development program will help accelerate planned new studies in chronic obstructive pulmonary disease, peanut allergy and grass allergy as well as in patients who have multiple allergic conditions. These areas are in addition to ongoing dupilumab clinical development in pediatric atopic dermatitis, pediatric asthma, eosinophilic esophagitis and nasal polyposis. Dupixent (dupilumab) is approved for the treatment of adults with moderate-to-severe atopic dermatitis in the U.S. and EU and a U.S. supplemental biologics license application was submitted for uncontrolled, persistent asthma for patients aged 12 and over in the fourth quarter of 2017.

The additional investment will also accelerate and expand development of REGN3500, an IL-33 antibody, with studies expected to be conducted in atopic dermatitis, asthma and chronic obstructive pulmonary disease. The increased funding for dupilumab and REGN3500 will be pursuant to the existing Antibody License and Collaboration Agreement between the companies.

"The ongoing collaboration between Sanofi and Regeneron underscores our commitment to partnering in the development of medicines to treat significant unmet medical needs," said Elias Zerhouni, MD, Global Head of R&D at Sanofi. "The expansion of these clinical programs for both cempilimab and dupilumab should enable us to quickly identify treatment opportunities in other disease areas."

Regeneron has agreed to grant a limited waiver of the "lock-up" in the Amended and Restated Investor Agreement between the companies, so that Sanofi may sell a small percentage of the Regeneron common stock it owns to fund a portion of the cemiplimab and dupilumab development expansion. This waiver will allow Sanofi to sell in private transactions to Regeneron up to an aggregate of 1.4 million shares of Regeneron common stock through the end of 2020, representing approximately 6 percent of the 23.9 million shares of Regeneron common stock Sanofi currently owns. As of October 20, 2017 there were 107.4 million shares of Regeneron capital stock outstanding. If Regeneron decides not to purchase the shares, Sanofi will be allowed to sell those shares on the open market, subject to certain volume and timing limitations. Further details on the updated agreements are available in Regeneron’s current report on Form 8-K filed today.

Cemiplimab and dupilumab were invented by Regeneron using the company’s proprietary VelocImmune technology that yields optimized fully-human antibodies. Other than the approved uses of Dupixent, cemiplimab, Dupilumab, and REGN3500 are under clinical investigation and their safety and efficacy have not been fully evaluated by any regulatory authority.

On January 8, 2018 PTC Therapeutics, Inc. (NASDAQ: PTCT) today provided a corporate update, which will be detailed as part of the company’s presentation at the 36th Annual J.P. Morgan Healthcare Conference on Wednesday, January 10th at 2:30 pm PT (Press release, PTC Therapeutics, JAN 8, 2018, View Source [SID1234525048]). Stuart W. Peltz, Ph.D., PTC’s Chief Executive Officer, will highlight the company’s 20-year commitment to bring best-in-class therapies to patients affected by rare disorders, the company’s 2018 strategic priorities, preliminary 2017 financial results and 2018 financial guidance. The presentation will be webcast live on the Events and Presentations page under the investors section of PTC Therapeutics’ website at www.ptcbio.com.

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Preliminary 2017 Unaudited Financial Results

PTC expects to report Translarna (ataluren) net product revenue for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) of approximately $145 million for 2017, an increase of 78% over the prior year. This strong performance, which achieves the upper end of the company’s guidance for the full year 2017, reflects the rapid uptake and the high unmet need in this community. PTC continues to be pleased by the greater than 90% compliance rate of patients on therapy.
PTC expects to report EMFLAZA (deflazacort) net product revenue for the treatment of Duchenne muscular dystrophy (DMD) of approximately $29 million for 2017, 16% higher than the upper end of the company’s guidance for the full year 2017.
PTC expects to report year-end cash and cash equivalents of approximately $191 million.
2018 Guidance

PTC anticipates full-year net product revenues to be between $260 and $295 million. PTC anticipates Translarna net product revenue for the full year 2018 to be between $170 and $185 million. PTC projects a 5-year (12/31/17-12/31/22) compound annual growth rate of 15% representing continued strong growth year-over-year of Translarna in existing countries and in expansion into new territories. PTC anticipates EMFLAZA net product revenue for the full year 2018 to be between $90 and $110 million.
PTC anticipates GAAP R&D and SG&A expense for the full year 2018 to be between $280 and $290 million.
PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2018 to be between $250 and $260 million, excluding estimated non-cash, stock-based compensation expense of approximately $30 million.
Corporate Highlights

Successful commercial launch of EMFLAZA for the treatment of Duchenne muscular dystrophy. PTC has established programs with the goal of ensuring that all eligible patients will have access to EMFLAZA regardless of financial or insurance status. PTC is committed to improving the standard of care for all Duchenne patients.
Continued strong growth of Translarna product revenue outside US in nonsense mutation Duchenne patients. PTC plans continued growth in Translarna ex-US business by increasing penetration in current countries, expanding into new geographies, and pursuing opportunities for label expansion.
As part of the US FDA appeal process for the Translarna NDA, a meeting is scheduled at the request of the Office of New Drugs and PTC plans to provide an update in the first quarter.
The SUNFISH trial in the spinal muscular atrophy (SMA) program transitioned to the pivotal portion in 2017 with FIREFISH anticipated to transition to the pivotal stage in the coming months. Survival data from FIREFISH study in Type 1 SMA patients will be presented at the upcoming SMA Europe International Scientific Congress in Krakow. The SMA program is a joint collaboration with Roche and the SMA Foundation.
PTC continues to expand its innovative pipeline with internal research programs in the company’s next generation readthrough platform, alternative splicing platform and key developments in oncology with two DHODH inhibitor compounds.
PTC to host an analyst day in the upcoming months to provide an update on its growing pipeline.
Non-GAAP Financial Measures:
In this press release, the unaudited financial results and financial guidance of PTC are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, non-GAAP financial measures exclude non-cash, stock-based compensation expense. This non-GAAP financial measure is provided as a complement to results reported in GAAP because management uses this non-GAAP financial measure when assessing and identifying operational trends. In management’s opinion, this non-GAAP financial measure is useful to investors and other users of PTC’s financial statements by providing greater transparency into the operating performance at PTC and the company’s future outlook. Quantitative reconciliations of these non-GAAP financial measures to GAAP financial measures are included in the table below.

PTC Therapeutics, Inc.

Reconciliation of Projected GAAP to Non-GAAP Full Year 2018 R&D and SG&A Expense (In thousands)

Low End of Range

High End of Range

Projected GAAP R&D and SG&A expense

280,000

290,000

Less: projected non-cash stock-based compensation expense

30,000

30,000

Total projected non-GAAP R&D and SG&A expense

$

250,000

$

260,000

Preliminary 2017 Financial Results:
PTC is currently in the process of finalizing its financial results for the 2017 fiscal year. The above information is based on preliminary unaudited information and management estimates for the full year 2017, subject to the completion of PTC’s financial closing procedures. In addition, the above information is subject to revision as PTC completes its financial closing procedures for fiscal 2017.