On March 2, 2020 ITM Isotopen Technologien München AG (ITM), a biotechnology and radiopharmaceutical group of companies reported, that it has expanded a long-term agreement for the supply of highly pure no-carrier-added (n.c.a.) Lutetium-177 (177Lu), which was originally concluded in 2018 between ITM AG and Endocyte, Inc., a Novartis company (Press release, ITM Isotopen Technologien Munchen, MAR 2, 2020, View Source [SID1234555073]). Under the agreement, ITM will provide Endocyte with supply of n.c.a 177Lu for its investigational 177Lu-PSMA-617 radioligand therapy for patients with metastatic castration-resistant prostate cancer. Pending approval of investigational drug candidate 177Lu-PSMA-617, ITM would continue to provide its proprietary n.c.a. 177Lu during the commercial phase, supporting the scalability and security-of-supply for patients world-wide.

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In addition, the parties have agreed that n.c.a. 177Lu supplied by ITM will be used in future pipeline investigations.

Sidonie Golombowski-Daffner, President of Advanced Accelerator Applications, the radioligand business of Novartis, stated: "We believe in the potential of targeted radioligand therapy to transform the lives of patients and aspire to make it broadly available to those in need. This supply agreement supports our future growth and ability to investigate radioligand therapies in a variety of cancers."

Steffen Schuster, CEO of ITM, commented: "We are very pleased to expand this long-term alliance, which allows us to further expand and support the development and increasing global patient access to targeted radioligand therapies. We are proud to contribute to these treatments by providing our highly pure n.c.a. 177Lu. By 2030, therapeutic radiopharmaceuticals are expected to account for 50% of the nuclear medicine market. Thanks to the extension of the Endocyte supply agreement, we are confident that we can make a significant impact to this growing demand."

On March 2, 2020 MorphoSys AG (FSE: MOR; Prime Standard Segment; MDAX & TecDAX; NASDAQ: MOR) reported that the U.S. Food and Drug Administration (FDA) accepted filing of MorphoSys’ Biologics License Application (BLA) and granted priority review for tafasitamab, the Company’s investigational anti-CD19 antibody, under review in combination with lenalidomide for the treatment of relapsed or refractory diffuse large B cell lymphoma (r/r DLBCL) (Press release, MorphoSys, MAR 2, 2020, View Source [SID1234555011]).The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of August 30, 2020. The FDA has informed MorphoSys that they are not currently planning to hold an advisory committee meeting to discuss the application.

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"We are extremely pleased that the FDA has accepted filing of our application and granted priority review, as we believe that the combination of tafasitamab and lenalidomide may provide an additional treatment option for patients suffering from DLBCL, who have relapsed after or are refractory to the current standard of care," said Dr. Malte Peters, Chief Development Officer of MorphoSys. "We would like to thank all patients participating in our clinical studies and we will continue to work relentlessly towards making tafasitamab available to patients."

The BLA submission is based on the primary analysis data from the L-MIND trial of tafasitamab in combination with lenalidomide in patients with r/r DLBCL and the retrospective observational matched control cohort Re-MIND evaluating efficacy outcomes of r/r DLBCL patients who received lenalidomide monotherapy. MorphoSys announced the submission of the BLA at the end of December 2019.

Priority Review is granted to therapies that the FDA determines have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. This designation shortens the FDA review period following the acceptance of the BLA to six months compared to ten months under Standard Review.

MorphoSys was granted Breakthrough Therapy Designation by the FDA for the combination of tafasitamab and lenalidomide in r/r DLBCL in 2017.

Tafasitamab is an investigational humanized Fc-engineered monoclonal antibody directed against CD19. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Under the terms of the collaboration and licensing agreement, FDA acceptance of MorphoSys’ BLA submission triggers an undisclosed milestone payment from MorphoSys to Xencor.

About L-MIND
L-MIND is a single arm, open-label phase 2 study, investigating the combination of tafasitamab and lenalidomide in patients with relapsed or refractory diffuse large B cell lymphoma (r/r DLBCL) after up to two prior lines of therapy, including an anti-CD20 targeting therapy (e.g. rituximab), who are not eligible for high-dose chemotherapy and subsequent autologous stem cell transplantation. The study’s primary endpoint is objective response rate (ORR). Secondary outcome measures include duration of response (DoR), progression-free survival (PFS) and overall survival (OS). In May 2019, the study reached its primary completion. Primary analysis data with a cut-off date of November 30, 2018 included 80 patients enrolled into the trial who had received tafasitamab and lenalidomide and had been followed-up as per protocol for at least one year. Efficacy results in this update were based on response rates assessed by an independent review committee for all 80 patients.

About Re-MIND
Re-MIND, an observational retrospective study, was designed to isolate the contribution of tafasitamab in the combination with lenalidomide and to prove the combinatorial effect. The study compares real-world response data of patients with relapsed or refractory DLBCL who received lenalidomide monotherapy with the efficacy outcomes of the tafasitamab-lenalidomide combination, as investigated in MorphoSys’s L-MIND trial. Re-MIND collected the efficacy data from 490 r/r DLBCL patients in the U.S. and EU. Qualification criteria for matching patients of both studies were pre-specified. As a result, 76 eligible Re-MIND patients were identified and matched 1:1 to 76 of 80 L-MIND patients based on important baseline characteristics. Objective response rates (ORR) were validated based on this subset of 76 patients in Re-MIND and L-MIND, respectively. The primary endpoint of Re-MIND has been met and shows a statistically significant superior best ORR of the tafasitamab/lenalidomide combination compared to lenalidomide monotherapy.

About tafasitamab
Tafasitamab is an investigational humanized Fc-engineered monoclonal antibody directed against CD19. In 2010, MorphoSys licensed exclusive worldwide rights to develop and commercialize tafasitamab from Xencor, Inc. Tafasitamab incorporates an XmAb(R) engineered Fc domain, which is intended to lead to a significant potentiation of antibody-dependent cell-mediated cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP), thus aiming to improve a key mechanism of tumor cell killing.
In January 2020, MorphoSys and Incyte Corporation entered into a collaboration and licensing agreement to further develop and commercialize tafasitamab globally. In the U.S., MorphoSys and Incyte will co-commercialize tafasitamab, outside the U.S., Incyte will have exclusive commercialization rights. The agreement is subject to clearance by the U.S. antitrust authorities under the Hart-Scott-Rodino Act as well as by the German and Austrian antitrust authorities and will become effective as soon as these conditions have been met.
Tafasitamab is being clinically investigated as a therapeutic option in B cell malignancies in a number of ongoing combination trials. An open-label phase 2 combination trial (L-MIND study) is investigating the safety and efficacy of tafasitamab in combination with lenalidomide in patients with relapsed/refractory DLBCL who are not eligible for high-dose chemotherapy (HDC) and autologous stem cell transplantation (ASCT). The ongoing phase 3 study B-MIND assesses the combination of tafasitamab and bendamustine versus rituximab and bendamustine in r/r DLBCL. In addition, tafasitamab is currently being investigated in patients with relapsed/refractory CLL/SLL after discontinuation of a prior Bruton tyrosine kinase (BTK) inhibitor therapy (e.g. ibrutinib) in combination with idelalisib or venetoclax.

On March 2, 2020 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company developing first-in-class antibody product candidates focused on emerging immune control mechanisms applicable to inflammation and immuno-oncology indications, reported operating results for the fourth quarter and year ended December 31, 2019 and provided pipeline updates (Press release, AnaptysBio, MAR 2, 2020, View Source [SID1234555042]).

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"We look forward to three Phase 2 clinical trial readouts from our wholly-owned etokimab and ANB019 programs and the expansion of our pipeline with ANB030 and ANB032 during 2020," said Hamza Suria, president and chief executive officer of AnaptysBio. "AnaptysBio is a capital-efficient antibody discovery and development engine that has been validated by the advancement of 7 internally-generated therapeutics to the clinic over the last 4 years, and we look forward to anticipated FDA approval of dostarlimab under our GSK partnership."

Etokimab (ANB020 Anti-IL-33) Program

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 topline data from an interim analysis of the ECLIPSE trial in the first half of 2020.

The Company previously announced data from its ATLAS trial, a Phase 2b randomized, double-blinded, placebo-controlled, multi-dose study in approximately 300 adult patients treated with etokimab in moderate-to-severe atopic dermatitis. Each of the etokimab dosing arms failed to meet the primary endpoint of the trial, which was demonstration of statistically greater improvement in the Eczema Area and Severity Index (EASI) relative placebo at week 16. AnaptysBio has discontinued development of etokimab in moderate-to-severe atopic dermatitis.

The Company has decided to postpone the initiation of its planned Phase 2b etokimab clinical trial in eosinophilic asthma, a multi-dose, randomized, double-blinded, placebo-controlled trial in 300-400 patients, until results are available from the ECLIPSE trial.

ANB019 (Anti-IL-36 Receptor) Program

In September, AnaptysBio announced positive topline data from an interim analysis of its Phase 2 clinical trial of ANB019 monotherapy in moderate-to-severe generalized pustular psoriasis, or GPP, also known as the GALLOP trial. In this interim analysis, both patients achieved the primary endpoint of disease score improvement at Day 29 and Day 113 without requiring rescue therapy, demonstrated rapid and sustained mJDA score improvement, with reduction of 58% at Day 8 and 63% at Day 113, and showed complete clearance of skin pustules by Day 8 and through Day 113, with CRP levels decreasing to nearly normal. Enrollment is ongoing in the GALLOP study, and the Company anticipates additional clinical data and a regulatory strategy update for the development of ANB019 in GPP during 2020.

The Company is also 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 topline data anticipated in the second half of 2020.

AnaptysBio has taken steps to enhance enrollment in the GALLOP and POPLAR trials, including expansion of clinical trial sites and countries.
ANB030 (Anti-PD-1 Agonist) Program

ANB030 is a wholly-owned antibody that binds PD-1 in an agonistic manner, leading to reduced T cell activity and anti-inflammatory effects in vivo. Genetic mutations in the PD-1 pathway are associated with increased susceptibility to various inflammatory conditions and we believe ANB030 has the potential to suppress inflammatory diseases by restoring insufficient PD-1-mediated negative signaling on activated T cells. The Company plans to focus future clinical development of ANB030 on certain autoimmune diseases where PD-1 checkpoint receptor function may be under-represented, submitted an Investigational New Drug Application (IND) in the fourth quarter of 2019 and plans to initiate a Phase 1 clinical trial in the first half of 2020. Preclinical data from the ANB030 was presented in June at the 2019 FOCIS Annual Meeting.
ANB032 (Anti-BTLA Modulator) Program

Our fourth wholly-owned program is an anti-BTLA modulator antibody, known as ANB032, which is broadly applicable to human inflammatory diseases associated with lymphoid and myeloid immune cell dysregulation. Mutations in the BTLA signaling pathway are associated with human inflammatory disease and we believe ANB032 silences pro-inflammatory signaling by modulating BTLA binding to HVEM. We anticipate filing an IND for ANB032 in the second half of 2020.
Dostarlimab (Anti-PD-1 Antagonist) Program Partnered with GSK

GSK has recently announced that a first BLA filing for dostarlimab, an AnaptysBio-generated PD-1 antagonist antibody under partnership with TESARO, a GSK company, occurred in the fourth quarter of 2019 for the treatment of endometrial cancer. AnaptysBio anticipates receiving a $10.0 million cash milestone payment upon acceptance of this BLA filing and a $20.0 million cash milestone upon first FDA approval of dostarlimab. Including additional cash milestones due upon future development and commercialization of dostarlimab, TSR-022, an AnaptysBio-generated TIM-3 antibody, and TSR-033, an AnaptysBio-generated LAG-3 antibody, AnaptysBio can potentially receive a total of $1.1 billion in aggregate milestone payments under this GSK partnership. In addition, AnaptysBio is due a 4% to 8% royalty from GSK, tiered upon global sales, for each of the aforementioned programs.
Board of Directors

In September, the Company appointed Laura J. Hamill to its board of directors. Most recently, Ms. Hamill served as Executive Vice President, Worldwide Commercial Operations, for Gilead Sciences, where she was involved in the strategic direction and long-term planning of the organization. Previously, Ms. Hamill held a number of US and international executive roles at Amgen, culminating with Senior Vice President and General Manager where she led ~$20B in U.S. commercial operations.
Fourth Quarter and Full Year Financial Results

Cash, cash equivalents and investments totaled $428.5 million as of December 31, 2019 compared to $500.2 million as of December 31, 2018, for a decrease of $71.7 million. The decrease relates primarily to cash used for operating activities.

Collaboration revenue was $3.0 million and $8.0 million for the three months and year ended December 31, 2019, which related to a milestone for initiation of a Phase 2 trial for TSR-033, the anti-LAG-3 antibody partnered with TESARO, a GlaxoSmithKline (GSK) company, compared to zero and $5.0 million for the three and year ended December 31, 2018.

Research and development expenses were $21.4 million and $99.3 million for the three months and year ended December 31, 2019, compared to $15.9 million and $56.2 million for the three months and year

ended December 31, 2018. The increase was due primarily to continued advancement of the Company’s etokimab and ANB019 clinical programs and additional personnel-related expenses, including share-based compensation.

General and administrative expenses were $3.8 million and $16.1 million for the three months and year ended December 31, 2019, compared to $3.7 million and $15.5 million for the three months and year ended December 31, 2018. The increase was due primarily to personnel-related expenses, including share-based compensation.

Net loss was $20.3 million and $97.3 million for the three months and year ended December 31, 2019, or a net loss per share of $0.75 and $3.60, compared to a net loss of $17.0 million and $61.7 million for the three months and year ended December 31, 2018, or a net loss per share of $0.64 and $2.50.
Financial Guidance
AnaptysBio expects its net cash burn in 2020 will be approximately $60.0 million, and that its cash, cash equivalents and investments will fund its current operating plan at least into 2023.

On March 2, 2020 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and reported financial results for the fourth quarter and full year ending December 31, 2019 (Press release, PTC Therapeutics, MAR 2, 2020, View Source [SID1234555058]).

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"2019 was a year of outstanding execution for PTC. I’m proud of the progress made in every area of the business," said Stuart Peltz, Ph.D., CEO of PTC Therapeutics. "We’ve significantly expanded our rare disease portfolio, strengthened our global commercial engine and added vital gene therapy manufacturing capabilities, all while delivering on our revenue guidance. The innovation in our diverse, multiplatform pipeline continues to create value for stakeholders."

Key Fourth Quarter and Other Corporate Highlights:

Expanding commercial footprint including continued revenue growth

Total revenue for the full year 2019 was $307 million. The Duchenne muscular dystrophy (DMD) franchise, consisting of Translarna (ataluren) and Emflaza (deflazacort), continues to grow with 2019 revenue of $291 million.

PTC continues to leverage its strong Latin American infrastructure to support the launch of multiple products. In 2019, Translarna received approval from the Brazilian health regulatory authority (ANVISA) for ambulatory nmDMD patients 5 years and older. In addition, Tegsedi (inotersen) received ANVISA approval for the treatment of stage 1 or 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). In 2019, PTC established an early access program for patients with Familial Chylomicronemia Syndrome (FCS) in Latin America. PTC anticipates filing for approval of Waylivra (volanesorsen) for the treatment of FCS from ANVISA in 2H 2020.

Real-world data from the Translarna STRIDE registry demonstrated that boys with nonsense mutation Duchenne muscular dystrophy (nmDMD) treated with Translarna and standard of care, preserved the ability to walk for 3.5 years longer than compared with those in a matched natural history cohort. Importantly, pulmonary function was also preserved in those treated with Translarna.

A recent publication on Emflaza adds to the growing body of evidence showing its relative benefit and supports the advantage of switching from prednisone to Emflaza. The data in the publication demonstrated that patients treated with Emflaza had better ambulatory function and were able to walk longer, had a lower risk of scoliosis, greater percentage lean body mass and lower weight than patients treated with prednisone.

Positive pivotal trials for risdiplam support planned global launch in broad range of SMA patient types

Following the U.S. Food and Drug Association’s (FDA) acceptance of the New Drug Application (NDA) for risdiplam, a Prescription Drug User Fee Act (PDUFA) date for risdiplam has been set for May 24, 2020.

Roche’s Marketing Authorization Application (MAA) filing with the European Medicines Agency (EMA) for risdiplam is expected mid-year 2020.

Data from part 2 of the FIREFISH trial will be presented during the American Academy of Neurology (AAN) 2020 Annual Meeting, from April 25 to May 1, 2020.

PTC gene therapy platform continues to make progress with regulatory filings and long-term lease of manufacturing facility

In January 2020, the EMA accepted the MAA for the potential approval of PTC-AADC for the treatment of Aromatic l-amino acid decarboxylase (AADC) deficiency. PTC expects the Committee for Medicinal Products for Human Use (CHMP) opinion by year end 2020.

PTC expects to submit a Biologics License Application (BLA) to the FDA in 2Q 2020.

PTC continues to anticipate that more than 300 global, addressable AADC patients will be identified by launch.

In 2019, PTC leased a state-of-the-art biologics manufacturing facility with supporting research and operations buildings in N.J. PTC expects manufacturing at this facility to begin in 2020.

Continued progress with PTC’s multi-platform, multi-product pipeline

The Phase 2 STAR trial in patients with nonsense mutation aniridia evaluating the effect of ataluren did not meet its primary endpoint of Maximum Reading Speed as measured by MNREAD. The results did show a trend in favor of ataluren and the results will be discussed with experts in the coming weeks, after which a decision will be made on a path forward for the program in this indication.

Topline data from the U.S. study to assess dystrophin levels in subjects with nmDMD after treatment with ataluren are expected in 2Q 2020.

In 2019, PTC declared a development candidate in its Huntington’s Disease program. The compound is currently in GLP safety toxicology studies and PTC expects to initiate a clinical trial by the end of the year. The Huntington’s Disease program was developed through PTC’s alternative splicing platform.

In 2020, PTC plans to initiate three clinical trials in two compounds that regulate inflammation and oxidative stress from its Bio-e (redox) platform. PTC acquired the Bio-e platform through an acquisition of assets from BioElectron Technology Corporation in 2019.

Potential registrational trial of PTC743 in patients with Mitochondrial Epilepsy (MEDS) expected to begin in 2Q 2020. MEDS is estimated to affect 5,000-6,000 patients in the U.S. and E.U. combined.

Potential registrational trial of PTC743 in patients with Friedreich Ataxia (FA) expected to begin in 3Q 2020. This is anticipated to be complementary to PTC-FA, a gene therapy candidate for the treatment of FA, which is expected to enter the clinic in 3Q 2020. FA is estimated to affect 25,000 patients worldwide.

PTC857 will enter into a Phase 1 healthy volunteer study in 3Q 2020. The first indication for PTC857 is planned to be GBA Parkinson’s disease based on strong pre-clinical rationale. This condition affects approximately 50,000-90,000 patients in the U.S.

Additional updates on PTC’s platforms and pipeline will be provided at PTC’s Analyst Day on June 16, 2020.

Fourth Quarter and Full-year 2019 Financial Highlights:

Total revenues were $96.5 million for the fourth quarter of 2019, compared to $86.3 million for the fourth quarter of 2018. Total revenues were $307 million for the full year 2019, compared to $264.7 million for the full year 2018.

Translarna net product revenues were $48.4 million for the fourth quarter of 2019, compared to $56 million for the fourth quarter of 2018. Translarna net product revenues were $190 million for the full year 2019, compared to $171 million for the full year 2018.

Emflaza net product revenues were $32.7 million for the fourth quarter of 2019, compared to $29.8 million for the fourth quarter of 2018. Emflaza net product revenues were $101 million for the full year 2019, compared to $92 million for the full year 2018.

GAAP R&D expenses were $81.8 million for the fourth quarter of 2019, compared to $53.6 million for the fourth quarter of 2018. GAAP R&D expenses were $257.4 million for the full year 2019, compared to $172 million for the full year 2018. The increase in R&D expenses reflects costs associated with advancing the gene therapy platform, increased investment in research programs, advancement of the clinical pipeline and the upfront $10M payment for the acquisition of the BioElectron assets.

Non-GAAP R&D expenses were $76.2 million for the fourth quarter of 2019, excluding $5.6 million in non-cash, stock-based compensation expense, compared to $49.7 million for the fourth quarter of 2018, excluding $4 million in non-cash, stock-based compensation expense. Non-GAAP R&D expenses were $236.6 million for the full year 2019, excluding $20.8 million in non-cash, stock-based compensation expense, compared to $155.9 million for the full year 2018, excluding $16.1 million in non-cash, stock-based compensation expense.

GAAP SG&A expenses were $63.5 million for the fourth quarter of 2019, compared to $48.7 million for the fourth quarter of 2018. GAAP SG&A expenses were $202.5 million for the full year 2019, compared to $153.6 million for the full year 2018. The increase in SG&A expenses was primarily due to continued investment to support our commercial activities.

Non-GAAP SG&A expenses were $57.7 million for the fourth quarter of 2019, excluding $5.8 million in non-cash, stock-based compensation expense, compared to $44.2 million for the fourth quarter of 2018, excluding $4.5 million in non-cash, stock-based compensation expense. Non-GAAP SG&A expenses were $181.2 million for the full year 2019, excluding $21.3 million in non-cash, stock-based compensation expense, compared to $136.4 million for the full year 2018, excluding $17.2 million in non-cash, stock-based compensation expense.

Change in the fair value of deferred and contingent consideration was $12.4 million for the quarter ended December 31, 2019, compared to $19.3 million for the quarter ended December 31, 2018. Change in the fair value of deferred and contingent consideration was $48.4 million for the year ended December 31, 2019, compared to $19.3 million for the year ended December 31, 2018. The change is related to the fair valuation of the potential future consideration to be paid to former equity holders of Agilis, as a result of our merger with Agilis which closed in August 2018. Changes in the fair value were due to the re-calculation of discounted cash flows for the passage of time and changes to certain other estimated assumptions.

Net loss was $77.7 million for the fourth quarter of 2019, compared to net loss of $48.3 million for the fourth quarter of 2018. Net loss was $251.6 million for the full year 2019, compared to net loss of $128.1 million for the full year 2018.

Cash, cash equivalents, and marketable securities was $686.6 million at December 31, 2019, compared to $227.6 million at December 31, 2018.

Shares issued and outstanding as of December 31, 2019 were 61,935,870.

PTC Reaffirms Full Year 2020 Guidance as Follows

PTC anticipates full year DMD franchise net product revenues to be between $320 and $340 million.

PTC anticipates GAAP R&D and SG&A expense for the full year 2020 to be between $610 and $640 million.

PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2020 to be between $545 and $575 million, excluding estimated non-cash, stock-based compensation expense of approximately $65 million.

Today’s Conference Call and Webcast Reminder:
Today’s conference call will take place at 4:30 pm ET and can be accessed by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 1732477. A live, listen-only webcast of the conference call can be accessed on the investor relations section of the PTC website at www.ptcbio.com. The accompanying slide presentation will be posted on the investor relations section of the PTC website. A webcast replay of the call will be available approximately two hours after completion of the call and will be archived on the company’s website for 30 days following the call.

On March 2, 2020 Seattle Genetics, Inc. (Nasdaq:SGEN) reported the cancellation of the Company’s presentation and webcast at the Cowen 40th Annual Healthcare Conference on Tuesday, previously scheduled to take place on March 3, 2020 at 9:20 a.m. Eastern Time (Press release, Seattle Genetics, MAR 2, 2020, View Source [SID1234555074]). Management will no longer be attending the conference as a precautionary measure related to travel amidst the evolving coronavirus situation.

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