PTC Therapeutics Corporate Update and Pipeline Progress at #JPM20

PTC Therapeutics, Inc.,  (NASDAQ: PTCT) today is providing a corporate update that will be presented at the 38^th Annual J.P. Morgan Healthcare Conference on Wednesday, January 15^th at 8:30 a.m. PST. Stuart W. Peltz, Ph.D., PTC’s Chief Executive Officer, will provide an update on the 2019 accomplishments and highlight value creating events in the upcoming year. Additionally, the Company is providing preliminary 2019 financial results and 2020 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

Corporate Highlights:

Gene Therapy Platform Update:

• PTC has submitted a Marketing Authorization Application (MAA) for the potential approval of a gene therapy treatment, PTC-AADC, for AADC deficiency with the European Medicines Agency (EMA). PTC expects the Committee for Medicinal Products for Human Use (CHMP) opinion in 2H 2020.
• In a recent interaction with the U.S. Food and Drug Administration (FDA) there was a request for additional information concerning the use of the commercial delivery system for PTC-AADC in young patients. Patients have been identified who are immediately available for treatment with the commercial delivery system. We anticipate that this will cause a short delay of the Biologics License Application (BLA) and should allow for submission to the FDA in 2Q 2020.
• PTC has identified 200 AADC patients to date and continues to anticipate that over 300 AADC patients will be identified by launch.
• PTC-FA gene therapy for Friedreich ataxia is progressing and is anticipated to enter the clinic in 3Q 2020.
• In order to control and accelerate its gene therapy platform, PTC secured a 15-year lease on ~185,000 sq. ft. of space, which includes a state-of-the-art biologics production facility with supporting research and operations buildings in NJ. PTC expects manufacturing to begin at this facility in 2020.

Risdiplam Regulatory & Clinical Updates:     

• The risdiplam Prescription Drug User Fee Act (PDUFA) date for a decision by the FDA is May 24, 2020. Risdiplam is expected to be indicated for spinal muscular atrophy (SMA) type 1, 2 & 3 patients, if approved.
• In November 2019, PTC announced that the pivotal part of the SUNFISH trial in SMA type 2/3 patients was successful, meeting its primary endpoint. Data from the pivotal part of SUNFISH will be presented at the SMA Europe Conference from February 5 – 7, 2020.
• Topline results from the pivotal portion of the open label FIREFISH study in type 1 SMA patients are anticipated in 1Q 2020.
• The SMA program has studied the broadest cohort of SMA patients in clinical trials to date. This program is a collaboration between PTC, Roche and the SMA Foundation.

Expanding commercial platform:

• The Duchenne muscular dystrophy (DMD) franchise, consisting of Translarna™ and Emflaza^®, continues to grow with 2019 unaudited revenue of approximately $291 million.
  • Translarna™ continues to grow with further penetration into existing territories, geographic expansion into new territories, increased awareness and earlier diagnosis. PTC plans to re-submit the Translarna New Drug Application (NDA) to the FDA with the data from the dystrophin study in mid-year 2020.
  • Growth in Emflaza^® revenue was based on a recent label expansion in the 2-5-year-old patient population, and reduced payer restrictions resulting from multiple publications showing benefit of Emflaza relative to prednisone. One such recent publication, which included real-world data from Cincinnati Children’s hospital, specifically highlighted the benefit of switching from prednisone to Emflaza.
• Tegsedi^® (inotersen) received approval from the Brazilian health regulatory authority (ANVISA) for the treatment of stage 1 or 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR), to delay disease progression and improve quality of life. The commercial launch for Tegsedi is ongoing with patient and physician services to support the launch, including medical education and diagnosis efforts.

Growing pipeline and R&D capabilities:

• In 2020, PTC plans to initiate three trials in its newly acquired redox platform with two unique compounds that regulate inflammation and oxidative stress.
  • These include potential registrational trials in mitochondrial epilepsy and Friedreich ataxia with PTC743. Additionally, a Phase 1 trial with PTC857 is also planned targeting GBA Parkinson’s disease.
• In late 2020, PTC expects to file an investigational new drug (IND) application for its development candidate in Huntington disease. PTC has several additional programs from the splicing platform in development with undisclosed targets.

Preliminary Unaudited 2019 Financial Results

• Total unaudited revenue for 2019 was approximately $306 million. This includes expected net product revenue for the Duchenne muscular dystrophy franchise of approximately $291 million for the full year 2019 and a $15 million milestone payment from Roche for the risdiplam NDA acceptance.
• PTC expects to report net product revenue for Translarna of approximately $190 million for full year 2019. As a reminder, the ANVISA approval in Brazil should lead to expanded market access in 2020 and beyond but led to a price discount that impacted 2019 net sales.
• Additionally, PTC expects to report net product revenue for Emflaza of approximately $101 million for the full year 2019.
• PTC expects to report 2019 year-end cash and cash equivalents of approximately $686 million.

PTC is currently in the process of finalizing its financial results for the 2019 fiscal year. The above information is based on preliminary unaudited information and management estimates for the full year 2019, 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 2019.

2020 Guidance

• PTC anticipates full year net product revenues for the DMD franchise to be between $320 and $340 million. New product launches, including Tegsedi, Waylivra, PTC-AADC and risdiplam, could contribute revenue in 2020. Remaining milestones and royalties from Roche on the SMA program are outlined in the table below.
• 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.
• The increase in R&D and SG&A expense are based in part on highly leverageable and scalable investments towards the $1.5 billion projected revenue in 2023, including gene therapy manufacturing, an increase in the number of programs advancing into the clinic and launches.

SMA Milestones/Royalties supporting information

• SMA program Milestone-based payments to PTC from Roche:

Event	Payment ($US)
Filing of a MAA in an EU country or with the EMA	$ 15,000,000
Filing of an NDA in Japan	$   7,500,000
First Commercial Sale in US	$ 20,000,000
First Commercial Sale in the EU	$ 20,000,000
First Commercial Sale in Japan	$ 10,000,000
Total Remaining	$72,500,000

• SMA program Royalties to PTC from Roche based on net sales:

Tier of Calendar Year Worldwide Net Sales in $US million	Percent (%) of Net Sales
0 – 500	8
> 500 – 1,000	11
> 1,000 – 2,000	14
> 2,000	16

• SMA program Sales-threshold-based payments to PTC from Roche:

Event Total Calendar Year Net Sales ($US)	Payment ($US)
> $ 500,000,000	$   25,000,000
> $ 750,000,000	$   50,000,000
> $ 1,500,000,000	$ 100,000,000
> $ 2,500,000,000	$ 150,000,000
Total Remaining	$ 325,000,000

https://www.biospace.com/article/releases/ptc-therapeutics-provides-corporate-update-and-highlights-pipeline-progress-at-2020-j-p-morgan-healthcare-conference/

Cytokinetics Announces Vision 2025 and Outlines 2020 Corporate Milestones

Cytokinetics, Inc. . (Nasdaq: CYTK) today announced its Vision 2025 and provided guidance for corporate milestones expected to occur in 2020. The company’s Vision 2025: “Leading with Science, Delivering for Patients,” articulates its five-year key imperatives enabling Cytokinetics to be the leading muscle biology biopharmaceutical company that meaningfully improve the lives of patients with diseases of impaired muscle function through access to novel medicines arising from its research.

Key imperatives for Vision 2025 include:

• Achieve regulatory approvals for at least two drugs arising from our pipeline
• Build commercial capabilities to market and sell our medicines reflective of their innovation and value
• Generate sustainable and growing revenues from product sales
• Double our development pipeline to include ten therapeutic programs
• Expand our discovery platform to muscle energetics, growth and metabolism
• Be the science-driven company people want to join and partner with

“Our company transformation began five years ago when we outlined our Vision 2020,” said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. “We effectively executed on those initiatives so that we are now well positioned to look forward to the next five-year horizon and the profound privilege to potentially bring forward to patients at least two new medicines from our pioneering muscle biology focused research. We expect to deliver on the promise of our science and to double our pipeline of novel mechanism drug candidates and expand the breadth of our discovery platform. As we execute against our vision, we will support disease advocacy groups elevating the patient voice and live by our values of integrity, fairness and compassion in all that we do.”

In addition, the company announced its expected 2020 milestones.

“To ensure that we deliver on the promise of our science for patients, we recognize that we must continue to plan and execute well in connection with expected milestones in 2020,” continued Mr. Blum. “This will be a pivotal year for Cytokinetics as we now expect trial results from GALACTIC-HF by the end of the year and we need to prepare commercial readiness and co-promotion plans accordingly. In addition, we expect to both advance and expand our pipeline to enable two other programs to potentially advance to late-stage trials while another two programs may advance in earlier-stage trials. We look forward to another highly productive year aligned with our vision.”

Expected 2020 Milestones

Cardiac Muscle Programs

Omecamtiv mecarbil (cardiac myosin activator)

• Second interim analysis of GALACTIC-HF (Global Approach to Lowering Adverse Cardiac Outcomes Through Improving Contractility in Heart Failure), the Phase 3 cardiovascular outcomes clinical trial of omecamtiv mecarbil, in Q1 2020.
• Topline results from GALACTIC-HF in Q4 2020.
• Complete enrollment in METEORIC-HF (Multicenter Exercise Tolerance Evaluation of Omecamtiv MecarbilRelated to Increased Contractility in Heart Failure), the second Phase 3 trial of omecamtiv mecarbil, in 2020.
• Conduct commercial readiness and develop co-promotion plan in collaboration with Amgen in 2020.

AMG 594 (cardiac troponin activator)

• Complete Phase 1 SAD/MAD study of AMG 594 in 2H 2020.

CK-3773274 (CK-274, cardiac myosin inhibitor)

• Conduct REDWOOD-HCM, the Phase 2 clinical trial of CK-274 designed to determine the safety and tolerability of CK-274 in patients with obstructive hypertrophic cardiomyopathy (oHCM), in 2020.

CK-3772271 (CK-271, cardiac myosin inhibitor)

• File IND and initiate Phase 1 study in 1H 2020.

Skeletal Muscle Programs

Reldesemtiv (fast skeletal muscle troponin activator, FSTA)

• Engage with regulatory and reimbursement authorities in 2020 to prepare for a potential Phase 3 clinical trial and registration program for reldesemtiv in patients with ALS (amyotrophic lateral sclerosis).

CK-3762601 (CK-601, next-generation FSTA)

• Advance CK-601 in IND-enabling studies in 2020.

Ongoing Research

• Continue research activities directed to the cardiac and skeletal sarcomere and our other muscle biology research programs.
• Expect to continue research in collaboration with Astellas directed to the discovery of next-generation skeletal muscle activators through 2020, subject to current negotiations.
• https://www.biospace.com/article/releases/cytokinetics-announces-vision-2025-and-outlines-2020-corporate-milestones/

STAAR Surgical Reports Preliminary Fourth Quarter Net Sales Growth of 25%

Achieves 33% ICL Unit Growth for Fiscal 2019

Provides Initial Outlook for Fiscal 2020

STAAR Surgical Company (NASDAQ: STAA), a leading developer, manufacturer and marketer of implantable lenses and companion delivery systems for the eye, today provided preliminary results for the fourth quarter and fiscal year ended January 3, 2020. The Company expects fourth quarter and fiscal year net sales to increase approximately 25% and 21% over the prior year periods, respectively. Total net sales for the fourth quarter and fiscal year are expected to be approximately $38.9 million and $150.2 million, respectively, as compared to Company outlook for $37.6 million and $149.0 million most recently provided on November 8, 2019. Fourth quarter GAAP earnings per share is expected to be approximately $0.05, exceeding the current $0.02 consensus analyst estimate.

“The preliminary sales and unit growth results we are reporting today demonstrate STAAR’s ability to deliver on our stated financial commitments that support continued market share momentum for our ICL family of products and paradigm change in refractive vision correction to our lens-based solutions,” said Caren Mason, President and CEO of STAAR Surgical. “For the 2019 fiscal year preliminary earnings per share nearly doubled to approximately $0.21 driven by 33% global ICL unit growth and operating expense discipline, underscoring STAAR’s standing as one of the few publicly-traded growth medical technology companies with GAAP earnings profitability. On the regulatory front we also announced today that we have won a new CE Mark approval for the use of our ICL as a supplemental lens for the many post-cataract-surgery IOL patients who may be unhappy because they find themselves back in glasses or disposable contact lenses. Following another year of strong performance in 2019, we now fully turn our attention to achieving the financial and operating targets we recently outlined for the next three-year planning period.”

The Company also today provided an initial outlook for fiscal year 2020 including total net sales growth, year-over-year, in the range of 16% to 20% with a mid-point of 18%, representing approximately $177.2 million in fiscal 2020 total net sales, and also reaffirmed expectations for a 25% total net sales CAGR for the fiscal 2020-2022 three-year planning period. STAAR expects net sales attributable to its non-core “Other Products” business segment will decline by approximately $3 million for fiscal year 2020 as the Company continues to phase out certain lower margin, non-strategic “Other Products” while simultaneously investing more resources in the core ICL business segment, which the Company expects will continue its strong trajectory of growth. The Company anticipates total net sales growth, on an annual basis, will accelerate over the three-year planning period driven by new product catalysts and increasing adoption by surgeons and patients of the Company’s proprietary EVO Visian ICL family of lenses. GAAP earnings per share for fiscal 2020 is expected to be similar to fiscal 2019 on a full-year basis as the Company incurs one-time expenses related to its EVO clinical trial in the U.S. and makes strategic sales and marketing investments ahead of accelerating sales growth in fiscal 2021 and 2022.

STAAR expects to report its complete 2019 financial results on its fourth-quarter and full-year earnings call on or about February 26, 2020 and provided today’s information due to investor meetings taking place January 13-14, 2020. The financial information in this release is unaudited and subject to adjustment in the final audited financial statements to be filed with the Company’s Annual Report on Form 10-K.

https://www.biospace.com/article/releases/staar-surgical-reports-preliminary-fourth-quarter-net-sales-growth-of-25-percent-/

Puma Biotechnology Releases Slides to be Presented at #JPM20

 Puma Biotechnology Inc. (NASDAQ: PBYI), a biopharmaceutical company, announced that its Chief Executive Officer and President, Alan H. Auerbach, will be presenting at the 38^th Annual J.P. Morgan Healthcare Conference at 12:00 p.m. PST on Wednesday, January 15, 2020. Mr. Auerbach will be providing a corporate update that will include, among other things, the company’s preliminary estimate that it sold approximately 4,900 bottles of NERLYNX in the United States in the fourth quarter of 2019. This preliminary estimate is subject to completion of the Company’s customary closing and review procedures and could change based on that process. The slides to be discussed during the presentation are currently available on the Investors section of Puma’s website at https://investor.pumabiotechnology.com/sec-filings/all-filings/default.aspx. A live webcast of the presentation will also be available on January 15, 2020, at www.pumabiotechnology.com.

https://www.biospace.com/article/releases/puma-biotechnology-releases-slides-to-be-presented-at-the-j-p-morgan-healthcare-conference/

AstraZeneca Faces $100M Impairment After Disappointing Epanova Data

AstraZeneca PLC (AZN.LN) said Monday that it expects to book a $100 million write-down in the fourth quarter after its decision to discontinue a study of its Epanova cardiovascular drug following a recommendation from an independent data-monitoring committee.

The company said it has decided to close the phase three trial for the drug due to its low likelihood of demonstrating a benefit to patients with mixed dyslipidaemia who are at risk of cardiovascular disease.

“We are disappointed by these results, but we remain committed to addressing the needs of patients in the cardiovascular space where we have an extensive pipeline,” Executive Vice President of BioPharmaceuticals R&D Mene Pangalos said.

The British pharmaceutical major said that it is reviewing the $533 million value it assigned to Epanova. The $100 million impairment, related to inventories, is expected to hit its core earnings in the final quarter of 2019.

https://www.marketscreener.com/ASTRAZENECA-4000930/news/AstraZeneca-Faces-100-Million-Impairment-After-Disappointing-Epanova-Data-Update-29829061/

Drugmakers Test New Ways to Pay for Six-Figure Treatments

Drugmakers are experimenting with new ways to get paid for their most-expensive medicines, as resistance to escalating prices builds and improvements are made in collecting and analyzing patient data.

Now that six-figure price tags are common, drug companies are finding creative ways to get reimbursed, from installment plans to subscriptions to more complex value-based contracts that tie payment to when a drug helps a patient. For years, pharmaceutical companies would typically set a price for a drug and then get paid per pill sold at that price, less any negotiated rebates.

Alnylam Pharmaceuticals Inc. now will charge full value for a nearly $600,000 new rare-disease drug only if a patient gets a benefit akin to what was seen in clinical testing, and it will make the drug cheaper for insurers if they cover more patients than expected. Sanofi SA is offering $99-a-month subscriptions for insulin. Novartis AG — which sells a gene therapy at $2.1 million, the most expensive drug in the world — is giving insurers the opportunity to pay over five years.

The drug-reimbursement innovation comes as cries for relief mount. Congress is considering plans to lower drug costs, while the Trump administration has proposed importing drugs from Canada. Earlier this month, drugmakers raised prices of hundreds of prescription medicines, The Wall Street Journal reported.

Meanwhile, health plans are controlling costs by restricting prescriptions for certain high-price medicines to a narrow set of patients.

Drugmakers “understand that if they come to the market with superhigh-cost drugs and aren’t willing to share the risk then they are going to face pushback and access challenges,” said Michael Sherman, chief medical officer of insurer Harvard Pilgrim Health Care.

It remains to be seen, however, how widely the innovative reimbursement programs will be adopted. Many previous experiments with installment-plan payments, for instance, were directed at drugs for rare diseases, not at more widely used treatments.

Dr. Sherman and other health-insurance officials worry the new efforts might give drugmakers cover to keep raising prices, limiting the overall impact on costs.

Insurer Cigna Corp.’s most popular version of value-based contracts refunds two-thirds of the cost to employers if a patient ends up taking a different anti-inflammatory therapy within the first 90 days — which happens in 25% of patients, said Steve Miller, Cigna’s chief clinical officer.

Value-based contracting is “a great lever to pull, but it’s just one more tool in our toolbox,” he said. “It’s definitely not going to revolutionize the system to make it more affordable.”

A big factor driving drug companies to explore new payment mechanisms, industry officials say, is rising employer, patient and political pressure to control health spending, with some prescription drugs costing hundreds of thousands of dollars a year.

Caught in the middle are health plans: They are trying to keep a lid on drug spending for employers while not inciting members by denying coverage.

“For payers, there really is a challenge in wanting to pay for these things,” said Walid Gellad, a drug-policy researcher at the University of Pittsburgh School of Medicine. “So if you can somehow make it easier, it’s going to be better for the payer and for the manufacturers.”

In November, Alnylam said it would calibrate the $575,000-a-year price of newly-approved Givlaari, depending on how patients do on the drug and how many take it. Givlaari treats acute hepatic porphyria, an inherited liver condition in an estimated 3,000 patients in the U.S. and Europe that often requires hospitalization.

Public and private health insurers that agree to participate in the program will pay full value only if patients show a benefit similar to clinical trials, Alnylam Chief Executive John Maraganore said. The company also will charge less if more patients than expected take the therapy.

The concessions may help Alnylam secure reimbursement from health plans that otherwise might recoil at such a high price tag, while maximizing prescriptions, Dr. Maraganore said. “We can work together without creating misaligned incentives around the cost of a new medicine.” he said.

Sanofi in June expanded its $99-a-month subscription program for insulins Admelog, Apidra, Lantus and Toujeo. Without the program, uninsured patients taking Lantus might face an annual bill of more than $4,000.

Sanofi said the program was used more than 52,000 times in 2019.

Novartis launched its gene therapy Zolgensma with an option for insurers to pay over five years in equal annual installments. Zolgensma treats an inherited disease called spinal muscular atrophy.

Gene therapies treating just 11 conditions are projected to cost $45 billion over the next five years and are “perfect candidates” for value-based contracts, according to a research report by CVS Health shared with The Wall Street Journal and that will be published soon.

Such contracts, previously used sparingly in part because of problems assessing how a patient fared on a drug, are becoming more popular, industry officials say.

Digitized medical records, combined with technology to analyze data and see how a patient does on a drug, is making it easier for health plans and drugmakers to agree on metrics for pegging payments.

Eli Lilly & Co. signed 15 such agreements with eight payers last year, according to Frank Cunningham, who leads Lilly’s managed health-care services. He said some agreements are linked to whether medicines successfully lead to patients showing up for work.

Amgen Inc. negotiated value-based contracts for migraine drug Aimovig that reward the drugmaker for factors like reducing emergency room visits, said Kave Niksefat, Amgen’s vice president and head of value & access.

“Tracking multiple metrics across multiple different health-care systems is something that is hopefully the blueprint for the next wave of value-based contracts,” he said.

https://www.marketscreener.com/ALNYLAM-PHARMACEUTICALS-8322/news/Drugmakers-Test-New-Ways-to-Pay-for-Six-Figure-Treatments-29828081/?countview=0

Accelerate Diagnostics sees FY 2019 revenue as high as $9.3M

Accelerate Diagnostics (NASDAQ:AXDX) reports preliminary financial results for Q4 and FY 2019.

Commercially contracted instruments were 137 in Q4 and 304 for FY 2019.

Total revenue for Q4 and FY 2019 is expected to be ~$3.5M and $9.3M, respectively.

FY 2019 Gross margin is expected to be roughly 50%.

Net cash used is expected to be ~$58M for FY 2019.

Full audited financial results for Q4 and FY 2019 will be filed in late February.

#JPM20

https://seekingalpha.com/news/3531165-accelerate-diagnostics-sees-fy-2019-revenue-high-9_3m