Melinta Therapeutics Successfully Completes Financial Restructuring

Company is well-positioned for success as it emerges from chapter 11

NEW YORK, NY, April 20, 2020—Commercial-stage antibiotics company Melinta Therapeutics (“Melinta”) and healthcare investment firm Deerfield Management Company, L.P. (“Deerfield”), announced today that Melinta has successfully completed its financial restructuring and has emerged from Chapter 11. 

In accordance with the pre-negotiated Chapter 11 plan of reorganization, Melinta is now privately owned by affiliates of Deerfield and has eliminated its debt obligations, resulting in a well-financed and strongly positioned anti-infectives company with plans for future growth. 

Melinta will continue to actively supply, distribute, and support its four marketed products for the treatment of certain antibiotic-resistant infections:  Vabomere® (meropenem and vaborbactam), Orbactiv® (oritavancin), Minocin® (minocycline) for Injection and Baxdela® (delafloxacin). In addition, with its new, solid financial footing, Melinta expects to enhance its portfolio with the addition of new commercial and clinical-stage pipeline candidates in support of its mission of serving the critical needs of patients in the hospital and hospital ecosystem.

“We welcome this partnership with Deerfield in continuing to best serve the needs of patients in the hospital and look forward to the new opportunities for innovation and growth that this partnership will bring,” said Jennifer Sanfilippo, Interim Chief Executive Officer and Director of the reorganized company. “Our antibiotics will remain a central component of Melinta’s portfolio, including our core brands Vabomere® and Orbactiv®, and we are excited at the prospect of augmenting this important portfolio with products that address high-need therapeutic areas.”

In partnership with the Melinta team, Deerfield intends to leverage its operational, business development, data analytics and market research expertise in order to continue to accelerate the growth and expansion of Melinta’s product portfolio.

“The Melinta team has demonstrated an ability to successfully deliver important antibiotics to treat serious infections and has shown resilience and dedication during the most challenging of times,” said Deerfield Partner Jonathan Leff. “Covid-19 is a wake-up call regarding the dangers of infectious diseases and the need for innovative anti-infective products to serve the public health. We are delighted to join Melinta in this journey.”

-####-


About Deerfield Management 

Deerfield is a healthcare investment management firm committed to advancing healthcare through investment, information and philanthropy.

About Melinta Therapeutics

Melinta Therapeutics, Inc. is dedicated to saving lives threatened by the global public health crisis of drug resistant bacterial infections through the development and commercialization of novel antibiotics that provide new therapeutic solutions. Its four marketed products are Vabomere® (meropenem and vaborbactam), Orbactiv® (oritavancin), Minocin® (minocycline) for Injection and Baxdela® (delafloxacin). This portfolio provides Melinta with the unique ability to provide providers and patients with a range of solutions that can meet the tremendous need for novel antibiotics treating serious infections. For additional information, including product information, visit www.melinta.com.

Contact:
Deerfield Management
Karen Heidelberger, 212-692-7140, [email protected]


Melinta Therapeutics

Susan Blum, 312-767-0296, [email protected]

Deerfield Closes $840 Million Healthcare Venture Fund

The Deerfield Healthcare Innovations Fund ll will invest in advancements in science for promising therapeutics and to improve ways healthcare is delivered to patients

(New York, NY, April 6, 2020)—Deerfield Management Company, L.P. today announced the closing of the Deerfield Healthcare Innovations Fund ll, L.P., which will invest in advancements in science that may lead to important therapeutic interventions and back new technologies and infrastructure to improve the way healthcare is delivered to patients.

“Now more than ever, these unprecedented times underscore the importance of supporting the critical work of our nation’s scientists and healthcare systems,” said James E. Flynn, managing partner of Deerfield. “Our uniquely supportive model allows us to provide leverage to innovative companies and accelerate the benefit to patients.”

The Healthcare Innovations Fund II will collaborate with more than 15 leading academic institutions to seed novel scientific research. The Fund also has access to innovative medtech incubators created in partnership with two medical device industry leaders.

The Cure, the recently announced innovation campus located at 345 Park Avenue South, will become the central location of Deerfield where much of the work of the Healthcare Innovation Fund II will take place. Many of the early-stage companies will utilize the Cure’s wet and dry labs, engineering, computing and shared resource spaces.

The companies will also benefit from on-site programming, including the Fellows Program, Break into the Boardroom and Women in Science. Newly curated programs to be featured involve the “Cure Lounge,” a thought leadership and executive club, and other educational and certification initiatives.

The Deerfield Healthcare Innovations Fund ll is supported by the entire Deerfield infrastructure. 

Deerfield has been investing in and supporting the healthcare industry for more than 25 years, and partner companies benefit from its organizational capabilities.

Today, Deerfield has more than 130 experienced healthcare-focused professionals with specialized knowledge in medicine, life sciences, drug and medical device development, healthcare markets, public policy, value-based care, financial instruments, tax, accounting, operations, corporate strategy, market access research, marketing, biostatistics and sector dynamics, all of which can be leveraged by its partners.

Together, Deerfield team members fully fund and manage the Deerfield Foundation, which to date has committed or invested over $49 million to global organizations seeking to improve the delivery of healthcare, with a focus on underserved children. The Deerfield Foundation recently committed approximately 10 percent of its 2020 fiscal budget toward COVID-19 emergency funding to organizations in need of urgent resources in order to provide care to their communities.


About Deerfield Management 

Deerfield is a healthcare investment management firm committed to advancing healthcare through investment, information and philanthropy.

Contact:

Media
Deerfield Management
Karen Heidelberger, 212-692-7140
[email protected]

-####-

Large-Scale Cell and Gene Therapy Contract Development and Manufacturing Organization to Launch in PA

The Center for Breakthrough Medicines expected to relieve the industry’s production constraints, providing patients better access to treatments

(King of Prussia, PA, and New York, NY, January 22, 2020)—The Discovery Labs and Deerfield Management Company have formed The Center for Breakthrough Medicines, a Contract Development and Manufacturing Organization (CDMO) and specialty investment company, to alleviate the critical lack of capacity that is preventing patients from accessing critically needed cell and gene therapies. The CDMO is occupying over 40 percent of The Discovery Labs’ 1.6 million square foot biotech, healthcare and life sciences campus in King of Prussia, PA.

The CDMO provides preclinical through commercial manufacturing of cell and gene therapies and component raw materials. It offers process development, plasmid DNA, viral vectors, cell banking, cell processing, and support testing capabilities all under one roof. The immense $1.1 billion facility will provide instant capacity as the largest known single source for accelerating the delivery and affordability of lifesaving and life-changing therapies from the bench to the patient’s bedside.

The Company has initiated a substantial hiring effort targeting the best and brightest of the life sciences community including, experts in CGMP manufacturing. The Company expects to hire over 2,000 team members within the next 30 months.

The CDMO has retained Nucleus Careers, a cloud-based specialty life sciences human capital recruiting and retention management expert, to buildout the entire team. Nucleus has proprietary recruiting and retention software designed for large scale human capital buildouts of high growth companies.

In addition to developing the world’s largest single-point cell and gene therapy manufacturing facility, The Discovery Labs is establishing THE COLONY which will provide custom built discovery labs, breakthrough funding, sponsored research agreements, housing and relocation for the world’s leading iconic experts in cell and gene therapy.

THE COLONY will seek to work hand in hand with scientists from both academic and pharmaceutical institutions to unlock and expedite groundbreaking therapies.

Marco A. Chacón, Ph.D., Founder of Paragon Bioservices and Chairman of The Discovery Labs states, “musicians, artists, members of religious communities and great thinkers throughout time have formed colonies where freedom of thought and expression combined with unlimited dreams and potential have resulted in the world’s greatest accomplishments. The United States of America is a perfect example.” Dr. Chacón went on to say, “the goal of THE COLONY is to unshackle the potential of the world’s greatest scientific minds.”

The ability for the industry’s greatest scientists to cohabitate, collaborate, cooperate, and communicate via technology and in person will create an exponential therapeutic “X FACTOR.” THE COLONY seeks to unlock institutional barriers prohibiting the world’s greatest scientists from moving at a pace necessary in today’s ever-changing therapeutic revolution. THE COLONY will partner with the institutions where the scientists currently work by providing equity, license fees, and revenue sharing.

“The Center for Breakthrough Medicines will be serving companies from the earliest stages through commercialization. Its exceptional scale and offering will quickly relieve the production bottleneck for advanced therapies by reducing the time, complexity, and cost of commercializing vitally needed gene and cell therapies,” noted Audrey Greenberg, Board Member and Executive Managing Director for The Discovery Labs.

The addition of this end-to-end manufacturing capability is expected to significantly enhance the offerings of The Discovery Labs in an area that has become one of the largest life sciences hubs in the world. Renovations are underway to construct a total of 86 plasmid, viral vector production, universal cell processing, CGMP testing, process development and cell banking suites. The viral vector and cell processing suites will be fully compliant with both U.S. Food and Drug Administration and European Medicines Agency standards. All suites will offer the flexibility to meet client-specific workflows and will be able to adapt quickly to meet demand. The Company is in the process of reserving capacity now for late 2020.

“Today brilliant scientists are advancing an unprecedented number of gene and cell therapy drug candidates. The real tragedy, however, is a scarcity of manufacturing know-how, which is complex and expensive,” said Alex Karnal, Partner and Managing Director of Deerfield Management and a Board Member of the Discovery Labs. “With its visionary business model, it is hoped that The Center for Breakthrough Medicines will help realize the promise of cell and gene therapies in time to treat the many patients who need them.”

The Discovery Labs provides a central campus where the world’s greatest scientists can collaborate on new therapeutic discoveries to eradicate diseases affecting small and large segments of the global population. The Center for Breakthrough Medicines will work with these leaders, life sciences companies, large pharmaceutical companies, and academic and government institutions.

This new manufacturing capability is a transformational addition to The Discovery Labs market offering and dovetails with The Discovery Labs biotech incubator, Unite IQ. Unite IQ offers immediate space to emerging life sciences companies and scientists giving them the ability to grow from startup to enterprise company on one campus. The incubator and accelerator space at Unite IQ provides a comprehensive home for startups with every resource needed to initiate business operations. Unite IQ tenants are expected to utilize the discovery, development, testing, and manufacturing capabilities of the Center for Breakthrough Medicines with seamless forward integration of processes and analytics, and seamless tech transfer from research lab to large scale production

The Emerging Field of Cell and Gene Therapy in Pennsylvania

The demand for clinical and commercial manufacturing capacity is acute and expected to remain that way. The current shortfall in manufacturing for cell and gene therapies is severely underserved with few approved products. There are currently approximately 1,100 advanced therapies in the pipeline pending FDA approval. This will greatly increase highly skilled manufacturing demand. Dr. Peter Marks, Director of the FDA Center for Biologics Evaluation and Research, states, “what keeps me up at night is will we be able to manufacture these on a scale that will allow us to bring the benefit of these therapies to patients?” He further added that “if we can help see cost of goods and ability to manufacture reproducibly improve, I think that’ll be a big thing.” All of this adds up to a supply constrained market that The Center for Breakthrough Medicines aims to help address.

With the potential to treat and even cure disabling, and deadly diseases, gene and cell therapies are ushering in a new era of medicine. These therapies may eventually be able to cure genetic conditions, such as cystic fibrosis, hemophilia A, and a range of cancers. The Philadelphia area has become the epicenter for the flourishing field of gene and cell therapy. Research from CBRE currently ranks the market among the top biotech clusters for medical research and health services. The cluster has become known worldwide as “Cellicon Valley” for its leadership in research and development of this rapidly evolving field. The Discovery Lab’s suburban Philadelphia location offers a talent rich environment due to the area’s preponderance of large pharmaceutical companies and the Philadelphia region’s position boasting the top 10 universities and primary school systems in nation.

Over the past three years, multiple Philadelphia companies have received approvals for major breakthroughs in cell and gene therapy. In 2017, the U.S. FDA approved the first-ever CAR-T cell therapy, Novartis’s Kymriah, which originated at the University of Pennsylvania. Shortly thereafter, the FDA gave landmark approval for the first-ever gene therapy to treat a genetic blindness condition to Spark Therapeutics, a start-up founded by researchers at Children’s Hospital of Philadelphia. These discoveries and others in the pipeline are attracting billions of dollars of venture capital. The Greater Philadelphia Region set a recent record in venture capital financing.

The Discovery Labs Center for Breakthrough Medicines joins more than 25 healthcare, life sciences and tech-enabled companies that already call The Discovery Labs King of Prussia home.

Contact Audrey Greenberg at [email protected] for more information about development services, manufacturing capacity, incubator space or leasing information at the property.

About The Discovery Labs

Part of MLP Ventures, The Discovery Labs is a global provider of world-class cGMP manufacturing, turnkey laboratory solutions, critical materials and office space that support therapeutic products and services to the biotechnology and pharmaceutical industry so that groundbreaking medicines get to the patients that need them. The location in eastern King of Prussia is a prototype for a global rollout of The Discovery Labs, providing Big Pharma, emerging life sciences, consumer and technology companies flexible, end-to-end technical real estate and business infrastructure for the customer’s entire lifecycle from discovery to delivery, including manufacturing capacity. It is the first fully integrated environment that merges technology and life sciences under one roof to drive innovation.

About Deerfield Management

Deerfield is a healthcare investment management firm committed to advancing healthcare through investment, information and philanthropy.

Media Contact:
Tony DeFazio, DeFazio Communications
(o) 484-534-3306 (c) 484-410-1354
[email protected]

Karen Heidelberger, Deerfield Management Company
212-551-1600
[email protected]

Women in Science

Launched in 2019, the Women in Science Translational Research Symposia were created to clarify the most targeted route to commercialization for female scientists and help advance their discoveries to market. The program is committed to addressing the gender gap in innovation and changing this paradigm through knowledge and network creation.

V-Wave scores FDA breakthrough status on its heart failure shunt

V-Wave, Ltd., recently announced that it received the prized FDA Breakthrough Designation for its heart failure shunt. Breakthrough designation is one of the highly sought pre-approval stamps that the FDA can place on a device.

According to the FDA’s website, it is granted when the device “provides for more effective treatment or diagnosis of life-threatening or irreversibly debilitating human disease or conditions” (than what is currently available). The program aims to provide patients and health care providers with more timely access to medical devices “by speeding up their development, assessment and review,” including prioritized review all the way through to market approval.

V-Wave’s minimally-invasive implanted interatrial shunt for the treatment of patients with severe symptomatic heart failure is designed to regulate left atrial pressure, the primary cause of breathing difficulty and hospitalization due to worsening heart failure.

“In addition to validation of the potential impact of this technology, breakthrough status will facilitate a timely regulatory review and solve a major issue with medical device investments, namely that reimbursement will effectively be secured immediately upon approval,” said Deerfielder, Andrew ElBardissi, MD, who serves on the Company’s board of directors.

The shunt is currently being evaluated in a global, randomized, controlled, double-blinded, 500 patient pivotal Investigational Device Exemption trial called RELIEVE-HF. The study is enrolling advanced heart failure patients with preserved or reduced left ventricular ejection fraction who

remain symptomatic despite the use of guideline directed medical and device therapies.

An ejection fraction is an important measurement of how well the heart is pumping and is used to help classify heart failure and guide treatment. In a healthy heart, the ejection fraction is 50 percent or higher – meaning that more than half of the blood that fills the ventricle is pumped out with each beat.

According to the Centers for Disease Control and Prevention, nearly 6 million adults in the United States have heart failure and about half of these individuals die within 5 years of diagnosis. Heart failure costs the nation an estimated $30.7 billion each year.

Achieving this status means that the device also met at least one of the following FDA criteria:

  1. It represents breakthrough technology;
  2. No approved or cleared alternatives exist;
  3. It offers significant advantages over existing approved or cleared alternatives; and
  4. Availability of this device is in the best interest of patients.

V-Wave, Ltd., a privately held medical device company, has been a Deerfield portfolio company since 2018.

Adapted from company news release: V-WAVE’S INTERATRIAL SHUNT RECEIVES FDA BREAKTHROUGH DEVICE DESIGNATION FOR HEART FAILURE:

https://www.fda.gov/medical-devices/how-study-and-market-your-device/breakthrough-devices-program

https://www.mayoclinic.org/diseases-conditions/heart-failure/symptoms-causes/syc-20373142

https://www.cdc.gov/dhdsp/data_statistics/fact_sheets/fs_heart_failure.htm

Deerfield Institute Report – Key insights into technology transfer offices

Translation of academic innovation has matured since the passage of the Bayh-Dole Act, 28 years ago. Universities and research institutes have contributed towards this to the tune of 380,000 disclosures, 206,000 new patent applications and 84,000 issued US patents.1 The market has responded in turn, generating 11,000 startups and 10,000 products that have yielded more than $1 billion in equity for the institutions.2 Deerfield Management has joined in this effort by entering into significant collaborations with leading academic institutions. In conjunction with these investments, the Deerfield Institute, the research division of Deerfield Management, surveyed 35 university technology transfer offices to endeavor to understand current trends impacting their organizations. The survey revealed insights that can shed light on the operations of these groups, provide guidance to those seeking to collaborate with academia and serve as a guide for continuous improvement in the practice of academic commercialization.

Revisiting Biosimilars: A Closer Look At The Commercial Barriers

In a past issue of this newsletter (September 2017) we took a broad look at the salient issues pertaining to the realm of biosimilars, including regulatory, legal, and commercial aspects of the debate. We refer readers here for an initial grounding if needed.  In the intervening time period, new developments have played out that highlight the significant barriers to commercial uptake that exist for these products.

Briefly, biosimilars are to biologics as branded drugs are to generics. However, both biologic drugs and biosimilars are by and large exceedingly more difficult to manufacture and thus bring to market as the production process is critical to the potency and release quality of these drugs within parameters established by the innovator product.  There are only yet 15 biosimilars approved in the US, and only five are actually commercially available, with the remainder being still blocked from the market due to patent exclusivity.  Contrast this to Europe, where 40 biosimilar products are approved.   

The cost of production for these products is not nearly at the level of generics. Take insulin, for example.  Insulin has a somewhat hybrid role in the US, as the first formulations were approved before the biologics approval pathway existed in the US, so its “biosimilars” are approved under what is known as the 505(b)2 pathway in the US, as opposed to the 351(k) pathway used by products whose innovator products are a true biologic (as determined by approval pathway). The market would seem ripe for a Lantus biosimilar, as the product has greater than $4bn in annual sales. Eli Lilly already has a biosimilar to Lantus on the US market, called Basaglar, which is annualizing greater than $600m through its first six quarters into launch.

Merck, through a collaboration with Samsung Bioepis had a tentatively approved biosimilar to Lantus from July 2017. However, a filing by Samsung Bioepis in October 2018 indicated Merck had cancelled development and commercialization contracts for the product. One analyst report cited the decision to pull out as being due to a review of the market environment and production costs of insulin biosimilars.  If a biosimilar as relatively “simple” as insulin can be difficult to manufacture at scale with attractive margins, then this certainly cannot bode well for more complex protein products to do the same. With Merck’s exit, only Mylan, in collaboration with Biocon, is developing another Lantus biosimilar. While district court litigation is ongoing, a launch for Mylan and Biocon’s product is expected in the 2020-2021 timeframe.

Similarly, Momenta recently announced it would be cutting its biosimilar efforts altogether, and simultaneously cutting half its staff while it refocuses on its non-biosimilar pipeline.

It’s a (rebate) trap

FDA Commissioner Scott Gottlieb further shed light on another major commercial obstacle for biosimilars, dubbed the rebate trap, in a March 2018 speech at the America’s Health Insurance Plans National Health Policy Conference[1]. The deep discounts offered on certain branded specialty drugs (often biologic), in the form of rebates and other payment or contractual mechanisms, in many instances can be upwards of 40% to attain preferred formulary positions from pharmacy benefit managers (PBMs) and health insurers. Often these negotiated discounts are volume-based, so the greater the utilization over competitive products, the greater the spread from the wholesale acquisition cost (WAC) to the net price to the plan, with PBMs and health insurers earning a percentage of the spread. Launched biosimilar products have generally been introduced to the market with roughly 15-20% discounts to the WAC of originator products, and thus are unable to displace the originator product. Biosimilars get stuck in a catch-22 situation, where they lack enough patient share for plans to consider moving them to a better formulary position but are hindered from getting more meaningful market share while they sit on a lower formulary tier. PBMs remain financially incentivized to limit the uptake of biosimilars to maintain the flow of rebate payments on originator products. Simply further discounting the biosimilar so that the WAC is on par with the net price of the originator is easier said than done for reasons noted earlier, namely that these drugs have turned out to be much costlier to develop and manufacturer than earlier predictions.

PBMs are attempting to move past the bad press they have received around the rebate trap and the general practice of collecting rebates. Express Scripts recently announced the launch of its Flex Formulary, which will consider authorized generics of branded drugs for inclusion on the formulary, in either a preferred or non-preferred position, and, importantly, discontinue coverage of the branded product. This could foreseeably open the door to more biosimilar adoption on this formulary.  The first products added to the Flex Formulary were Epclusa and Harvoni, two hepatitis C drugs made by Gilead Sciences. 

Humira in the hotseat

Gottlieb has gone so far as to suggest a competitive bidding scheme for biologics would be ideal[2], which is more akin to the experience in the EU. For example, Remicade sales have fallen over two-thirds in the three years since the introduction of its first biosimilar in the EU. Another closely watched drug is Humira, the world’s top-selling drug, as four biosimilars have just launched in the EU. One analyst report has said AbbVie, the maker of Humira, has won its first tender in Europe by offering an 80% discount off the price prior to the launch of biosimilars[3]. The deep discounting shows the extent to which the company is willing to go to hold onto market share.

Humira biosimilars in the US are still a pipedream, protected by a patent thicket the company has created with additional patents on formulations changes and extending life as new indications are approved. AbbVie has forged confidential legal settlements with several biosimilar makers, that will keep biosimilar copies of Humira at bay until 2023. In the meantime, AbbVie and Amgen are likely to continue to find themselves under increased scrutiny over the practice of repeated price increases for their top-selling drugs. A recent article cited nearly a 140% overall price increase for both of those drugs since January 2013[4]. Without biosimilar competition, and assuming continued price increases on par with recent history, both drug makers could see themselves in the hot seat in the court of public opinion.

[1] https://www.fda.gov/NewsEvents/Speeches/ucm599833.htm

[2] https://www.fda.gov/NewsEvents/Speeches/ucm613452.htm

[3] https://www.statnews.com/pharmalot/2018/11/01/abbvie-humira-biosimilars-prices/

[4] https://www.statnews.com/2018/11/14/humira-abbvie-amgen-enbrel-price-hikes-biosimilars/

Inaugural Approvals For RNA-Based Medicine

In the case of another historic first in the world of healthcare, this summer saw the first US and EU regulatory approvals for an RNA interference (RNAi) therapeutic, in the form of Alnylam’s Onpattro (patisiran). The drug, given as an infusion, was approved to treat polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients. A rare and often fatal disease, hAATR is characterized by the buildup of abnormal amyloid protein in peripheral nerves, the heart, and other organs. Up until this approval, the American College of Cardiology’s recommendations for treatment were merely supportive care and clinical trials, pointing to the level of unmet need for this patient population[1].

Still, it has been anything but smooth sailing for Onpattro, nor for the field of RNA-based medicines.  Like gene therapy, it has seen an earlier wave of enthusiasm come crashing down in the wake of technical challenges and safety issues. While Onpattro will not be a panacea to all the known issues, it is important to note where and how it has succeeded where others have failed and to understand what it means for other candidates in the pipeline. 

How it works

To understand how RNAi works, transport yourself to your high school biology class where you learned about the “central dogma” theory that explains the relationship between DNA, RNA and proteins – that DNA in the nucleus forms genes which are the template for transcription to RNA, which in turn is the template for translation to proteins. Those proteins then serve as a central player in most biological systems. In individuals without hAATR, the liver produces the TTR protein, used to transport vitamin A and a thyroid-binding protein in the body. Patients with hATTR have a mutation in the gene for TTR, which leads to the creation of a defective and unstable TTR protein. Onpattro binds to the mutated mRNA sequence that causes the defective protein, and cuts out that sequence, effectively halting the production of the misfolded and defective protein[2]. Of note, this approach targets the upstream cause of the defect, and not simply the downstream symptoms that are the manifestation of the defect.

Location, location, location

RNAi does not simply work by targeting the mutated mRNA of interest – it must additionally be specifically targeted to the tissue(s) of interest, which is the tougher nut to crack. The liver, the target organ of interest in hATTR, happens to be better suited for targeted drug delivery given its unique vasculature – notably that it has both the hepatic artery and hepatic vein, allowing it to effectively double dip on whatever has been infused into the blood stream, compared to other organs. It will be more challenging to deliver RNAi to other organs, which thus far has been a challenge to do at therapeutically appropriate levels. 

Onpattro is encased in a lipid nanoparticle to help carry the drug to the liver and enter the cell.  The lipid nanoparticle disrupts the cell membrane to allow Onpattro in to the cell, and thus can trigger an immune response, so patients must prophylactically take a steroid, acetaminophen, and antihistamines. There is thus also the need to find delivery vehicles that are slightly more sophisticated to obviate any immune responses.

How did we get here?

Pharma entered the RNAi fray in the early 2000s with some big dollar risk-taking in a hot field that would go on to earn a Nobel prize for its scientific discoverers, only to encounter future obstacles. Novartis and Roche paid their way into accessing Alnylam’s platform in 2005 and 2007, respectively. Merck paid $1.1bn to acquire Sirna Therapeutics, Alnylam’s main competitor at the time. Prior to that transaction, Sirna had a research deal with GlaxoSmithKline as well.  Early attempts in ophthalmology and other liver targeted attempts encountered issues with RNA degrading before reaching its target, and thus required extremely high dosing to show any efficacy. Novartis and Roche parted ways with Alnylam in 2010, and Merck eventually sold Sirna IP to Alnylam in early 2014 at a massive discount to its earlier purchase. In the interim, Alnylam soldiered on with some restructuring and a deal with Sanofi dating back to 2012 that helped keep it afloat[3].

Looking ahead

Alnylam has said the list price for one year of treatment in the US is $450,000 and has at least one value based agreement, with Harvard Pilgrim Health Care.  It plans to officially launch Onpattro later this year and will face competition in the US from Ionis and Akcea’s also newly approved Tegsedi (inotersen), and possibly also Pfizer’s Vyndaqel (tafamidis) before the end of this year.

Alynlam and others are pursuing experimental RNAi treatments in other indications, including acute hepatic porphyrias, cardiovascular disease, hepatitis B, alpha-1 antitrypsin deficiency, primary hyperoxaluria, delayed graft function, and alcohol use disorder[4]. Notably, several of these are also liver-targeted indications with various delivery methods to help with targeting.

[1]   http://blogs.sciencemag.org/pipeline/archives/2017/09/20/alnylam-breaks-through

[2]   https://www.fda.gov/Drugs/NewsEvents/ucm615953.htm

[3]   http://cienp.org.br/wp-content/uploads/2018/03/Alnylam-prepares-to-land-first-RNAi-drug-approval.pdf

[4]   https://www.wsj.com/articles/fda-approves-first-drug-based-on-gene-silencing-research-1533923359

DFB Healthcare Acquisitions Corp. Closes $250 Million Initial Public Offering

NEW YORK, Feb. 21, 2018 /PRNewswire/ — DFB Healthcare Acquisitions Corp. (NASDAQ: DFBHU) (“DFB Healthcare”) announced today that it closed its initial public offering of 25,000,000 units. The offering was priced at $10.00 per unit, resulting in gross proceeds of $250,000,000.

The Company’s units began trading on the NASDAQ Capital Market under the ticker symbol “DFBHU” on February 16, 2018. Each unit consists of one share of the Company’s common stock and one-third of one warrant, each whole warrant enabling the holder thereof to purchase one share of common stock at a price of $11.50 per share. Once the securities comprising the units begin separate trading, the common stock and warrants are expected to be listed on the NASDAQ Capital Market under the ticker symbols “DFBH” and “DFBHW”, respectively.

DFB Healthcare is sponsored by Deerfield/RAB Ventures, LLC, which was jointly founded by Deerfield Management Company L.P. (“Deerfield”) and DFB Healthcare’s management team, led by CEO Richard Barasch. Deerfield is an investment firm focused exclusively on the healthcare industry, and its investment activity spans both public and private securities. Mr. Barasch has more than 30 years of experience in healthcare services, health insurance, and related industries, and was CEO of Universal American Corp. from 1995 until the Company’s acquisition by WellCare Health Plans in April 2017.

Goldman Sachs & Co. and Deutsche Bank Securities acted as joint book runners for the offering and Leerink Partners acted as co-manager. DFB Healthcare has granted the underwriters a 45-day option to purchase up to an additional 3,750,000 units at the initial public offering price to cover overallotments, if any.

A registration statement relating to these securities has been filed with, and declared effective by, the Securities and Exchange Commission on February 15, 2018. This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction. The registration statement can be obtained at www.sec.gov.

About DFB Healthcare Acquisitions Corp.

DFB Healthcare Acquisitions Corp. is a newly organized blank check company formed for the purpose of effecting a merger, capital stock exchange, asset acquisition, stock purchase, reorganization or similar business combination with one or more businesses. It has not identified any business combination target and has not, nor has anyone on its behalf, initiated any substantive discussions, directly or indirectly, with respect to identifying any business combination target. Its acquisition and value creation strategy will be to identify and acquire a business in the healthcare sector.

The offering was made only by means of a prospectus, copies of which may be obtained from Goldman, Sachs & Co., Attention: Prospectus Department, 200 West Street, New York, NY 10282, or by telephone toll-free at 1-866-471-2526 or by email at [email protected], from Deutsche Bank Securities Inc., Attention: Prospectus Group, 60 Wall Street, New York, New York 10005-2836 (Tel: (800) 503-4611; Email: [email protected]), and from Leerink Partners LLC, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, by telephone at 800-808-7525, ext. 6132 or by email at [email protected].

Contact

DFB Healthcare Acquisitions Corp.
Chris Wolfe, (212) 965-2400
[email protected]

New And Noteworthy Approvals From 2018

The FDA is on track for another record setting year of new drug approvals.  Below we recap some significant approvals from the past year, spanning therapeutics, medical devices, and digital tools.

Viz.ai | February 2018
Analysis of CT results and highlights cases that may have experienced a stroke

What you should know
Created a new regulatory classification for artificial intelligence driven platforms for clinical decision support.

Dexcom | March 2018
Integrated continuous glucose monitoring (iCGM) system

What you should know
First iCGM that can integrate with other compatible medical devices and electronic interfaces, including dosing systems, insulin pumps, and blood glucose meters.

IDx-DR | April 2018
Autonomous detection of diabetic retinopathy

What you should know
First AI-based diagnostic system that can provide a screening decision without clinician interpretation.

Hospira, a subsidiary of Pfizer | May 2018
Treatment of anemia caused by chronic kidney disease, chemotherapy, or use of zidovudine in patients with HIV infection

What you should know
First biosimilar to Epogen/Procrit.

Novartis | May 2018
Relapsed or refractory (r/r) large B-cell lymphoma

What you should know
New indication added following first approval for the CAR-T product in 2017.

Portola Pharmaceuticals | May 2018
Reversal of anticoagulation in patients treated with rivaroxaban or apixaban

What you should know
“Generation 1” approval for smaller batch product, available in only limited launch. Generation 2 product has YE2018 PDUFA data.

Amgen | May 2018
Preventive treatment of migraine in adults

What you should know

First approval of new class of migraine agents known as calcitonin gene-related peptide (CGRP) blockers.

Mylan | June 2018
Reduce the risk of infection during cancer treatment

What you should know
First biosimilar to Neulasta.

GW Pharmaceuticals | June 2018
Rare, severe forms of epilepsy

What you should know

First FDA-approved drug that contains a purified drug substance derived from marijuana.

Senseonics | June 2018
Implantable continuous glucose monitoring system

What you should know
First implantable product approved, capable of 90-day use.

Abbott | July 2018
Continuous glucose monitoring system

What you should know
Allows adult patients to make diabetes treatment decisions without obtaining a blood sample from the fingertip (fingerstick). Also has a companion app for smartphone use.

Alnylam | August 2018
Hereditary transthyretin-mediated amyloidosis in adult patients

What you should know
First RNA interference (RNAi) approval.

Apple | September 2018
Atrial fibrillation-detecting algorithm and ECG built into Apple Watch

What you should know
De Novo approval for incorporation of these features into a consumer-facing electronic device.

Merck | October 2018
HPV vaccine

What you should know
Extended the approved age range of the vaccine to include men and women ages 27 to 45.

Celltrion | November 2018
B-cell non-Hodgkin’s lymphoma (NHL)

What you should know
First biosimilar to Rituxan.

Loxo Oncology and Bayer | November 2018
Solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion

What you should know
Tissue agnostic approval – can be used in any tumor type with a specific gene fusion status.