A QIDP candidate with a good safety profile is rare
On the subject of the Qualified Infectious Disease Product designation it's worth noting that the FDA has already granted several designations.
Most of the designations found with an initial search involve products which target Community-acquired or Hospital-acquired Pneumonia - the subject of an April 2012 report which demonstrated that a short course of aminoglycoside therapy in combination with the routine therapy results in a shorter average stay in hospital. Here are extracts from three recent QIDP PRs -
..... "SAN DIEGO, Jan. 7, 2013 (GLOBE NEWSWIRE) -- Trius Therapeutics, Inc. (Nasdaq:TSRX), a biopharmaceutical company focused on the discovery and development of innovative antibiotics for serious infections, announced today that the U.S. Food and Drug Administration (FDA) has designated the company's Phase 3 antibiotic candidate, tedizolid phosphate, as a Qualified Infectious Disease Product (QIDP). Trius received the designation for its current Phase 3 program of tedizolid for acute bacterial skin and skin structure infections (ABSSSI) as well as the planned Phase 3 program for hospital-acquired/ventilator-associated bacterial pneumonia (HABP/VABP)."
"Tedizolid recently completed enrollment of its second ABSSSI Phase 3 trial, designated ESTABLISH 2, which examined the efficacy and safety of a six-day course of tedizolid administered once a day versus a 10-day course of linezolid (Zyvox®) administered twice a day in patients recruited across sites in North and South America, Europe, Australia, New Zealand and South Africa. For both tedizolid and linezolid, drug was initially administered as an intravenous (IV) infusion with the option to switch to oral therapy."
..... "BOSTON, Jan. 3, 2013 /PRNewswire/ -- Paratek Pharmaceuticals, Inc. today announced that the U.S. Food and Drug Administration (FDA) has designated the Company's lead antibiotic candidate, omadacycline (formerly known as PTK 0796), as a Qualified Infectious Disease Product (QIDP) for both intravenous (IV) and oral formulations in the treatment of acute bacterial skin and skin structure infections (ABSSSI) and community-acquired bacterial pneumonia (CABP). The QIDP designation provides Paratek access to certain incentives, including priority review associated with a New Drug Application (NDA) submission, eligibility for fast-track status for smaller population studies targeting certain resistant organisms, and a five-year extension of exclusivity under the Hatch-Waxman Act upon FDA approval of omadacycline.
The QIDP designation is provided under the Generating Antibiotic Incentives Now Act (GAIN Act), which was enacted in July 2012 under the Food and Drug Administration Safety and Innovation Act (FDASIA), formerly known as Prescription Drug User Fee Act V (PDUFA V).
Paratek has completed clinical studies necessary to advance oral and IV formulations of omadacycline into Phase 3 development. These studies will be conducted under two Special Protocol Assessment, or SPA, agreements, one in ABSSSI and one in CABP. Paratek is currently planning additional studies of omadacycline for the urinary tract infection (UTI) indication."
..... "NEW HAVEN, CT, October 10, 2012 — Rib-X Pharmaceuticals, Inc., today announced that the Food and Drug Administration (FDA) designated radezolid as a Qualified Infectious Disease Product (QIDP) for oral and intravenous (IV) use for the indications of acute bacterial skin and skin structure infections (ABSSSI) and community-acquired bacterial pneumonia (CABP). This is the second drug candidate in Rib-X's pipeline to receive QIDP designation. Rib-X announced in September that the FDA designated delafloxacin as a QIDP for ABSSSI and CABP.
Radezolid is a next-generation oxazolidinone designed to be a potent antibiotic with intravenous (IV) and oral formulations and a safety profile permitting long-term treatment of resistant infections, including those caused by methicillin-resistant Staphylococcus aureus (MRSA). Radezolid has completed two Phase 2 clinical trials with an oral formulation in uncomplicated skin and skin structure infections (uSSSI) and in community acquired bacterial pneumonia (CABP). A Phase 1 study with an IV formulation was recently completed in healthy subjects. Rib-X recently presented data at ICAAC from a positive Phase 1 intravenous (IV) dosing study conducted in healthy subjects and an in vivo long-term safety study versus linezolid."
It's of particular relevance to the potential of Arikace that two of these three PRs mention studies where the control drug was Linezolid, when one considers the following extract from its Wiki entry -
"As of 2009, linezolid is a "black triangle drug" in the United Kingdom, meaning that it is under intensive postmarketing surveillance by the Commission on Human Medicines of the Medicines and Healthcare products Regulatory Agency."
A recent advisory panel recommendation for the approval of another drug candidate reinforces the implication that strains of bacteria which are resistant to the drugs routinely used to treat pneumonia pose a serious problem -
"Nov 12: An FDA panel voted 9 to 6 against recommending telavancin for first-line treatment of nosocomial pneumonia (NP), but voted 13 to 2 for the treatment of NP when other alternatives are not suitable. There were concerns about toxic effects on kidneys ."
When rehdvm concluded that the telavancin recommendation had caused the INSM share price to drop he'd overlooked the fact that Arikace has a massive competitive advantage in a key area - it entails a far safer route of delivery. Rehdvm told us that telavancin is an inhaled therapy, but in fact Arikace is the only therapy mentioned in this post delivered by a route which bypasses the bloodstream.
The scale of the problem (and opportunity) is reflected in this extract from a 2004 report -
"About 2 million people acquire bacterial infections in U.S. hospitals each year, and 90,000 die as a result.
About 70 percent of those infections are resistant to at least one drug. The trends toward increasing numbers of infection and increasing drug resistance show no sign of abating."
That IDSA document - Bad Bugs, No Drugs - is extremely informative. It was clearly part of an initiative which led to the 2012 QIDP / Generating Antibiotics Incentives Now legislation. It's well worth reading for those here who are looking to get a grip on the likely use of Arikace. There are several detailed case studies, and it's worth noting that death from pneumonia is often the outcome of diseases which originate nowhere near the lungs.
One of the barriers identified back then was that therapies for chronic diseases generate far more revenue than do antibiotics, given the speed with which antibiotics eliminate the infection. It's reassuring that the safety profile of Arikace looks likely to position it both as a long-term maintenance therapy and as an emergency therapy.
The analysis of that final nine-month safety study requested by the FDA is due to start this month. The preliminary report is scheduled for Q2, but one imagines that Insmed will know well in advance if the data is clean. Will Insmed then initiate the planned Arikace Expanded Access Program?
Terry assures us that clean safety data is already priced in. Terry is wrong - unsurprising when one considers that he has been demonstrating how to lose money in biotechs since the mid-nineties.