For drug developers, AIM is seen as the seed investment ground for companies at the pre-clinical or the early clinical stage of development.
Recently, however, a handful of hopefuls have pushed drugs into the final, make-or-break phase.
They include GW Pharma, whose stock is up 66% in the year to date, boosted by its phase III success with a cannabis-derived treatment for an acute form of childhood epilepsy.
Sadly, GW cancelled its listing on the UK’s growth market effective December 5 and is now quoted solely on the NASDAQ market for tech and biotech stocks.
Here we look at four lesser-known juniors companies that have drugs in phase III that are looking to 2017 as a pivotal year.
Motif Bio
It is fair to say 2016 hasn’t been the easiest of years for Motif Bio Plc (LON:MTFB). However, financially and operationally the company actually made progress and next 12 months.
Late November was the turning point for the developer of iclaprim, a next generation antibiotic.
It successfully raised US$25mln via a transatlantic placing of shares and warrants and listed on NASDAQ.
The firm will receive a net US$21.4mln that will be used to complete its REVIVE-1 phase III clinical study of its lead drug.
“iclaprim is set to fill a major market void in the wake of a global public health crisis driven by rampant antibiotic resistance,” said Northland Capital analyst Vadim Alexandre.
The results of the REVIVE-1 study are expected in the second quarter, while data from a separate clinical trial, called REVIVE-2, will be published at some point in the final six months of 2017.
Amryt Pharma
Here’s another junior druggie that has been very busy but doesn’t seem to have garnered the market plaudits it deserves.
In 18 months it has it has made two acquisitions, managed two stock market listings and raised €43mln.
Oh, and it has put a heavy-hitting team together that supports a very ambitious growth strategy.
Earlier this month it landed a €20mln European Investment Bank debt facility that will help fund a late stage clinical study of a potentially breakthrough treatment called Episalvan for a rare skin disease.
The cash will pay for a phase III trial to demonstrate efficacy and safety, which is expected to get underway in the first-quarter of 2017.
Early data from patients with Epidermolysis Bullosa (EB) will be available by mid-2018 with full commercial launch of the product expected the year after.
EB is an inherited disorder where the skin becomes very fragile. The market for the new treatment is thought to be worth US$1.5bn a year.
The funding package is important as it spares investors dilution that would have come if Amryt Pharmaceuticals (LON:AMYT) had issued shares to bankroll the development of Episalvan.
The product is approved for use in adults in a type of gash or ulcer called a partial thickness wound.
It is a case now of proving the treatment works for EB. It does have competition in the EB market in the form of a drug coming to the market developed by NASDAQ-listed Amicus Therapeutics.
Some experts say Amicus’s product has a slightly inferior profile to Episalvan, yet it is valued by analysts at €326mln (US$350mln).
Amyrt’s market capitalisation currently stands at €48mln, so there’s a valuation disconnect there.
The anomaly hasn’t escaped chief executive Joe Wiley and the team, who, longer term, may look to list the business on NASDAQ (while keeping its stock market quote here).
Amryt expects phase III trials of Episalvan to begin early next year and is already making plans for a commercial launch the year after.
ImmuPharma
Earlier this month it the company confirmed it has completed patient recruitment for its phase III clinical trial of Lupuzor, a potentially breakthrough treatment for lupus.
It has delivered on its plan to have 200 sufferers of the life-threatening auto-immune disease taking part in the study by the end of the year.
In regulatory terms, Lupuzor is in a strong position, already having received fast-track status from the all-powerful US Food & Drug Administration (FDA), which speeds up the approval process by shortening review periods.
ImmuPharma PLC (LON:IMM) has also received regulatory approval to complete its trials under what’s known as a special protocol assessment.
This effectively guarantees the FDA will accept the trial results if they meet or exceed certain criteria.
Systemic Lupus Erythematosus (SLE), the most severe form of the disease, which Lupuzor is targeting, is debilitating and painful.
It sees the body's immune system turn against itself for reasons still not fully understood.
Symptoms include headaches, abdominal pain, chest pain, depression, psychosis and in some cases death.
It can affect various organs, including the heart and liver and is much more common in women, appears most often in African-Americans, Asians and Hispanics. It is estimated 5mln people globally have it.
Only one treatment - Glaxo's Benlysta - has been approved over the last 50 years. And, according to analysts, the product has proven a disappointment, in that it is only marginally effective while having side-effects.
Yet it still generates sales of around US$400mln, a figure that is predicted to grow to US$1bn by 2020.
Faron Pharmaceuticals
The Finnish company recently embarked on a final phase clinical study in Japan for a condition called acute respiratory distress syndrome (ARDS).
ARDS is a disease with a reported mortality rate of around 30-45% for which there is currently no approved pharmacological treatment.
It is characterised by widespread capillary leakage and inflammation in the lungs, most often as a result of pneumoniafor exmample, following a pandemic influenza), sepsis (organ failure), or significant trauma.
It is an orphan disease, which means that any treatments designed to treat it generally get fast-tracked by the regulatory authorities as an inducement to target a disease that otherwise would not be cost effective for the drugs companies to address.
Traumakine is Faron Pharmaceutical Oy's (LON:FARN) flagship drug candidate and it is attracting a lot of interest.
The treatment is already in phase III trials in Europe and licences have been picked up by three companies in Asia, including a deal with Korean firm Pharmbio.