NEA-backed Night­star Ther­a­peu­tics sets sights on an $86M IPO to fund PhI­II gene ther­a­py study

David Fel­lows

Two months af­ter bank­ing a $45 mil­lion crossover round from some top biotech in­vestors, Ox­ford gene ther­a­py spin­out Night­star Ther­a­peu­tics has pen­ciled in an $86 mil­lion IPO to push its lead drug through Phase III test­ing.

Night­star is a reti­nal gene ther­a­py com­pa­ny— of­fi­cial­ly set up as a new sub­sidiary to Night­StaRx — ad­vanc­ing new ther­a­pies aimed at dis­eases that cause blind­ing. First up is NSR-REP1, de­signed to treat choroi­deremia by cor­rect­ing mu­ta­tions in the CHM gene. Ac­cord­ing to the F-1, the biotech has da­ta on 32 pa­tients show­ing their gene ther­a­py ei­ther main­tained or im­proved vi­su­al acu­ity in 90% of the pa­tients in the study. The Phase III pro­gram, set to start in ear­ly 2018, will re­cruit 140 pa­tients and di­vide it in­to high- and low- and no-dose groups, while run­ning a par­al­lel nat­ur­al his­to­ry study to de­tail ex­pect­ed rates of de­cline.

Their next clin­i­cal-stage pro­gram is fo­cused on XL­RP, which “ac­counts for ap­prox­i­mate­ly 15% of all cas­es of re­tini­tis pig­men­tosa, an in­her­it­ed X-linked re­ces­sive reti­nal dis­ease char­ac­ter­ized by a lack of pro­tein trans­port that leads to a loss of pho­tore­cep­tors.” That’s in a Phase I/II study, and there’s a pre­clin­i­cal ther­a­py in view as well.

David Mott, NEA

As of mid-2017, the biotech re­ports that it had burned through about $40 mil­lion, rel­a­tive­ly mod­est for a three-year-old com­pa­ny look­ing to kick off a Phase III study and an IPO.

The com­pa­ny is led by an ex­pe­ri­enced group of bio­phar­ma vets. David Fel­lows, who held se­nior-lev­el mar­ket­ing po­si­tions at J&J and Al­ler­gan, is CEO. CMO Aniz Girach came from Throm­bo­Gen­ics and CSO Gre­go­ry Robin­son once helped Shire on sci­en­tif­ic li­cens­ing for rare dis­eases.

NEA (rep­re­sent­ed by a pro­lif­ic David Mott), Syn­cona, Welling­ton Man­age­ment are the prin­ci­pal share­hold­ers, though the com­pa­ny didn’t ac­tu­al­ly spell out the shares they own in the F1, as re­quired.

The com­pa­ny plans to list as $NITE.

They’ll be head­ed in­to a good-though-not-great mar­ket for biotech IPOs. We’ve seen a string of new of­fer­ings over the last few months, but it doesn’t come close to the 2014 boom, when gene ther­a­py IPOs were gold­en.

Com­mu­ni­cat­ing the val­ue of pre­ci­sion med­i­cine

By Natasha Cowan, Content Marketing Manager at Blue Latitude Health.
Many stakeholders are confused by novel precision medicines, including patients and healthcare professionals. So, how can industry help them to navigate this complexity?

Precision medicine represents a new paradigm in healthcare. It embodies the shift from treating many patients with the same therapy, to having the tools to identify the best treatment for every patient.

Mer­ck buys a fledg­ling neu­rode­gen­er­a­tive biotech spawned by an old GSK dis­cov­ery al­liance. What’s up with that?

Avalon Ventures chief Jay Lichter has a well-known yen for drug development programs picked up in academia. And what he found in Haoxing Xu’s lab at the University of Michigan pricked his interest enough to launch one of his umbrella biotechs in San Diego.
Xu’s work laid the foundation for Avalon to launch Calporta, which has been working on finding small molecule agonists of TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) for lysosomal storage disorders. And that pathway, they believe, points to new approaches on major market neurodegenerative diseases like Parkinson’s, ALS and Alzheimer’s.

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In­vestors could emerge from Neil Wood­ford de­ba­cle with £1B loss, in­ter­nal analy­sis re­veals

When Link Fund Solutions announced that it is closing Woodford Equity Income Fund permanently and kicking out Neil Woodford, it was implied that investors probably won’t get back everything they entrusted to the fund manager. But nobody knew just how much they would lose.

An internal analysis commissioned by Link suggested that the collective loss could amount to £1 billion — out of a fund last valued at £3.1 billion — Citywire has revealed.

GSK's asth­ma bi­o­log­ic Nu­cala scores in rare blood dis­or­der study

GlaxoSmithKline’s asthma drug Nucala, which received a resounding FDA rejection for use in chronic obstructive pulmonary disease (COPD) last year, has shown promise in a rare blood disorder.

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Spe­cial re­port: Twen­ty ex­tra­or­di­nary women in bio­phar­ma R&D who worked their way to the top

What differentiates a woman leader in biopharma R&D from a man?

Not much, except there are fewer of them in senior posts. Data suggest women are not more risk-averse, family-oriented or less confident than their male counterparts — indeed the differences between the two sexes are negligible. But a glance at the top R&D positions in Big Pharma leaves little doubt that upward migration in the executive ranks of biopharma R&D is tough.

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FDA Vas­cepa re­view spot­lights new safe­ty sig­nals, pos­si­ble min­er­al oil spoil­er as Amarin hunts a block­buster ap­proval

An in-house FDA review of Amarin’s Vascepa raises a set of hurdles the biotech will have to clear if the biotech expects to get the long-awaited FDA approval that could set it on a path to superstar status. But it appears that Amarin has survived another potential setback without introducing a major new threat to its prospects.

The stakes don’t get much higher, with analysts saying a win this week for Amarin could lead to billions in new sales — provided the agency stamps it with an OK. And investors liked what they say in the FDA review this morning, bumping the stock $AMRN 17%.

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FDA puts Sol­id Bio’s lead gene ther­a­py pro­gram on hold — again — af­ter an­oth­er pa­tient is hurt by SGT-001

Solid Biosciences continues to be plagued by safety issues.

Close to 18 months after the gene therapy biotech was able to quickly shed an FDA hold on their lead Duchenne muscular dystrophy program for SGT-001, regulators have stepped back in to force another halt after another patient was hit hard by a set of serious adverse events remarkably similar to the first set.

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FDA slaps a hold on an AML tri­al as Mark­er scraps a fail­ing ovar­i­an can­cer pro­gram, sink­ing shares

The FDA has placed a hold on a Phase II AML trial from the small immuno-oncology biotech Marker Therapeutics. Marker disclosed the issue two weeks after responding to FDA concerns, adding it to the Q3 release Tuesday. The company also announced it was scrapping a Phase II ovarian cancer program it determined was unlikely to succeed.

The agency’s concern centers around two reagents used in manufacturing for their trial for acute myeloid leukemia patients who have received a stem cell transplant. The reagents are from third parties and not present in the final product, Marker said.

Eli Lil­ly-backed biotech grabs $100M to dis­patch an­ti­body-oligonu­cleotide con­ju­gates af­ter mus­cu­lar dy­s­tro­phy

Hold up your hand. Make a fist. Now open it. And again.

If you can do it fully and with ease, then the proteins in your hand are likely working properly. If you can’t then they may not be. In people with myotonic muscular dystrophy, something more atomic is going on.

In those folks, the problem is RNA. Certain base pairs repeat far beyond normal, up to 11,000 superfluous letters in some cases. The extended strands form “clumps.” Proteins misform and can’t function properly. They often allow one movement but not the reverse, a condition called myotonia that gives the dystrophy its name.